Eliminating Parkinson’s may eventually be possible with a ‘stunning’ single treatment

[Researchers found] a new therapeutic approach for Parkinson’s disease and other neurodegenerative diseases. Just a single treatment to inhibit [the protein] PTB in mice converted native astrocytes, star-shaped support cells of the brain, into neurons that produce the neurotransmitter dopamine. As a result, the mice’s Parkinson’s disease symptoms disappeared.

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In the treated mice, a small subset of astrocytes converted to neurons, increasing the number of neurons by approximately 30 percent. Dopamine levels were restored to a level comparable to that in normal mice… By two different measures of limb movement and response, the treated mice returned to normal within three months after a single treatment, and remained completely free from symptoms of Parkinson’s disease for the rest of their lives.

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“I was stunned at what I saw,” said study co-author William Mobley, MD, PhD, Distinguished Professor of Neurosciences at UC San Diego School of Medicine. “This whole new strategy for treating neurodegeneration gives hope that it may be possible to help even those with advanced disease.”

What is it about PTB that makes this work? “This protein is present in a lot of cells,” Fu said. “But as neurons begin to develop from their precursors, it naturally disappears. What we’ve found is that forcing PTB to go away is the only signal a cell needs to turn on the genes needed to produce a neuron.”

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