Endpoints

Today on Endpoints, Dr. Danielle Boyce joins us to explore how data science is transforming our understanding of ALS and why the ALS Research Collaborative (ARC) Study presents an exciting opportunity for research. As ALS TDI’s Principal Investigator for Real-World Evidence, Dr. Boyce brings extensive expertise in analyzing big data to uncover key insights about ALS. Her work focuses on harnessing the vast amount of information collected through ALS TDI’s ARC Study, turning data into discoveries that could drive ALS research forward.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

25 years ago, Steven Ascher and Jeanne Jordan began filming a documentary about a new, one-year-old organization and the family that founded it. That organization was called the ALS Therapy Development Foundation, and its mission was to find treatments for ALS. It was started by Jamie Heywood, whose brother Stephen had been diagnosed with the disease at the age of 29. The film they made, So Much So Fast, came out in 2006. It received critical acclaim and was screened at the Sundance Film Festival. It documents five years in the lives of the Heywood family as they dealt with Stephen’s progressing disease while simultaneously building what would eventually become ALS TDI, the world’s most comprehensive drug discovery lab dedicated solely to ALS. Today, on Endpoints, Steven and Jeanne join us to talk about what it was like during the earliest days of ALS TDI, what it's been like watching the organization evolve, and how their own story helped inspire the film. Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

In February 2024, ALS TDI launched the ALS Trial Navigator, a set of online tools designed to help people with ALS find clinical trials. Users can fill out a questionnaire to be matched with trials that meet their criteria, search a global database of studies, and even find sites close to them on an interactive map. Dr. Nadia Sethi, an ALS advocate who became a clinical trial expert after her husband Sundeep was diagnosed with the disease, was instrumental in the conception, design, and rollout of the tool. As we approach one year since the launch of the ALS Trial Navigator, Dr. Sethi joins us to talk about what went into creating this comprehensive database of ALS clinical trials and how her experience helped inspire it. In a first for this show, she also had some questions for the host – Jonathan and Nadia worked together closely during the design of the tool and continue to collaborate to maintain it.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Chris Ritter, the co-owner of Oakland United Beerworks, never thought he'd make a profession out of brewing. He had always loved beer but was a lawyer by training. Brewing was his son Andrew's great passion. From a young age, Andrew always knew he wanted to make beer. After college, he got into the industry and eventually became the lead brewer of his own brewery. Everything changed for the Ritter family, however, when Andrew was diagnosed with ALS. When he passed away at the age of just 31, his parents made the difficult decision to keep the brewery going as a tribute to his memory. They have also dedicated themselves to helping end ALS as longtime participants and advocates for the Ales for ALS™ program – which supports research at ALS TDI through craft beer. Today, Chris joins us to share how he's working to honor his son's legacy and stay active in the fight against ALS.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

We need more – likely many more – effective treatments to end ALS. Progress in ALS research has been too slow for too long but, in 2024, we're witnessing significant advances and a new pace of progress. The past few years have see more treatments for ALS in clinical trials than ever before. Still, most drugs that reach trial fail, in any disease. However, the more experimental treatments that make it to trials – with good preclinical evidence to support them – the more likely there are to be successes. Recently, we have even seen approvals for new ALS treatments, such as tofersen, a genetic treatment developed by Biogen for SOD1-related ALS, sold under the brand name Qalsody. Dr. Stephanie Fradette was one of the people behind milestone. As the VP Head of Neuromuscular Development at Biogen, she oversees the company's research and development of treatments for ALS and related diseases. Today, on Endpoints, Dr. Fradette joins us to talk about the current state of ALS research ­- the biggest challenges facing researchers, the developments that give her hope for the future, and how the ALS research community could better serve people with the disease.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

In 1999, Stephen Heywood, who was 29 years old at the time, was diagnosed with ALS. His family was devastated when they learned that there were no treatments that could slow or stop his disease. His brother Jamie decided that he had to do something about it, founding the organization that would eventually become ALS TDI – the world's first nonprofit biotech – in the basement of his parents' Newton, MA home. The first hire Jamie made as he worked to get this new organization off the ground was Rob Bonazoli. In those days, Rob was responsible for, in his words, “everything non-scientific.”  Part of this included building the team that would carry out Jamie's lofty research goals. Many of the people he helped recruit at that time are still with the organization twenty-five years later. Two of those early employees were Ken Thompson, now ALS TDI's Vice President of Facility Operations, and Fernando Vieira, our CEO and Chief Scientific Officer. Today, on Endpoints, we're joined by Rob – as well as Ken and Fernando – to talk about what it's been like to see ALS TDI grow from its humble beginnings to one of the world's leading ALS research institutions.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Kids Quest to Cure ALS is an organization for kids, by kids. Their mission is to get young people involved in the fight against ALS. The group was founded by kids in the US and Canada – all of whom have been affected by a loved one's ALS diagnosis. They are encouraging people under the age of 18 across both countries to band together to help raise funds for ALS research and bring awareness to the disease. Today, on Endpoints, we're joined by several members of the Kids Quest steering committee to tell us more about the inspiration behind their mission, and why it's important to involve young people in the fight against ALS.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

One of the most common misconceptions about ALS is that it only affects older white men. In reality, the disease can affect anyone of any age – and of any ethnicity. Many Shades of ALS, a team within the I AM ALS organization, is working hard to dispel some of these misconceptions. Their mission is to bring attention to people of color living with ALS and the unique challenges they often face – as well as providing resources for their mental, physical, and social well-being. Juan Reyes is a veteran living with ALS, an advocate, and co-chair of Many Shades of ALS team. Lakeia Nard is a member of the team who lost her son, King'nazir, to a rare form of pediatric ALS and also runs her own nonprofit, Melanin Children Matter. Today, on Endpoints, they join us to talk about how the team came together, its mission, and how the ALS research and medical communities could be better serving people of color with the disease.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

When dealing with a disease like ALS, it can be hard to know where to turn for reliable information and advice. While your doctor and care team might be able to provide some answers to your questions, there are many topics that they might not be equipped to handle. There are many resources available on the internet, but it can be hard to know if you've found a reputable source. Roon is an app that help solve this problem. It provides answers to questions about disease, including ALS, in the form of short video clips featuring researchers, doctors, caregivers, people with the disease, and more. They provide information about topics like treatments, current research into the causes of the disease, and advice about to day-to-day life. Today, on Endpoints, we're joined by Roon's co-founder and CEO Vikram Bhaskaran to tell us more about the app, and how his own personal experience with ALS helped inspire it.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk about PTC Therapeutics phase 2 trial for PTC 857. There are currently no treatments to stop or reverse ALS but the ALS Therapy Development Institute is working to change that. To learn more about ALS TDI and our research to end ALS, visit ALS.netSupport the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Andrea Lytle Peet's passion for running started simply enough. At age 30, she had just moved to Washington, D.C. to be with her husband, David. Stuck in their house during the snowy D.C. winter, she started walking on a treadmill in their basement to keep active. Soon, she had signed up for a local 5k. Then she stepped up to a 10 miler. Then came marathons and triathlons. Within three years, she had begun training for a half ironman. Then, at the age of 33, everything changed when she was diagnosed with ALS. In four months, she went from the strongest she had ever been to walking with a cane. However, this would not be the end of her athletic career. Despite having ALS for almost a decade, she is still able to ride a recumbent bike, which she has used to continue to participate in marathons all over the country. Recently, she reached a major milestone – completing at least one marathon in all 50 states. To commemorate this accomplishment and share her story to inspire others, Andrea is releasing two documents of her journey – a film, Go On, Be Brave and a memoir, Hope Fights Back.  Today, on Endpoints, Andrea and David join us to talk about how she achieved these accomplishments in the face of such adversity, and why they believe it's important to share her story with the world.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Finding more accurate ways to measure ALS progression is one of the most important challenges facing scientists today. More sensitive, objective ways of measuring how the disease is affecting people could help make clinical trials faster and more efficient. Achieving this is one of the primary goals of the ALS Research Collaborative (ARC) – a program at ALS TDI that seeks to learn more about ALS by gathering data about the disease and sharing it with researchers all over the word. One way we do this is to collect movement data by sending people with ALS wearable accelerometers – devices that track movement much like a smart watch. By wearing these devices on each wrist and ankle, participants can generate data about how their disease is affecting their movement over time. Recently, researchers from Massachusetts General Hospital partnered with ALS TDI scientists to analyze these data and demonstrate that these devices can be used as a reliable measure of ALS progression. A paper detailing their findings titled, At-Home Wearables and Machine Learning Sensitively Capture Disease Progression in Amyotrophic Lateral Sclerosis, was published in the journal Nature. To tell us more about this research project and what it means for people with ALS, we're joined by Dr. Anoopum Gupta, a Neurologist at MGH and the paper's lead author.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

ALS TDI's ALS Research Collaborative (ARC) is a global initiative that partners with people with ALS to gather data about the disease to help us better understand the underlying biology of ALS and accelerate the discovery of treatments. Recently, ALS TDI announced a partnership with Unite Genomics, a healthcare data analytics company, that will allow us to integrate a new data source into the program – electronic health records (EHRs).   Today, on Endpoints, we're joined by Unite Genomics CEO Taner Dagdelen, to tell us more about what this collaboration entails, what researchers can learn from EHR data, and what this means for present and future ARC participants. Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. I'm Jonathan Gang. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk about Novartis Pharmaceuticals' phase 2 trial for BLZ945.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk about AL-S Pharma's phase 2 trial for AP-101. That there are currently no treatments to stop or reverse ALS but the ALS Therapy Development Institute is working to change that. To learn more about ALS TDI and our research to end ALS, visit als.net.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space – all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk about Annexon's phase 2 trial for ANX005.  That there are currently no treatments to stop or reverse ALS but the ALS Therapy Development Institute I is working to change that. To learn more about ALS TDI and our research to end ALS, visit http://www.als.netSupport the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

In March 2023, ALS TDI announced the launch of the ALS Research Collaborative (ARC). ARC's goal is to learn more about the underlying biology of ALS to help accelerate treatments. The study is powered by the participation of people with ALS, who generously share data about their disease, their genetics, and their progression. This data then informs ALS TDI's research to find treatments for ALS. It is also shared with the broader ALS research community through the ARC Data Commons, an online portal allowing researchers to access years of data dating back to when the program was founded as the Precision Medicine Program, in 2014. This program is powered by participants – people with ALS all over the world who have chosen to share their data with ALS TDI in order to help accelerate research. ALS TDI works hard to make sure that participation is as easy as possible for these volunteers – nearly every aspect of the program can be completed from participants' homes. Today, on Endpoints, we're joined by Alan Premasiri, ALS TDI's Manager of Clinical Operations, to talk about what it's like to participate in ARC, how ALS TDI works to support our participants, and how their invaluable contributions help move our science forward. To learn more about ARC and sign up to join the program, visit ALS.net/ARCSupport the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Scott and David Lloyd first joined us on Endpoints in 2020. The two brothers shared their story about how David's ALS diagnosis inspired them to pursue a lifelong dream – racing high-performance cars. This passion grew into Racing for ALS, an organization that has raised almost $1 million to date through high-performance driving events, all in support of ALS research, care, and services. Since that first conversation, much has changed for the Lloyd Brothers and Racing for ALS. The organization has grown significantly, going from putting on one annual event, Dave's Race, to several across the country. However, their mission remains the same – to spread awareness, raise funds, and rally the performance-driving community in support of ending ALS. Today, Scott returns to Endpoints to say more about what's changed over the past three years, what's stayed the same, and what's next for Racing for ALS.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk about MedicNova's phase 2/3 trial for Ibudilast.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

For more than 20 years, riders have been coming to the Tri-State Trek for an unforgettable weekend of riding bikes, seeing friends, and supporting ALS Research. The Trek is ALS TDI's signature fundraiser, and it has raised more than $10 million dollars to support our research to end ALS. It has also become something of a family affair, with riders who have been showing up for years, or even decades. Rinaldo and Sara Dorman have been an essential part of the Trek for 11 years. Rinaldo is the captain of Team America, one of the Trek's biggest and most dedicated teams. His wife Sara is a longtime volunteer who serves as the leader of the lunch crew – a job that requires feeding hundreds of hungry, sweaty cyclists every day. Rinaldo and Sara joined us to discuss how they got involved with the Trek, why they keep coming back year after year, and why they're especially excited for this year's ride on June 24-25th.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

When we talk about ALS, we often make a distinction between types of the disease: 90% of cases appear to be sporadic – meaning there is no family history of the disease. The opposite is true of the other 10% – known as familial ALS or genetic ALS. In these cases, the disease can sometimes be traced back in a family for generations.  Behind this phenomenon are a number of inherited mutations in genes with names like SOD1, FUS, C9orf72. With the advent of widely available genetic testing, people with a family history of ALS are now able to find out if they carry these mutations – and are thus likely to develop the disease. When Jean Swidler was a child, she lost her grandmother to ALS. Then as, an adult, her mother was diagnosed with the disease. Eventually, Jean herself underwent genetic testing and discovered that she carries the C9orf72 repeat-expansion mutation.  Since then, Jean has become a tireless advocate for asymptomatic carriers of ALS-related genetic mutations. Today, she joins us to talk about her story, her advocacy, and how she believes the medical and research establishment could be better serving people in her situation. Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk us through AB Science's phase 3 trial for masitinib.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts, presented by the ALS Therapy Development Institute. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk us through Cytokinetics' phase 3 trial for reldesemtiv.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Normally, you'd expect the subject matter of the videos you see while scrolling through TikTok to be light – funny skits, cute animals, dances, maybe a recipe or two. However, when one of Brooke Eby's videos comes into your feed, you'll notice it's a little different from your average clip. Her TikToks may still be funny and full of jokes, but the subject matter – living with ALS as a young woman – is anything but light.  Brooke was 28 years old when she experienced her first symptoms – weakness in her left foot, which continued to spread throughout her leg. At the time, she was living in New York City and working in tech. It would be four years before she received her diagnosis at the age of 33. Of course, this was a shocking development for an active, young woman like her at first. Every person with ALS finds their own way to process such a devastating diagnosis – for Brooke, the solution was to turn to her strong sense of humor and look for the lighter side of things. Since she started sharing her story on TikTok, her videos have attracted more than 18,000 followers to her account and received millions and millions of views. Today, Brooke joins us on Endpoints to talk about living with ALS as a young woman, and how she's working to spread ALS awareness with humor on social media.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Welcome to the latest episode of Endpoints Shorts. In these bite-size podcasts, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk us through Biogen's Tofersen Trial for presymptomatic people with SOD1 gene mutations.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

ALS TDI's primary mission is to discover treatments for everyone with ALS. Every day, our scientists are testing drugs to find the next compound with the potential to make it to clinical trials and beyond. Currently, one of our most promising leads is a group of molecules called copper complexes. These copper complexes, also referred to as redox metabolism modulators, have demonstrated signs of efficacy in ALS TDI's cellular and animal models of ALS. Currently, our scientists are hard at work looking for a lead molecule to advance toward clinical trials. Today, on Endpoints, we're joined by Dr. Fernando Vieira, ALS TDI's CEO and CSO, to tell us more about what copper complexes are, how ALS TDI discovered their potential for treating ALS, and where they are in the drug development process.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Today we have the first installment of a new series we're calling Endpoints Shorts. In these bite-size episodes, we'll be tackling important scientific concepts for people interested in ALS research, current clinical trials, and news from around the ALS space ­– all in 15 minutes or less. Our guide throughout this series will be Dr. Nadia Sethi, ALS TDI's Director of Community Outreach and Engagement. Today, Nadia joins us to talk us through the Ionis' Pharmaceuticals Fusion Clinical Trial.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Project Relate is an app for Android phones and tablets that aims to use the power of voice recognition technology to help people with dysarthria, or difficulty speaking, in their day-to-day lives. The app is a product of Project Euphonia – a program founded by Google and Harvard researchers in collaboration with ALS TDI. Although it's since expanded to include other conditions that might affect someone's speech, the project was founded using data collected through ALS TDI's Precision Medicine Program (PMP). Today, on Endpoints, we're joined by Pan-Pan Jiang, a Technical Project Manager for Project Relate, to tell us more about how the app works, how it can help people with ALS, and how ALS TDI's PMP data was instrumental in its development.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Antibodies are an essential part of our bodies' defenses against diseases. They are specialized proteins that attack and kill foreign invaders like infectious bacteria and viruses. Antibodies are normally naturally produced by our immune system in response to an infection. However, in recent years, scientists have learned to produce antibodies in a lab to treat a variety of diseases.  Several Antibody treatments have been investigated in ALS, including tegoprubart, which was invented by ALS TDI and recently successfully completed a phase 2a clinical trial. Dr. Silvia Pozzi is Assistant Professor at the Université Laval's Brain Research Center in Quebec City specializing in ALS Research. She recently was an author on a paper reviewing the current state of antibody therapies for ALS.  She joined us on Endpoints to discuss the current outlook for these therapies in the ALS space – as well as what the future might hold for these treatments.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

ALS can happen to anyone. Although many outside of the ALS community often think of it as a disease that affects older people, onset can occur at any age, in any person, for reasons that are not understood. Take, for example, Ajay Sampat. He's 36 years old, he has a wife and two young children, and works as a neurologist specializing in sleep disorders. Earlier this year, he was diagnosed with ALS. Facing a terrible diagnosis like ALS at such a young age is, of course, unimaginable for anyone who hasn't experienced it. But, with the help of his family and friends, Ajay is working to make sure he can live as fully as possible with the time he has. Today, Ajay joins us to talk about what it was like going through all this, and how checking things off his bucket list since he received his diagnosis has been helping him process it all.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.

Celecoxib is a non-steroidal anti-inflammatory drug, or NSAID. Ciprofloxacin is an antibiotic that is used to treat a number of bacterial infections. The former has been trialed unsuccessfully in ALS. However, according to preclinical evidence and a phase 1 trial conducted by Neurosense, a biotech company based in Israel and Cambridge, MA, these two FDA-approved drugs may hold some promise for people with ALS when combined into one treatment. Neurosense's formulation of these two drugs is known as Prime C. The first participants were recently enrolled in a Phase 2b trial of the treatment, and it is also currently undergoing a pharmacokinetic trial in healthy volunteers. Recently, several members of Neurosense's team joined Endpoints to help us learn more about the company's mission, the science behind prime C, and what's next for the potential ALS Treatment. Episode Guide: 1:22 – NeuroSense's Company Story 5:58 – The Science Behind Prime C 15:13 –Details about Prime C in Clinical Trials Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

20 years. More than 5000 miles, give or take. Thousands of riders. Countless hugs, tears, and cheers from the side of the road. And, if everything goes according to plan, $10 million raised for research to end ALS. The Tri-State Trek has built quite a legacy over the past two decades, and there is no better time to celebrate it than June 24-26th 2022 – the year that will mark the 20th annual Trek, as well as the return to its traditional three day, 270-mile format. The Trek started back in 2003, with 16 cyclists pedaling from Boston to New York and raising $30,000 for ALS research along the way. Since then, it has grown to become the marquee fundraising event for the ALS Therapy Development Institute, with hundreds of riders each year raising hundreds of thousands of dollars to help end ALS. It's also become something of a yearly family reunion – from the cyclists who return to ride year after year, to the volunteers who make the event run smoothly, to the supporters who show up to cheer everyone on, the event has fostered a strong community throughout its 20 years. To celebrate the 20th Trek, we talked to Ride Marshall Kevin Porter and Bike Mechanic Ric Starry – two people who have been volunteering for the ride since nearly the beginning, and the namesakes of the Starry-Porter award for outstanding trek volunteers – about what keeps them coming back, year after year. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

There are currently no effective treatments available for ALS. Because of this many people living with ALS turn to Clinical Trials, both to get a chance to receive a potentially beneficial treatment and to help push forward the scientific effort to end the ALS. It's not an easy commitment –participants may have to travel to faraway sites to receive treatment, undergo uncomfortable or even painful procedures, and face the chance that, in many trials, they may be randomized into the placebo group. José Fajardo, a radio producer and executive living in Hawaii who was diagnosed with ALS in 2019, elected to take on some remarkable challenges in order to join a clinical trial, including traveling for regular site visits all the way from Hawaii to Texas. Today, he joins us on Endpoints to talk about his story, how he decided a clinical trial was right for him, and what his experience was like. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Regulatory T cells, also known as Tregs, are specialized cells that are part of the immune system. As the name implies, they help regulate the body's immune natural immune response, among other functions According to some studies, the dysregulation of TRegs may play a role in several neurodegenerative disease. Coya Therapeutics is a Texas based biotech company working to utilize TReg therapies to treat these diseases – including ALS. Their ALS treatment completed a Phase 2a study in 2021, with results to be published later in 2022. They are also planning a phase 2b study later this year. Today, on Endpoints, we're joined by Dr. Adrian Hepner, Coya's Chief Medical Officer, to talk about how TRegs might be able to help people with ALS, give an update on the Phase the phase 2a study, and discuss plans for the next steps. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Sally Stewart knows wine. It's the focus of her career, and her great passion. As a sommelier, she's worked in great restaurants in places like Portland, Oregon, Telluride, Colorado, and New York City. Her career was in a great place – she had a resumé that included restaurants at a world-famous ski resort, a restaurant with a Michelin star, and even one founded by food legend Wolfgang Puck. That all changed when, at the age of 31, she was diagnosed with ALS. However, Sally found a way to adapt – and, even when she couldn't work in restaurants anymore because of her progression, to stay involved with her passion. Today, she's the co-owner of the Denver Wine Merchant, a store in Colorado that sells fine wines, beers, and liquor, hand-picked by Sally and her Co-owner/partner Stephen. This year, they also had a particularly special product on the shelves – an ALES for ALS beer from Nederland, Colorado's Knotted Root brewing company, whose help sales support the critical research at ALS TDI. Today, Sally joins us on Endpoints to talk about her story, making an ALS-inspired career pivot, and how she's working to support others dealing with the disease. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

In 2018, ALS TDI began working a big collaboration with a partner you've probably heard of – Google. At the time, Google was looking to put their artificial intelligence technology to use to help develop better speech recognition software for people with conditions that affect their ability to talk. ALS TDI was the perfect partner to launch this effort with, having already collected a wealth of voice recordings as part of our Precision Medicine Program. Thus, Project Euphonia was born. Utilizing our PMP data, Google researchers began working to train their AI speech recognition tools to better understand people with ALS-affected speech, as well as other conditions. Today, this collaboration is beginning to bear fruit, with the early testing of Project Relate, the first app made available to the public from Project Euphonia. Today, on Endpoints, we're joined by Google's Julie Cattiau, a product manager for Project Euphonia, to tell us about the history of this collaboration, what people can expect from the new app, and what might be coming in the future Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

One of our most powerful new tools in recent years for in vitro experiments are something called induced pluripotent stem cells, or iPSCs. iPSCs are created with a technique that allows scientists to take living cells, often skin cells, from an adult human and revert them to the state of a stem cell, which can then be grown into any kind other kind of cell in the body – including cells affected by ALS like motor neurons. To further explain what an iPSC is and how we use them in our research to end ALS, we're joined today by Dr. Kyle Denton, ALS TDI's director of Cell biology. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Much of what causes the 90% of Amytrophic Lateral Sclerosis (ALS) cases that occur sporadically remains a mystery. However, one risk factor that is supported by a large body of evidence is service in the military. In recent decades, a number of studies have shown that veterans' have a significantly higher change of being diagnosed with ALS than the general population. Studies have found that 1 in 6 people living with ALS have served in the military in some capacity, and veterans have a roughly 50 percent higher risk of dying from ALS than the general population. Exploring this connection is an important part of our work at ALS TDI – and it has led to many important steps in both our own science and the wider ALS research community. To explain more about what we know about the connection between the military and ALS, as well as ALS TDI's history working with veterans to advance the cause of ALS research, we're joined by ALS TDI's Senior Director of Development, Carol Hamilton.  Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

When it's used right, social media can be a powerful tool to spread awareness. Before Tavares Speers was diagnosed with ALS, he already had a big social media following. His tens of thousands of Instagram followers would give him enormous numbers of likes on every picture he posted – images of him out partying with friends, hanging at the beach, or out to dinner with his then-partner, now husband Erik. Today – more than a year after his diagnosis – Tavares' feed looks a little different. Mixed in with the pictures of the “self-described” “happy-go-lucky guy” having a good time, there's a lot of content promoting ALS awareness, including telling his followers about ALS TDI's Precision Medicine Program (PMP). Today, Tavares joins us to talk about his story, and how he puts his status as an influencer to work for a great cause – supporting ALS TDI's research to find treatments for everyone living with ALS. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

After an ALS diagnosis, it can be hard to know where to turn. There are doctors and clinics that offer medical support, and services organizations that can help with getting equipment and things like securing travel to medical appointments. There are advocacy groups that work to change policies to help people with ALS, and research organizations like ALS TDI that are leading the way in finding treatments to end the disease However, Steve Cochlan recognized a gap in what was out there after he was diagnosed with ALS in 2019. As a deeply religious man, he felt that he needed spiritual sport to face what lay ahead of him – and when he couldn't find an established group that could provide it, he started one himself. Thus, the ALS Family of Faith was born. The group connects people – both religious and non-religious – with trained chaplains who can provide them with spiritual and emotional support as they navigate the challenges of living with ALS. Steve joins us today on Endpoints to discuss his story and the mission of the ALS Family of Faith. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Recently, ALS TDI took an opportunity to reintroduce ourselves. If you follow us on social media, read our emails, or check our website you may have noticed that we have a new way of describing our role in the effort to end ALS – that we are the Drug Discovery Engine for ALS. What do we mean by drug discovery engine? It means that our mission is to discover and invent new ALS treatments, and work to advance those treatments into human trials – and to do it over and over again until there are treatments for everyone living with ALS. Recently, our CEO and Chief Scientific Officer Fernando Vieira gave a presentation at one of our ALS TDI Town Halls to explain more about what we're doing to fulfill this mission, and what it means to be the Drug Discovery Engine for ALS. Today, on endpoints, we're featuring that presentation for anyone who wasn't able to attend or wants to learn more about ALS TDI and our research to end ALS. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

If you aren't already, you should be tuning in to I'm Dying to Tell You. It's a podcast about inspiration, full of compelling interviews with people who have faced incredible challenges – including many people from the ALS community. However, one of the most incredible things about the show is the story of the podcast itself – it's hosted by Lorri Carey, who has been living with ALS since 2004. Lorri brings her own experience dealing with a debilitating, terminal illness to every interview on her show. Today, she joins us to tell us about her own ALS story, how her podcast got started, and what she's learned from talking to so many deeply inspiring people. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Biomarkers are an important part of medical research and drug development. A biomarker is a broad term that describes some sort of measurable property of the body, especially one that can tell us something about a disease. Common examples are glucose levels for type one diabetes and blood pressure for hypertension. Today on Endpoints, ALS TDI's former CEO Dr. Steve Perrin joins us to talk about what a biomarker is and how they're used in drug research. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

The Complement system is an intriguing potential pathway for treating ALS. It's an essential part of the immune system – and many researchers believe its dysregulation could play a role in some cases of ALS. A class of drugs known as “complement inhibitors” seek to treat ALS by targeting certain proteins that are part of the complement system, also known as the complement cascade.  On Endpoints, we've previously discussed one complement inhibitor that's in trials as a treatment for ALS, Alexion's Ravulizimab. Today, we're looking at another potential treatment – Pegcetacoplan, a C3 inhibitor that's currently in a phase 2 trial for ALS sponsored by the company Apellis. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

For years, Lou Gehrig has been recognized as both a face of ALS and a legend of Baseball. But, until now, he did not have an official day dedicated to him by the MLB, unlike some of his fellow legends like Jackie Robinson and Roberto Clemente. That’s where the Haberstrohs come in. Tom, Steve, and Chuck Haberstroh are three brothers, with two big shared passions – baseball, and ending ALS. Their mother, Patty Haberstroh, was diagnosed with ALS in 2017. Since then they’ve gone on to raise awareness and money for the disease through fundraisers like the ALS Pepper Challenge. Recently, along with a group of other diehard baseball fans who were connected to ALS, they worked with the MLB to create an annual Lou Gehrig Day to both celebrate his legacy as a ballplayer and raise funds and awareness  for ALS. The first annual Lou Gehrig Day will take place throughout the league on June 2nd, 2021.  Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Today on Endpoints, we’re joined by Dr. Leigh Hochberg. He's the the Director of the Center for Neurotechnology and Neurorecovery at Mass General Hospital’s Department of Neurology and a neurologist in its Neurocritical Care and Stroke Services, a Professor of Engineering at Brown University, and the Director of the Veterans Affairs Center for Neurorestoration and Neurotechnology in Providence, Rhode Island. He’s also one of the leaders of the Braingate Consortium – a collaborative team of researchers from several hospitals and academic institutions working together on devices that aim to allow people with limited mobility to control devices directly with their brains. This technology – which also goes by the name Braingate – has already helped people with ALS, as well spinal cord injury and brainstem stroke survivors, do things like move a computer cursor using only the power of their minds.  Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Today we’re joined by Lisa Cross Bonahoom. Lisa is a teacher, singer, actor, director, choreographer, and dancer who was diagnosed with ALS about three years ago. She brings us a story of how this disease has changed her life – but also how it’s inspired her to become an activist and fundraiser. It’s a story of how her family has inspired her to keep up the fight. And, especially, it’s a story of how she’s kept dancing through it all.SIGN IN TO LISTEN  Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Biomarkers are an important part of medical research and drug development. A biomarker is a broad term that describes some sort of measurable property of the body, especially one that can tell us something about a disease. Common examples are glucose levels for type one diabetes and blood pressure for hypertension. Today on Endpoints, ALS TDI CEO Dr. Steve Perrin joins us to talk about  what a biomarker is, how they’re used in drug research, and a promising possible biomarker for ALS and other neurodegenerative diseases: Neurofilament Light Chain. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Finding out a loved one has ALS can be hard enough. But for people whose family members test positive for one of the genetic mutations behind familial ALS, it can bring a whole other set of issues to deal with – knowing that they might also inherit the mutation, and that they might pass it down to your children. Daniel Barvin knows this firsthand – a number of people in his family have passed from ALS, and he himself is a presymptomatic carrier, meaning it may only be a matter of time until he himself begins to show signs of the disease. Today, he joins us on Endpoints to discuss what it’s like to know that ALS is likely part of his future, and what he’s doing to change things – both for himself, others with ALS, and his own newborn son.   Donate to ALS research in honor of Daniel here.  Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Today, we’re happy to be joined by representatives from one of our partners in the industry, Alexion – a biotech specializing in rare diseases – to talk about the company and it’s upcoming CHAMPION-ALS trial. Champion-ALS is a phase Three trial of Ravulizimab, also known by the brand-name Ultomiris, a drug that targets part of the immune system called the complement system. The drug is already approved for use in some rare blood disorders, but there is evidence that the complement system may also play a role in ALS pathology. Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.

Joe Lynch hosts a podcast called “The Logistics of Logistics.” Normally this show covers topics about the logistics and supply chain industries. But recently Joe decided to switch up the format for one special episode after his friend Larry Cepuran was diagnosed with ALS. Joe and Larry talked about the story of their friendship, Larry’s diagnosis, and how he’s adjusted to life with the disease. They also touch on why they’ve decided to encourage their listeners to support our research at ALS TDI. Today, we’re presenting their conversation in full as a special guest episode. Find out more about Joe's podcast at https://www.thelogisticsoflogistics.com/ Support the show: https://www.als.net/donate/ See omnystudio.com/listener for privacy information.