ALS Therapy Development Institute

About Us

We are the world's foremost drug discovery center focused solely on ALS. As a nonprofit biotech we operate without regard to profit motive or politics. Led by people with ALS and drug development experts, we are funded by a global network of supporters unified to end ALS.

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The ALS Therapy Development Institute is our Cambridge, MA-based laboratory space, 100% dedicated to innovative ALS research. It is funded by our global community.

Our innovative science and cutting edge approach have resulted in:

  • The identification of a promising treatment for ALS: AT-1501
  • The largest database of preclinical drug study findings in animal models of ALS, with more potential treatments screened each year.
  • The world's first Precision Medicine Program designed to speed up drug discovery and therapeutic development for ALS.
  • The only database integrating genetics, demographics, voice recordings, and accelerometer-derived movement data from hundreds of people with ALS.
  • The development of algorithms and tools that assess ALS clinical progression and increase the pace of ALS clinical research.
  • The overall improvement of world-wide standards of preclinical drug evaluation in ALS.
  • The highest number of independent preclinical drug validation experiments conducted in current ALS development pipelines.

Our Mission

The ALS Therapy Development Institute and its scientists actively discover and develop treatments for ALS. We are the world's first and largest nonprofit biotech focused 100 percent on ALS research. Led by people with ALS and drug development experts, we understand the urgent need to slow and stop this disease.

ALS Therapy Development Institute

Our History

Stephen Heywood was diagnosed with ALS at age 29. After finding no treatment options, his brother, James Allen Heywood, realized that a gap existed in the preclinical development of therapeutics for ALS. In 1999, he founded the ALS Therapy Development Institute in the basement of his family's Newton, MA home to bring effective treatments to people living with ALS as quickly as possible. Although Stephen is no longer with us, this same sense of hope and family drives every decision made at the Institute today.

 

2016
  • The ALS Therapy Development Institute entered into a new research partnership with Massachusetts General Hospital/Harvard Medical School and the University of Massachusetts Medical School as part of ‘ALS ONE’, the goal of which is to identify a treatment for ALS by 2020.
  • The ALS Therapy Development Institute announced a new research collaboration with Denali Therapeutics to investigate potential new endpoints for use in ALS clinical trials. The Institute and Denali will evaluate and analyze data sets collected by the Institute through the first-of-its kind Precision Medicine Program.
  • By July 2016, 300 people were enrolled in the Precision Medicine Program, 5326 accelerometer datasets and 472 DNA fingerprints captured, 268 fibroblast lines created and 12 iPSc lines created and 248 genomes sequenced. 2016 also saw the extension of PMP, designed based on discoveries that signal the opportunity to advance subtype identification by adding specific types of data from more people with ALS than originally enrolled. In addition, findings from the first enrollment cohort signaled the opportunity to identify potential new clinical trial endpoints, such as the use of accelerometers, which may speed up all clinical trials worldwide if proven effective. Therefore, the Institutional Review Board (IRB) responsible for the PMP approved the expansion of enrollment to an additional 450 people with ALS.
  • ALS Unfiltered series was launched in cities across the US, bringing expert-led presentations and candid conversations on ALS, current drug research, clinical trials, resources and more to individuals free of charge.
2015
2016
2015
  • Launched ALS.net, a global network of affiliates who are working to build ALS awareness and funds for research taking place at the ALS Therapy Development Institute.
  • The ALS Therapy Development Institute directed via its wholly owned subsidiary, Anelixis Therapeutics, $1.5 million of Ice Bucket Challenge funds towards the development of a potential treatment for ALS that targets the innate immune system. Anelixis Therapeutics contracted Lonza, a leading global biologics API manufacturer, to develop and manufacture clinical material of anti-CD40L for future phase I patient trials.
  • Nancy O'Dell, host of Entertainment Tonight joined ALS.net as an official spokeswoman in an effort to help raise awareness and funds for ALS research. Nancy’s mother Betty lost her battle to ALS in 2008.
  • More than 250 people took part in the 3-day Tri-State Trek bike ride from Boston, MA to Greenwich, CT and raised over $800,000 for ALS research.
  • ALS.net Ambassador Sarah Coglianese launched the #WhatWouldYouGive campaign and raised over $170,000 for ALS research at the ALS Therapy Development Institute.

  • PepsiCo and Albertsons Companies donated $100,000 to fund ALS research at the ALS Therapy Development Institute as part of the #ReasonstoCare campaign.

  • ClubCorp and its family of clubs raised over $812,000 for ALS research through its philanthropic events program, ClubCorp Charity Classic.
  • By the end of 2015, the Precision Medicine Program had enrolled 230 people, captured 2200 accelerometer datasets and 200 DNA fingerprints, created 220 fibroblast lines and sequenced 140 genomes.
2014
2015
2014
  • Successfully integrated the Augie's Quest brand and team into the Institute bringing new leadership and fundraising experience
  • Successfully Enrolled 30 PALS in Phase 2A clinical trial of TDI-132 (Gilenya®).
  • Ales for ALS™ program expanded to 65 brewers and 2 beerfests, raising $300,000 for ALS research.
  • Raised $4 million through Ice Bucket Challenge phenomenon created by ALSTDI supporters Pete Frates, Pat Quinn and Anthony Carbajal.
  • Enrolled first 20 PALS into Precision Medicine Program, announced enrollment expanded to 300 thanks to Ice Bucket Challenge funding.
  • Announced $3 million in Ice Bucket Challenge funding to be spend on clinical stage programs targeting CD40L (w/Anelixis) and misfolded SOD1 (w/Neurimmune).
  • Annual leadership summit hosted by institute attracts speakers from Cytokinetics, Voyager Therapeutics, PatientsLikeMe, Polliwog Holdings, The Scripps Research Institute and the University of Florida.
2013
2014
2013
  • Entered into collaborations with 3 biotech companies on several different potential treatments for ALS; to-BBB (Netherlands), Anido Pharma (USA) and Neurimmune SA (Switzerland).
  • FDA approves Phase 2A clinical trial of TDI-132 in ALS patients. Enrollment begins in four states (Massachusetts, Georgia, California and Texas).
  • Launched the Precision Medicine Program at the Institute with the hiring of three additional scientists to the team, including Matvey Lukachev, Ph.D., a translational research expert who has been part of several teams successful at bringing drugs to market for other diseases indications.
  • 50 of the top craft brewers in the USA join Ales for ALS™ a unique beer brewing program to fund ALS research, raising $150,000 in first year.
  • Robert Sepucha, Jr., Michael M. Smith and Jennifer Gore-Dwyer appointed to the Institute's Board of Directors.
  • 9th annual leadership summit hosted by ALSTDI features speakers from the CF Foundation, UCB Pharma, Stanford University, University of California, Third Rock Ventures, to-BBB and others.
  • The Institute creates Anelixis Therapeutics as a subsidiary to help advance potential treatments for ALS in partnership with private investors.
2012
2013
2012
  • Expanded the Institute to 26,000 square feet with a move to a larger, most modern facility in Cambridge's Kendall Square. Move allows the institute to hire more scientists and take on ever bigger projects to accelerate drug development for patients today.
  • Entered into separate drug development aimed collaborations with Regenesance (Netherlands) and Neurotune (Switzerland) and a technology development collaboration with the Gladstone Institutes (USA).
  • Celebrated the 10th year of the Tri-State Trek which has grown from 16 riders to more than 400. To date the event has raised $5.1 million for ALS research.
  • Announced intent to fund and conduct clinical research program on TDI-132.
  • The annual leadership summit hosted by the Institute attracted speakers from UMass Medical, University of British Columbia, The Kauffman Foundation, the Wall Street Journal, UCB Pharma, Neurotune and Vertex Pharmaceuticals.
2011
2012
2011
  • Historic collaboration unites AD, FTD and ALS community in effort to characterize first publicly available TDP43 mouse model at TDI.
  • 7th Annual Summit includes: Stan Appel, Chris Austin, Robert Blum, Doug Onsi, Steve Perrin, Jeff Rothstein, George Scangos and Henri Termeer (pictured). Leadership Awards goto: Sharon Isles, Megan Mishork, Thomas Ohlson and Frank Provost.
  • Preclinical research on TDI-132 (fingolimod) shows it to be a potential treatment in 2011. TDI announces it will pursue clinical trial alone.
  • ALS TDI enters into important research agreement with Biogen Idec & UCB Pharma to investigate TDI-846 (aCD40L) as a potential treatment for ALS.
  • ALS TDI works with patients and families to relaunch website with new databases about clinical trials and past experiments.
  • Congressman Michael Capuano presented with Iron Horse Award during inaugural White Coat Affair gala in Boston.
  • Total revenue in 2011 is $7,997,504 and TDI is again recognized by CharityNavigator as top rated ALS org in USA.
2010
2011
2010
  • ALS TDI and Aestus Therapeutics initiate a collaboration on potential treatments for ALS.
  • 'Corey', the GeneTitan is installed in the genomics center at ALS TDI.
  • Five members of the ALS TDI Research & Development Team gave presentations at each of these meetings, a record for the Institute.
  • MDA's Augie's Quest provides more than $2.5 million in support to ALS TDI, extending its historic partnership.
  • 6th annual Leadership Summit includes presentations from Drs. Merit Cudkowicz, Gilmore O'Neil, Steve Perrin, Clive Svendsen, and Fernando Vieira. Leadership Awards presented to Steve Saling, Carla Zilbersmith, Tim LaFollete and Eric Valor.
  • Created by the Institute as an online social networking community based on Facebook, YFALS launches with PALS Corey Reich, Melissa Erickson, Michael Winston, Alyssa Reardon, Alex Grausnick, Kristen Sauer, Tom Lafollete and Meagan Mishork. Campaign draws attention of thousands through 4ALS Awareness Campaign and other events.
  • The US Department of Defense awards $1.6 million grant to ALS TDI to study disease and develop potential treatments.
  • The Institute's commitment to transparency and financial responsibility is recognized by CharityNavigator for second year in a row. Named top rated ALS organization in the country.
  • The Institute realized $8,051,161 in total public support in 2010, making it the 3rd most successful fundraising year on record.
2009
2010
2009
  • Scientists submit a paper regarding the discovery of ALS TDI 00846 to Nature Genetics entitled "From Transcriptome Analysis to Therapeutic Anti-CD40L Treatment in the SOD1 Model of Amyotrophic Lateral Sclerosis".
  • Asklepios and ALS TDI collaborate 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.
  • ALS TDI launches a "virtual lab tour" on its website and adds an "Ask a Researcher" button to every web page on its site to provide a direct link between patients, families, and researchers.
  • Michael Goldsmith, an ALS TDI Ambassador and ALS Patient, helps to inspire the creation of Major League Baseball's 4 • ALS Awareness Campaign. A joint effort between Major League Baseball, ALS TDI, MDA's Augie's Quest, the ALS Association and Project A.L.S., culminates in a League-wide awareness initiative on July 4th, 2009; the 70th anniversary of Lou Gehrig's retirement from baseball.
  • Board Member, Stanley Appel, M.D., tells a congressional hearing that "ALS is not an incurable disease, it is an underfunded one".
  • February 6th - Sean Scott, president of ALS TDI, passes away from ALS.
  • Based on significant progress made as chief scientific officer, Steve Perrin, Ph.D., is named as the Institute's CEO.
  • 5th Annual Leadership Summit is held at Hotel Marlowe in Cambridge and webcast live over the internet. Presentations were given by Drs. John McCarty, Jude Samulski, Zuoshang Xu and Erika Holzburg. Steve Perrin makes first public presentation of data on ALS TDI 00846 to peers and ALS community.
  • ALS TDI redesigns its website, and launched www.als.net in April.
  • ALS TDI raises $9,360,831, bringing the total raised and spent on research to more than $56 million over the past 10 years.
2008
2009
2008
  • ALS TDI enters into partnership with Allen Institute for Brain Science. Begins 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 SOD1 database.
  • Friends for Faye Fund, one of the first family funds started at ALS TDI, reaches $1 million in total fundraising.
  • 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.
  • Tri-State Trek event surpasses $1 million in total fundraising.
  • ALS TDI scientists publish guidelines for researchers using the G93A-SOD1 mouse model of ALS in the journal, Amyotrophic Lateral Sclerosis. The paper's title is "Design, Power and Interpretation of Studies in the Standard Murine Model of ALS".
  • RGK Foundation makes $1 million, two-year grant to ALS TDI.
  • US Department of Defense makes $1.1 million grant to ALS TDI for research.
  • 4th Annual Leadership Summit held at the Holiday Inn at MGH in Boston and features talks from Drs. Sharon Hesterlee, Stanley Appel, Michael Strong, Jean-Pierre Julien, and Tom Caltagirone. For the first time ever, the event is broadcast live over the internet, funded by and at the challenge of Jeff Cronon, an ALS patient in Atlanta, GA.
  • Prize4Life selects ALS TDI model standards as basis for its $1 million biomarker and therapeutic prize.
  • ALS TDI begins to hold Monthly Webinars. In its first year, several hundred PALS and CALS join together online each month to receive updates from the lab and ask questions about research and trials.
  • With $11,022,032 in donations in 2008, ALS TDI continued to grow capacity, science team to include more than 30 full-time professional researchers.
2007
2008
2007
  • ALS TDI becomes ALS Therapy Development Institute (ALS TDI) following the announcement of a three-year, $18 million grant from MDA's Augie's Quest.
  • 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 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. They are tasked with speeding up the processing of RNA and genotyping.
  • Steve Milne Family Fund reaches its $1 million goal. Commits to hitting its next target in half the time.
  • 3rd Annual Leadership Summit fills the room at the Museum of Science in Boston. Drs. Ray Onders, Dale Lange, and Hans Kiersted make presentations.
  • After eight years leading ALS TDI, James Heywood steps down as CEO and joins the board.
  • Steve Perrin, Ph.D., becomes the organization's first chief scientific officer.
  • ALS TDI raises an astonishing $9,687,302 in 2007 - making the Institute one of the best funded private research organizations in the world focused on a single disease.
2006
2007
2006
  • Augie Nieto, diagnosed with ALS in 2005, commits to raising $18 million and becomes chairman of the board.
  • So Much So Fast, a documentary about the Heywood's and ALS TDF, premiers at the Sundance Film Festival.
  • 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.
  • November 26th - after eight years living with ALS, Stephen Heywood, the inspiration for ALS TDF, passes away.
  • Sean Scott is named president of ALS TDF.
  • Leadership Summit expands dramatically in its second year, moving to the Hotel @ MIT. Drs. John Lincecum, Robert Bowser, and Dan Benjamin give updates on their work. The event features talks from newly hired science team members, and a screening of So Much So Fast.
  • ALS TDF has a record breaking fundraising year- recognizing $5,181,635 in donations.
2005
2006
2005
  • James Heywood articulates the Apollo Projects, an ambitious vision for ALS TDF which includes novel discovery and drug development approaches combined to move drugs into clinic faster.
  • Nearly 100 cyclists, including many PALS, participate in the Tri-State Trek, a 270-mile bike ride from Boston to NY.
  • A Biosafety Level 2 Lab is constructed at ALS TDF. The secure lab allows expansion of gene therapy and cell-based treatment pipelines.
  • The 1st Annual "A Cure is Coming!" walk is held. Dozens of patients and families walk from Lexington Battle Green to the Old North Church in Boston to raise ALS awareness.
  • ALS TDF purchases a powerful Zeiess X1 microscope and builds up its molecular imaging capabilities thanks to an allocation from the Jeff Hadley Fund.
  • Mass spectrometry core created. The goal is to monitor drug levels attained in any tissue type.
  • Passion for Life Fund, inspired by PALS Mary Lou Krauseneck, reaches its $1 million fundraising milestone.
  • 1st Annual Leadership Summit held in Cambridge at the Massachusetts Biotechnology Council followed by a lobster dinner and awards at the Heywood's home in Newton, where the Foundation first began to operate.
  • ALS TDF raises $3,509,483 - spends 79% on R&D. This makes the Foundation the largest funded R&D program exclusively focused on ALS.
2004
2005
2004
  • ALS TDF staff attend 2004 Society for Neuroscience in San Diego.
  • ALS TDF moves to its current location, a 16,000 square foot space in Cambridge's biotech hub, Kendall Square.
  • Gene Expression Core launched at ALS TDF with the purchase of a Taqman. This high-end device is capable of measuring the expression of genes, aiding in the search for biomarkers of disease.
  • Scientists at the Institute innovate a novel device known as an intrathecal catheter, which delivers potential therapeutics directly to the spinal cord of the mouse.
  • His Brother's Keeper, a book by Jonathan Weiner, chronicles Stephen’s illness ad Jamie's founding of ALS TDF.
  • ALS TDF hires industry trained discovery experts, establishing a dedicated Discovery Biology Core.
  • ALS TDF raises $3,447,963 in its 5th year of existence.
2003
2004
2003
  • 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.
  • ALS TDF raised $3,990,771 in 2003.
  • The Fran Delaney Fund hits their $1 million cumulative fundraising milestone in less than 3 years.
  • PGA Tour Golfers Jeff Julian and Tom Watson, together with Tom Watson's caddy, Bruce Edwards, start "Driving 4 Life", a fundraising effort based on golf. Campaign raises $1 million by end of the year.
  • 58 drugs evaluated throughout 2003, including 8 advanced efficacy screens.
  • Ronya Kozmetsky makes $1 million pledge to ALS TDF after the passing of her husband, George, to ALS a year earlier.
  • "Friends and Family community" grows to include 70 separate and active funds. Group surpasses $2 million in annual grassroots fundraising.
  • ALS TDF research team attends Society for Neuroscience meeting in New Orleans.
  • With a second $500,000 grant, the ALS Hope/Hobler Foundation becomes the second foundation to provide $1 million in funding to ALS TDF.
2002
2003
2002
  • ALS TDF moves across the Charles River into a larger research facility located in the Harvard Partners building.
  • Established 4 external research collaborations with academics and more than 25 partnerships with biotechs and pharmaceutical companies.
  • The Friends and Family Community, the Foundation’s main fundraising arm, doubles its income to $1.8 million.
  • 28 drugs evaluated and 3 preliminary hits in the SOD1 mouse model.
  • ALS TDF screens Dapsone, WHI-P131, Celebrex, Chlorpromazine, Copasone, Gusperimus, Quinacrine, Riluzole, Thalidomide, CVF, DDC llG, Indomethacin, Leflunomide, Memantine and others.
  • Ten ALS 101 sessions held throughout the year.
  • An anonymous donor provides a $300,000 grant funding the development of a new bioinformatics systems at ALS TDF.
  • Alex & Brit d'Arbeloff make $1,000,000 donation to ALS TDF.
  • ALS TDF raised $4,335,417 in 2002.
2001
2002
2001
  • ALS TDF opens first lab inside New England Medical Center.
  • An anonymous donor makes $1 million grant to ALS TDF.

  • 27 drugs tested in SOD1 mouse; 2 hits.
  • ALS TDF's Founder and CEO, James Allen Heywood, begins to hold "ALS 101" sessions for ALS patients. The goal is to provide information and encourage discussion among stakeholders regarding drug development.
  • ALS TDF receives approval to conduct the world’s first stem cell trial for ALS. Stephen Heywood and 2 others participate.
  • ALS Hope/Hobler Foundation makes $500,000 gift to ALS TDF. The Foundation renames its main drug-screen program the "ALS Hope Drug Discovery Center".
  • Sean Forrester Scott joins ALS TDF as volunteer following his mother's diagnosis. ALS runs in his family and Sean himself has a 50/50 chance of developing it.
  • 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 raised $4,018,069 in 2001.
  • www.als-tdf.org gets facelift; designed to include results from research trials and better connect patients and researchers together.
2000
2001
2000
  • The Foundation applies for Phase 1 Stem Cell Transplant trial. If approved, it would be the first of its kind for ALS in the world.
  • "Neurobase" developed to track leads and gather information to support research and development.
  • "ALS In-Vivo Screening Program" launched in the Fall. Scaled up to become largest in the world.
  • The virtual web-based bulletin board, "The ALS Forum", is launched.
  • Alex & Brit d'Arbeloff make $300,000 donation to ALS TDF.
  • ALS TDF's "Friends & Family Community" goes live. Web based fundraising community launched as one of the first in the no-profit world.
  • Will Hubben, pALS established the ALS/MND Research Mail List (later, after his death, it is renamed in honor as the Hubben Digest). Within its first year, the mailing list grows to over 1,000 email addresses.
  • Heywood brothers and ALS TDF featured in New Yorker magazine exposé "Curing the Incurable".
  • 1st annual "Night at Fenway" event held.

  • ALS TDF raised $1,562,126 in 2000.
1999
2000
1999
  • James Allen Heywood starts the ALS Therapy Development Foundation three months after his brother Stephen is diagnosed with amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease). A nonprofit biotech, ALS TDF is dedicated to drug development for patients today. Stephen is the first donor, writing a check for $10,000 and telling his brother to "get going".
  • First therapy concept developed: replace EAAT2 protein using gene therapy.
  • Alex & Brit d'Arbeloff make $100,000 donation to ALS TDF.
  • The Uptake Therapy Project kicked off in partnership with Dr. Jeffrey Rothstein at John Hopkins University.
  • First annual Belly Dance Extravaganza takes place at Karoun in Newton, MA.
  • www.als-tdf.org is launched.

  • ALS TDF raises $434,761 in its first year of existence.

 

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Favorable Mentions

In my opinion, the research ALS TDI is doing will bring us closer to a cure than anything else going on.

Bob Hebron

Bob Hebron

We have met the people who work at ALS TDI and their passion shines through, their personal connections to this disease drive them. We believe they are one of the most progressive and effective insitutes around, and they will be part of the cure.

Jeremy Truman

Jeremy Truman

Jose supported ALS TDI because "of the Institute's single-minded focus on finding a solution for ALS, and doing so as a nonprofit biotech company with top-tier talent. Clear Objective + Solid Strategy + Stellar People = Desired Results."

José Cofiño (PALS, Died 2017)

José Cofiño (PALS, Died 2017)