Nobody knows ALS better than people living with the disease. Therefore, it’s crucial for scientists to utilize the perspectives of people with lived experience in their research. Earlier this year, ALS TDI joined with Her ALS Story—a group of women diagnosed with ALS before the age of 35—to put on a series of Town Hall webinars that highlighted the importance of this connection. The three-part series featured discussions about ALS risk factors, lesser-known symptoms, and stories of diagnosis. In each session, researchers from ALS TDI’s ALS Research Collaborative, or ARC Study, presented information about our current scientific understanding of each topic, while Her ALS Story members shared their own personal experiences. Today, on Endpoints, we’ll hear some highlights from each episode of the series. We’ll also be joined by Dr. Danielle Boyce, ALS TDI’s Principal Investigator, Real World Evidence, and Alan Premasiri, our Director of Clinical Operations, to discuss what they learned from the discussions and how conversations like these can help shape the way they approach their research. If you'd like to learn more about ALS TDI's research and how we are incorporating lived experience to advance research, consider attending the ALS TDI Summit on October 17th, 2025 in Boston, MA or online. Register here: https://fundraise.als.net/alssummit/Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.
Brain-computer interfaces, or BCIs, aim to enable people to control various adaptive devices directly with their thoughts. There are many BCI devices currently in various stages of development, with the potential to help people with ALS and similar conditions across many different activities of daily life. One particular area in which BCI devices may be able to support people with advanced ALS symptoms is communication. Many people with ALS rely on devices such as smartphones, tablets, and laptops to write or speak. All tools like these, collectively known as augmentative and alternative communication or AAC devices, need some kind of input to work. This could mean using a keyboard, a touchscreen, or, for people with advanced paralysis, an eye-gaze device. As ALS progresses, however, even eye gaze devices may become difficult for some people to use. BCIs—by allowing people to control AAC devices with their minds—could be a potential solution to this problem. Today, on Endpoints, we’re joined by two experts to discuss BCI technology and, in particular, its potential to help people with ALS continue to communicate. The views expressed in this podcast are those of the speakers and do not represent official endorsements by any organization. Kate Nilsen currently serves as an advisor to Synchron, a company developing a brain-computer interface (BCI) device. This device is investigational and is currently in clinical trials approved by the U.S. Food and Drug Administration (FDA). For those interested in staying informed about Synchron’s ongoing work and updates, please join the Synchron community: https://link.edgepilot.com/s/4239f747/ca21cbCpKkOSLI5ZXNqTdQ?u=https://www.synchronbci.com/StayConnected.htmlSupport the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.
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.
Note: Since this recording, David Lloyd passed away in July of 2023. His legacy lives on through Racing for ALS, and in the more than $1 million he helped to raise to support ALS Research. 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.
Please note that the study described in the podcast episode, the COURAGE-ALS Phase 3 Study for reldesemtiv, was terminated on March 31st, 2023 for futility – meaning that an unblinded analysis was conducted on data by an independent committee while the trial was still ongoing, and it was determined that there was no evidence of an effect. 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 ALS Research Collaborative (ARC) Study. 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 ARC data was instrumental in its development.Support the show: https://www.als.net/donate/See omnystudio.com/listener for privacy information.
