MDA has awarded $2 million to the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., to help scientists complete the preclinical testing of four promising new compounds in ALS TDI's drug development pipeline.
The grant, made through MDA's Augie's Quest ALS fundraising initiative, raises MDA's total grant contributions to ALS TDI to more than $23.4 million since 2007.
About the new funding
ALS TDI plans to test the four new compounds in the SOD1 ALS research mouse model.
The new funds also will enable the nonprofit biotech to add the TDP43 research mouse model to its database so as to include it in its preclinical testing program. Most ALS research to date, including that conducted at ALS TDI, has utilized research mice with mutations in the SOD1 gene, known to cause human ALS. The TDP43 mouse model, developed in 2009 with MDA support, carries a mutation in the gene for the TDP43 protein, also known to cause ALS in humans. (For more, see New ALS Mouse.)
"MDA is happy to continue funding research at ALS TDI into 2011," said Jane Larkindale, director of translational research at MDA. "The company is making a significant impact on ALS research and therapy development, both through identification of new therapeutic targets and through the testing of potential therapeutic agents in models of the disease. We are very excited by its progress."
To learn more about ALS TDI's research program, see ALS TDI Conference Summarizes ALS Research Progress.
About MDA and ALS TDI
MDA and ALS TDI forged a historic partnership in January 2007 when they launched the largest ALS drug discovery project to date, a three-year, $36-million collaboration to identify biochemical targets in ALS and find drugs that hit them. MDA's initial $18-million investment was matched by ALS TDI.
In January 2010, MDA renewed its partnership with ALS TDI with a grant totaling $2.5 million (see Muscular Dystrophy Association Renews Partnership). Based on the biotech's extraordinary progress, MDA awarded it a supplemental grant of $855,600 in December of that same year.
"It's humbling to have MDA continue its support of ALS TDI at such an enormous level," said Steve Perrin, CEO and chief scientific officer at the Institute. "It's a testament to the efficiency and innovativeness of the multidisciplinary team working at the Institute today. This grant will provide crucial new resources that we desperately need to continue to home in on subtle aspects of the disease and to execute an even bigger and bolder efficacy screening program."
Meaning for people with ALS
If test results from the four compounds show promise, the new experimental therapies are likely to advance to human clinical trials.
The addition of the TDP43 mouse model to ALS TDI's preclinical testing program will provide a powerful new tool for TDI scientists, enabling them to gain greater insight into the ALS disease process.
The new funding supports the ultimate goal of both MDAand ALS TDI: to speed drug development and testing, and move potential therapeutic agents through to human clinical testing and, ultimately, to the clinic.