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Children's Hospital Boston and Pfizer Enter Into Novel Collaborative Research Agreement for Muscular Dystrophy Therapeutics
Date:11/9/2011

BOSTON, Nov. 9, 2011 /PRNewswire-USNewswire/ -- Children's Hospital Boston and Pfizer Inc. (NYSE: PFE) have launched a new collaborative program aimed at identifying potential drug therapies for Duchenne muscular dystrophy (DMD). At the heart of the one-year collaborative research agreement is a novel exchange of expertise that brings the respective strengths of academia and industry together in a framework that could hold promise for benefitting DMD patients in the future.

The agreement focuses on the work of the laboratory of Louis Kunkel, Ph.D., Director of the Program in Genomics at Children's Hospital Boston and discoverer of the dystrophin gene that underlies the biology of DMD. In March 2011, Kunkel and colleagues announced the results of their efforts to screen 1,200 chemicals already approved for human use in a zebrafish model of DMD for any that might have a restorative effect on muscle tissue. Kunkel's team – one of several at Children's engaged in research on rare or orphan diseases – found that several had the desired effect in the zebrafish model, including compounds owned by Pfizer.

Through the agreement, Pfizer will, via its Orphan and Genetic Diseases Research Unit, provide Kunkel access to select proprietary compounds, as well as relevant data about these compounds. Pfizer is also committing internal resources to the project such as medicinal chemistry.

In collaboration with Pfizer's Carl Morris, Ph.D., Kunkel's laboratory will test the compounds provided by Pfizer in the DMD zebrafish model, with an eye toward identifying candidates for further preclinical development.

"This agreement brings together the best of two worlds, namely Pfizer's ability to develop promising new compounds and our ability to test them in physiologically relevant models," Kunkel said. "To have Pfizer show this level of interest in DMD is ve
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SOURCE Children's Hospital Boston
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