Funded Studies
Outcomes of Original Grant:
Neurotrophic factors including neurturin (NTN) have been shown to effectively augment the function and prevent the death of dopaminergic nigrostriatal neurons in animal models of Parkinsons’s disease (PD). Thus, NTN administration may potentially provide symptomatic benefit as well as modify the disease course in PD. CERE-120, a gene transfer vector to deliver NTN to the striatum, was demonstrated to be safe and well tolerated in a Phase I clinical trial in subjects with advanced PD. In a Phase II, double-blind trial, Cere-120 was demonstrated to be safe but did not demonstrate efficacy against the pre-specified primary endpoint at 12 months. However, further analysis at 18 months suggested a modest clinical benefit.
Objectives for Supplemental Investigation:
This follow-on study will continue the evaluation of patients enrolled in the Phase II trial of Cere-120. The goal of this study is to continue to evaluate the safety of Cere-120, while at the same time collecting additional efficacy data in an open labeled study.
Importance of This Research for the Development of a New PD Therapy:
This study will provide additional data on the long-term effects of trophic factor gene therapy in PD patients. Outcomes from this study will inform future efforts to test trophic factor therapies in PD patients