ALS Therapy Development Institute

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The ALS Therapy Development Institute (ALS TDI) is the world's foremost drug discovery lab focused solely on ALS. As a nonprofit biotech we operate without regard to profit or politics.

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 develop effective treatments for ALS.

The ALS Therapy Development Institute (ALS TDI) is the world's foremost drug discovery lab focused solely on ALS. As a nonprofit biotech we operate without regard to profit or politics.

Our Impact

With preclinical, clinical, and translational research all happening under one roof, we rapidly pursue the best ideas for slowing, stopping, and ending ALS. Our cutting-edge approach has resulted in:

The rigorous screening of over 400 potential treatments for ALS—more than any other ALS research lab in the world.
The world's first Precision Medicine Program, a partnership with people living with ALS, designed to speed up drug discovery and therapeutic development for ALS.
ALS TDI becoming the first nonprofit biotech in any disease to develop a potential treatment, AT-1501, and bring it from our own labs, through FDA review and into clinical trial.
The improvement of worldwide standards in preclinical drug screening in models of ALS.

Learn About Our Research Approach

Why We Started

Stephen Heywood was diagnosed with ALS at age 29. After finding no treatment options, his brother, Jamie Heywood, realized that a gap existed in the preclinical development of therapies for ALS. In 1999, Jamie founded the ALS Therapy Development Foundation, now ALS TDI, in the basement of his family's Newton home in an effort to bridge this gap and bring effective treatments to people with ALS as quickly as possible.

Although Stephen is no longer with us, this same sense of hope, family and innovation drives every decision made at ALS TDI today.

Explore Our 20-Year History

Video: Meet the Team Working to End ALS

Meet the team working to end ALS at the ALS Therapy Development Institute (ALS TDI). When you make a donation, you are helping us get closer to finding cures for this devastating disease.

What We Did In 2020

Goal: We acquired a compound library of 36,000 small molecules that will be screened rapidly in ALS-related cells to identify better quality leads, earlier on in the drug screening process.

Accomplishment: We began to screen drugs from the compound library. The drugs are screened using neural cells derived from human induced pluripotent stem cells (iPSCs). Some of the early hits have been advanced to lead discovery using in vivo models.

Goal: We will establish a zebrafish model of C9orf72-mediated ALS in collaboration with researchers at the University of Sheffield in the United Kingdom.

Accomplishment: We imported a zebrafish model of C9orf72-mediated ALS in collaboration with researchers at the University of Sheffield in the United Kingdom to characterize and optimize for drug screening in house.

Goal: We will enroll 100 more people in our Precision Medicine Program (PMP), allowing ALS TDI researchers to study data on symptom progression, genetics and other risk factors, biomarkers, and interventions to enhance the ALS drug development pipeline and to make clinical trials faster.

Accomplishment: We enrolled 85 people in our PMP, enabling participants to access helpful tools while allowing ALS TDI researchers to study data on symptom progression, genetics and other risk factors, biomarkers, and interventions.

And, we collaborated with Google to optimize the application of digital symptom measurement tools, like accelerometers and voice recordings collected from the PMP. Our goal is to ready these types of tools for use in ALS clinical trials.

And we did this!

Accomplishment: In September 2020, ALS TDI scientists published a paper in Frontiers in Pharmacology, where we shared the discovery of a previously unexplored drug target for C9ORF72-mediated ALS.

2021 Accomplishments

2022 Goals

What We Did

What We're Doing

We discovered "redox metabolism modulators" – a collection of drug-like molecules that we believe can protect against ALS related cell death.

We will perform further in vivo experiments to identify and optimize a lead small-molecule drug candidate to advance toward clinical testing.

We worked to further unlock the potential of our new drug target discovery, Type-1 PRMTs, for C9orf72-related ALS. We began testing treatments against this target, with the hope of identifying one that we can advance to animal testing. Findings may also be relevant to other forms of ALS.

We will work to validate up to five new animal models of ALS to test Type-I PRMT inhibitors. These include models that will allow us to further test their potential to treat C9orf72-related ALS, as well as investigate whether they may be effective in other forms of the disease.

We announced a partnership with Dr. Richard Bedlack and the Duke ALS Clinic to enroll people who represent the rare instances of ALS reversals into the Precision Medicine Program (PMP). Our goal is to learn more about the underlying biological pathways behind these possible reversals and to use this understanding to inform treatment discovery and development.

The PMP will continue enrolling and analyzing the data of individuals who represent ALS reversals. By comparing this data with those of fastprogressing, slow-progressing, and other variants of ALS, we hope to identify biological differences that may reveal potential drug targets or other avenues for treatment.

We continued to collaborate with Google to apply a voice scoring machine learning algorithm to recordings contributed by PMP participants. We began sharing these scores with participants to give them a numerical measure of their own speech wellness.

The algorithm scores will be shared with each participant to give them a numerical measure of their own speech wellness. We will also share the algorithms with the ALS research and clinical communities so that these new tools can be used as possible biomarkers of ALS disease progression.

We tested 26 treatments in animal models of ALS. Those that showed potential will be assessed for tolerability, side effects, and efficacy. The treatments that show the most promise will be further advanced - allowing us to continue to fuel our pipeline.

In addition to screening possible Redox Metabolism Modulator drug candidates in animal models of ALS, we will also continue to fuel the pipeline through in vivo screens of more than 20 other promising drug candidates.

We Put Your Money Where Our Research Is

87 cents of every dollar donated goes directly to finding treatments and cures at ALS TDI, compared to the average 75 cents at other research labs.

Charity Navigator has awarded ALS TDI its highest rating—four stars—for a record six years in a row. This exceptional designation sets ALS TDI apart from its peers.

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