San Diego—Ceregene, Inc., announced today that CERE-120, a gene therapy product in development for the treatment of Parkinson’s disease, was well tolerated and appeared to reduce symptoms by approximately 40 percent (p<0.001), as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS) motor “off” score, in an open-label Phase I study in 12 patients with advanced disease. Initial results of the study were presented by William J. Marks Jr., M.D., principal investigator of the study and associate professor of Neurology at the University of California, San Francisco (UCSF) today at the American Neurological Association annual meeting in Chicago.
The study was supported in part by a grant from The Michael J. Fox Foundation for Parkinson’s Research. Based on the initial results, the foundation announced plans to partially fund a Phase II study with a $1.9 million grant. “We were encouraged by the results of the Phase I trial,” said Deborah W. Brooks, president and CEO of The Michael J. Fox Foundation. “Based on these and on the intriguing efficacy observations, we’re eager to continue to support research in Phase II that will more definitively assess the potential of CERE-120 to treat PD.”
All 12 patients enrolled in the study underwent stereotactic neurosurgery to deposit CERE-120 into their putamen. The putamen is a region of the brain that degenerates, reducing dopamine production in Parkinson’s disease patients. This trait has been closely linked to major motor deficits.
CERE-120 was delivered at two different doses, with patients receiving the low dose demonstrating approximately 40 percent improvement in UPDRS motor “off” scores by nine months and patients receiving the four-fold higher dose showing a similar effect three months sooner. Patients also demonstrated a 50 percent reduction in hours of “off” time (i.e., time when normal Parkinson’s medication was ineffective and symptoms were troubling to the patient) and a doubling of good quality “on” time without dyskinesias (i.e., time when a patient is functioning well) according to self-reported diaries.
NTN (neurturin) is a member of the same protein family as glial cell-derived neurotrophic factor (GDNF) and the two molecules have similar pharmacological properties. GDNF has previously been tested in Parkinson's disease patients.