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Brain-Cell Growth Protein Shows Promise For Parkinson’s Patients in Early Human Trial

A protein that promotes the growth of brain cells may have great promise for people with Parkinson disease, according to the results of a small experimental study involving five patients.

A report on this novel approach to the treatment of Parkinson among patients in the later stages of the disease appears this week in the journal Nature. When the protein GDNF was drip-fed into the area of the brain known as putamen, all five patients in the study showed marked improvement.

One 64-year old patient, in constant pain and barely able to walk when the two-year research effort began, now walks miles each day, eats his own food, and has even taken up bowling.

“We’re seeing marked improvements across the board,” said Steven Gill, a neurosurgeon at the Institute of Neurosciences in Bristol, England, who directed the study.

In the new trial, GDNF was delivered continuously to the putamen through tubing connected to a mini-pump implanted under the skin. GDNF, the acronym for glial cell-line-derived neurotrophic factor, has been shown in research on mice and primates to nourish the brain cells that wither during PD.

The drug eliminated periods of immobility that had occurred as much as 20% of the time before treatment, according to Dr. Clive Svendsen of the University of Wisconsin-Madison, a member of the research team. It reduced or stopped the involuntary movements common to the disease, he said. Also, the senses improved for three patients who had lost the ability to taste or smell.

Dr. Gill claims that this is the first time a treatment for Parkinson has reversed, rather than simply stalled, disease progression. The rejuvenated brain cells began to sprout. They produced more of the chemical dopamine, which is essential for normal movement.

Much more work with GDNF needs to be done, the doctors agreed. The use of a very small group of patients is typical of the first clinical trial of a substance to be conducted on human beings. Such a “phase one” trial provides evidence of the safety of a drug being tested, and of any side effects. The results of this study, Dr. Svendsen said, show that GDNF “is worth studying very carefully”.

Comments on the results from other doctors ranged from the enthusiastic to the sceptical.

Dr. Michael Zigmond of the University of Pittsburgh School of Medicine, who was not part of the research team, was quoted as saying: “I consider this study to be the most exciting advance in the treatment of Parkinson disease that has come about in years. I think the findings hold tremendous promise for going beyond” treating the disease’s symptoms to treating the underlying disease itself.

Parkinson researcher Warren Olanow of Mt. Sinai School of Medicine in New York City was more reserved. “Theoretically it’s very nice,” he was quoted as saying, “but a long-term infusion might cause unknown side effects. You have to take [the results] with a grain of salt.”

Source Date: Apr 04 2003