NIH Grant to Boost IU-Led Spinal Research

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The chair of the Indiana University School of Medicine Department of Dermatology has received more than $430,000 from the National Institutes of Health. The IGNITE grant will support testing by Elliot Androphy and his team for a potential treatment of spinal muscular atrophy.

The genetic disease affects voluntary muscle movement.

Androphy says "the IGNITE funding covers tests to determine the compounds' toxicity, if they reach the blood-brain barrier, how quickly they are metabolized and other characteristics. My colleagues and I have done a lot of screening and validation, and a lot of the chemistry has been covered by other grants from the NIH. But we need to conduct these tests, which are very expensive." He is working with colleagues at Brigham and Women's Hospital/Harvard Medical School in Boston and the University of Missouri Medical School.

The IU Research and Technology Corp. has filed patent applications on the compounds. "This intellectual property could lead to a therapy for spinal muscular atrophy, and in order to get a drug on the market, it needs to be patented and protected," Androphy said. "IURTC also is working with us to find a partner for some development work. Our hope is to take the best candidate to a clinical trial, which is an expensive step. Having a pharmaceutical company partner could move this forward as quickly as possible."

He calls the process to obtain this first round of funding "very rigorous" and adds "knowing that we received the highest score of the session has strengthened our belief that we can impact people's lives by reducing the pain and suffering caused by spinal muscular atrophy."

IU says, if researchers hit predetermined milestones, the grant will last three years.

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