ALS Therapy Development Institute

ALS Therapy Development Institute
Abbreviation ALS TDI
Founded 1999
Founder
Type non-profit biotech
Purpose "to discover and develop effective treatments for ALS, Lou Gehrig's disease."[1]
Location
Key people
Affiliations International Alliance of ALS/MND Associations
Website ALS TDI

The ALS Therapy Development Institute (ALS TDI) is a non-profit biotechnology company seeking effective treatments for amyotrophic lateral sclerosis (ALS). It is a registered 501(c)(3) non-profit organization in the United States. It operates the world's largest research and development program focused on ALS.

ALS TDI has conducted pre-clinical investigations (using the G93A ALS mouse model) and efficacy studies on more potential treatments than any other research facility. Many of the findings from these tests are posted online with data freely available. The institute has worked with dozens of collaborators from both academia and industry, most recently entering into agreements with Gene Logic, Microbix, Aptagen and the Allen Institute for Brain Science. Through its partnerships and the organization of significant research talent at its facility, the Institute is focusing resources on using powerful genomic and proteomic information to stop the disease.

History

In 2007, ALS TDI changed its name to ALS TDI from ALS Therapy Development Foundation (ALS TDF) corresponding with a major grant and research partnership with the MDA's Augie's Quest. ALS TDI was founded in 1999 by James Heywood, Robert Bonazoli, and Melinda Marsh Heywood after James' brother, Stephen Heywood, was diagnosed with the disease. Dr. Tennore Ramesh joined ALS-TDF when his sister in law was diagnosed with ALS and set up the research facility and served as chief scientific officer from inception until 2003.[2] James Heywood served as the d'Arbeloff Founding Director and CEO of the Institute from 1999–2007, when he joined the board. Following Heywood's move to the Board, Sean F. Scott was named the Institute's President. Scott's family has a history of ALS caused by an inherited form of the neuro-degenerative disease. Augie Nieto, a fitness pioneer and ALS patient himself, worked with Scott and MDA VP of Translational Research, Sharon Hesterlee, to bring together the MDA and ALS TDI in 2007. Today, the Institute is run by Steven Perrin, Ph.D., a genomic expert with more than 15 years of experience in the biotech and pharma industries.

Perrin leads a team of more than 30 industry trained scientists and technicians that conduct research in the Institute's 16,000-square-foot (1,500 m2) facility in biotech rich Cambridge, Mass. Several additional organizations are in the process of mimicking the ALS TDI model, including the Scleroderma Foundation, the Myelin Repair Foundation, Autism Speaks, Michael J. Fox Foundation, and CHDI, an organization searching for medicines to treat Huntington's disease.[3] The Institute has raised and spent nearly $60 million on research into effective treatments for ALS and published several works that have helped to further the field of neuro-degenerative research, including:

ALS, also known as Lou Gehrig’s disease, is a neuro-degenerative disorder that paralyzes the body but leaves the mind intact. ALS is not rare; 350,000 patients have it worldwide. Patients live on average two-to-five years following diagnosis. There is no cure and no effective treatment.

ALS TDI practices open-source science and maintains a patient and research-focused discussion forum. Some of the Institute's funding comes from grassroots fundraising events organized by ALS patients, their families and supporters.

The Institute, and the story of its origins were captured by the award-winning documentary "So Much So Fast".

In 2014, Augie's Quest officially transitioned from MDA to ALS TDI.[6]

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.[7]

Evaluation

ALS TDI 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.

However, many ALS researchers have raised questions about the utility and predictive power of the SOD1 G93A familial ALS mouse model for understanding and finding drugs to treat sporadic ALS in humans. A critical issue is whether the pathogenesis of the SOD1 G93A mutation (one of 120 in the SOD1 gene) is idosyncratic and further, whether pathogenesis caused by familial ALS SOD1 mutations (<4% of cases) is similar to sporadic ALS (>90% of cases). Critically, no pre-clinical compound, even those validated by ALS TDI, have actually proved beneficial even in early stage clinical trials.

References

  1. ↑ "Charity Report for the ALS Therapy Development Institute". Better Business Bureau. 2014.
  2. ↑ http://www.als.net/aboutus/ourstory.asp
  3. ↑ http://journals.lww.com/neurologynow/Fulltext/2010/06050/The_CEO_of_ALS_Research.11.aspx
  4. ↑ "Design, power, and interpretation of studies in the standard murine model of ALS". Amyotroph Lateral Scler 9 (1): 4–15. 2008. doi:10.1080/17482960701856300. PMID 18273714.
  5. ↑ http://www.nature.com/ng/journal/v42/n5/abs/ng.557.html
  6. ↑ "Augie’s Quest transitioning to the ALS Therapy Development Institute".
  7. ↑ "ALS ONE, an unprecedented partnership between leading Massachusetts ALS institutions, launches".

External links

This article is issued from Wikipedia - version of the Friday, April 29, 2016. The text is available under the Creative Commons Attribution/Share Alike but additional terms may apply for the media files.