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.

2019

2020

What We Said

Goals for 2020

What We Did

Accomplishments

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.

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.

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

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.

We will begin analyzing samples from the PMP’s In-Home Blood Collection Initiative to discover and validate potential biomarkers.

We began analyzing samples from the PMP’s In-Home Blood Collection Initiative to discover and validate blood-based biomarkers of ALS disease progression. We explored neurofilament light chain, among other biomarkers, because there is evidence that this could predict rate of ALS progression.

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.

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 did this!

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.

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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