Wednesday, November 9, 2011

Children's Hospital, Pfizer team up on muscular dystrophy program

By Michelle Lang

Children’s Hospital Boston is working with Pfizer Inc. on a drug discovery program focused on Duchenne muscular dystrophy (DMD), a fatal muscular disease that affects newborn boys and currently has no approved treatments.

The collaboration gives Children’s Hospital’s Program in Genomics, and its lab director Louis Kunkel, access to Pfizer’s compounds and data on such compounds from its Orphan and Genetic Diseases Research Unit. The research team had screened 1,200 chemicals that may be able to restore muscle tissue, and Pfizer compounds were among those that showed encouraging results, a news release from the hospital indicated. Now, researchers from both Pfizer and Kunkel’s teams will test the company’s compounds in an effort to find ones for further development.

“This agreement helps line up the pieces of the drug development pipeline for the benefit of children and adults with DMD,” Erik Halvorsen, director of technology and business development for Children’s, said in a statement. “It is rare to see an industrial partner allow this kind of access, and we are very pleased to have established this novel arrangement with Pfizer.”

Pfizer announced in September that it is growing its Massachusetts presence with a new lease for a Cambridge research center that will employ 400 people, with plans to hire more. In June, the New York-based company announced that its new Centers for Therapeutic Innovation will have its world headquarters in Boston. The establishment of the Centers for Therapeutic Innovation helped the company establish partnerships with a number of Boston-area medical centers, include Children’s Hospital Boston.

Among New England biotechs developing potential treatments for DMD are Acceleron Pharma Inc., which is launching clinical trials for its drug treatment, ACE-031; and Tivorsan Pharmaceuticals, a Rhode Island protein therapeutics firm focused on developing biglycan protein as a DMD treatment.