Engineered Protein Yields Hopeful Results

A drug the preliminary tests of which three years ago failed to produce the hoped-for results has emerged this fall as a possible means of regenerating damaged cells in the brains of people with Parkinson’s and other brain ailments. The drug is what is known as a “bio-engineered protein” called GDNF.

In previous trials, GDNF (which stands for “glial cell line-derived neurotrophic factor”) failed to live up to the promise it had offered of helping damaged brain cells to restore themselves. Within recent months a researcher at the University of Kentucky Medical Center, utilizing a new and ingenious method of delivering the drug directly to the affected areas of the brain, has produced evidence in a small number of Parkinson’s patients that GDNF can be effective.

The lead investigator is Dr. Greg Gerhardt, director of the University of Kentucky’s Parkinson’s Research Center, one of the medical institutions selected by the National Institutes of Health as a Morris Udall Center. His bold innovation as a delivery system is a pump implanted in the chest of a patient, which directs a measured amount of the protein at regular intervals directly into the substantia nigra, the part of the brain affected by the loss of dopamine.

Dr. Gerhardt says it is too early to draw conclusions from this small test, but he is looking forward to larger research projects. The project, reported in the October issue of the medical journal “Brain”, was financed by Amgen, Inc., manufacturer of GDNF.