Five-year initiative targets Parkinson's

orking with scientists from across the nation, the Medical Center is coordinating a national effort to pioneer a new treatment for Parkinson's disease.

The five-year program, funded with $8.8 million from the National Institute of Neurological Diseases and Stroke and additional support from the National Institutes of Health, was conceived by Howard Federoff, professor of neurology and director of the Center for Aging and Developmental Biology.

The program is the first to focus on gene therapy as a treatment for diseases such as Parkinson's, Alzheimer's, Huntington's, or Lou Gehrig's in which neurons die.

While the idea of manipulating genes to boost human health--gene therapy--has been around for more than two decades, implementing the idea has proven difficult for researchers. Only a handful of patients worldwide have been treated with the technique.

"Our goal is to bring forward, in the most rigorous possible fashion, a gene therapy for patients with Parkinson's disease," says Federoff. "We're proceeding deliberately and cautiously to develop and test new treatments, in a coordinated progression from basic research to preclinical evaluation to clinical testing. Our intention is to lay the groundwork for a future study where new approaches can actually be tested in people."

To accomplish the program's goals, Federoff has brought together many of the world's foremost Parkinson's experts. The new group, known as the Parkinson's Disease Gene Therapy Study Group, is made up of researchers from the University, Northwestern, Rush-Presbyterian Medical Center in Chicago, Yale, and University of California campuses in Irvine, Los Angeles, and San Francisco.

"This is an unprecedented comprehensive program, and everyone involved has made a substantive commitment to this effort," says Federoff.

Neuroscientists in the program will work on ways to shuttle genes into cells, along with methods to turn on and off those genes exactly when and where they want, including using viruses to alter brain cells to counter the disease's side effects.

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