MDA Awards $2 Million to ALS TDI

by Amy Madsen on Wed, 2011-03-02 14:13

Through MDA's Augie's Quest program, the ALS Therapy Development Institute has been awarded an additional $2 million for drug development research

Article Highlights:
  • MDA has awarded $2 million to the ALS Therapy Development Institute to support the Institute's ongoing drug development research.
  • This latest award will support preclinical testing of several different potential therapeutic agents in the SOD1 research mouse model of ALS, and will help expand ALS TDI's research program to include the TDP43 mouse model of the disease.
  • The new funding comes via MDA's Augie's Quest, an ALS research fundraising initiative, and brings the total amount MDA has awarded the nonprofit biotech to more than $23.4 million since 2007.

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.

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