A 62-year-old Parkinson’s disease patient died of an unrelated heart attack three months after participating in an experimental Parkinson’s drug trial. When researchers opened up his brain to take a look, they discovered something very encouraging-if not for the heart attack, he might have recovered.
The experimental drug used on the patient was GDNF (glial cell line-derived neurotrophic factor), made by Amgen, the second trial of which was stopped due to safety concerns.
When the brain was analyzed, it showed a regrowth of the nerve fibers once destroyed by the progressive degenerative disease.
Only one side of the brain was injected with GDNF, which made it easy to compare the hemispheres. Dopamine-containing nerve fibers had sprouted in the putamen.
“This is the first neuropathological evidence that infusion of GDNF in humans causes sprouting of dopamine fibers, in association with a reduction in the severity of Parkinson’s disease,” said Professor Seth Love of Bristol University, United Kingdom.
Parkinson’s disease, which afflicts famed heavyweight Mohammed Ali and actor Michael J. Fox, attacks the cells that produce dopamine. Loss of muscle control and tremors, slow movement, lack of coordination and balance are all problems associated with the disease.
A cure has yet to be found, but dopamine-boosting medicines have shown positive results in helping patients manage their physical difficulties.
Researchers in the initial experiment on GDNF injected the drug directly into damaged parts of the brain.
Within months, patients exhibited dramatic improvements in motor function, an improvement that continued through four years of treatment. Patients retained the improvement even after being taken off the medication.
The discovery in the dead man’s brain will revitalize the research on GDNF and its potential contribution to a cure of the debilitating disease. Scientists expect studies to resume once the safety concerns are properly addressed.