Podcasts about gilenya

Play Episode Listen Later Jul 24, 2023 36:20

Experiencing difficulty walking is one of the most common mobility challenges faced by people living with MS. The good news is that there are things you can do -- both with a physical therapist and by yourself, at home -- that can make a real difference. Joining me to discuss improving your gait, avoiding falls, and what experts are learning from the latest research in this area is the Director of Research for the Department of Health Care Sciences at Wayne State University School of Medicine, Dr. Nora Fritz. We're also sharing the results of a study that looked at whether disease-modifying therapies had an impact on the number of hospitalizations or visits to the doctor someone with MS might experience over a 22-year period. We'll tell you about a study that measured the outcome for people with relapsing-remitting MS who were treated with autologous hematopoietic stem cell transplantation (aHSCT) compared with the outcome for people with relapsing-remitting MS who were treated with Gilenya, Tysabri, or Ocrevus. We'll share the results from a study that analyzed 56 other studies to determine the prevalence of sexual dysfunction among women living with MS. We'll tell you about a nationwide study that's about to get underway that will try to answer the question, 'Should people with MS over the age of 65 discontinue their disease-modifying therapy?' And we'll remind you about how you can join us in Napa Valley this Saturday, July 29th, for Crush MS. We have a lot to talk about! Are you ready for RealTalk MS??! This Week: Improving your balance and gait :22 A research team analyzed the risk of hospitalization over a 22-year period for people with MS who were on a disease-modifying therapy and compared those results to people with MS who weren't on a DMT 1:21 Researchers compared patient outcomes associated with autologous hematopoietic stem cell transplantation (aHSCT) and compared them with outcomes associated with Gilenya, Tysabri, and Ocrevus 4:40 A research team in Iran analyzed results from 56 separate studies about sexual dysfunction in women with MS 8:29 Should someone over the age of 65 discontinue their disease-modifying therapy? 11:40 Dr. Nora Fritz discusses gait, balance, fall prevention and more 15:20 Share this episode 33:49 I'll be at Crush MS! Will I see you there? 34:09 SHARE THIS EPISODE OF REALTALK MS Just copy this link & paste it into your text or email: https://realtalkms.com/308 ADD YOUR VOICE TO THE CONVERSATION I've always thought about the RealTalk MS podcast as a conversation. And this is your opportunity to join the conversation by sharing your feedback, questions, and suggestions for topics that we can discuss in future podcast episodes. Please shoot me an email or call the RealTalk MS Listener Hotline and share your thoughts! Email: jon@realtalkms.com Phone: (310) 526-2283 And don't forget to join us in the RealTalk MS Facebook group! LINKS If your podcast app doesn't allow you to click on these links, you'll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com STUDY: Disease-Modifying Drugs for Multiple Sclerosis and Subsequent Health Service Use https://pubmed.ncbi.nlm.nih.gov/34949130 Tremlett's MS Research Explained: Disease-Modifying Drugs for Multiple Sclerosis and Subsequent Health Service Use https://tremlettsmsresearchexplained.wordpress.com/category/ms-disease-modifying-drug-research STUDY: Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis https://pubmed.ncbi.nlm.nih.gov/37437240 STUDY: Prevalence and Risk of Developing Sexual Dysfunction in Women with Multiple Sclerosis (MS): A Systematic Review and Meta-Analysis https://bmcwomenshealth.biomedcentral.com/articles/10.1186/s12905-023-02501-1 RealTalk MS Episode 255: Aging With MS with Dr. John Corboy https://realtalkms.com/255 Crush MS https://crushms.org If you're living with MS, email info@crushms.org for special deep discount code Crush MS 10% Discount Code for everyone is Realtalkmsdiscount (case sensitive) Join the RealTalk MS Facebook Group https://facebook.com/groups/realtalkms Download the RealTalk MS App for iOS Devices https://itunes.apple.com/us/app/realtalk-ms/id1436917200 Download the RealTalk MS App for Android Deviceshttps://play.google.com/store/apps/details?id=tv.wizzard.android.realtalk Give RealTalk MS a rating and review http://www.realtalkms.com/review Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 308 Guest: Dr. Nora Fritz Privacy Policy

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59. Future of ADCs w/ David Epstein - CEO @ Seagen

BIOS

Play Episode Listen Later Jun 12, 2023 43:43

David Epstein joined Seagen as Chief Executive Officer and a member of the Board of Directors in 2022, bringing more than 30 years of drug development, deal making, commercialization and people leadership experience on a global scale. Prior to Seagen, he was executive partner at Flagship Pioneering. From 2010 to mid-2016 he served as Chief Executive Officer of Novartis Pharmaceuticals, a division of Novartis AG.Previously, David started and led Novartis' Oncology and Molecular Diagnostic units. Over the course of his career, he led the development and commercialization of over 30 new molecular entities, including major breakthroughs such as Glivec, Tasigna, Gilenya, Cosentyx and Entresto.David holds a B.S. Degree in Pharmacy from Rutgers University College of Pharmacy and an MBA in Finance and Marketing from the Columbia University Graduate School of Business. He serves as a member of the board of directors for OPY Acquisition Corp. I and Senti Biosciences, Inc.w/ Special Guest Host: Brian Fiske - Co-Founder & CSO @ Mythic TherapeuticsBrian Fiske co-founded Mythic in 2017 & currently serves as Mythic's Chief Scientific Officer. Prior to Mythic, he was a co-founder and Chief Technology Officer at Ohana Biosciences and a Senior Associate at Flagship Ventures, where he also co-founded KSQ Therapeutics. During his time at Flagship, Brian successfully led R&D teams to key milestones, recruited 25+ FTEs across all levels of seniority, and raised $34M across multiple rounds of financing. In 2016, he was nationally recognized for healthcare entrepreneurship by Forbes 30 under 30.Prior to Flagship, Brian completed his PhD in biology in Matt Vander Heiden's lab at MIT where he published over 10 papers in the field of cancer metabolism. He also worked closely with Agios Pharmaceuticals (NASDAQ: AGIO) on their cancer metabolism programs, which have since translated into four clinical programs that include two approved drugs. He holds an AB summa cum laude in Biochemical Sciences with a secondary field in Economics from Harvard University.Alix Ventures, by way of BIOS Community, is providing this content for general information purposes only. Reference to any specific product or entity does not constitute an endorsement nor recommendation by Alix Ventures, BIOS Community, or its affiliates. The views & opinions expressed by guests are their own & their appearance on the program does not imply an endorsement of them nor any entity they represent. Views & opinions expressed by Alix Ventures employees are those of the employees & do not necessarily reflect the view of Alix Ventures, BIOS Community, affiliates, nor its content sponsors.Thank you for listening!BIOS (@BIOS_Community) unites a community of Life Science innovators dedicated to driving patient impact. Alix Ventures (@AlixVentures) is a San Francisco based venture capital firm supporting early stage Life Science startups engineering biology to create radical advances in human health.Music: Danger Storm by Kevin MacLeod (link & license)

Stem Cell Transplant Versus DMTs & DMTs Ease SPMS Disability Early

Play Episode Listen Later Nov 18, 2022 15:29

Multiple Sclerosis News Today's multimedia associate, Price Wooldridge, reads a news article on how stem cell transplant is superior to Gilenya and Tysabri at preventing relapses and easing disability in RRMS patients.  He also reads another report on how early use of DMTs may lead to lower levels of disability but does not appear to slow disability progression over time. =================================== Are you interested in learning more about multiple sclerosis? If so, please visit: https://multiplesclerosisnewstoday.com/ ===================================== To join in on conversations regarding multiple sclerosis, please visit: https://multiplesclerosisnewstoday.com/forums/

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Episode 268: What You Need to Know About Open Enrollment with Nicole Vasquez

Play Episode Listen Later Oct 17, 2022 34:04

If you're on Medicare, or you get your health insurance through the healthcare.gov marketplace or one of the state health insurance exchanges, then you might already know that the annual open enrollment period is about to get underway or, in some cases, is already underway. Health insurance policies change. Physician networks change. Drug formularies often change. Deductibles change. And if you're living with MS, you can't afford to go through open enrollment assuming that the health insurance policy you have this year will be the best coverage for you next year. Nicole Vasquez returns to the podcast to discuss the things you should carefully consider during open enrollment. Nicole is one of the National MS Society's MS Navigators and she'll be sharing information and resources that you can use to make the best health insurance decisions for yourself and your family. Can you spare 5 minutes? I have a quick favor to ask each of you. I'll also tell you about the Supreme Court decision that, for now, clears the way for Gilenya generics. You'll hear about a new cell therapy being developed that will use nanotechnology to eliminate Epstein-Barr Virus (and I'll remind you why that would be a massive step forward in ending MS). And we'll tell you about a University of Illinois research team that leveraged artificial intelligence to predict MS by observing how people walk. We have a lot to talk about! Are you ready for RealTalk MS??! Sharing some listener feedback :22 This Week: Open enrollment 3:06 Will you do me a quick favor? 5:06 Supreme Court clears the way for Gilenya generics 7:25 Cell therapy will use nano-technology to eliminate EBV 8:25 Research team uses AI to identify people living with MS 10:55 MS Navigator Nicole Vasquez gets you ready for open enrollment 13:57 Share this episode 32:32 Download the RealTalk MS app for your iOS or Android device 32:52 SHARE THIS EPISODE OF REALTALK MS Just copy this link & paste it into your text or email: https://realtalkms.com/269 ADD YOUR VOICE TO THE CONVERSATION I've always thought about the RealTalk MS podcast as a conversation. And this is your opportunity to join the conversation by sharing your feedback, questions, and suggestions for topics that we can discuss in future podcast episodes. Please shoot me an email or call the RealTalk MS Listener Hotline and share your thoughts! Email: jon@realtalkms.com Phone: (310) 526-2283 And don't forget to join us in the RealTalk MS Facebook group! LINKS If your podcast app doesn't allow you to click on these links, you'll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com Take the iConquer MS Caregiver Survey https://realtalkms.com/caregiver STUDY: A Vision-Based Framework for Predicting Multiple Sclerosis and Parkinson's Disease Gait Dysfunctions -- A Deep-Learning Approach https://ieeexplore.ieee.org/document/9896159 Join the RealTalk MS Facebook Group https://facebook.com/groups/realtalkms Download the RealTalk MS App for iOS Devices https://itunes.apple.com/us/app/realtalk-ms/id1436917200 Download the RealTalk MS App for Android Deviceshttps://play.google.com/store/apps/details?id=tv.wizzard.android.realtalk Give RealTalk MS a rating and review http://www.realtalkms.com/review Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 268 Guest: Nicole Vasquez Tags: MS, MultipleSclerosis, MSResearch, MSSociety, RealTalkMS Privacy Policy

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Higher Covid-19 Risk Tied to 2 MS Therapies & With MS Good Planning Is Key

MS-Perspektive - der Multiple Sklerose Podcast mit Nele Handwerker

Play Episode Listen Later Jul 20, 2022 11:15

Multiple Sclerosis News Today's multimedia associate, Price Wooldridge reads an article about how fully vaccinated MS patients who are taking Ocrevus or Gilenya have a higher risk of COVID-19 infection than those on other therapies. He also reads “While Living With MS, Good Planning Is Key to Avoiding Chaos”, a column from Beth Ullah's “ Through the Looking Glass.” =================================== Are you interested in learning more about multiple sclerosis? If so, please visit: https://multiplesclerosisnewstoday.com/ ===================================== To join in on conversations regarding multiple sclerosis, please visit: https://multiplesclerosisnewstoday.com/forums/

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Interview mit dem MS-Patienten und Autoren von Alex Blog #133

Play Episode Listen Later Apr 25, 2022 40:48

Heute interviewe ich den MS-Patienten und gleichnamigen Autoren von Alex Blog, der auch aufgrund der Erkrankung zu sich selbst gefunden hat. Hier geht es zum Blogbeitrag: https://ms-perspektive.de/interview-mit-dem-ms-patienten-und-autoren-von-alex-blog Diesmal habe ich Alex zu Gast im Interview. Er erhielt 2014 die Diagnose Multiple Sklerose, ca. 12 Jahre nach einem ersten deutlichen Schub, einer halbseitigen Lähmung. Wir sprechen über seine Texte, die bei der DMSG Niedersachen auf Alex' Blog erschienen sind. Sie decken insgesamt vier Jahre ab, in denen er seinen Weg mit der Erkrankung und zu sich selbst gefunden hat. Heute ist Alex sehr reflektiert und hat sich gelöst davon immer etwas zu „müssen“ hin zu „ich darf“. Auf Alex' Blog lernst Du die Geschichte eines Mannes von tausend Gesichtern der MS kennen. Und ob diese Geschichte interessant für Dich ist, findest Du im Beitrag heraus. Inhaltsverzeichnis Vorstellung Diagnose und aktueller Status Alex' Blog Tipps Wünsche und Ziele Blitzlicht-Runde Verabschiedung Vorstellung Ich bin Alexander Gottschalk, 39 Jahre alt und stamme ursprünglich aus Magdeburg. Beruflich bin ich nach Hannover gegangen und heute sehr aktiv bei der DMSG Niedersachsen. Mittlerweile bin ich Autor von dem Blog: „Ich, mein Freund. Mein Weg zu mir selbst unter erschwerten Bedingungen.“ Im Blog geht es um meinen Weg. Wie ich gelernt habe mich selbst neu wahrzunehmen und mit der MS anzufreunden und wie ich mir neue Sichtweisen auf die Welt und die Menschen erarbeitet habe. Die MS hat einen unglaublichen Entwicklungsschub bei mir ausgelöst und mir ermöglicht, mich selbst neu zu entdecken, nachdem ich viele, viele vermeintliche Verluste hinnehmen musste. In den Artikeln arbeite ich mit vielen Sprachbildern, die ich aufgrund meiner Erfahrungen hoffentlich auch für andere verständlich in Szene setzen kann. Das Schreiben über meine Erfahrungen hilft mir, das Fortschreiten der Krankheit selbst zu verarbeiten. Ich beginne dabei natürlich am Anfang, als es mir sehr schlecht ging bzw. ich mir Fragen stellte, die ich nicht befriedigend beantworten konnte. Das Ganze entwickelt sich vor allem durch die Unterstützung meiner Therapeuten und meinem Durst nach Wissen. Es geht darum sich nicht selbst aufzugeben, Möglichkeiten in diesem jungfräulichen Neuland der Krankheit zu entdecken, die bisherige Erfahrungen nicht in der Lage waren, abzudecken. Diagnose und aktueller Status Seit wann hast du die Diagnose und was war der Anlass für die Diagnose? Die endgültige Diagnose erhielt ich 2014, als die Fatigue überhandnahm, und ich mich immer mehr in mich selbst zurückzog. Wie hast Du die Diagnose aufgefasst? Und wie war es für Dein Umfeld? Ich war froh, dem an mir zerrenden Kind einen Namen geben zu können, hatte auch Unterstützung von meiner damaligen Frau. Von Anfang bin ich sehr offen damit umgegangen. Mein Arbeitgeber und meine Familie waren natürlich betroffen. Allerdings stieß ich nur selten auf Verständnis. Es fiel mir damals sehr schwer, meine Eigenartigkeiten zu erklären. Ich wusste ja selber noch nicht sehr viel über die Krankheit und schon gar nicht über mich selbst. Wie geht es Dir aktuell mit der MS? So lala, es gibt solche und solche Tage. Ich bin froh, dass ich mich auf die Suche nach mir selbst begeben habe, somit kann ich sehr viel besser mit meinen Körperlichkeiten und Erschöpfungszuständen umgehen. Jede Krankheit beginnt bekanntlich im Kopf und durch meine Beschäftigung mit mir selbst ist die MS nicht mehr so sehr wirksam hinsichtlich Selbstmitleid und Rückzug. Es gibt natürlich Tage, da haben die Einschränkungen, speziell die Fatigue, prägende und einnehmende Qualität. Aber das kann ich mittlerweile zulassen, ohne daran zu verzweifeln. Welche Behandlung wurde Dir zu Beginn der Diagnose empfohlen und bist Du der Empfehlung gefolgt? Tecfidera, ja ich bin der Empfehlung gefolgt. Ich bin selber kein Arzt und trotz aller vergangenen Widrigkeiten habe ich immer ein großes Urvertrauen gegenüber Fachleuten gehabt und habe es immer noch. Die Frage ist ja auch, was wäre, wenn ich keine Medikamente nehmen würde, wie würde es dir dann jetzt gehen? Hast Du im Laufe der Zeit Erfahrungen mit verschiedenen verlaufsmodifizierenden Therapien gemacht? Ja habe ich, Gilenya und aktuell Ocrevus, muss aber dazu sagen, dass ich diese Medikamente gar nicht merke, so soll das wohl auch sein, nur bei Tecfidera am Anfang hatte ich die bekannten Flushs. Warst Du bei einer oder mehreren Reha-Maßnahmen wegen der MS? Ja, ich habe mittlerweile zwei Rehas gemacht. Nach der Ersten wurde ich gefragt, ob ich denn Therapeut oder Patient wäre. Das hat also wirklich was gebracht! Na ja und nach der zweiten war die Krankheit bereits so fortgeschritten, dass ich danach zum Rentner wurde. Glücklicherweise, darf ich sagen. Welche symptomatischen Therapieangebote nutzt Du und wie zufrieden bist Du damit? Physiotherapie, Ergotherapie, Psychotherapie, Logotherapie und zu Hause mache ich auch noch ein bisschen. Habe zwischenzeitlich auch mal Funktionstraining gemacht, aber das wurde mir ziemlich schnell einfach zu anstrengend. Danach war immer der ganze Tag vorbei weil Akku leer. Die ersten vier Sachen reichen mir durchaus, machen Spaß, geben ein wenig Struktur, sind aber auch anspruchsvoll Besonders die Psychotherapie will ich nicht mehr missen, das hilft mir meine Depression aufzulösen und mich nun nicht weiter geistig und substanziell selbst zu vergiften. Wie versuchst Du, auf den Verlauf der MS Einfluss zu nehmen? Indem ich lerne, mit ihr umzugehen. Ich halte nicht viel davon mir viele verschiedene Mittelchen reinzutun und mich an jeden Nahrungsergänzungsstrohhalm zu klammern und zu hoffen. Mich selbst bewusst wahrnehmen und auf mich hören, ist sehr viel mehr wert, finde ich. Hilfe und Hilfsmittel annehmen und entschleunigen. Wenig Stress und öfter Neinsagen hilft ungemein. Alex' Blog Wie kamst Du auf die Idee, Deine Gedanken und Gefühle aufzuschreiben? Als ich meinen Schwerbehindertenausweis beantragt hatte, habe ich einen Widerspruch einlegen müssen, weil ich mit dem Ergebnis des Antrags nicht zufrieden war. Ich hatte damals ursprünglich 30 GdB bekommen, aber auch damals schon fühlte ich mich nicht richtig eingestuft und somit habe ich einen Widerspruch geschrieben. Damit fing alles an und irgendwie, ich weiß nicht genau, habe ich dann immer mal wieder was geschrieben. Ja und dann verselbstständigte sich das und ich musste einfach schreiben, auch weil ich gemerkt habe, dass ich nach jedem Text ruhiger wurde. Welche Rolle spielen die MS und die dazugehörigen Symptome in Deinen Beiträgen auf Alex' Blog? Nun, beides sind natürlich Grundlage für meine Gedanken und Überlegungen. Ich bin beruflich geprägt Probleme zu lösen, so als Speditionskaufmann. Früher waren es Transport und Logistikprobleme. Das hilft mir wirklich sehr, denn irgendwie muss ich ja mit dem, was ich habe, energiesparend umgehen. Jetzt, nach ca. dreieinhalb Jahren habe ich meinen Alltag entsprechend neu reglementiert, eben so dass die MS und deren Symptome nicht allzu sehr im Vordergrund stehen. Waren die Texte von Anfang an für alle lesbar oder wie hat sich das Projekt "Alex' Blog" entwickelt? Nein, es hat eine ganze Weile gedauert, bis ich mich überhaupt einer Selbsthilfegruppe angeschlossen habe, der DMSG. Und die Oberindianer dort gaben mir die Möglichkeit, meine Texte zu veröffentlichen, nachdem sie einige gelesen hatten. Erst gab es nur die Texte an sich, sie wurden gleich veröffentlicht, nachdem ich sie eingereicht habe. Und nun gibt es mittlerweile vertonte Texte, also einige, nicht alle, die nach und nach auf einer neueren professionell erstellten Seite veröffentlicht werden. Daran waren zwei Leute von der DMSG, eine Grafikerin, ein IT'ler, der professionelle Sprecher und natürlich ich beteiligt. Welche Rückmeldungen bekommst Du zu Deinen Texten? Durchweg positive, die ersten Texte sind natürlich ein wenig finster. Da haben die Leute vielleicht aus Verständnislosigkeit nach dem Sinn meiner Unternehmung gesucht. Ich habe überlegt ob ich vielleicht irgendwo in der Mitte meiner Entwicklung anfange. Also mit einem Text und wo man sieht, dass es mit mir bergauf geht. Aber das finstere Vorher gehört eben auch dazu. Bei manchen Texten wurde geweint, bei anderen gelacht und oft blieb das Lachen im Halse stecken. Mir wurde gesagt, dass eine Entwicklung auch in meinem Schreibstil erkennbar ist. Über welchen Zeitraum Deines Lebens erstrecken sich die Texte auf Alex' Blog und hast Du vor ein festes Ende zu setzen? Es beginnt mit meinem Widerspruch gegen das Sozialamt im Februar 2019. Es soll ein festes Ende geben, geplant ist das für dieses Jahr. Wann genau und ob überhaupt, ich mache mir da selbst keine Auflage. Ich lasse mir mittlerweile Zeit beim Schreiben und auch beim Einordnen meiner Erfahrungen. Es ist, glaube ich kein klassischer Blog im Sinne von Tagebuch, sondern ein Erfahrungsbericht, wenn man so will. Schon gar kein Ratgeber, eher eine Sammlung von Geschichten, die zufällig was mit MS zu tun haben. Was haben die Texte mit Dir selbst gemacht? Tja, das klingt jetzt bestimmt irgendwie komisch, aber sie haben mich reifer werden lassen. Gelassener. Ich verfluche mich nicht mehr sondern kokettiere mit meinen vermeintlichen Unzulänglichkeiten, ich nenne sie auch Special Effects. Wie hast Du Dich im Laufe der Zeit verändert und worauf bist Du besonders stolz? Ich bin sehr viel aufmerksamer mir selbst gegenüber geworden. Und auch unsere Mitwelt nehme ich viel differenzierter wahr. Ich schaue auf das, was ist und nicht, was sein sollte oder sein müsste. Am meisten bin ich darauf stolz mich selbst nicht mehr so ernst zu nehmen, fünf auch mal gerade sein lassen zu können und mich selbst nicht mehr so unter Druck zu setzen. Die Erkenntnis, dass ich nicht muss, sondern darf. Tipps Was war dein tiefster Tiefpunkt mit der MS und wie hast du dich wieder empor gekämpft? Ganz klar, meine Scheidung. Der Verlust von Haus und Hof, und die plötzliche Erkenntnis, dass ich nicht mehr so belastbar bin, wie es früher der Fall gewesen ist. Es hat eine Weile gedauert, aber meine Strategie weg von der Lethargie hin zu mehr Spaß war und ist das genaue Hinsehen und Hinfühlen auch und gerade in unangenehmen Situationen. Das schafft Verständnis und Wissen, härtet ein wenig ab, zeigt Möglichkeiten auf und macht vor allem flexibel. Wie lange hat es gedauert bis Du wieder freundlicher auf die Welt schauen konntest? Dezember 2020 bis hin zu Neujahr 2021. Also knapp zwei Jahre nachdem ich mit der Schreiberei angefangen habe, sechs Jahre nach meiner endgültigen Diagnose und 19 Jahre nach meinem ersten Schub bei dem ich als Azubi halbseitig gelähmt war. Ich hatte 11-12 Jahre keine gravierenden Symptome also auch keinen Grund, mich so intensiv mit mir zu beschäftigen. Wünsche und Ziele Gibt es einen großen unerfüllten Wunsch? Na klar! Gesehen und angenommen werden!! Als der Mensch, der ich heute bin. Geborgenheit, Nähe, Zugewandtheit, Trost, eine Freundin. Welche Entwicklung wünschst du Dir im Bereich der MS in den kommenden 5 Jahren? Ich bin generell sehr vorsichtig mit Wünschen. Am Ende kriegt man auch noch, was man sich vorstellt. Natürlich möchte ich, dass ein Mittelchen gegen die MS gefunden wird, welches alle Narben zurückbildet und Symptome verschwinden lässt. Mich irgendwie wieder der werden lässt, der ich mal war. Blitzlicht-Runde Was war der beste Ratschlag, den du jemals erhalten hast? Trau dich und guck mal ein bisschen genauer hin. Wie lautet dein aktuelles Lebensmotto? Da gibt es einige. Wer sich Sorgen macht, leidet zweimal, ist wohl der aktuell Prägnanteste. Und: Das größte Geschenk an die Zukunft ist, der Gegenwart alles zu geben. Mit welcher Person würdest du gern einmal ein Kamingespräch führen und zu welchem Thema? Mit mir selbst vor 20 Jahren. Thema: Was ist das Leben? Vervollständige den Satz: „Für mich ist die Multiple Sklerose... “ …der Beginn meines zweiten Lebens. Welche Internet-Seite kannst du zum Thema MS empfehlen? Puh, das Forum der Amsel ansonsten die DMSG-Seiten. Ich selbst meide sowas aber und konzentriere mich auf meine Therapien und mich selbst. Welches Buch oder Hörbuch, das du kürzlich gelesen hast, kannst du uns empfehlen und worum geht es darin? Die Brautprinzessin von William Goldman. Das ist ein Märchen für Erwachsene, es geht natürlich um Liebe. Sehr fantasievoll, sprachgewandt und einfach schön. Verabschiedung Hast du einen Tipp, den Du Deinem jüngeren Ich geben würdest, für den Zeitpunkt der Diagnose? Fliehe bloß nicht wieder in irgendwelche Süchte. Such dir gleich Hilfe und stell dich deinen Dämonen. Möchtest du den Hörerinnen und Hörern noch etwas mit auf dem Weg geben? Das Gleiche, was ich mir selber sagen würde, steck den Kopf nicht in den Sand. Du kriegst alle Hilfe, die Du willst, wenn Du es zulässt. Und vor allem, du bist nicht allein! Wo findet man dich im Internet? Bei der DMSG Niedersachsen gibt es eine Sparte „junge DMSG“. Da ist Alex‘ Blog zu finden. Vielen Dank, liebe für Deinen offenen Einblicke, gerade auch in die schweren und dunklen Seiten Deines Lebens und toll, wie Du es geschafft hast Dich wieder herauszukämpfen. Weiterhin alles Gute auf Deinem Weg. Bis bald und mach das beste aus Deinem Leben, Nele Mehr Informationen und positive Gedanken erhältst Du in meinem kostenlosen Newsletter. Hier findest Du eine Übersicht zu allen bisher interviewten MS-Patienten.

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How Novartis Built a Hit Factory for New Drugs

Soonish

Play Episode Listen Later Mar 12, 2022 60:13

When you hear people use the phrase "It's a hits-driven business," they're usually talking about venture capital, TV production, videogames, or pop music—all industries where you don't make much money unless you come up with at least one (and preferably a string of) massively popular products. But you know what's another hits-driven business? Drug development. This week, we present the fourth and final episode in the Persistent Innovators miniseries, originally produced for InnoLead's Innovation Answered podcast and republished here for Soonish listeners. It's all about the giant Swiss pharmaceutical company Novartis, maker of more than a dozen blockbuster drugs like Cosentyx for psoriasis, Entresto for heart failure, and Gilenya for multiple sclerosis. Because companies lose patent protection on their old drugs after 17 years, they must constantly refill their pipeline of new drugs—and Novartis has done that by placing a huge bet on the Novartis Institutes for BioMedical Research (NIBR), its 2,000-person R&D lab based in Soonish's hometown of Cambridge, MA. In this episode you'll meet Tom Hughes, a biotech entrepreneur and former Novartis executive who helped to set up NIBR in the early 2000s, as well as NIBR's current president, Jay Bradner. They explain why the decision to build NIBR was initially controversial even inside Novartis, and how the labs are structured today to take big but manageable risks and ensure that the company can capitalize on biology's growing understanding of the molecular and genetic underpinnings of disease."I find from the top down, our chairman to our CEO, to every commercial leader, there is a tolerance and an appetite for bravery in drug discovery that is really refreshing and honestly very empowering," Bradner says of Novartis. "If you looked at the type of programs in our portfolio, they're not for the faint of heart. And this is for a very specific reason. We worry that if we don't try to [do it] well, then who will?""What Makes Novartis a Persistent Innovator?" was first published by Innovation Answered on February 28, 2022. You can hear the entire miniseries at innovationleader.com or in your podcast player of choice.Logo photo by Sangharsh Lohakare on UnsplashFull transcript available at http://www.soonishpodcast.org/505-novartis

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Mavenclad Works Best at Reducing Relapses & Managing Fatigue Through Medication

Play Episode Listen Later Mar 11, 2022 11:59

Multiple Sclerosis News Today's multimedia associate, Price Wooldridge, covers how MS patients on Mavenclad are less likely to experience a disease relapse than those on other oral DMTs like Gilenya, Tecfidera, or Aubagio. He also reads “The Double-Edged Sword of Fatigue Medications”, from Beth Ullah's column "Through the Looking Glass". =================================== Are you interested in learning more about multiple sclerosis? If so, please visit: https://multiplesclerosisnewstoday.com/ ===================================== To join in on conversations regarding multiple sclerosis, please visit: https://multiplesclerosisnewstoday.com/forums/

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Greater Cancer Risk Linked to Certain Oral DMTs & Supplements for MS

Play Episode Listen Later Mar 7, 2022 12:17

Multiple Sclerosis News Today's multimedia associate, Price Wooldridge, reads a news article about how S1P receptor modulators such as Mayzent and Gilenya may be associated with a greater likelihood of skin cancer. He also reads “The Supplements of Mice and Men”, from John Connor's regular column "Fall Down, Get Up Again". =================================== Are you interested in learning more about multiple sclerosis? If so, please visit: https://multiplesclerosisnewstoday.com/ ===================================== To join in on conversations regarding multiple sclerosis, please visit: https://multiplesclerosisnewstoday.com/forums/

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Stopping Fingolimod for Pregnancy Raises Relapse Risk & Singing Can Benefit People with MS

Play Episode Listen Later Dec 3, 2021 12:26

Multiple Sclerosis News Today's multimedia associate, Price Wooldridge, discusses how women with relapsing-remitting MS who stop fingolimod – sold as Gilenya – to conceive or in early pregnancy have a higher relapse risk. Also, Price reads “Once More, With Feeling: How Singing Can Benefit MS Patients”, a column by Jamie Hughes. =================================== Are you interested in learning more about multiple sclerosis? If so, please visit: https://multiplesclerosisnewstoday.com/ ===================================== To join in on conversations regarding multiple sclerosis, please visit: https://multiplesclerosisnewstoday.com/forums/

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Gilenya

MS Podcast UK

Play Episode Listen Later Mar 9, 2021 11:39

giylenya and worlds gone mad. --- Send in a voice message: https://anchor.fm/dave-westley4/message

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Episode 158: A Deep Dive Into MS Disease-Modifying Therapies with Dr. Scott Newsome

Play Episode Listen Later Sep 7, 2020 37:18

Disease-modifying therapies are the cornerstone of any effective MS treatment plan. But with more than 20 approved prescription medications available today, the choices can seem overwhelming. People living with MS have questions -- Do you start out with a safe but less effective medication, or do you hit MS hard with a high-efficacy drug that may carry more risks? When is the right time to consider switching medications, and how do you have that conversation with your neurologist? Do things like other health conditions affect which disease-modifying therapy you're on? And what about aging? My guest is Dr. Scott Newsome, an Associate Professor of Neurology, and Director of Neurosciences Consultation and Infusion Center at Johns Hopkins Medicine. Dr. Newsome also serves as the Co-Director of the Multiple Sclerosis Experimental Therapeutics Program at Johns Hopkins. And I'm devoting this entire episode of RealTalk MS to taking a deep-dive into disease-modifying therapies with Dr. Newsome. We'll also give you the details about two major events taking place this week -- the MSVirtual2020 conference and the Keep Moving Forward benefit celebrating the MS Movement. We have a lot to talk about! Are you ready for RealTalk MS??! ECTRIMS + ACTRIMS = MSVirtual2020 :24 Don't miss Keep Moving Forward 1:08 My Interview with Dr. Scott Newsome 1:38 Share this episode 33:06 Leave a rating & review 33:26 Please Support the National MS Society COVID-19 Response Fund 34:45 SHARE THIS EPISODE OF REALTALK MS Just copy this link & paste it into your text or email: realtalkms.com/158 ADD YOUR VOICE TO THE CONVERSATION I've always thought about the RealTalk MS podcast as a conversation. And this is your opportunity to join the conversation by sharing your feedback, questions, and suggestions for topics that we can discuss in future podcast episodes. Please shoot me an email or call the RealTalk MS Listener Hotline and share your thoughts! Email: jon@realtalkms.comPhone: (310) 526-2283 And don't forget to join us in the RealTalk MS Facebook group! LINKS If your podcast app doesn't allow you to click on these links, you'll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com Give RealTalk MS a Rating and Review National MS Society's Ask An MS Expert Video Replay National MS Society COVID-19 Response Fund Join the RealTalk MS Facebook Group Download the RealTalk MS App for iOS Download the RealTalk MS App for Android Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 158 Hosted By: Jon Strum Guests: Dr. Scott Newsome Tags: MS, MultipleSclerosis, MSResearch, MSSociety, Tecfidera, Ocrevus, Kesimpta, Copaxone, Gilenya, RealTalkMS Privacy Policy

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The Approval of a Generic Form of the MS Treatment Gilenya in the US

Play Episode Listen Later Jan 2, 2020 2:56

MS News Today's columnist and forums moderator, Ed Tobias, discusses the approval of a generic form of the MS treatment Gilenya in the U.S. Are you interested in learning more about Multiple Sclerosis? If so, please visit https://multiplesclerosisnewstoday.com/

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Episode 119: Gaining Access to MS Medications with Lisa Aquillano, PharmD, BCPS, MSCS

Play Episode Listen Later Dec 9, 2019 33:56

What can you do when the MS medication you need can cost $80,000, $90,000 or even $100,000 a year, and you don't have health insurance? Or you have health insurance, but your insurance company won't approve the specific disease-modifying therapy that your neurologist thinks will be best for you? My guest is Lisa Aquillano, a Clinical Pharmacy Specialist in Multiple Sclerosis at Emory University Hospital in Atlanta, Georgia. And we're talking about specific steps that you can take to overcome the obstacles standing in the way of gaining access to your MS disease-modifying therapy. We're also talking about a remarkable program for people living with MS and their care partners. But the window to register for this program is just 24 hours long! And it's coming up in 2 days! We'll tell you all about the program and let you know exactly how to register. We'll tell you about what may be an important new discovery of a molecule that completely resolves MS in mice. We'll also tell you about a study that shows that people living with MS who are also dealing with depression are much more likely to develop debilitating disability sooner. And we'll tell you how and why Google is cracking down on shady stem cell clinic ads. We have a lot to talk about! Are you ready for RealTalk MS??! ___________ CAN-DO Program: 24-Hour Registration Opens Dec. 12 2:15 MicroRNA Resolves Mouse Model of MS 5:39 FDA Approves 3 Generics for Gilenya 8:06 Depressed MS Patients Suffer More Serious Physical Disability Earlier 9:39 Google Cracks Down on Stem Cell Clinic Ads 12:25 My Interview with Lisa Aquillano 14:48 Share This Episode 32:19 How to Subscribe to RealTalk MS, Download the RealTalk MS App, and Listen with Alexa 32:43 ___________ SHARE THIS EPISODE OF REALTALK MS Just copy this link & paste it into your text or email: http://realtalkms.com/119 ___________ ADD YOUR VOICE TO THE CONVERSATION I've always thought about the RealTalk MS podcast as a conversation. And this is your opportunity to join the conversation by sharing your feedback, questions, and suggestions for topics that we can discuss in future podcast episodes. Please shoot me an email or call the RealTalk MS Listener Hotline and share your thoughts! Email: jon@realtalkms.comPhone: (310) 526-2283 ___________ LINKS If your podcast app doesn't allow you to click on these links, you'll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com Give RealTalk MS a Rating & Review The Can-Do Program STUDY: Oral Administration of miR-30d from Feces of MS Patients Suppresses MS-Like Symptoms in Mice By Expanding Akkermansia Muciniphila STUDY: Diability Worsening Among Persons with Multiple Sclerosis and Depression: A Swedish Cohort Study Give RealTalk MS a Rating & Review Download the RealTalk MS App for iOS Download the RealTalk MS App for Android ___________ Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 119 Hosted By: Jon Strum Guests: Lisa Aquillano, PharmD, BCPS, MSCS Tags: MS, MultipleSclerosis, MSResearch, MSSociety, CanDoMS, Gilenya, Depression, Stemcells, RealTalkMS Privacy Policy

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Ralph Kern, BrainStorm - Part of the NameTag Series

Solebury Trout Talks

Play Episode Listen Later Aug 7, 2019 24:51

Ralph Kern, MD, MHSc, is Chief Operating Officer and Chief Medical Officer at BrainStorm. He brings significant industry and neurodegenerative disease experience to the organization. His biotech experience includes senior medical roles at Genzyme, Novartis, and Biogen. At Novartis he was Vice President and Head of the Neuroscience Medical Team, leading the global launch of Gilenya® in relapsing remitting multiple sclerosis (rrMS). At Biogen he was Senior Vice President and Head of the Worldwide Medical Organization. His team launched Zinbryta® in rrMS and Spinraza® in spinal muscular atrophy (SMA), and developed the medical and scientific strategy for MS, SMA, and Alzheimer’s disease.

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Episode 67: MS Research with Dr. Larry Sherman

Play Episode Listen Later Dec 3, 2018 24:40

Research is the engine that drives us toward better understanding MS, better treating MS, and one day, curing MS. My guest on the podcast is Dr. Larry Sherman, who plays a vital role on the front lines of MS research. We're talking with Dr. Sherman about some of his most significant research and his unique research lab. We're also talking about two important victories for MS Activists. We'll tell you about the EMA approval of Gilenya for treating pediatric MS, the FDA approval of a generic for Aubagio, new technology that will enable people with MS to pilot their wheelchairs by flexing a couple of facial muscles, and the rehab technique that can benefit Olympic athletes and people living with MS. We have a lot to talk about! Are you ready for RealTalk MS?! ___________ MS Activists Have Something to Celebrate 0:22 National Neurological Conditions Surveillance System Will Study Data for MS 4:48 European Medicines Agency Approves Gilenya for Pediatric MS 6:41 FDA Approves Generic for Aubagio 8:21 New Technology Lets You Pilot a Wheelchair By Flexing Facial Muscles 9:32 Motor Imagery with Verbal Cues & Music May Lead to Improved Walking, Fatigue & Quality of Life for People Living with MS 11:31 My Interview with Dr. Larry Sherman 15:34 ___________ LINKSIf your podcast app doesn’t allow you to click on these links, you’ll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com Download the RealTalk MS App for iOS Download the RealTalk MS App for Android National MS Society: Get Involved & Advocate For Change Limbitless Solutions Project Xavier STUDY: Effects and Mechanisms of Differently Cued and Non-Cued Motor Imagery in People with Multiple Sclerosis: A Randomised Controlled Trial Oregon National Primate Research Center Give RealTalk MS a Rating & Review ___________ Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 67 Hosted By: Jon Strum Guest: Dr. Larry Sherman Tags: MS, MultipleSclerosis, MSsociety, ACA, MSActivist, Gilenya, Aubagio, Limbitless3D, RealTalkMS

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Episode 66: Embracing Carers with EMD Serono's Scott Williams

Play Episode Listen Later Nov 26, 2018 30:33

We're into the final few days of National Family Caregivers Month, and my guest on the podcast is Scott Williams, Vice President, Head of Global Patient Advocacy and Strategic Partnerships at EMD Serono. We're talking with Scott about EMD Serono's commitment to MS caregivers. We're also talking about creating a caregiver protocol that becomes part of the conversation in the neurologist's office as soon as someone is newly diagnosed with MS. We'll tell you about the FDA's new warning about stopping Gilenya. We'll share news about two different clinical trials that are exploring two different cell therapies for treating progressive MS. And we'll share some amazing statistics that demonstrate the reach and effectiveness of the National MS Society's MS Navigator program, a remarkable one-on-one MS support program. We have a lot to talk about! Are you ready for RealTalk MS?! ___________ Can We Talk About Establishing a Caregiver Protocol? 1:04 FDA Warns About Severe MS Worsening After Stopping Gilenya 4:54 Phase 2 Clinical Trial Planned for NurOwn Cell Therapy for Treating Progressive MS 6:30 ATA190 Cell Therapy Shows Promising Phase 1 Clinical Trial Results for Treating Progressive MS 7:43 MS Society's MS Navigator Program Demonstrates Reach & Effectiveness 10:36 Scott Williams, Vice-President, Head of Global Patient Advocacy and Strategic Partnerships Discusses EMD Serono's Commitment to MS Caregivers 14:08 ___________ LINKSIf your podcast app doesn’t allow you to click on these links, you’ll find them in the show notes in the RealTalk MS app or at www.RealTalkMS.com Download the RealTalk MS App for iOS Download the RealTalk MS App for Android FDA Warns About Severe Worsening of Multiple Sclerosis After Stopping the Medicine Gilenya BrainStorm Cell Therapeutics Announces Submission of IND for NurOwn in Progressive Multiple Sclerosis Epstein-Barr Virus-Specific T Cell Therapy for Progressive Multiple Sclerosis Ask An MS Navigator RealTalk MS Episode 19: MS Navigators -- A Seriously Remarkable Service That You Need to Know About DOCUMENTARY: Seeing MS From the Inside Out Embracing Carers Give RealTalk MS a Rating & Review ___________ Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 66 Hosted By: Jon Strum Guest: Scott Williams Tags: MS, MultipleSclerosis, MSsociety, Gilenya, ProgressiveMS, MSInsideOut, EmbracingCarers, Caregiving, RealTalkMS

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FirstWord Pharmaceutical News for Friday, July 13 2018

Play Episode Listen Later Jul 13, 2018 2:14

Today in FirstWord:

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Episode 32: MS Rehabilitation & Symptom Management

Play Episode Listen Later May 14, 2018 40:18

Living your best life while you're living with MS means learning how to manage and overcome the physical and psychological limitations that MS tries to impose. This week's guest, Dr. Kathleen Zackowski, Senior Director of Patient Management, Care, and Rehabilitation Research for the National MS Society, takes us on a deep dive into MS rehabilitation & symptom management. We're also talking about the FDA approval of Gilenya for treating Pediatric MS, President Donald Trump's announced "blueprint to lower (prescription) drug prices," and next week's International Progressive MS Alliance Scientific Congress on Rehabilitation & Symptom Management. And...we're announcing some very special podcast episodes coming your way next week!! We have a lot to talk about! Are you ready for RealTalk MS? ____________ The RealTalk MS Podcast & Alexa 1:35 FDA Approves Gilenya For Pediatric MS 3:13 President Trump Announces His Prescription Drug Strategy 4:30 MS Rehabilitation & Symptom Management 6:37 International Progressive MS Alliance Scientific Congress 9:35 Special Upcoming Podcast Episodes 10:32 Interview with Dr. Kathleen Zackowski 11:21 ___________ LINKSIf your podcast app doesn’t show these links, you’ll find them in the show notes at www.RealTalkMS.com The RealTalk MS Podcast Alexa Skill FDA Approves Gilenya for Pediatric MS The International Progressive MS Alliance Give RealTalk MS a Rating & Review ___________ Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 32 Guest: Dr. Kathleen Zackowski Hosted By: Jon Strum Tags: MS, multipleSclerosis, Alexa, Gilenya, MSRehabilitation, Rehabilitation, AllianceCongress2018, RealTalkMS

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Episode 30: Dr. Matthew Miles & MS Research Australia

Play Episode Listen Later Apr 30, 2018 41:29

We have a lot to talk about in this podcast episode! First, a big announcement...the RealTalk MS Podcast has its own Amazon Alexa Skill! If you already own an Amazon Echo, Dot, or any Alexa-enabled device, you can access all sorts of convenient features for listening to RealTalk MS! (And if you don't yet have an Alexa-enabled device, this might be one more reason to get one!) My guest on the podcast is Dr. Matthew Miles, the CEO of MS Research Australia, the largest non-profit organization dedicated to funding, coordinating, and advocating for MS research in Australia. We're talking with Matthew about some of the amazing research collaborations that MS Research Australia is driving in Australia and around the world. (NOTE: During our conversation, Dr. Miles references the MS Research Australia website as "www.msra.org". The correct website is www.msra.org.au. Sorry for the confusion!) We're also talking about the results of MS research studies that were announced at the just concluded American Academy of Neurology Annual Meeting. These are studies that you need to know about! ____________ The Alexa Announcement 2:22 Introducing Matthew Miles & MS Research Australia 5:48 Study Shows MS Progresses Faster in African-Americans 6:46 Gilenya Shown To Be Effective in Delaying MS Progression & Disease Activity in Pediatric MS 8:00 MS Disease-Modifying Drugs May Lose Effectiveness As You Age 9:46 Interview with Dr. Matthew Miles 11:20 ___________ LINKSIf your podcast app doesn’t show these links, you’ll find them in the show notes at www.RealTalkMS.com The RealTalk MS Podcast Alexa Skill STUDY: MS Progresses Faster in African-Americans STUDY: Gilenya Effective in Delaying MS Progression & Disease Activity in Pediatric MS Patients MS Disease-Modifying Therapies May Be Less Effective As You Age MS Research Australia Give RealTalk MS a Rating & Review ___________ Follow RealTalk MS on Twitter, @RealTalkMS_jon, and subscribe to our newsletter at our website, RealTalkMS.com. RealTalk MS Episode 30 Guest: Dr. Matthew Miles Hosted By: Jon Strum Tags: MS, multiple sclerosis, Alexa, Matthew Miles, MS Research Australia, AAN, Gilenya

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RealTalk MS Episode 15: We Are Illmatic

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later Jan 15, 2018 24:57

Jon's guest on the podcast is Victoria Reese, founder of the We Are Illmatic campaign. We Are Illmatic is building awareness and providing support for women of color who are living with MS in their lives. We have good news about the U.S. Defense Department's MS Research Program, and the passage of the bipartisan RAISE Caregivers Act. We'll tell you how an MS patient's handwriting may predict a decline in movement, sensory, and cognitive skills. And we'll share some interesting research about a hidden benefit associated with Gilenya. Are you ready for RealTalk MS?

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Multiple Sclerosis Discovery -- Episode 90 with Dr. Daniel Hartung

Play Episode Listen Later Jul 21, 2016 20:29

[intro music] Host – Dan Keller Hello, and welcome to Episode Ninety of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m Dan Keller. Welcome to the weird world of the U.S. pharmaceutical market. A few outrageous cases of drug price gouging have made the headlines, but in multiple sclerosis, a more serious concern is the steady annual rise in cost of all disease-modifying therapies, or DMTs. So says Dr. Daniel Hartung, a researcher at the Oregon State University/Oregon Health and Science University College of Pharmacy. In a recent study, he found that MS drug prices over time outpaced both inflation and similar biologics. It’s not just the new drugs. As each more expensive DMT comes to market, the prices of older drugs also race to catch up. It’s affecting the drugs available to patients and causing other concerns. Interviewer – Carol Morton Can you tell me what questions you were asking and why? Interviewee – Daniel Hartung Sure. So the study that we did had its origin after having some conversations with some neurologists at OHSU about increasing frequency of seeing their patients facing larger and larger, not only cost sharing and copays from the insurance companies for drugs for MS, but also increasing restrictions, typically from insurance companies in kind of what medications they were supposed to take first prior to perhaps failing one, then going to another medication for MS. And so this is all kind of happening in the context of what they were seeing as just higher prices for some of these medications. And so what we decided to do is…no one's really done this…is look at in a systematic way the trajectory of pricing for MS drugs, essentially since their approval until we went through the end of 2013. And to look at what the just general trend was, try to figure out if there were certain specific factors that were associated with higher prices over time, like the approval of newer agents, things like that. That was kind of the general objective of the study. MSDF And then how did you go about conducting this study? Is it hard to find that data? Dr. Hartung It can be. So I'm fortunate to have access to some data set that has longitudinal pricing data for pharmaceuticals for the past 30 years or so. And so from my perspective, it wasn't difficult. But essentially we used this data set that collected average wholesale price, as well as wholesale acquisition cost, so kind of the two usual, most common (I'll call them) sticker prices for drugs. And so this data set for all medications, it kind of tracked pricing of medications over time. And so that was the core data set for our analysis. MSDF And so you pulled the multiple sclerosis disease-modifying therapies out of that. How many did you look at? Dr. Hartung So in our study we looked at 11 medications for MS. They included the three what are typically called platform therapies that have been on the market for about 20 years now. Those include Avonex, Copaxone, and Betaseron, and just followed them through time, through the approval of several other new agents, like Tysabri. And then there's in the last five to six or seven years, the FDA has approved several agents that can be taken orally, Gilenya, Aubagio, and Tecfidera now. And there was a couple other kind of miscellaneous agents that were kind of variants of the interferons and things like that. MSDF And then what did you find? Dr. Hartung Well, there are several interesting things, but I think one of the most striking things is that the prices for the platform therapies, Avonex, Betaseron, and Copaxone, were pretty stable for at least 10 years from their approval in early to mid-90s. And then, essentially what we observed is that new agents that came on the market, starting with Rebif in about 2001, came out, and they were usually priced about 20% to 30% higher than the existing therapies. And what we observed is that when these new agents came out or approved, that these higher prices, the cost or the price of kind of the platform therapies quickly escalated to almost match the price of the newer agents that were approved. And this pattern kind of repeated itself and actually became more intense when the newer oral agents came on the market in the last five or six years. So the cumulative effect of that is in the early 2000s, Copaxone, Betaseron, and Avonex were priced about $10,000 to $15,000 a year. And at the end of our study, all of the agents that are currently approved were priced between $50,000 and $60,000 per year. And so we tried to quantify kind of the rate of increase and compare that with other kind of benchmarks: inflation, prescription drug inflation. What we found is that the price increase for those agents was well above what you'd expect for not only just general inflation, but also prescription drug inflation. MSDF MS drugs, the cost of all of them, not just the new ones, are increasing at a rate higher than any other drug category? Dr. Hartung In addition to looking at kind of standard metrics of inflation, we compared the price increases for the platform therapies to what we considered kind of comparable biologics. So we looked at a class of medications called tumor necrosis factor inhibitors, which are used for immunologic conditions like rheumatoid arthritis. And what we found is that the price increases for the platform therapies for MS increased substantially and significantly above price increases for those medications for the tumor necrosis factor inhibitor. So from our study, from our perspective, prices increased higher than they did for these TNF inhibitors. We haven't really compared them across other classes of drugs, but there are some new publications that have looked at price increases for other agents, such as in other classes like insulin, drugs for diabetes, and cancer agents as well. The numbers are slightly different, but the trajectories look pretty similar. So in the last, you know, 10 years, there's been almost it seems like a logarithmic increase in the price of many of these agents and classes. MSDF So is this a case of a system that has incentives that maybe aren't as well matched to patient needs as they should? What's going on here? Dr. Hartung I mean, that's a good question. Definitely there's a system. The market-based system for pharmaceuticals in the United States is incredibly dysfunctional in that it's very dissimilar from any other kind of consumer market for technology, phones, cars, things like that, where you typically see prices go down after a while. And you don't see that in health care or in drugs. You see just prices increase. And so there's a dysfunction that just kind of is core to the economics of health care. And then I think there is an element of pharmaceutical industries pricing these agents essentially what the market will bear. Now my opinion is that a lot of the aggressive increases in price were initially seen with some of the cancer agents. And so I think that in that field there is a kind of pushing of the envelope for many anti-cancer drugs that's now has proliferated to other classes of drugs, including MS agents. The other element that's kind of unclear and adds to the murkiness to this is that, you know, our study and other studies that have looked at what I'm calling pricing of the agents use average wholesale or WAC and with some sort of adjustments for rebates or discounts. So typically third party payers or pharmaceutical benefits managers will negotiate with pharmaceutical industry to lower the cost of the agent for the payer. But all that information is typically proprietary, and so it's really difficult to know what the actual cost of the medication is, unless you're paying cash. If you're paying cash, then the cost is going to be pretty close to the price that's set. So people who don't have insurance are paying the most, and the people with insurance, Medicaid, any sort of governmental insurance, they're paying typically AWP minus a certain proportion or WAC plus a proportion percentage essentially based on the rebate that they get. So that adds a little bit of kind of uncertainty. Pharmaceutical industry may come back to say that, you know, we're giving pretty good discounts on certain medications in certain payers, but from the data we have and the pricing data, there's just been this aggressive increasing in prices. And we don't know if it's being mitigated by increasing rebates and discounts over time. So it's complicated. MSDF What do you hope people will do with this information? It does sound like a complicated system that's almost unapproachable for the individual patient or individual doctor. What can people start doing now? Where does the responsibility or responsibilities lie? Dr. Hartung You know, I think that the data we generated in our study has been useful for some of the advocacy groups in the multiple sclerosis community. So the National Multiple Sclerosis Society has been using it to try to, you know, advocate or perhaps political reforms or some other meaningful reforms in kind of how these things are reimbursed, things like that. Drug prices has been in the news quite a bit over the last several years, and now even more with the election season in full tilt. And so I think a lot of the candidates are talking about potential solutions to the issue. From the patient's perspective, they're in a real quandary in a sense that even a sharp move with the Affordable Care Act to a lot of high deductible, high cost sharing plans where if your monthly cost of a MS agent is $5,000, you pay 20% of it until you hit your deductible. You know, that's $1,000 at the pharmacy, and that's a pretty big out-of-pocket cost that you face. So I think that there's some, you know, movement in the advocacy groups to try to…especially working with insurance companies to make sure that access is open because these medications are incredibly individualized. And there's not really good predictors of who will respond to each type of medication, and they're all different. Some of them are administered subcutaneously, intramuscularly, orals, and so there's some patient preferences that fall into play here as well as the price. And so I think there's been some movement and some discussion making sure that access to all the agents is relatively easy for patients. But from a solutions to the pricing situation, you know, I think we're still kind of in discussion phases about what we can do as a country to kind of deal with this issue because it's not exclusive to the MS drugs. MSDF So what's next with you? Are you following up on this? Dr. Hartung So from our perspective, the group that I worked with, the two neurologists' project, we just submitted a grant, well, it was in January, that we hope to be competitive and hope to get that's looking at how these high drug prices actually affect patients in terms of their medication taking and potentially adverse outcomes because they're not taking their medication. Either they're hitting access restrictions from insurance companies or they just can't afford or have problems with the cost sharing or something like that, and so trying to quantify how this is affecting patients. And so from a research perspective, I think that's kind of our next move. My colleagues, my two neurologist colleagues, they're really active in kind of speaking with representatives at the state about the issue, bringing it to increased visibility from our elected officials as well as making sure that the MS Society is aware of kind of the current status of the pricing trajectory. So we've been updating our graph that we published as new agents come online and things like that. MSDF Can you give us a couple of the updates you've made since the study? Dr. Hartung They haven't been dramatic, but there's been a couple new agents that have been approved. And I guess most notably is that the first generic drug for MS was approved, I believe, last April. So a generic for Copaxone came online. I think there's two manufacturers of it. When it came online, there was one. And so I think it was priced just modestly lower than the brand name Copaxone. But something interesting also just dealing with Copaxone, which is the number one MS drug in terms of sales, so when Copaxone lost its patents and lost its kind of patent disputes, in preparation for that, Teva released a different formulation of Copaxone. So Copaxone is traditionally a daily injection. And so they released a three-times-a-week higher strength injection and basically switched everyone from the once-a-day to the three-times-a week 40-mg injection. And so I think a large proportion of patients who were originally on the once-daily Copaxone were switched to the 40-mg three-times-a-week Copaxone. So that really to some extent mitigated if there's any sort of savings due to this new generics in the field, kind of really mitigated any kind of savings due to the new generic as most people are now on the 40-mg three-times-a-week product. And the generic is not substitutable for the 40-mg three-times-a-week product. So that's a very common tactic in pharmaceutical industry approach to try to like sustain their franchise with a particular drug that's going off patent. But the big questions are the ones that don't have a good answer. Essentially, what do patients do about this? What do we do as a society to deal with this issue? And you know, there's been proposals that have been put out by different elected officials and other folks about, you know, we should allow Medicare to aggressively and directly negotiate with pharmaceutical industry on price. We should allow importation of medications from other countries, similar industrialized countries like Canada. So the United States pays by far and away the highest prices than any other country in the world. And so many people think that we should be able to import these drugs that are the same drugs that are going to Canada into the United States. You know, some people suggest that there should be some sort of forms of price control. You know, maybe medications shouldn't be allowed to increase 10% a year or something like that. And so all of these are being kind of discussed and played out and the pros and cons are weighed. And whenever you talk about limiting price increases, the usual response you get from industry is that any constraint on the amount of money that they're able to make and the profits that they're able to make for their shareholders is going to have some sort of effect on kind of future innovation potentially. Whether that comes to bear or not is unclear, but that's usually the number one response you get is that we need to have these high profits in place because it's an incredibly risky endeavor that we're doing. Only a very small proportion of drugs that are under development actually make it through the developmental process and are approved and make it to market. So any constraint on profits is going to have an effect in terms of future innovations and future breakthrough medications and things like that. Incentives are a big…they are real. And so that is something that needs to be weighed carefully in kind of any solution, essentially. I don't think it's the best solution, but just people are talking about a wide variety of things, I think. MSDF I appreciate your raising all these issues and going through the study. Is there anything else that I haven't asked that you wanted to add or emphasize as take-home lessons? Something to mitigate the rage, I don't know… [laughter]? Dr. Hartung Yeah, well I mean there's been a lot with all this, you know, the Valeant Pharmaceutical issue and the other company, Martin Shkreli guy who's castigated for increasing the price of this drug for toxoplasmosis by like 5,000% and buying the company and jacking up the price. That's a separate phenomenon of what is happening. But I think the outrage over that type of exploitation of the dysfunctional pharmaceutical market kind of masks and kind of hides the other issues that are happening on a consistent and aggressive basis in terms of just regular 6% to 10% increases in price on a year-to-year basis for drugs that a lot of people use, like drugs for diabetes or MS products, cancer agents, things like that. And so, you know, you have these really highly visible cases of really dramatic increases that are kind of morally outrageous. They draw your attention from the real and kind of moderate but aggressive and year in, year out, increases that are seen across the board in a lot of different agents. And that's where our focus should be essentially. MSDF That's helpful. Well, thank you so much. Dr. Hartung Yeah. My pleasure. [transition music] MSDF Thank you for listening to Episode Ninety of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. MSDF’s executive editor is Carol Cruzan Morton. Msdiscovery.org is part of the nonprofit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is Vice President of Scientific Operations. Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances. [outro music] We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org. For Multiple Sclerosis Discovery, I'm Dan Keller.

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Multiple Sclerosis Discovery -- Episode 84 with Dr. Ilya Kister

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later May 20, 2016 15:45

[intro music] Host – Dan Keller Hello, and welcome to Episode Eighty-four of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m Dan Keller. People with MS take disease modifying therapies, or DMTs, for years. But is it possible to stop the drugs at some point or at least take a drug holiday? I spoke last fall at the ECTRIMS meeting in Barcelona with Dr. Ilya Kister, an assistant professor in the MS Care Center at the New York University School of Medicine. He has looked at various studies and registries that shed light on the question, and he discusses the utility and limitations of using observational data from big data sets. Interviewer – Dan Keller People know a lot about starting DMTs, but not about stopping. And, I take it, there's not much been looked at yet in terms of could you stop and what happens. Interviewee – Ilya Kister Yes, that’s a question that patients often ask, and clinicians certainly wonder about. Is it safe to stop the drug? When is it safe to stop it? And all the literature that I’ve seen on stopping the DMTs has basically analyzed the reasons for stopping them. The reasons for non-adherence—why did patient not want to continue—but there is very little data on actually what happened in terms of disease course. It’s just an observational study, you know. Do those patients continue to have relapses? Do they have more relapses or less? The only exception is natalizumab, where we have, you know, more than a dozen—probably two dozen—articles looking at what happens when you stop the drug. But that’s a little kind of almost an exceptional circumstance. There is a question of disease rebound and such. With the other drugs, very little to no data. So, so one wonders whether it’s an okay thing to do. MSDF What are the pros versus cons of stopping? Dr. Kister I think you can make almost equally appealing arguments on both sides. The arguments to continue the drugs, the main ones, are that relapses are unpredictable, and even though they’re less common as people age, we do see patients in practice, even in their 60s, who have relapses. And there was a recent study that showed that about 30% of secondary progressive MS patients have relapses. So, presumably, the drugs which work to decrease the risk of relapse would be helpful to reduce the risk of relapse even in those circumstances as well. But that’s not entirely clear, because they were never shown to be beneficial, truly, in the secondary progressive patients or in the older patients, because older patients are, by and large, excluded from all the studies. So we really don’t have any high-level data on these subpopulations. So the reasons to continue would be to try to prevent relapses, even in older patients. And the reasons to stop would be that the relapses are kind of few and far between. It may be not worth the hassle, and maybe the disadvantages of continuing in DMT long-term outweigh the theoretical risk of decreasing relapse rates. So it’s in a clinical equipoise situation, as far as I am concerned. MSDF How have you looked at this issue? Dr. Kister This is just kind of our individual practice, and many people may agree or not agree with it. This is not really based on our studies, but generally speaking, patients after age 60 who haven’t had relapses or MRI activity for at least five years, I do have a discussion with them and kind of feel them out whether they’re interested in stopping or not. And the reactions vary widely. You know, some people are very attached to their drug. They feel like it’s helping them and protecting them and has done good for them, and they don’t even want to think about stopping. And some people are very tired from being treated for many years. They don’t necessarily see the advantages of it, and they’re very willing to consider stopping and take you up on the offer. They just need a blessing to do this, because the doctor says to stop. You know, there are people in between who are kind of vacillating and not sure. But this is a population that I would consider stopping the drug. But now, about two weeks ago, we received the news that we have funding for study, wherein we’ll randomize patients. Some will continue on whatever drug they were on, and some will stop. And then this way we’ll actually collect, in a more rigorous fashion, the data of actually what happens to those patients. And that’s a study where the primary investigator is Dr. John Corboy from the University of Colorado in Denver. And there are six sites across the states that were approved for this, and where NYU is one of the six sites, and maybe a few more sites will be added. So this is our best hope, I think, to conduct, not a randomized clinical trial of starting a drug, but a randomized clinical trial of stopping a drug, which has been done in other fields, most important in oncology, a little bit in psychology, but not in neurology or in MS, as far as I know. MSDF But short of that, you've done a database study and looked at people who have stopped? Dr. Kister Yes, though that was a study that was just presented at this ECTRIMS meeting. And there we used a very large international registry called the MS Base, which has over 30,000 patients enrolled in it, so, and dozens of countries. And it's open to any investigator in the world who is interested, and he can contribute patient data. Obviously, it's patient consent, and many patients are interested in contributing their data to the registry. So because the registry is so large, we were able to include for this study almost 500 patients who met our criteria, which were fairly rigorous. We required that patients be on some drug for three years; have no relapses for at least five years, because we want to exclude active patients; and be followed for at least three years. Three years is more than most clinical trials, which are one to two years. But we really wanted to see what happened to them this time. And we excluded people who went from one DMT to another within three months. So this was the crux of this study. We looked at this—485 patients to be exact—and we followed them. And the minimum was three years, but the median was almost five years. And we found that in this population during this followup of almost five years, 36% of patients had at least one relapse. And 31% of patients had a confirmed disability progression, meaning three months apart they had a worsening of EDSS. And almost half of the patients have restarted a DMT, but not right after stopping, but two years or more after. That was the average time to restart. So that was the main kind of result. So when you talk to the patient, you try to kind of lay out the data for them, you know, this is the numbers you can use, I think. Even though somebody hasn't had relapses for five years or more, they still are at risk of relapses. And what we found was a predictor of relapses was age and EDSS. The younger patient and less disabled patients who we think are typically probably more in the relapsing phase, rather than in the secondary progressive phase, were more at risk for relapses. So for younger patients, I would be much more wary of stopping the drug, even if they have been relapse-free for years, than in an older patient. So that's one result of the study. But there was a second component of this study which was interesting, I thought, wherein we compared the people who stopped the drug with the people who continued on the drug, and we matched them. There is a technique called propensity score matching. So we matched the people who stopped and the people who stayed. And the two groups were almost identical. All the parameters, like age, disability, how long they've been on the drugs, proportion of times they've been on the drug, their gender—very, very similar according to most of the variables. And we followed them through time, and the mean followup for both groups was about five years. And we found, a little bit counterintuitively, that people who stopped the drugs did not have any more relapses than people who did not stop the drug. If you think that the drugs are protective, you will expect some effect; we didn't see any effect whatsoever. There was absolutely no effect. But interestingly enough, the people who stayed on the drug tended to progress, to show confirmed disability progression, a little less. They were at less risk of disability progression, about 40% compared to people who stopped. So it's a little hard to interpret this data. It may be that the drugs actually have some cumulative effect and maybe continue, and that does delay disability progression. That would be a very favorable interpretation as far as clinicians are concerned and the rational to continue. But it may be that people who stayed on the drug were really in some what we call unmeasured confounders. They had some reasons why they stayed, and they are not really entirely comparable to the people who stopped. Maybe they were a little more, for whatever reasons, considered to be more active by the clinician, and that's why they kept them on the drugs. So maybe there're intrinsically different groups with intrinsically different disability progression, and that is the reason for the finding. So this is where we stand right now, and this goes to show kind of the utility and the limitations of using observational data sets. The utility is that we're able to basically run that kind of a pseudo-trial, if you will, comparing the stoppers and stayers, and run it for many years. We actually have data six, seven years after stopping the drug, which is almost not possible with randomized clinical trials. And we're able to use this data. In fact, to power the clinical trial that I talked about earlier, because we can predict how many people are expected to have relapses at this age and such. And the limitation that there are known unmeasured confounders, and that there're biases in who continues to be observed and who is not, and we cannot control for that without randomization. MSDF Now, from your study, it looked like people who had been off of a DMT for more than two years had a higher relapse rate. Is there any possibility of having a drug holiday? Or, when someone comes off drug, a silent insult happens that you only see later, so you really have to not give them a holiday? Dr. Kister Well, it's a hard question to answer. They had a higher risk of disability progression, not relapses in this study. The curves begin to diverge after about two years. It was more of a long-term effect. So, you know, one wonders. But the counter argument to what you are describing is maybe there's a cumulative effect, that you really have to stay on the drug for long periods of time in order to see. And if you stop and have a holiday, you kind of wash out that possibility. So the answer is, we really don't know whether it's okay or not to give holidays. It's definitely not okay in actively relapsing patients, especially if they're on strong drugs like natalizumab or even Gilenya or even interferon. That's pretty clear. So but as far as the patients who hadn't had relapses for a long period of time, we don't know. It remains to be seen. MSDF Is there a continuing effect of any drugs, such as monoclonals, like alemtuzumab, where you might get a tail effect even after stopping it, which would essentially be your accumulative effect? Dr. Kister I think that is, you know, a very important point that we talked about stopping the drugs, but we really have to specify which drug we're stopping. Because drugs like alemtuzumab have been shown to have an effect that lasts for four years or more. And I think at this conference they will show data for even longer term effect of alemtuzumab. I've seen some posters to that effect. So those drugs have an effect on the immune system that persists. Some chemo treatments as well, you know, a stem-cell transfer. It's not something you do every year; it's something you've done once, and you see the effect that lasts for a long period of time. So I think a lot depends on the mechanisms of the drug, you know, how long they're expected to affect the immune system for. Something like natalizumab that washes out within three months or so, and you don't really see, you know, effect on the receptor level than you'll be after about three or four months. We don't really…you wouldn't expect it to work beyond that time, and it really doesn't. It only lasts that long. And other drugs, there is a sustained loss of T cells and B cells for a long period of time, and perhaps that's why there's a clinical effect that lasts for many years. MSDF Have we missed anything? Or is there anything important or interesting to add on the topic? Dr. Kister I think your interest was in observational data sets, and I think MS Base registry and others, like NARCOMS registry, they show the power of, kind of all of the people power. It's not the big pharma who is collecting the data, which is very important and has a big role, obviously. It's actual clinicians and actual patients who volunteer their data. And I think patients should be gratified to see that their data is used to actually come up with some insight as to advantage them, come back to the patients and answer some of the questions they had. So I think those databases are very important. MSDF I appreciate it. Thank you. Dr. Kister Thank you very much. [transition music] MSDF Thank you for listening to Episode Eighty-four of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. MSDF’s executive editor is Carol Cruzan Morton. Msdiscovery.org is part of the nonprofit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is Vice President of Scientific Operations. Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances. [outro music] We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org. For Multiple Sclerosis Discovery, I'm Dan Keller.

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Multiple Sclerosis Discovery -- Episode 51 with Dr. Luke Lairson

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later Aug 21, 2015 16:31

[intro music] Host – Dan Keller Hello, and welcome to Episode Fifty-One of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m your host, Dan Keller. This week’s podcast features Dr. Luke Lairson of Scripps Research Institute, who discusses discovery of small molecules to induce remyelination and, in particular, some muscarinic receptor antagonists currently approved for other indications. But first, here are some new items in the MS Discovery Forum. According to our curated list of the latest scientific articles related to MS, 114 such articles were published in the first two weeks of August. We selected a few of these articles as our Editor’s Picks. One is a longitudinal study of gray matter lesions and cortical atrophy in MS published in PLOS ONE. The investigators obtained MRIs at baseline and five years later from subjects with clinically isolated syndrome, early and late relapsing-remitting MS, and secondary progressive MS, and examined lesion placement and cortical thinning in the different disease subtypes. To see this publication and the other articles we selected, go to msdiscovery.org and click on Papers. Our Drug-Development Pipeline includes continually updated information on 44 investigational agents for MS. During the past week, we added 1 new trial, we updated information on 2 other trials, and we added 12 other pieces of information. The drugs with important additions and changes are ATX-MS-1467, daclizumab, dimethyl fumarate, fingolimod, glatiramer acetate, interferon beta-1a, and interferon beta-1b. To find information on all 44 compounds, visit msdiscovery.org and click first on Research Resources and then on Drug-Development Pipeline. [transition music] And now to the interview. Dr. Luke Lairson is an assistant professor at the Scripps Research Institute and a principal investigator at Calibr, the California Institute for Biomedical Research, in La Jolla. We spoke at the research institute. Interviewer – Dan Keller Dr. Lairson, we're talking about the potential for remyelination. And your institution is taking a very systematic and maybe novel approach. Can you describe how you're going about looking at possible ways to induce remyelination? Interviewee – Luke Lairson Sure. So we're using a phenotypic assays to look for small molecules that selectively induce the differentiation of the precursor cell population which is required for remyelination, which are the so called oligodendrocyte precursor cells or OPCs. We developed imaging-based assays where we could look for small molecules that selectively induce that differentiation phenotype. MSDF And how are you going about screening compounds, and what is your institute set up to do? Dr. Lairson So at Scripps and Calibr we have the capacity to screen on a million compound scale capacity to look at molecules. In this particular assay, we do it in a 3D four-well format, which limits us to screening collections on the scale of hundreds of thousands. And to date, we've screened about 200,000 compounds with this assay. MSDF In terms of multiple sclerosis, what are you looking at now? Dr. Lairson From our preliminary screen of our collection of bioactive compounds, including FDA-approved drugs and drugs in late-stage clinical development, we identified a series of compounds for which OPC differentiation had not been previously reported, which are muscarinic receptor antagonists, which are clinically approved drugs and which work in the central nervous system. And we demonstrated a number of these compounds work in two different rodent models of remyelination in MS. And we're currently developing these as a lead class of compounds as a combination therapy when combined with existing immunosuppressant drugs, including Gilenya. MSDF What compounds have you focused on most? There's a multitude of approved antimuscarinic agents. Dr. Lairson Right. So this is actually a a critical point. So we identified a number of compounds which had antimuscarinic activity, which were active in our OPC differentiation assay. We used pharmacology to demonstrate that the antagonism of M1 or M3 receptor subtype is a required component of the mechanism of these drugs. However, we think that there's a second target, and it's a dual mechanism action through which these drugs are acting, which we're currently trying to elucidate what that second target is to fully characterize the mechanism. That second target actually provides the opportunity to identify compounds that have a potential to have a better therapeutic index in vivo. So the lead compound we published was benztropine. Jonah Chan's lab at UCSF later showed that clemastine also works, which was in our paper, as well. So these are drugs that have been demonstrated to work in vivo. What we did after we published that is we then looked at every compound that we could get our hands on that had antimuscarinic activity – specifically targeting M1/M3 receptor subtypes – and then characterized their activity in the OPC differentiation assay and, as well as profiling their potency on the M1/M3 receptor subtypes, with the goal of looking for compounds that have an optimal therapeutic index in terms of on-target toxicity. So benztropine induces OPC differentiation in the low micromolar range, but it antagonizes M1 and M3 receptors in the low nanomolar range so there's a discrepancy there. And we think that the on-target toxicity of antimuscarinic activity is going to limit the therapeutic potential of these drugs. We've since identified other FDA-approved drugs for which that index is improved and we compounds with approximately 100-fold improvement in therapeutic index, which we've demonstrated in the EAE model are active. And we're currently evaluating them in combination with immunosuppressant drugs to identify an optimal combination, which could well be benztropine or clemastine but may be another FDA-approved drug. MSDF You're at very early stage in terms of clinical utility of these things in MS. But is there any way to separate out the negative antimuscarinic effects that affect people taking drugs for overactive bladder and various other things from their therapeutic effect? Or is it really intrinsic to attacking that receptor? Dr. Lairson That's the key point. So we do think that it's that on-target toxicity which is going to potentially limit this class of compound, which is why we're looking for these other compounds and where we have an improved therapeutic index of inducing remyelination versus antagonizing those receptor subtypes. And likely this class of drugs – and any class of drugs that induces remyelination – is going to have to be used in combination with immunosuppressant drugs. It will require a careful clinical evaluation to figure out which combination will be the most effective and what doses will be safe. MSDF But you also have been doing T-cell assays I take it in looking at benztropine in your work. So what goes on with immune modulation? Is there any effect there? Dr. Lairson So we did extensive studies with benztropine to evaluate its activity in not just T-cell biology but also macrophage biology both in vitro and in vivo. And we found benztropine had no affect on in vitro or in vivo T cell numbers or activity in terms of cytokine production. It has no affect on macrophage polarization in vitro or in vivo, including looking at spinal cords of animals. We don't think that it's acting through a peripheral immune system effect. We can't rule out an important concept that came out at a recent meeting was we need to look at the affect of these compounds on microglial cell activation in the brain and also in astrocyte activation. MSDF So it looks like it's a pure remyelination effect at this point and not really an immunosuppressive effect, which would argue for having to use it in conjunction with today's drugs for MS. Dr. Lairson Correct, that's our current reasoning, yeah. MSDF Have you looked at other models other than cuprizone? Dr. Lairson Yeah, we looked at the EAE…PLP-induced EAE model of relapsing-remitting MS, and we've looked at the MOG model of progressive MS and the cuprizone model. Yeah, those are the models we've looked at to date. MSDF With similar results? Dr. Lairson The compounds we've evaluated have all been active in in those models. MSDF Do you have an idea of the mechanism of action how this is actually working in the oligodendrocyte precursor cells? Dr. Lairson Downstream of the muscarinic receptor…so as I said, based on pharmacology, these classes of compounds – these neurotransmitter receptor modulating agents – are notoriously pleiotropic in that they had multiple receptor subtypes in the brain. So benztropine, for example, it hits nicotine and histamine receptors in this dopamine reuptake inhibitor in addition to being an antimuscarinic. We've shown that those activities are not responsible for inducing OPC differentiation. However, as I said, we've identified multiple compounds that do inhibit muscarinic receptors – specifically receptor subtypes 1 and 3 – that do not induce OPC differentiation. So we think there's a second target; we're actively trying to identify what that second target and downstream mechanism is. MSDF Do you think the same compound would attack both targets, or are you going to need to give multiple compounds to hit multiple targets very selectively as I would think would be the hope? Dr. Lairson The existing compounds we have the argument is that they are hitting both of these targets to induce the differentiation. In that, there's a number of compounds that do hit the M1/M3 receptors that do not induce differentiation, which argue that you need both. The compounds we've identified fortuitously hit both of the necessary targets. MSDF In the antimuscarinic field, often the goal is to be very selective and limit activity at different receptors, but it sounds like you want some overlap here. Dr. Lairson Exactly. We've also initiated some medicinal chemistry where we're trying to see if we can dial in potency for the second target. So we know if benztropine is active on muscarinic and low nanomolar can we improve its potency in the OPC assay by dialing in potency on that second target? MSDF By modifying the molecule? Dr. Lairson Yeah, so making analogs of existing active antimuscarinic agents and then evaluating their activity in the OPC differentiation assay, as well as evaluate their antimuscarinic activity. MSDF Are you hoping that the same active part of the molecule hits both receptors, or have you ever considered making a bifunctional molecule that would be best at both receptors? Dr. Lairson If we knew what the other receptor was, we could potentially address that, or you could argue a bifunctional versus having two unique compounds so it would be you'd have to evaluate that in vivo I think, yeah. The other argument for moving away from this is that as soon as you make a change to that compound it's no longer an approved drug, and you have to go through the rigor of bringing that to the clinic. MSDF Now in your screening, you're using a lot of drugs – a lot of compounds at this point – that have already passed phase 1 screening or phase 1 clinical testing, and this has shown safety. Does that speed up do you think the approval process if these things look active? Dr. Lairson It does. So UCSF has actually initiated a phase 2 trial to evaluate clemastine in MS. So they were able to immediately proceed from a screening result to a clinical trial because it's an approved drug. MSDF And just about Calibr, your institute. You have full facilities for taking this from screening up to what stage? Dr. Lairson Up to the rodent proof of concept stage. So we have a high throughput screening facility, as I mentioned, and then we have a medicinal chemistry group, a pharmacology group where we can do pharmacokinetics in-house and then in core biology. So we take it to the rodent proof of concept. MSDF Do you do any synthetic chemistry, or this is all screening of existing molecules? Dr. Lairson Yeah, we do significant amount of synthetic chemistry so we have a group of 20 chemists – medicinal chemists – that are making analogs. And we do a significant amount of contract research to get compounds and analogs made. MSDF What have we missed, or what do you think is important to add, if anything? Dr. Lairson The other aspect of our program is we've identified novel compounds for which their mechanism of action is unclear. So we've identified multiple scaffolds. We've focused on three of those, which have been subjected to medicinal chemistry optimization. So we identified screening hits, which we were unable to evaluate in the rodent models due to their pharmacokinetic property. But we've now identified analogs of those compounds which are potent in the OPC assay, in which we can achieve reasonable levels in the brain. So we're currently evaluating those in these preclinical rodent models. And once we demonstrate efficacy there, we'll then go and evaluate their mechanism of action. MSDF You test these compounds first in a phenotypic sense to see if they actually do something that you want done. And then you trace it back to mechanism of action? Dr. Lairson Correct. That's our general approach for a lot of assays. So rather than looking at a validated, biochemical target, typically we'll just look for small molecules that induce the cell fate decision that we're interested in. For me, personally, it's the most interesting part of the project is I’m figuring out how those compounds that we rule out known mechanisms how they're actually acting. So we do the mass spec-based proteomics to figure out the specific protein target. And then we use standard cellular molecular biology techniques to elucidate the downstream mechanism of action. MSDF So I suppose the phenotype you're looking for in the case of this research we've been discussing is remyelination. How do you look for that activity? Dr. Lairson The remyelination activity in vitro? We use a co-culture assay, which we collaborate with Rusty Gage at Salk where we pre-differentiate neurons in a dish and then co-culture with our oligodendrocyte precursors plus or minus drug and then look at the ability of those drugs to enhance the rate of myelination of co-cultured axons. MSDF And you just stain the cells in vitro looking for myelin production or myelin basic protein? Dr. Lairson Exactly, yeah. So we just look at myelin basic protein co-localization with axon. MSDF Very good. I appreciate it, thanks. Dr. Lairson Absolutely. Thanks very much for your interest. [transition music] MSDF Thank you for listening to Episode Fifty-One of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. Msdiscovery.org is part of the non-profit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is vice president of scientific operations. Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances. We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org. [outro music]

ceo president ms 3d discovery fda papers multiple sclerosis m1 california institute ucsf m3 la jolla mris plos one mog scripps opc salk biomedical research scripps research institute eae research resources dan keller rusty gage calibr gilenya accelerated cure project msdf

Multiple Sclerosis Discovery -- Episode 49 with Dr. Hugh Rosen

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later Jul 21, 2015 22:38

[intro music] Host – Dan Keller Hello, and welcome to Episode Forty-nine of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m your host, Dan Keller. This week’s podcast features Dr. Hugh Rosen of the Scripps Research Institute. But first here are some new items in the MS Discovery Forum. If you’re an MS researcher, you may want to keep an eye on our Bulletin Board section, where we post a variety of news items that may be of interest. One of the items we posted this week is directly related to Dr. Rosen’s work. It’s a notice that a phase 3 trial of a sphingosine 1-phosphate receptor modulator called RPC1063 has started recruiting twelve hundred patients with relapsing remitting MS in the US. RPC1063 had its origins in Dr. Rosen’s lab. We also recently added a notice of another clinical trial to the Bulletin Board. That one’s a phase 2 trial of oral laquinimod in primary progressive MS. And a third new Bulletin Board announcement is a request for information from the Patient Centered Outcomes Research Institute to identify patient registries and research groups with established cohorts of patients for potential collaborative research opportunities on comparative effectiveness research in MS treatment. To read any of these announcements, go to msdiscovery.org and click first on Professional Resources and then on Bulletin Board. And if you have an announcement you think may be of interest to MS researchers, please send it to editor@msdiscovery.org. We won’t post purely promotional press releases, but if we judge the notice to be of general interest, we’ll be happy to post it at no charge. In other news, it was a relatively slow week in published MS research. According to our curated list of the latest scientific articles related to MS, only 22 such articles were published last week. Typically at least 40 MS-related peer-reviewed articles are published weekly, and we’ve seen some weeks with more than a hundred. To see the weekly lists going back to March 2012, go to msdiscovery.org and click on Papers. Our Drug-Development Pipeline includes continually updated information on 44 investigational agents for MS. This past week we added 2 new trials and 7 other pieces of information. The drugs with important additions are dalfampridine, fingolimod, masitinib, and natalizumab. To find information on all 44 compounds, visit msdiscovery.org and click first on Research Resources and then on Drug-Development Pipeline [transition music] Now to the interview. Dr. Hugh Rosen studies chemical and biological approaches to the molecular mechanisms regulating lymphocyte trafficking. I met with him in his office at the Scripps Research Institute in La Jolla, California. Interviewer – Dan Keller We're talking about mostly new compounds, S1P1 receptor compounds; the prototype now I suppose is fingolimod. What's in development and do they appear to offer advantages? Interviewee – Hugh Rosen So, firstly, let me disclose that I am a cofounder of a biotechnology company called Receptos that has licensed an S1PR1 agonist from the Scripps Research Institute, so I have and my institution have a significant interest in this particular field. Sphingosine 1-phosphate receptors act in a number of ways to modulate immune tissue damages in both autoimmune diseases and in viral infections. They've proven to be particularly efficacious in multiple sclerosis. Gilenya, of course discovered by Yoshitomi in Japan and developed by Novartis, has proven to be a clinically useful compound in the treatment of relapsing-remitting multiple sclerosis. And it appears to do so, at least in part, by altering the ability of lymphocytes to recirculate, and thus lymphocytes to reach the target tissues where they, in fact, produce demyelinating damage to the white matter of the central nervous system, and then the signs and symptoms of multiple sclerosis. So clearly these are useful compounds. Gilenya, of course, is not a selective small molecule, it is an agonist of four of the five high affinity receptors for sphingosine 1-phosphate – S1P1, 3, 4, and 5 – and some of the associated side effects may be attributable in part to activity of Gilenya on other receptors like the S1P3 receptor that are not required for modulation in the treatment of multiple sclerosis. MSDF I see that it's referred to as an immunomodulator, not necessarily referred to as a receptor agonist. Does it not have pure agonist effect? Does it have any effects either because of the other receptors or at that same S1P1 receptor? Dr. Rosen No. In fact, Gilenya when phosphorylated is a full agonist of the sphingosine 1-phosphate receptors, and the newer compounds that are much more selective are also agonists of the sphingosine 1-phosphate 1 receptor. And some of the effects on them for cyto-mediated by downmodulation of the receptor, but I don't use the term modulators or immunomodulators because of the activity on the sphingolipid receptors per se, I use the term immunomodulator because of some of the unique advantages that we've demonstrated in model systems and in man about altering the activity of the sphingosine 1 receptor, because one of the beauties of immunomodulation is to blunt the immune response that causes collateral damage to the tissues whilst leaving sufficient of the immune response intact to allow protection from opportunistic pathogens – bacteria, viruses, and yeasts. So one of the most striking features that we found – and these have been in some experiments done as a collaboration between my laboratory and the laboratory of Professor Michael Oldstone here at Scripps – has been in the area of influenza; pandemic influenza causes significant collateral tissue damage by having an overactive immune response. What we show is that the sphingosine 1-phosphate 1 receptor blunts that immune response and blunts the amplification of cytokines and chemokines so that you protect from the collateral tissue damage, but you leave intact the ability to mount protective, sterilizing T cell and B cell immunity to the virus. So you can eradicate the virus, sterilize it, you can provide a long-term memory both on the T-lymphocyte side as well as on the antibody side; there's class switching, there's affinity maturation, there are good protective immunity that is produced, and all this while blunting the immune response. This is the Holy Grail as we think about treating patients, because the window for patients with autoimmune diseases like multiple sclerosis is that window between effective blunting of the immune response and the prevention of deleterious opportunistic infections that can have life-threatening consequences. So one of the advantages that I suspect we will see over time is that the sphingosine 1-phosphate agonists will prove to be particularly well-tolerated and have a wide window between the ability to limit tissue damage and progression of RRMS, and the need to protect patients from intercurrent infections or subclinical infections that become expressed later. MSDF Do the other sphingosine 1-phosphate receptors interfere with lymphocyte trafficking also, or do they have other effects which nonselective ligands would then induce these adverse effects through them, or do they also have some effect in terms of trafficking? Dr. Rosen They don't have significant effects on lymphocyte trafficking the way that S1PR1 does, both from the chemical approaches and the genetic evidence. S1P1 is clearly a toggle switch for lymphocyte trafficking. S1P2 is involved in the maintenance of hearing and in the function of vascular smooth muscle, so it regulates blood pressure. S1P3 is involved in cardiac contractility and also in the control of coronary artery caliber and the control of the airways, so S1P3 agonism is not a useful thing, it's actually quite deleterious. S1P4 and 5 have really no rate-limiting functions, at least of which I am aware, so there may be some redundancy and may not play a critical role in the modulation of health and disease. MSDF Do you see compounds coming along which will be more selective and therefore not lead to the adverse effects so much? And if so, are these compounds chemically similar or do they have different structures to attach to the receptor, the S1P1? Dr. Rosen These are clearly different structures, they're structurally very distinct from Gilenya and from each other. Novartis have a backup called siponimod. Actelion had a compound but it's only being used in psoriasis called ponesimod. Receptos has a compound now known as ozanimod – formerly known as RPC1063 – that is in two phase 3 studies for relapsing-remitting multiple sclerosis, a two-year study called RADIANCE and a one-year study called SUNBEAM, both of which are enrolling twelve hundred patients each. MSDF And the RADIANCE trial results looked pretty good; I mean, you had 85, 90% effects at 12 to 24 weeks or even at a year in terms of relapse rate. Does this look like the next compound to emerge? Dr. Rosen I think it's likely that ozanimod will be the next compound to be submitted for the regulatory process here in the United States and probably in Europe as well. The pleasing thing about the phase 2 data for ozanimod was, in fact, both the strong efficacy signal and a very well-tolerated safety profile; in fact the adverse effect profile of ozanimod and placebo were, in fact, indistinguishable and overlapping in the phase 2 studies. In addition, this very well-tolerated, favorable safety profile has been replicated in a highly successful phase 2 study in ulcerative colitis called TOUCHSTONE that was released recently. So clearly this is a mechanism of immunomodulation that could well prove to be useful for relapsing-remitting multiple sclerosis, but also in a range of other autoimmune diseases where treatments are hard to come by. MSDF Even with Gilenya, I think there have been reports of a couple of cases of progressive multifocal leukoencephalopathy, so it gives a nice balance between immune surveillance and inhibiting T cell trafficking, but it seems like not a perfect balance. Does it look like that margin will be narrowed in the future with other compounds? Dr. Rosen It's possible that it will be. I think the critical point to bear in mind is that real-world experience in tens of thousands of patients with hundreds of thousands of patient-years is really ultimately what is required to define these very rare events that on occasions do occur, and preexisting treatments with other immune-modifying agents such as Tysabri, for instance, may predispose to issues being seen later with PML. And I think that we always have to say that long-term patient experience and physician comfort are ultimately the best guides to the risk-benefit ratio. MSDF I think you've identified something like four compounds in development, those are some that I had seen. Are there others, or these are really the ones to focus on at this point for people to keep an eye on? Dr. Rosen There may well be others that are further behind. There have been a number of others that have had safety signals, particularly liver enzyme elevations, and significant first-dose cardiac effects. Arena have a compound that has recently completed a phase 1 multiple-dosing study and will go on to phase 2. So, you know, there are additional compounds and there will be additional compounds. Ultimately, patients do best when the best compounds appear, and the only way one knows that is to test them in man over the long-haul and define that risk-benefits for patients. And, you know, these multiple efforts really reflect the fact that a field has advanced, and that advancing field really does improve through intelligent intervention our ability to offer patients a better set of choices and a better set of long-term outcomes, which is what we're all about. MSDF We're still focusing here on RRMS, none of this applies to the progressive phase. Is there anything coming along there? Dr. Rosen You know, there's been one trial in primary-progressive; this was the Gilenya trial which didn't meet its endpoints. It may be that the mechanisms in rapidly progressive MS are a little different and that we don't yet, I think, understand the pathogenesis of that rather different presentation. So I'm not aware of a good alternate approaches to that, but that doesn't mean that the understanding isn't there for that to happen over time, it simply means that I'm not yet aware of it. MSDF Finally, in secondary-progressive MS, we can understand what's going on, what led to it; if you limit relapses, that's good. But does it look like primary and secondary really may be overlapping but not the same disease? Dr. Rosen I think there may be balances of pathogenesis where you can intervene more easily in some than in others. Clearly the sphingosine 1-phosphate agonists work particularly well by inhibiting the movement of lymphocytes into the brain. The movement of lymphocytes from the perivascular cuff into the parenchyma, into the white matter, where the demyelination proceeds. However, in parallel in multiple sclerosis, there are also events where there is collateral damage to neurons; we see axonal severing, we see elements of neuronal loss. Certainly with the sphingosine 1-phosphate agonists, there is some evidence that there is a diminution of cortical thinning over time with treatment, and that may be a really good thing. I think that the neurodegenerative components is one that is hard to get a handle on right now, and that I think that these differences will become more obvious with early treatments of the immunopathology of multiple sclerosis. And that may well separate the autoimmune inflammatory damage and its sequelae from neurodegenerative mechanisms that may be entrained, and I think we will learn a lot from looking at those subsets of patients over time, particularly as more, better, and earlier treatment modalities allow the avoidance of significant damage in most patients. MSDF Is there anything important we've missed or you'd like to add? Dr. Rosen You know, I think for all of us who try to work at this interface of therapeutics, we do so because disease is, in fact, personal. We all know patients, we've all seen the multigenerational impact and depredations of multiple sclerosis on friends and family. And I think this is the very strong underlying motivator that drives us as scientists and as physician scientists to really try and bear in mind that the basic mechanisms and the basic therapeutic approaches that we pursue ultimately need a safe and effective human face to change the lives of patients in a positive way. MSDF Very good. Thank you. [transition music] Thank you for listening to Episode Forty-nine of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. MSDF’s executive editor is Robert Finn. Msdiscovery.org is part of the non-profit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is vice president of scientific operations. Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances. We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org. [outro music]

united states ceo california president europe japan ms discovery arena holy grail papers rosen multiple sclerosis radiance novartis la jolla touchstone scripps sunbeam scripps research institute bulletin board pml research resources tysabri rrms dan keller actelion professional resources robert finn gilenya msdf accelerated cure project receptos

Multiple Sclerosis Discovery -- Episode 43 with Dr. May Han

Multiple Sclerosis Discovery: The Podcast of the MS Discovery Forum

Play Episode Listen Later May 27, 2015 18:12

[intro music] Hello, and welcome to Episode Forty-Three of Multiple Sclerosis Discovery, the podcast of the MS Discovery Forum. I’m your host, Dan Keller. This week’s podcast features an interview with Dr. May Han, who discusses issues related to following patients with clinically isolated syndrome. But first, here are some new items on the MS Discovery Forum. We recently posted an article on a surprisingly strong association between a certain gene variant and non-response to interferon beta in people with RRMS. The study is a meta-analysis of three independent cohorts in Italy, France, and the U.S., and it comes from the labs of Philip De Jager and Filippo Boneschi. You’ll find this article by clicking first on News & Future Directions and then on New Findings. This past week we published the latest in our series of data visualizations. This month’s visualization is a series of word clouds illustrating how key terms in the MS clinical-trial literature have changed between 1993 and 2014. To find this visualization, first click on Research Resources, then on Data Visualizations, and then on Word Cloud. According to our curated list of the latest scientific articles related to MS, 30 such articles were published last week. To see last week’s list, go to msdiscovery.org and click on Papers. We selected one of those papers as an Editors’ Pick. It’s study of the association between depressive symptoms and walking ability in people with RRMS. Are you attending the annual meeting of the Consortium of Multiple Sclerosis Centers in Indianapolis this week? If so, please come visit us at the Accelerated Cure Project’s booth. We’ll be demonstrating some of our latest data visualizations along with other features of the MS Discovery Forum. You’ll find the booth in the hallway close to the main entrance to the exhibit hall, and we look forward to meeting you. [transition music] Now to the interview. Dr. May Han is an assistant professor in Neurology and Neurological Sciences at Stanford University. I spoke with her about following patients with clinically isolated syndrome, as well as her approach to patients with MS across the course of their disease. But first, she addressed some unmet needs in MS. Interviewer – Dan Keller Dr. Han, you told me that we’re good at the diagnosis of MS in general, but still there’s a vast area that we don’t know about. What are some of those unmet needs? Interviewee – May Han So it’s been over 150 years since Charcot first described multiple sclerosis, and I have to say that we have come a long way in understanding and treating this disease. But as you have mentioned, there are still areas where we have no idea, there are gaps in our understanding of this disease. One of these areas that is clinically very relevant and is very challenging is in the day and age where we have a dozen disease-modifying therapies for MS patients, and yet we don’t have a good way, a scientific way of selecting the most effective therapy for a particular patient is what I find quite challenging in the clinics. MSDF What gives you clues or how do you approach this essentially algorithm of deciding where to begin and how to move on to other medications if the first one’s not working well? Dr. Han Currently, of course, we follow the guidelines. So for any relapsing-remitting patients, our logic is to go for the safest medication that we think are going to be most effective, which means we go with the first-line therapies. So we have the convention ABC drugs such as beta-interferon family of therapies and glatiramer acetate, plus the newer oral medications such as Tecfidera and fingolimod or Gilenya that we use for the first-line therapy; not a whole lot of science in choosing these medications for a particular patient, but what we would do is initially we would educate the patient about these disease-modifying therapies and then select the medication together with the patient to see what would be most appropriate and the patient could be most compliant for a particular medication. To give you an example, certain patients have aversion to needles, in which case we go with the oral medications. We also have in mind what the preference of the patient, such as whether they could be able to follow it through for years on end with a particular medication. Ideally, we would like to have zero relapses or MRI activity when a patient is on a disease-modifying therapy, but as we all know none of these medications are 100% foolproof, and they can still have some degree of MRI activity or infrequent relapses on this medication. However, if a patient is clearly not responding to a therapy either in terms of not being compliant, being intolerant to the mode of administration, or if they’re having worsening disease activity, we would decide to go on to stronger medications or second-line of therapy. MSDF Do you initially discuss a plan of action, a stepwise pattern of medication prescribing, or do you wait until something needs to be changed to bring it up with patients? Dr. Han That is a very good question. I’m sure it varies among clinicians, but, however, I would like to paint the picture to the patient the best that I can. So, let’s say for example, if a patient who is a newly-diagnosed MS patient who has very few MRI lesions, I would discuss with them what the most appropriate medication could be. We would decide a medication and we would also give them an outline of what the followup plan would be and when we would be deciding to switch to a different therapy, and if so, which medications would be most likely appropriate for them, and also how we would monitor them. So by doing this, it gives the patient a better picture of their path and what to watch out for, and in my experience we have a better outcome with these patients. MSDF Do you find that once you achieve success in limiting relapses and lesions that the medication is fairly stable for a long time, or do you have to have an armamentarium that you keep moving through? Dr. Han So my model if a patient is responding to a medication, unless they have other side effects or reasons to switch, I would like to get the most mileage out of the medication as much as I can for a particular patient. However, if a patient, for example, has JC virus positivity, in which case even if they’re responding to Tysabri really well, there is a cutoff time point where we have to sit down and consider whether this patient should be switched onto a different medication to prevent the development of opportunistic brain inflammation such as PML, in which case what the next medication would be. And so we would sit down and talk the pros and cons; this conversation was started even before the patient was started on medication, but that would be the checkpoint. MSDF I suppose another aspect is do medications start to fail patients even after a long period of stability, or do they usually continue to be stable if the medication is working for some period of time? Dr. Han This is also a very pertinent question. MS patients, as we know, is very heterogeneous. Some of the patients, if they are stable on a medication, they would continue to do well on a medication for several years up to decades. However, some patients would have an initial improvement or stabilization of their disease, however in the later stages they would have worsening disease. And it is really unclear whether because their disease per se is getting worse or whether their body is rejecting the medication secondary to the immune response. And that is also one area that we should do research on to better understand this condition. MSDF When you say reject the medication, are you actually referring to an immune rejection such as with, say, interferon; I would think it would be less likely they would actually mount an immune response to a small molecule. Am I clear on that or not? Dr. Han I think we have quite a lot of information in terms of beta interferon therapies, because we clearly know that patients do tend to develop antibodies against beta interferon, especially the therapy. However, even that we don’t really know if all those antibodies are attacking the drug or whether they are just there. So just by finding the antibody alone is not enough to say that the patient is not responding to it; I think we need to use it hand-in-hand with the clinical response as well as the MRI activity. Getting to the second part of your question whether there’ll be less intolerance or rejection to the therapy if it were small molecules, but I don’t think we understand at the cellular or molecular level. For small molecules there could be receptor down-regulation, there could be availability or cellular sequestration, or even the prodrug being converted to an active drug, or how the breakdown process occurs. So when a patient does not respond anymore to a medication, we just know that the clinical response is worse, and we don’t really know whether it is because the disease activity has worsened or other aspects, pharmacodynamic or kinetic aspects of the system has changed in such a way that they no longer respond. So, again, we do need to do more research to have a better understanding. MSDF You have called it MS comes in many different flavors. Have you found that any medications are particularly good for different constellations of symptoms, or is everything about equal no matter how they present? Dr. Han Very good question as well. I think in the experimental models people know that MS, or central system autoimmunity, can have a bias towards one type of inflammation as opposed to the other. For example, some would say that certain medications are better to treat Th1 as opposed to the Th17 type of inflammation, however in human beings there’s no clear-cut Th1 MS or Th17 MS. I don’t think people have done enough studies to clearly decipher the immune profiles of patients. So the answer is we don’t know. MSDF Finally, let’s talk about the need for biomarkers especially very early in the disease when someone’s presenting with CIS which may or may not become MS. Where does that stand and how acute is the need? Dr. Han The need is there, especially if you look at it from a patient who just had an initial attack. If you tell them that we don’t really know whether this is a one-time thing or whether you’re going to develop MS, and we’ll have to wait and see for three-plus years. So for these three years, the patient’s life is very much consumed by the “is it going to be MS” kind of question. And it does affect their physical-mental wellbeing as well as their quality of life. I think we’ve come a long way with the advancement of the MRI studies in such a way that if a patient has MRI lesions together with the first-time attack, we could almost clearly say that this is going to blossom into MS. However, for patients who are radiographically clean and who just had one episode, it would be very, very helpful to have some kind of blood biomarkers to predict whether this could be a single event or whether it could be a central nervous system inflammatory disorder. MSDF You picked three years as a period of waiting, watching. Are they out of the woods after that, or how late can it blossom into full MS? Dr. Han It’s always a bell-shaped curve. There are patients who would declare themselves sooner than three years, there are also patients who would take several years before they have the second attack. I have one patient who had an initial attack of optic neuritis and nine years later she had the second attack. During that period, she had had MRI scans for three years which were clean. So, I guess, one is never completely out of the woods, but at the same time it is also not prudent to perform unnecessary tests on a patient. So I think we have to focus on what is the safety net and pick a period of time, but at the same time it is very important to educate a patient to symptoms to watch out for, how to get help, and to work very closely with the primary care physician or a neurologist so in case the symptoms show up they will not be ignored or delayed to receiving treatment. MSDF Is there anything we’ve missed or is important to add? I’m sure it’s a gigantic field, but is there anything glaring that should be added? Dr. Han I would like to encourage people in the field to also focus on the secondary-progressive stage of MS. We know that relapsing-remitting MS patients with or without therapy eventually would end up having secondary-progressive MS, so it’ll be really important to decipher whether during the secondary-progressive stage there is no inflammation but only the early neurodegeneration, or how the immune system and the central nervous system interact and how we can change it, or at least modulate it, to either delay or to prevent neurodegeneration. The third area that I think is very important is to try to understand the regenerative aspects of the central nervous system. As I have given you the example, if we have two patients who have had similar lesion burden or even lesions that are approximately the same in similar areas, a patient can be severely devastated, neurologically devastated, whereas the other may have minimal neurologic deficits. And we would always say that it depends on the brain reserve, or neural reserve, but we don’t quite know what it is. Is it the stem cells, is it the nervous system being more resistant to insult and how the immune system interacts with it? And I think this is also a big area that we should focus on, of course, to prevent further damage, but also once the damage is done to limit the damage and perhaps to regenerate it. And I think that people always have within themselves the ability to heal. MSDF Good, thank you. Dr. Han Thank you. [transition music] Thank you for listening to Episode Forty-Three of Multiple Sclerosis Discovery. This podcast was produced by the MS Discovery Forum, MSDF, the premier source of independent news and information on MS research. MSDF’s executive editor is Robert Finn. Msdiscovery.org is part of the non-profit Accelerated Cure Project for Multiple Sclerosis. Robert McBurney is our President and CEO, and Hollie Schmidt is vice president of scientific operations. Msdiscovery.org aims to focus attention on what is known and not yet known about the causes of MS and related conditions, their pathological mechanisms, and potential ways to intervene. By communicating this information in a way that builds bridges among different disciplines, we hope to open new routes toward significant clinical advances. We’re interested in your opinions. Please join the discussion on one of our online forums or send comments, criticisms, and suggestions to editor@msdiscovery.org. [outro music]

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