This trial follows positive results from a phase I trial of the gene therapy CERE-120 in 12 patients in which preliminary data showed the treatment to be well tolerated and to provide a 35 to 40% reduction in Parkinson’s symptoms.
The phase II trial is now enrolling 51 patients with advanced Parkinson’s disease. Patients will be followed for 12 months for safety and efficacy.
“Patients in the advanced stage of Parkinson’s disease begin to run out of effective therapeutic options, and CERE-120 may have the ability to alleviate this great unmet medical need-especially given its potential to offer measurable and lasting benefit after just a single treatment,” said William Marks Jr., associate professor of Neurology at the University of California, San Francisco.
The Michael J. Fox Foundation for Parkinson’s Research funded a portion of the phase I clinical trial and chose to contribute funding for the phase II trial.
CERE-120 is composed of an adeno-associated virus vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and functioning normally.