Gene Therapies Against Parkinson Disease On Clinical Trials

Filed in archive Drugs, Vaccines and Therapeutics on December 15, 2005

The Michael J. Fox Foundation for Parkinson's Research (MJFF) handed over a three-year grant of approximately $740,000 to Ceregene, Inc., to help fund Ceregene's Phase I clinical study of CERE-120, a novel gene therapy product using the neurturin gene. In pre-clinical tests, CERE-120 has shown potential to slow or stop the progression of Parkinson's Disease, a debilitating condition resulting when brain cells cease to produce dopamine, a chemical crucial for the cellular communication that controls muscle movement.

Scientists and neurosurgeons from the University of California, San Francisco and Chicago's Rush University Medical Center have commenced the first attempts to use to treat Parkinson's Disease using CERE-120. In this particular study, the brains of 12 patients are being injected with a harmless virus that carries the gene for one growth factor, called neurturin, in an effort to protect and/or rescue the remaining dopamine-producing neurons, or nerve cells.

There are two other gene therapy approaches currently on trial:

UCSF researchers and Avigen, Inc aims to extend the therapeutic effect of Parkinson's medication, levodopa, through gene therapy designed to spur production of an enzyme that converts levodopa into dopamine. Three patients so far have received the therapy, with 12 more to be enrolled.

In September, Neurologix Inc. announced promising preliminary results from 12 patients received gene therapy involving the gene encoding for glutamic acid decarboxylase (GAD), an Enzyme which synthesizes the major inhibitory neurotransmitter in the brain, (gamma)-aminobutyric acid (GABA). Results indicate that the gene therapy seemed safe, with no adverse reactions and, resulted to a statistically significant improvement in motor function on the side of their body correlating to the treated part of the brain.