The study, a phase 1 clinical trial designed primarily to assess the safety of administering a protein known as glial cell line-derived neurotrophic factor or GDNF, was reported today (March 31) in the journal Nature Medicine.
The study, according to co-author Clive Svendsen of the University of Wisconsin-Madison, induced a remarkable uptick in the motor skills of five Parkinson's patients in advanced stages of the disease, as well as the ability of their brains to store the neurotransmitter dopamine, a key chemical that helps the brain control muscles.
"Nobody's ever put a growth factor directly into the brain before," said Svendsen, a neuroscientist at UW-Madison's Waisman Center. "Our main concern was the safety issue, and it is important to keep in mind the limited scope of the trial, but the clinical results we observed were impressive."
The study was carried out at the Frenchay Hospital, Institute of Neurosciences, in Bristol, England. It was coordinated by neurosurgeon Steven S. Gill and neurologist Peter Heywood.
In the United States, Parkinson's afflicts about 1.5 million people. It is caused by the death of the brain cells that produce dopamine, the chemical messenger that helps control muscle movement. It is a chronic, progressive and ultimately fatal disease characterized by uncontrollable shaking and an inability of the brain to command muscles to move in a prescribed way.
The new study raises hope that GDNF, which has long been studied in rats and primates, may one day become a new therapy for alleviating the symptoms of Parkinson's patients.
"GDNF is known to protect dopamine neurons from cell death," Svendsen said. "We know from rat models that GDNF has strong positiv
Contact: Clive Svendsen
University of Wisconsin-Madison