ALS Therapy Development Institute Awarded $1M Grant

Last update: 8:15 a.m. EDT Oct. 28, 2008 CAMBRIDGE, Mass.,, Oct 28, 2008 /PRNewswire-USNewswire via COMTEX/ -- RGK Foundation grant will support the creation of potential gene therapies to treat ALS

The ALS Therapy Development Institute announced today that it has been awarded a $1 million grant from the RGK Foundation of Austin, Texas. The grant, to be awarded over 18 months, will support the institute's on-going efforts to utilize the latest gene therapy technology to develop an effective therapeutic to slow or stop the progression of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease.

"We are grateful and honored to have the trust and support of the RGK Foundation. It is imperative that the latest gene therapy technologies and therapeutic validation strategies be brought to bear on this relentless disease. This investment by the RGK Foundation is just the latest in their long commitment to supporting innovative research at ALS TDI and elsewhere," said Dr. Steve Perrin, Chief Scientific Officer at ALS TDI.

The grant will support research designed to identify and develop gene specific viral vectors at ALS TDI, including in vitro validation and the development of sufficient vector stocks to conduct full in vivo validation projects. Support from individuals and organizations, such as the RGK Foundation, enables the institute to advance increasing numbers of high-priority therapeutic targets through its rigorous validation process. Currently, the research team at ALS TDI has more than 40 different research projects going on in its 16,000 square foot research center in Cambridge, Massachusetts.

The Kozmetsky family and the RGK Foundation have been supporting the important work of ALS TDI over several years. In 2003, Ronya Kozmetsky co-founder of the RGK Foundation, provided the institute with $1 million to build the Kozmetsky Drug Testing and Knowledge Nexus Initiative. This investment allowed ALS TDI to further its drug discovery and testing capabilities and upgrade knowledge sharing capabilities through an online ALS Forum and Internal Knowledge Management System (LIMS). At the end of 2006, ALS TDI was planning a major new initiative to jumpstart therapeutic development in ALS, and the RGK Foundation provided a $50,000 grant to facilitate our transition into the world's largest ALS research organization, in partnership with MDA and Augie's Quest. The partnership with the MDA and Augie's Quest, a three-year, $18 million funding and scientific collaboration, was initiated at the beginning of 2007. Today, ALS TDI is aggressively pursuing the most comprehensive research plan ever attempted to discover viable treatments for ALS, with projects investigating nearly every aspect of discovery research and therapeutic development.

ALS is a neurodegenerative disease resulting in progressive paralysis and is considered fatal. The disease typically strikes without an identifiable cause, indiscriminately affecting a new family every 90 minutes in the U.S. That incidence rate is similar to that of multiple sclerosis, but the typical survival prognosis given to a new ALS patient is only 2-5 years from the date of diagnosis. Currently there is no known cure for ALS and only one FDA-approved drug, with marginal efficacy, for treating the disease.

About the ALS Therapy Development Institute The ALS Therapy Development Institute (ALS TDI) ( http://www.als.net), based in Cambridge, Massachusetts, operates the world's largest research and development program focused exclusively on ALS. The Institute has a staff of more than 30 scientists and research technicians, working on behalf of ALS patients to discover and advance therapeutics for treating, and ultimately curing, ALS. The nonprofit biotechnology institute excels in identifying novel disease targets, discovering compounds that may act against these targets, and screening potential treatments for clinical development. CONTACT: Robert Goldstein of ALS TDI, +1-617-441-7295, rgoldstein@als.net SOURCE ALS Therapy Development Foundation

http://www.als.net