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Ice Bucket Challenge Advances Antibody Toward Clinical Trial for ALS

Posted: May 28, 2015 in Other News

ALS Therapy Development InstituteALS Therapy Development Institute

CAMBRIDGE, MA – The ALS Therapy Development Institute announced it is directing, via its wholly owned subsidiary, Anelixis Therapeutics, $1.5 million toward the development of a potential treatment for ALS that targets the innate immune system. The funding comes from the $4 million the ALS Therapy Development Institute received from the 2014 social media phenomenon known as the “ALS Ice Bucket Challenge.” The entire sum of $4 million raised directly by the Institute through last year’s Ice Bucket Challenge will be spent on ALS research.

“Inappropriate activation of the immune system is a prominent feature in autoimmune diseases and neurological diseases such as Multiple Sclerosis and ALS. Antibodies that block CD40L activation on T lymphocytes have demonstrated benefit in these diseases, and we are encouraged that it has worked so well in animal models of ALS,” says Steve Perrin, Ph.D., CEO and CSO of the ALS Therapy Development Institute.

The anti-CD40L antibody targets a protein which has been implicated as a trigger in converting immune cells to an active state. Blocking the expression of this protein has been shown via rigorous preclinical experiments to slow disease progression and improve survival times of mSOD1 mice. Those results have been presented publicly and published in the peer-reviewed journal, Nature Genetics (Lincecum & Vieira, et al, 2010). Anelixis Therapeutics has contracted Lonza, a leading global biologics API manufacturer, to develop and manufacture clinical material of anti-CD40L for future phase I patient trials.

“The Ice Bucket Challenge gave the Institute a boost in funding, which we were able to deploy quickly to advance promising programs like this one. Everyone who donated to our Institute made the development of this treatment possible,” adds Dr. Perrin.

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