ALS Therapy Development Institute
|Purpose||"to discover and develop effective treatments for ALS, Lou Gehrig's disease."|
|Affiliations||International Alliance of ALS/MND Associations|
The ALS Therapy Development Institute (ALS TDI) is a non-profit biotechnology research organization focused on finding effective treatments for amyotrophic lateral sclerosis (ALS). With a staff including more than 30 scientists, it operates the world's largest research and development program focused on ALS.
ALS TDI was founded as the ALS Therapy Development Foundation (ALS TDF) in 1999 by James Heywood, Robert Bonazoli, and Melinda Marsh Heywood after James' brother, Stephen Heywood, was diagnosed with the disease. The foundation was initially funded through a donation from Stephen, as well as one from Alex and Brit d'Arbeloff. The Foundation's first therapy concept was to replace EAAT2 protein using gene therapy.
In 2001, the Foundation opened its first lab at the New England Medical Center and tested 27 drugs in the SOD1 mouse with two successes. The organization also received approval to conduct the world’s first stem cell trial for ALS. Then, in 2004, the Foundation moved to a 16,000-square-foot (1,500 m2) location in Cambridge, Massachusetts with an in-house lab. ALS TDF constructed a biosafety level 2 lab in 2005, allowing for the expansion of "gene therapy and cell-based treatment pipelines."
In 2005, the Foundation became the beneficiary of the Tri-State Trek, a 270-mile bike ride from Boston to New York founded in 2003. Fitness pioneer and ALS patient Augie Nieto also became chairman of the board. The Trek has grown to include over 400 participants and has raised more than $7 million for research.
Heywood served as founding director and CEO of the Foundation through 2007, when he joined the board. Following Heywood's move to the Board, Sean F. Scott, who ALS runs in his family, was named CEO. Augie Nieto worked with Scott, as well as with Sharon Hesterlee, the Vice President of Translational Research at MDA, to bring together the two organizations in 2007. As a result, TDI would received $36 million from MDA through Nieto's initiative, Augie's Quest. In addition, the organization replaced the "Foundation" part of its name with "Institute".
Steven Perrin, previously only Chief Scientific Officer, was appointed CEO in 2009 following the death of Scott. In addition, Stanley Appel, a board member, tells a congressional hearing that "ALS is not an incurable disease, it is an underfunded one".
MDA and Augie's Quest contributed another $2.5 million to the Institute in 2010. The Young Faces of ALS (YFALS) program was also created.
In 2011, the Institute moved to a new 26,000-square-foot (2,400 m2) facility, also in Cambridge, allowing for the hiring of more scientists and a bigger lab.
In 2016, the Institute announced the ALS One partnership with Massachusetts General Hospital/Harvard Medical School and the University of Massachusetts Medical School to find a treatment for ALS within four years.
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The Institute has raised and spent more than $100 million on research into effective treatments for ALS and practices open-source science. After the discovery that the multiple sclerosis drug Gilenya might also be a treatment for ALS, the Institute enrolled 30 people in a Phase 2A clinical trial the drug in 2013.
ALS TDI launched the Precision Medicine Program in partnership with Denali Therapeutics in 2013 "to identify subgroups of ALS, potential treatments for them using patient data, genomics and iPS cell technology". By 2015, over 300 people had been registered and pre-screened in the Program, significantly funded by money raised in the Ice Bucket Challenge.
The Institute has performed independent validation of previously published pre-clinical efficacy studies in the mutant SOD1 G93A mouse model. Using large cohorts and rigorous trial design, ALS TDI demonstrated that several promising pre-clinical compounds such as minocycline and creatine, which showed dramatic effects in high-profile publications, could not be independently reproduced. Through "statistical analysis of the large gene expression database from the SOD1 G93A mouse model," ALS TDI was able to develop AT-1501, a potential effective treatment for the disease. AT-1501, an antibody that acts as an immuno-suppressing drug, targets and protects nerves from ALS progression.
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- Seiffert, Don (2016-04-12). "Cambridge biotech aims at a better way to test new drugs for ALS". Boston Business Journals. American City Business Journals. Retrieved 2017-01-13.
- Leuty, Ron (2016-04-04). "Strength in numbers — or why a hot, young biotech and a nonprofit are taking aim at new ALS targets". Boston Business Journal. American City Business Journals. Retrieved 2017-01-14.
- Riemer, Emily (2015-08-04). "ALS research already benefiting from Ice Bucket Challenge's return". WCVB. Retrieved 2017-01-14.
- "AT-1501". ALS Therapy Development Institute. Retrieved 2017-01-14.