Parkinson's disease is set apart from other neurodegenerative disorders by our knowledge of the key biochemical defect. Dopamine replacement therapy is effective in treating major symptoms of the disease. However, despite the availability of symptomatic therapies, patients experience a functional decline as a result of the progressive nature of the illness. A major unmet need in the treatment of PD is the identification of therapies that slow the rate of progression of neuronal degeneration. Proper investigation of therapies with neuroprotective potential requires infrastructure and collaboration among several sites to evaluate disease modifying effects over several years. The goal of this competing renewal is to report on the contributions of the Institute for Neurodegenerative Disorders (IND) as a clinical center for the NET-PD. IND has played an active role in the recruitment and retention of subjects for LS-1, FS-1 and FS-TOO over the past several years. We enrolled a total of 31 subjects into the program with a retention rate of on average 84%. Our success in our recruitment and retention stems from our relationship with advocacy groups, experience in advertising, a stable and responsive clinical research team and our relationship with the community neurologists in Connecticut who refer, on average, 10 PD patients per month to our research program. As active members of the PD research community, we propose to continue our collaboration with other NET-PD sites, the Central Coordination Center (CCC) and the Statistical Center toward the goal of successfully completing the current trials.
At IND we have a commitment to developing improved treatments, including the development of agents with neuroprotective potential, for individuals with Parkinson's disease. Determining whether creatine has a disease modifying effect would provide a major opportunity for the treatment of PD. Understanding the course of progression of PD during the early stages of disease is also an important advance for the field.
|Wills, Anne-Marie A; PÃ©rez, Adriana; Wang, Jue et al. (2016) Association Between Change in Body Mass Index, Unified Parkinson's Disease Rating Scale Scores, and Survival Among Persons With Parkinson Disease: Secondary Analysis of Longitudinal Data From NINDS Exploratory Trials in Parkinson Disease Long-term Study 1 JAMA Neurol 73:321-8|
|Elm, Jordan J; NINDS NET-PD Investigators (2012) Design innovations and baseline findings in a long-term Parkinson's trial: the National Institute of Neurological Disorders and Stroke Exploratory Trials in Parkinson's Disease Long-Term Study-1. Mov Disord 27:1513-21|