What is the ALS Therapy Development Institute?
The ALS Therapy Development Institute (ALS TDI) is the world's most comprehensive drug discovery lab focused solely on ALS.
Led by drug development experts and people with ALS, our Watertown, Massachusetts-based lab is funded by a global network of supporters unified to end ALS. Our mission is to discover and invent effective treatments for ALS.
What does it mean to be a nonprofit biotech? Why is ALS TDI set up this way?
Most companies in the biotech industry are for-profit enterprises. Instead of relying on donations, they receive their funding from investors or venture capital. In addition to developing drugs to cure disease, they also must consider what decisions and practices will generate the best financial return for these investors.
As a nonprofit, we are only answerable to our ALS community stakeholders – we only worry about finding effective treatments for ALS, rather than providing a financial benefit to shareholders.
What kind of research does ALS TDI do?
ALS TDI is 100 percent focused on research to invent and discover treatments to slow, stop, and reverse ALS. The ALS TDI lab executes research encompassing all areas of ALS target and drug discovery research – including preclinical, clinical, and translational – under one roof. This makes our research extremely efficient and allows for seamless collaboration across all areas.
ALS TDI’s main role is to be the drug discovery engine for ALS. We do the time-intensive research that is needed to invent and discover treatments that show enough efficacy to be advanced to trials.
What is preclinical drug discovery?
Preclinical drug discovery is everything that happens in the process of inventing new drugs and treatments for a disease before trials in humans. This begins with research into the drivers of a disease progression and why they lead to certain symptoms, and what kinds of treatments might help slow or stop the disease. It also encompasses testing potential drugs for effectiveness and safety in cellular, and animal models of the disease. The goal is, at the end of this process, to advance promising drugs into clinical trials.
Why does ALS TDI focus solely on ALS?
ALS TDI was founded in 1999 by the family of Stephen Heywood, who was diagnosed with ALS at the age of 29. After finding no treatment options his brother, James “Jamie” Allen Heywood, realized that a gap existed in the preclinical development of therapeutics for ALS. Jamie started ALS TDI in the basement of his family's Newton, MA home in an effort to bridge this gap and bring effective treatments to people living with ALS as quickly as possible.
Today, ALS TDI is the world's most comprehensive ALS drug discovery lab focused solely on ALS. Although Stephen has since passed, the same sense of hope and urgency drives every decision made in our lab.
How is ALS TDI working to end ALS?
ALS TDI’s mission is to invent new drugs and treatments for ALS and advance them into clinical trials. Because each case of ALS is unique, it will take multiple treatments to end the disease. The current therapies in the clinical pipeline, though promising for some, will not come close to addressing the treatment needs for everyone with ALS. Therefore, we are constantly working to discover new potential drugs – and we will not stop until there are treatments for every person living with ALS.
Our relentless research to find treatments is informed by our ALS Research Collaborative (ARC) – a global initiative to accelerate ALS research by gathering comprehensive data from people with ALS and sharing it with researchers around the world.
What does a "drug discovery engine" mean?
Unlike a traditional biotech company, our mission is to make sure there are as many promising treatments in clinical trials as possible. We work to invent drugs in our own lab, and partner with others to help advance their treatments as well.
Once we have discovered and optimized a potential treatment, we then hand it off to a partner company to advance it to clinical trials. This allows us to continue working to discover new treatments, rather than dedicating our resources toward supporting a single drug as it moves through human trials. As the drug discovery engine for ALS, it is our mission to keep feeding the clinical pipeline for ALS drugs until there are treatments for every person living with ALS.
What drugs have the ALS Therapy Development Institute helped advance to human clinical trial?
ALS TDI has recently helped to advance three drugs into human trials
- Tegoprubart (formerly known as AT-1501), which we invented in our lab, succesfully completed a phase 2 clinical trial in May 2022, sponsored by Eledon Pharmaceuticals.
- AP-101, which we validated in collaboration with Neurimmune, Inc, and is currently in a phase 2 clinical trial sponsored by AL-S Pharma AG.
- CuATSM, which we independently validated, is completed a phase 2/3 clinical trial sponsored by Collaborative Medicinal Development, LLC in 2021.
What other treatments are in ALS TDI’s Pipeline?
ALS TDI researchers are dedicated to discovering and developing ALS therapies that can be advanced to human trials. These are the most advanced drug programs in our preclinical pipeline:
- Copper Complexes: Evidence shows that copper mismanagement in ALS and other neurodegenerative diseases can result in cellular dysfunctions. ALS TDI is investigating copper complexes as potential treatments for ALS – focusing on their ability to regulate oxidative stress and copper balance. From this cutting-edge program, TDI1831 has emerged as the lead candidate. Our initial focus is to advance TDI1831 specifically for ultra-rare ALS cases caused by mutations in a copper transporter gene, paving the way for precision-targeted treatments.
- Type 1 PRMT Inhibitors: In 2020, ALS TDI researchers discovered that type 1 PRMT inhibitors can alter ALS-linked proteins associated with the C9orf72 mutation, paving the way for targeted treatments. Recent studies indicate that type 1 PRMT inhibitors may also treat other types of ALS. ALS TDI is advancing these inhibitors into animal testing to assess their potential for human trials.
For more information about all of ALS TDI's active research programs, click here.
Can ALS TDI provide me or a family member with treatments or enroll me in a clinical trial?
Because the treatments we work on at ALS TDI are all in the preclinical stage of development and have not been tested for safety or efficacy in humans, we cannot provide access to them. Similarly, because our development model is to license treatments that we discover to partners who can then advance them into clinical trials, we cannot directly help anyone to enroll in a trial.
We do, however, provide a powerful suite of tools to help people with ALS find and learn more about clinical trials – the ALS Trial Navigator.
Developed with invaluable input based on lived experience from the ALS community, the ALS Trial Navigator houses a comprehensive database of global ALS clinical trials and observational studies and helps to personalize the search process. It includes a suite of tools for connecting people with ALS, their caregivers, and asymptomatic gene carriers with trials they could potentially enroll in. These include:
- Guided Trial Finder: A tool that walks users through a questionnaire to determine their preferences and potential eligibility for clinical trials. After answering a series of questions about their needs, concerns, and disease history, they are provided with a customized list of studies that meet their criteria.
- ALS Trial Map: An interactive map that lets users locate trials with sites near them, anywhere in the world.
- Trial Browser: A searchable list of available ALS trials and studies, featuring a variety of filtering options.
We also encourage people living with ALS who are participating in clinical trials to enroll in our ALS Research Collaborative (ARC) to track their progression and assess whether their clinical trial participation is affecting their progression over time.
The ALS Research Collabroative (ARC), formerly known as the Precision Medicine Program, is the most comprehensive and longest-running translational research study in amyotrophic lateral sclerosis (ALS). Through ARC, researchers at ALS TDI partner with people with ALS to gather data to learn more about this disease. People from around the world share their data on movement, lifestyle, medical history, genetics, biomarkers, voice recordings, and patient cell biology.
These data are also shared with ALS researchers around the world through the ARC Data Commons. This database provides researchers the opportunity to ask more targeted questions about the connections between ALS symptoms, genetics, and disease biology. By providing a large, ever-expanding dataset, and powerful tools to filter and visualize the data, the ARC Data Commons enables scientists to answer these complex questions – that previously might have required months or years of research – in a matter of minutes.
By participating in ARC, you can access your own disease progression data while also helping to find effective treatments for ALS. Unlike other research studies, enrolling in ARC gives you full access to your personal data, helping you make informed decisions about your own ALS treatment and care. You can sign up for ARC here.
How can I support research at ALS TDI?
There are many ways to support ALS TDI. The simplest is to make a donation or participate in a fundraising event such as the Tri-State Trek. You can track down a beer at a brewer participating in our Ales for ALS™ program, or attend a Brewfest for ALS. You can organize your own fundraiser with our own fundraising platform or on Facebook. You can even simply share our story and mission with your friends and family, and let them know why you support our work to end ALS.
How can I learn more about ALS TDI’s research to end ALS?
There are many ways to learn more about what we do. You can visit our research page or read some of the articles on our blog. We have a monthly email newsletter with information about research, events, and community stories. You can subscribe to the Endpoints Podcast, available on our website as well as iTunes and Spotify.
Does ALS TDI focus on advocacy?
As the Drug Discovery Engine for ALS, ALS TDI’s sole mission is inventing and discovering drugs until everyone with ALS has effective treatments – our focus must always remain on research. While its vital that our focus remains on research, we support the efforts of organizations that lead advocacy initiatives. We always encourage members of our ALS community to remain informed and involved in potential legislation and advocacy efforts that could improve the lives of those impacted by ALS.
ALS TDI does not take a public position regarding the regulatory approval of drugs to be marketed for the treatment of ALS. Alongside the community, we look forward to advocacy efforts that explore avenues for access to treatments.