TDI-132 Shows Promising Results in the Treatment of ALS

March 4, 2012
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ALS may be the most deadly disease many people have never heard of.

ALS may be the most deadly disease many people have never heard of. Amyotrophic lateral sclerosis (ALS), also referred to as Lou Gehrig’s disease affects at least 30,000 people in the United States and 450,000 people worldwide.  It is a progressive neurodegenerative disorder that causes muscle atrophy, paralysis, and ultimately death with a three to five year survival rate.

While the incidence rate of ALS is similar to that of Multiple Sclerosis (MS) with 5,000 new cases per year, more people die from ALS because there are virtually no effective treatments.

Founded in 1999 by the Heywood family, the ALS Therapy Development Institute seeks to provide an urgent response to the crisis that is ALS.  Dr. Steve Perrin, ALS TDI’s CEO and CSO, spearheads the development of computational biology capabilities and information management systems to more clearly understand the molecular mechanisms associated with disease onset and progression in neurodegenerative diseases.

A non profit organization, ALS TDI collaborates with biotech and pharma companies to address the unmet need in neurodygeneration.

In ALS, nerve cells (neurons) waste away or die, and can no longer send messages to muscles. This eventually leads to muscle weakening, twitching, and an inability to move the arms, legs, and body. The condition slowly gets worse. When the muscles in the chest area stop working, it becomes hard or impossible to breathe on one’s own.

ALS TDI is in phase II clinical trials with two drugs, one of which has previously been approved by the FDA to treat MS and is marketed by Novartis. Gilenya, or TDI 132, blocks lymphocytes in blood and has shown promising results in mice, on several disease measures.

ALS TDI operates on a $10M annual budget with zero funding from NIH. Much of their funding is derived from CRO relationships, foundations, and individual donors.

Dr. Perrin presented the company’s drugs and technologies at the BIO CEO & Investor Conference in New York February 12-13.

 

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