Gendelman discussed his and graduate student Eric Benner's Parkinson's discoveries Wednesday at an Omaha seminar hosted by Nebraskans for Research. It was the first time Gendelman had publicly discussed the matter since December, when StatePaper exclusively reported limited details about the potential vaccine.
"This is kind of the first comprehensive effort to tackle Parkinson's research in this way," Gendelman said.
Parkinson's disease, a degenerative brain disorder, afflicts more than 1 million Americans with muscle stiffness, tremors, slowness of movement, poor balance and walking problems. Actor Michael J. Fox and former Attorney General Janet Reno have the disease.
Few new details about the potential vaccine beyond what StatePaper has already reported were available Wednesday. Gendelman said he must keep quiet about specifics or risk losing the patent. (As with all patents, approval isn't a sure thing.)
Unfortunately, things that must be kept quiet include how the vaccine would work, and on what kind of patient.
"The crux of how the vaccine works is all under patent," Gendelman said.
But Gendelman was able to release these new points of interest:
- If the patent is approved, proving the concept behind the potential Parkinson's vaccine will take about five years. Laboratory work on cell cultures is already underway. Experiments on mice that have been given a Parkinson's-like condition could begin soon. The timetable for human trials will depend, as always, on the Food and Drug Administration.
- The illegal trade in the street drug heroin actually has a positive side effect. A chemical used to purify heroin -- methylphenyltetrahydropyridine, or MPTP -- gives mice a condition close enough to human Parkinson's disease to be useful for research.
- Cells from aborted human fetal tissue are not being used in the Parkinson's vaccine research. (Gendelman uses fetal cells in other research projects.) If fetal cells were used in the vaccine research, Gendelman said, they would be used to prove the vaccine works, not to manufacture the vaccine itself.
As StatePaper reported in December, the research into the potential vaccine got started with Benner's idea to use a vaccine to get immune cells, called T-cells, into the area of the brain affected by Parkinson's. Once in the damaged environment, these T-cells actually secrete chemicals that reduce the damage that's occurred. For reasons that are still unknown, these T-cells only do this in damaged areas, not healthy ones.
Collaboration between Gendelman, Benner and scientists in New York and Israel has been key in developing the ideas behind the potential vaccine. Benner has worked with the renowned Movement Disorders Division at Columbia University's Columbia-Presbyterian Medical Center in New York. Gendelman's contributions come in part from his Fulbright Scholar experiences studying spinal-cord regeneration at the famous Weizmann Institute of Science in Israel. Gendelman's ideas on the potential vaccine also are an outgrowth of his "Dr. Jekyll and Mr. Hyde" approach to other degenerative brain disorders like Alzheimer's disease and AIDS-related dementia.
You can read more about Gendelman's theories in this story, but here's the gist. Gendelman says brain cells called glia, in the vast majority of cases, act like the good Dr. Jekyll, helping the brain function. But sometimes the glia can be transformed into the evil Mr. Hyde, hurting the brain. In the classic story, the evil persona isn't permanent - Mr. Hyde turns back into Dr. Jekyll. The analogy to that story is fitting, because Gendelman's research has found ways to turn the evil glial cells back into good ones.
Also notable in this is the rarity of having a graduate student join a world-renowned researcher as co-discoverer of a patent. Even in the unlikely event the patent isn't approved, just being listed on the application will be one heck of a resume booster for Benner.
"He's unbelievable. We let this kid roll and he's performed spectacularly," Gendelman said. "He had a significant role in the discovery."
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This story originally appeared in Nebraska StatePaper on May 30, 2001.
