Parkinson's Disease Neuroprotection Trial

Carter, Julie

Abstract

The NET-PD program used a rational, scientific approach for the selection and evaluation of potential therapies for the prevention of Parkinson's disease (PD) progression. Since its original award, the NET-PD program has successfully completed two futility studies, known as Futility Study 1 (FS-1) and Futility Study 2 (FS-TOO) which evaluated the therapeutic potential of four compounds (creatine, minocycline, coenzyme Q10 (CoQ10), and GPI-1485) [5, 6]. The results of FS-1 and FS-TOO informed the design of a large, simple clinical trial for efficacy of creatine treatment in slowing clinical decline in participants with early, treated Parkinson's disease, known as LS1. LS1 is a multi-center. Phase 3, double-blind clinical trial of creatine for the prevention of PD progression that tests the hypothesis that daily administration of creatine (10gm/day) is more effective than placebo in slowing clinical decline in PD between baseline and the 5 year follow-up visit against the background of dopaminergic therapy and best PD care.

Public Health Relevance

While current estimates indicate that approximately six million people worldwide (over 1/2 million Americans) have been diagnosed PD, there is neither a treatment to slow progression nor a cure. Morbidity and mortality associated with PD burden results in an estimated six billion dollars in healthcare costs in the United States annually. Treatments to slow progression of PD remain an unmet need, and NET-PD is one of the largest, scientifically grounded and most coordinated effort to date to address this need.