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Neurotrophic factor reverses Parkinson's symptoms in monkeys

A lentiviral vector that carries a neurotrophic factor into the brain seems to reverse symptoms of Parkinson's disease in monkeys, raising hopes that it could also be effective in humans.

October 31, 2000

A lentiviral vector that carries a neurotrophic factor into the brain seems to reverse symptoms of Parkinson's disease in monkeys, raising hopes that it could also be effective in humans. Researchers based in Switzerland and the US have developed a lentiviral vector that delivers the gene for glial-derived neurotrophic factor (GDNF) directly to cells in the brain. GDNF boosts nutrients in the brain as well as increasing the production of dopamine. This offsets the low levels of dopamine that lead to the symptoms of Parkinson's disease. Professor Jeffrey H Kordower, Director at the Research Center for Brain Repair at Rush-Presbyterian-St Luke's Medical Center in Chicago, explained: "By giving GDNF, we can stimulate dopamine production and prevent both the structural and functional consequences of cell degeneration that are characteristic of Parkinson's disease."

In the study, published in 27 October Science, lenti-GDNF was injected into the brain of aged monkeys — a primate model for Parkinson's disease. After three months, analysis with positron emission tomography (PET) and neuroanatomical, neurochemical and molecular biological techniques, showed a dramatic increase in the production of dopamine, similar to levels found in a healthy monkey.Professor Kordower and his team also demonstrated a reversal of symptoms of the disease by using lenti-GDNF in a group of younger, healthy monkeys that had been treated with the chemical MPTP to initiate a Parkinson's disease-like state.

Professor Kordower commented: "The study suggests a new approach to forestall disease progression in newly diagnosed Parkinson's disease patients by delivering potent trophic factors with effects that are long-term and non-toxic." He hopes that clinical testing in humans will begin within the next five years.

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