Parkinson's Disease
Ceregene is currently evaluating its clinical development plan for CERE-120 (AAV-neurturin), which has a goal of restoring lost motor function and protecting against further loss of function, thus potentially significantly improving quality of life in Parkinson's patients. Parkinson’s disease (PD) is a devastating and progressive movement disorder that affects at least one million people in the United States. It is a neurodegenerative disease characterized by the loss of dopamine neurons within the brain. Loss of these neurons leads to motor problems such as difficulty in initiating movement, debilitating tremors and postural instability. PD is a progressively degenerative disease for which there is currently no cure.
Treatment of Parkinson's Disease
Drugs that enhance dopamine function are able to significantly correct motor problems early in the course of PD, but over time these drugs universally become ineffective. In time, patients suffer long periods where movement is not possible despite optimized medication and where the serious side effects of the dopamine drugs can no longer be avoided. Thus, the need to develop more effective treatments for PD is widely recognized.
Ceregene’s Parkinson's Disease Treatment Approach
Our approach to treating Parkinson's disease is different from all others. Using our proprietary gene delivery technology, a single gene transfer procedure is potentially all that is needed to provide significant symptomatic improvement, while also possibly partially reversing and delaying further disease progression. We are using neurturin (NTN) as the neurotrophic factor to treat the dopamine-containing neurons that degenerate in PD. NTN is a member of the same protein family as glial cell-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties, and thus both have been shown to restore the health of the dopamine motor neurons that degenerate in Parkinson’s disease, while also protecting them from further degeneration. Our therapy, CERE-120, is composed of an adeno-associated virus (AAV) vector designed to deliver the gene for NTN. CERE-120 is delivered by a neurosurgical dosing procedure to the dying brain region in PD patients, providing a stable, long-lasting supply of NTN in a highly targeted fashion.
Data from a double-blind, controlled, multicenter Phase 2 trial in 58 patients with advanced Parkinson’s disease were released in November 2008. The trial failed to demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Both groups showed an approximate 7 point improvement in the protocol-defined primary endpoint (Unified Parkinson’s Disease Rating Scale - motor off), relative to a mean baseline of approximately 39 points. Both groups had a substantial number of patients who demonstrated a meaningful clinical improvement from baseline. CERE-120 appeared to be safe and well tolerated. Ceregene is currently evaluating future development plans for CERE-120.
Genzyme Corporation has licensed the ex-North American rights for the development and commercialization of CERE-120 from Ceregene, an agreement that was announced in June 2007.
We are pleased to be partnered with the Michael J. Fox Foundation for Parkinson's Research (MJFF) to improve the treatment of PD patients. Founded in 2000, The MJFF is dedicated to ensuring the development of a cure for Parkinson's disease through an aggressively funded research agenda. To date, the MJFF has funded tens of millions of dollars supporting treatment-related research in numerous university and corporate laboratories, either directly or through partnerships. The MJFF provided partial funding for both our Phase 1 and Phase 2 trials of CERE-120 for PD in the United States.