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At the ALS Therapy Development Institute (ALS TDI), our mission is to End ALS. The only way to achieve this is through innovative research. Our scientific programs reflect our commitment to exploring multiple strategies, attacking ALS from every angle until, one day, the therapeutic needs of every person living with ALS are met completely.
Take a look at the programs that, with your help, we have built and continue to grow each year, since our foundation.
Look how we've grown
  •   In-home Human Blood Collection for Biomarker Discovery 2019
    • ALS TDI expands our Precision Medicine Program by including an in-home blood collection protocol. Collecting blood samples improves capacity to discover blood-based biomarkers for ALS.
  •   Preclinical Electrophysiology 2019
    • ALS TDI begins looking closely at electrophysiology in mouse models to examine disease progression and to determine the efficacy of potential treatments at slowing ALS. Electrophysiology is the study of electrical properties of cells and tissues.
  •   A.I. Application for Clinical Assessment 2018
    • ALS TDI partners with with experts in artificial intelligence to analyze the “big data” collected from our PMP, making significant strides towards the development of unbiased, quantitative and sensitive measures of ALS disease progression.
  •   Human Induced Pluripotent Stem Cell Biology 2016
    • ALS TDI begins to reprogram cells from the PMP to iPSCs to provide a nearly unlimited source of cells that can be differentiated to cell types of interest, including motor neurons, microglia, oligodendrocytes, and skeletal muscle.
  •   Human Fibroblast Banking 2015
    • ALS TDI collects and banks skin samples from participants enrolled in our PMP. These types of cells can be reprogrammed into induced pluripotent stem cells (iPSCs).
  •   Human Genome Sequencing 2014
    • Using data gathered from our Precision Medicine Program (PMP), ALS TDI takes a closer look at genetic causes of ALS through whole genome sequencing.
  •   Clinical Drug Development Operations 2012
    • Based on promising preclinical results, ALS TDI begins operations to invent a drug for humans that would target CD40lg.
  •   Misfolded SOD1 Disease Biology Discovery 2009
    • ALS TDI examines mechanisms of SOD1-related ALS, a genetic form of the disease which causes superoxide dismutase protein to misfold and accumulate into clumps (aggregates).
  •   Whole Transcriptome Analyses (mouse and human) 2006
    • ALS TDI takes a closer look at RNA and gene expression in the cells of both mice and people with ALS.
  •   Assessment of Alternative Animal Models (TDP43, C9ORF72, Profilin1, SOD1) 2005
    • ALS TDI begins to explore other models of ALS with different genetic mutations. Since ALS is complex, it is important to develop and validate more models in which to test potential treatments.
  •   Cell Biology for Compound Mechanism Validation 2004
    • ALS TDI works to demonstrate that the potential treatments we are testing are having an impact on their intended biological target.
  •   Preclinical Pharmacodynamics 2003
    • ALS TDI looks more closely at the effects that different drugs have on the body of the SOD1 mouse model and their mechanism of action.
  •   Preclinical Pharmacokinetics 2002
    • ALS TDI looks at how the drugs being tested move throughout the body of the SOD1 model including the study of how substances are absorbed, distributed, metabolized, and excreted.
  •   Efficacy Testing in SOD1 Mice 2001
    • ALS TDI brings SOD1 mice in-house to begin screening drugs in an effort to find treatments to slow or stop ALS disease progression. The SOD1 mouse model is the most widely used mouse model in ALS.
Our Milestones
Working to End ALS
  • In May 2022, Eledon Pharmaceuticals announced that tegoprubart – a drug invented at ALS TDI – completed phase 2 trials. The drug was demonstrated to be safe and well-tolerated in people with ALS and hit its target pathways in humans.
  • Google and ALS TDI announce a preprint publication of a paper describing their research to create artificial intelligence tools for scoring ALS-related symptom severity.
Moving to Watertown
  • With the science programs having grown immensely, ALS TDI moves to up and coming biotech hub, Watertown, MA. This new space allowed ALS TDI to tailor a lab around, what is today, the most comprehensive research program in ALS.
  • ALS TDI publishes paper on Roles of Arginine Methylation in C9orf72-Mediated ALS and FTD.
  • ALS TDI begins collaboration with Duke ALS clinic to study ALS reversals.
  • Fernando G. Vieira, M.D. appointed as Chief Executive Officer
An Unprecedented Year
Always Pushing Forward
  • ALS TDI enhances its longitudinal blood-based biomarker discovery program to identify biomarkers of disease progression.
  • ALS TDI publishes a paper showing evidence of the neuroprotective role of SOD1 protein aggregates and the toxic role of soluble misfolded proteins in the SOD1 mouse model.
  • Augie's Quest donates the over $4.5 million raised for them through Orangetheory's #AQuestforMoreLife campaign to ALS Research at ALS TDI.
First of Our Kind
  • ALS TDI becomes the first nonprofit biotech in any disease to discover and develop a potential treatment, tegoprubart, bringing it from our labs, through FDA review, and into clinical trial.
  • ALS TDI partners with Google to leverage their expertise in artificial intelligence to analyze the “big data” collected from our Precision Medicine Program, making significant strides towards the development of unbiased, quantitative, and sensitive measures of ALS disease progression.
  • In collaboration with Neurimmune, ALS TDI publishes a paper supporting the further development of α-miSOD1 antibody as a candidate treatment for forms of ALS involving misfolding of SOD1.
  • In collaboration with Virginia Tech, ALS TDI publishes a paper on findings demonstrating that sensory neurons are directly affected by two ALS-inducing factors, suggesting important roles for this neuronal subpopulation in ALS-related pathogenesis.
  • Dr. Fernando Vieira becomes Chief Scientific Officer at ALS TDI in August 2018.
Heating Up ALS Research
  • ALS TDI co-hosts the 2017 International ALS Symposium with the ALS Hope Foundation in December 2017 in Boston.
  • ALS TDI publishes a paper validating the efficacy of Copper ATSM (CuATSM) at slowing down ALS disease progression in the SOD1 mouse model.
  • The #ALSPepperChallenge is started by the Haberstroh Family in honor of Patty Haberstroh.
Uniting for the Fight
  • ALS TDI enters into a new research partnership with Massachusetts General Hospital/Harvard Medical School and the University of Massachusetts Medical School as part of “ALS ONE”, with the goal of identifying a treatment for ALS by 2020.
  • ALS TDI announces a new research collaboration with Denali Therapeutics to investigate potential new endpoints for use in ALS clinical trials. ALS TDI and Denali will evaluate and analyze data sets collected through ALS TDI's first-of-its kind Precision Medicine Program.
  • By July 2016, 300 people are enrolled in the Precision Medicine Program, 5326 accelerometer datasets and 472 DNA fingerprints are captured, 268 fibroblast lines are created, 12 iPSc lines are created, and 248 genomes are sequenced. Findings from the first enrollment cohort signal the opportunity to identify potential new clinical trial endpoints. The Institutional Review Board (IRB), responsible for the Precision Medicine Program, approve the expansion of enrollment to an additional 450 people with ALS.
Leading By Example
  • Via its wholly-owned subsidiary Anelixis Therapeutics, ALS TDI directs $1.5 million of funds raised from the Ice Bucket Challenge towards the development of a potential treatment for ALS that targets the innate immune system.
  • Anelixis Therapeutics contracts Lonza, a leading global biologics Active Pharmaceutical Ingredient (API) manufacturer, to develop and manufacture clinical material of anti-CD40L for future Phase I patient trials.
  • By the end of 2015, the Precision Medicine Program enrolls 230 people, captures 2200 accelerometer datasets and 200 DNA fingerprints, creates 220 fibroblast lines, and sequences 140 genomes.
  • ALS TDI publishes a paper detailing a new video protocol developed by in-house scientists to evaluate disease progression in the SOD1-G93A Mouse Model of ALS.
  • ALS TDI publishes a paper highlighting the challenges of pharmacological interventions of cellular stress responses in animals with ALS.
  • The ClubCorp Charity Classic raises over $800,000 for ALS research through its philanthropic events program.
  • YFALS Ambassador Sarah Coglianese launches the #WhatWouldYouGive campaign and goes on to raise over $600,000.
  • PepsiCo and Albertsons Companies donate $100,000 as part of the #ReasonstoCare campaign.
Ice Buckets Make Waves
  • The Ice Bucket Challenge, created and promoted by ALS TDI supporters Pete Frates, Pat Quinn, and Anthony Carbajal, goes viral and raises $4 million for ALS TDI.
  • ALS TDI announces that $3 million of Ice Bucket Challenge funding will be spent on clinical stage programs targeting CD40L (via Anelixis Therapeutics) and misfolded SOD1 (via Neurimmune).
  • The Precision Medicine Program enrolls its first 20 people with ALS and increases enrollment to 300, thanks to funds raised in the Ice Bucket Challenge.
  • ALS TDI publishes a paper examining the emerging disease phenotype of a transgenic mouse model that overexpresses a mutant human TDP-43 (hTDP-43) gene under mouse prion promoter control.
  • ALS TDI publishes a paper revealing that dexpramipexole is ineffective in two models of ALS-related neurodegeneration.
  • Augie's Quest joins forces with ALS TDI, bringing new leadership, experience, and funding to the cause.
The Telemedicine Era Begins
  • ALS TDI enters into collaborations with three biotech companies on several different potential treatments for ALS: to-BBB (Netherlands), Anido Pharma (USA), and Neurimmune SA (Switzerland).
  • The Food and Drug Administration (FDA) approves the Phase 2A clinical trial of TDI-132 in ALS patients and enrollment begins in four states (Massachusetts, Georgia, California, and Texas).
  • ALS TDI launches the first ALS-focused Precision Medicine Program, partnering with people with ALS to share and gather data on medical and family histories, genetics, biomarkers, and patient cell biology in order to better understand ALS.
  • ALS TDI creates Anelixis Therapeutics as a subsidiary to help advance potential treatments for ALS by pursuing partnerships with private investors.
Moving On Up
  • ALS TDI moves to a larger, more modern facility in Cambridge's Kendall Square. The 26,000 square foot lab space allows ALS TDI to hire more scientists, take on larger projects, and accelerate drug development.
  • ALS TDI enters into drug development collaborations with Regenesance (Netherlands) Neurotune (Switzerland) and a technology development collaboration with the Gladstone Institutes (USA).
  • ALS TDI announces its intent to fund and conduct a clinical research program on TDI-132.
Pursuing Clinical Trials
  • A historic collaboration unites the Alzheimer's, frontotemporal dementia, and ALS communities in an effort to characterize the first publicly available TDP43 mouse model at ALS TDI.
  • After preclinical research shows TDI-132 (fingolimod) to be a potential treatment, ALS TDI announces its intent to pursue a clinical trial.
  • ALS TDI enters into a research agreement with Biogen Idec and UCB Pharma to investigate TDI-846 (aCD40L) as a potential treatment for ALS.
Gene Titan Joins the Lab
  • ALS TDI and Aestus Therapeutics initiate a collaboration on potential treatments for ALS.
  • A brand new GeneTitan, named “Corey” after YFALS Ambassador Corey Reich, is installed in the genomics center at ALS TDI.
  • ALS TDI publishes a paper in Nature Genetics regarding the discovery of potential therapeutic ALS TDI 00846.
  • Augie's Quest, then associated with the Muscular Dystrophy Association, provides more than $2.5 million in support to ALS TDI.
  • The US Department of Defense awards a $1.6 million grant to ALS TDI to study and develop potential treatments for ALS.
Coining our Call to Action
  • ALS TDI collaborates with Asklepios on the development of novel adeno-associated viral vectors to be used in drug development research.
  • Applied Proteomics becomes a strategic partner in ALS TDI's effort to characterize protein expression changes associated with disease onset and progression.
  • Sean Scott, president of ALS TDI, passes away from ALS on February 6th, 2009. His picture is placed in the lab and work continues on in his memory.
  • ALS TDI publishes a paper on study results which show no potential benefits of chronic lithium dosing in G93A-SOD1 mouse model of ALS.
  • Board Member Stanley Appel, M.D., coins the phrase "ALS is not an incurable disease, it is an underfunded one" in a congressional hearing regarding ALS therapeutic development.
  • Steve Perrin, Ph.D., is named Chief Executive Officer of ALS TDI, building on his significant progress made as chief scientific officer.
Tri-State Trek Hits $1 Million
  • ALS TDI partners with the Allen Institute for Brain Science and begins a massive in situ hybridization experiment.
  • ALS TDI partners with 6 clinics to collect blood and tissue samples from ALS patients for cross-referencing against gene expression pathways identified in the SOD1 database.
  • ALS TDI collaborates with California Stem Cell. Work suggest that stem cells can engraft and survive in spinal cord of SOD1 mice. Groups extend collaboration work to develop cell-based drug delivery tools to treat ALS.
  • ALS TDI's science team grows to include more than 30 full-time professional researchers.
  • ALS TDI publishes a paper detailing guidelines for researchers using the G93A-SOD1 mouse model of ALS. Based on this work, Prize4Life awards a $1 million biomarker and therapeutic prize to ALS TDI.
  • ALS TDI's signature event, the 270-mile Tri-State Trek, surpasses $1 million in total fundraising.
  • US Department of Defense makes a $1.1 million grant to ALS TDI for ALS research.
  • RGK Foundation makes $1 million, two-year grant to ALS TDI.
  • Friends for Faye Fund, one of the first family funds started at ALS TDI, reaches $1 million in total fundraising.
From Foundation to Institute
  • The ALS Therapy Development Foundation (ALS TDF) is renamed the ALS Therapy Development Institute (ALS TDI).
  • ALS TDI becomes one of the best funded private research organizations in the world to be focused on a single disease.
  • ALS TDI completes the first ever comprehensive gene expression database of the SOD1 mouse in partnership with Gene Logic.
  • ALS TDI purchases a Laser Capture Microdisection (LCM) to investigate cell-specific changes in gene and protein expression.
  • ALS TDI launches a novel gene therapy pipeline and partners with Microbix to develop targeted vectors.
  • "Yoda" and "Darth", two BioMek automation robots, become permanent members of the team at ALS TDI and are tasked with speeding up the processing of RNA and genotyping.
  • After eight years leading ALS TDI, James Heywood steps down as Chief Executive Officer and joins the board.
  • Steve Perrin, Ph.D., becomes the organization's first Chief Scientific Officer.
  • The Steve Milne Family Fund reaches its $1 million fundraising goal for ALS TDI.
Committed to the Cure
  • The In Vitro Validation Team is organized at ALS TDF. Their primary charge is to validate the activity of small molecule, protein biologics, and gene therapy products prior to advancing them into full in-vivo (animal) testing for efficacy.
  • So Much So Fast, a critically-acclaimed documentary about the Heywood family and ALS TDF, premiers at the Sundance Film Festival.
  • After eight years living with ALS, Stephen Heywood passes away on November 26th, 2006. He remains the inspiration and motivating force behind ALS TDF to this day.
  • Sean Scott is named President of ALS TDF.
  • Augie Nieto becomes Chairman of the Board at ALS TDF. Augie, a fitness mogul diagnosed with ALS in 2005, commits to raising $18 million in three years for ALS research at ALS TDF.
Leveling Up in the Lab
  • ALS TDF becomes the largest nonprofit research and development (R&D) program exclusively focused on ALS.
  • A Biosafety Level 2 Lab is constructed at ALS TDF allowing for further expansion of gene therapy and cell-based treatment pipelines.
  • ALS TDF purchases a powerful Zeiess X1 microscope and builds up its molecular imaging capabilities, with funding from the Jeff Hadley Fund.
  • The Mass Spectrometry Core created to monitor drug levels attained in any tissue type.
  • The Passion for Life Fund, inspired by a person with ALS, Mary Lou Krauseneck, reaches its $1 million fundraising milestone.
Express Yourself - Gene Expression Core
  • The Gene Expression Core is launched at ALS TDF after the purchase of a Taqman, a high-end device capable of measuring gene expressions and aiding in the search for disease biomarkers.
  • ALS TDF moves to a new location, a 16,000 square foot lab and office space in the Cambridge biotech hub, Kendall Square.
  • Scientists at ALS TDF innovate a novel device known as an intrathecal catheter, which delivers potential therapeutics directly to the spinal cord of the mouse.
  • ALS TDF hires industry trained drug discovery experts, establishing a dedicated Discovery Biology Core.
Advanced Efficacy Screening
  • The lab tests 58 potential treatments, including advanced efficacy screenings of 8 drugs.
  • ALS TDF executes a combination drug trial of Ritonavir and Hydroxyurea at the University of San Francisco in partnership with the Larry L. Hillblom Foundation.
  • The ALS Hope/Hobler Foundation grants ALS TDF another $500,000, becoming the second foundation to provide $1 million in funding to ALS TDF.
  • Ronya Kozmetsky makes a $1 million pledge to ALS TDF after the passing of her husband, George.
On the Move
  • ALS TDF moves across the Charles River into a larger research facility located in the Harvard Partners building.
  • ALS TDF establishes four external research collaborations with academics and more than 25 partnerships with biotechs and pharmaceutical companies.
  • The lab tests 28 potential treatments in the SOD1 mouse model.
  • An anonymous donor provides a $300,000 grant funding the development of a new bioinformatics systems at ALS TDF.
  • Alex and Brit d'Arbeloff make a $1,000,000 donation to ALS TDF.
First Lab Opened
  • ALS TDF opens its first lab inside the New England Medical Center. New funding allows the lab to move to a larger space at the Massachusetts College of Pharmacy by the end of the year.
  • ALS TDF's Founder and Chief Executive Officer, James Allen Heywood, begins to hold "ALS 101" sessions for people with ALS with the goal of providing information and encouraging discussion about drug development.
  • The lab tests 27 potential treatments in the SOD1 mouse model.
  • ALS TDF receives approval to conduct the world's first stem cell trial for ALS. Stephen Heywood and two other people with ALS participate.
  • The ALS Hope/Hobler Foundation makes a $500,000 gift to ALS TDF. ALS TDF renames its main drug-screening program the "ALS Hope Drug Discovery Center."
Largest In-Vivo Screening Program
  • ALS TDF establishes the world's largest in-vivo screening program, using mouse models to evaluate compound formulation and dosage.
  • ALS TDF applies for a Phase 1 Stem Cell Transplant trial. If approved, it would be the first of its kind for ALS in the world.
  • ALS TDF develops Neurobase to track drug leads and gather information to support research and development.
  • The Heywood brothers and ALS TDF are featured in New Yorker magazine exposé, Curing the Incurable.
Doors are Open
  • James Allen Heywood starts the ALS Therapy Development Foundation three months after his brother Stephen Heywood is diagnosed with ALS. As a nonprofit biotech, ALS TDF dedicates itself to drug development for patients today.
  • Stephen Heywood becomes ALS TDF's first donor, writing a check for $10,000 and telling his brother to "get going."
  • Alex and Brit d'Arbeloff make a $100,000 donation, getting ALS TDF off the ground.
  • The lab's first therapy concept is developed: replace EAAT2 protein using gene therapy.