A new trial coming for previously failed Parkinson's treatment

Technology Review updates us on efforts to try an experimental gene therapy using glial-derived neurotrophic factor (GDNF), a protein that helps keep neurons alive, against Parkinson's disease. A team led by Krystof Bankiewicz is in the final stages of gaining approval from the FDA in the hopes that this trial can address problems with the protein in previous tests.

There was some hope back in 1993 that GDNF could help dopamine-producing neurons survive rather than degenerate with the disease. Trials using various delivery methods, however, have been unsuccessful. So, what makes this time different? Better, and more precise, delivery, Bankiewicz tells Technology Review. "They all turned out to be negative, because the delivery was never controlled," Bankiewicz says.

This time around, researchers will deliver a gene encoded with GDNF in the the putamen, the area of the brain involved in Parkinson's. They'll do it with a virus brought to the brain through convection-enhanced delivery, which uses pressure to drive it deep into the regions that are targeted. And, as a chaser, they'll include an MRI contrast agent to track where the treatment is going.

Once the treatment arrives, Bankiewicz tells Technology Review, it should ride along a trail of axons to other areas of the brain.
Not everybody is convinced, though, that GDNF is the answer to Parkinson's. "It still remains to be seen whether GDNF really is something that helps people with Parkinson's disease," Andrew Feigin, a neuroscientist at North Shore University Hospital, tells Technology Review.

- read the article in Technology Review

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