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 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.

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.

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.   

We also partner with other biotech companies and academic researchers in the ALS space to validate the results of their experiments and, where data supports it, to advance their own drugs into human clinical trials. It’s what makes us the drug discovery engine for ALS.

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:

  1. AT-1501, which we invented in our lab, and is currently in a phase 2 clinical trial sponsored by Eledon Pharmaceuticals.
  2. 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.
  3. CuATSM, which we independently validated, is currently in a phase 2/3 clinical trial sponsored by Collaborative Medicinal Development, LLC

Moving forward, it is our ambitious goal to advance at least one new treatment into clinical trials every two years.

What other treatments are in ALS TDI’s Pipeline? 

We are constantly working to discover new treatments for every person living with ALS. Some of our most promising current projects are:

  1. Type-I PRMT Inhibitors: In late 2020, ALS TDI scientists published a paper detailing a new drug target for C9orf72-related ALS. We are currently working to identify molecules that targeted at Type I PRMTs that might be effective for C9orf72-related ALS and other ALS subtypes.
  2. Redox Metabolism Modulators: ALS TDI scientists have discovered a number of drug-like molecules that appear to be protective against ALS in cellular models of the disease as part of our induced pluripotent stem cell (iPSC) program. The next step is to test these potential drug candidates in our animal models of ALS for safety and efficacy.

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 many resources to help our community find and enroll in clinical trials, including a database of ALS clinical trials, as well as recorded town halls about how to select a trial for you or a loved one, and what it’s like to participate in a trial

We also encourage people living with ALS who are participating in clinical trials to enroll in our Precision Medicine Program (PMP) to track their progression and assess whether their clinical trial participation is affecting their progression over time. 

What is the Precision Medicine Program (PMP)?

The Precision Medicine Program (PMP) is the most comprehensive and longest-running translational research study in amyotrophic lateral sclerosis (ALS). Through the PMP, 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.

By participating in the PMP, you can access your own disease progression data while also helping to find effective treatments for ALS. Unlike other research studies, enrolling in our PMP 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 the PMP 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.