Parkinson's disease (PD) is the second most common neurodegenerative disorder. While a number symptomatic therapies have been developed, no treatment has been identified that halts or slows this progressive disorder. For a number of reasons, we believe that it is appropriate to begin a prospective clinical trial of one or more of the more promising agents to determine whether they offer benefit.
The aim of this application is to participate as a clinical center in a collaborative study of neuroprotective agents. The agents and protocol will be specified by NINDS. UCSF has a long history in providing subspecialty care for subjects with PD. The UCSF Parkinson's Disease Clinic &Research Center (PDCRC) has been involved in a number of clinical trials to elucidate the neurologic dysfunction underlying PD as well as testing a variety of novel treatments for PD. This research has included a number of industry sponsored treatment trials for subjects with early to advanced PD. A large diverse, local population provides a reliable source of motivated subjects for clinical research. The experience of the UCSF PDCRC in evaluating, selecting and following subjects with PD make it an ideal candidate for a multicenter, prospective trial of a potential neuroprotectant therapy. The ideal neuroprotective treatment should be effective, inexpensive, well tolerated, safe, easy to administer, and have few interactions with common medications. A number of studies over the last decade in humans and experimental animals have demonstrated a local immunologic response mediated by microglia and a number of inflammatory cytokines. Moreover, several animal models of PD have demonstrated that anti-inflammatory medications can interfere with the local immunologic response, and that they may preserve function of dopaminergic neurons. Although emerging understanding of the pathogenesis of PD may provide an alternative candidate for a neuroprotectant therapy, we propose that an anti-inflammatory medication would be a rational and practical candidate for the Parkinson's disease Neuroprotection Trial.
|Wills, Anne-Marie A; Pérez, 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|
|Writing Group for the NINDS Exploratory Trials in Parkinson Disease (NET-PD) Investigators; Kieburtz, Karl; Tilley, Barbara C et al. (2015) Effect of creatine monohydrate on clinical progression in patients with Parkinson disease: a randomized clinical trial. JAMA 313:584-93|
|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|
|Hegde, Manu; Glass, Graham A; Dalmau, Josep et al. (2011) A case of slow orthostatic tremor, responsive to intravenous immunoglobulin. Mov Disord 26:1563-5|
|Faulkner, L R; Juul, D; Pascuzzi, R M et al. (2010) Trends in American Board of Psychiatry and Neurology specialties and neurologic subspecialties. Neurology 75:1110-7|
|Christine, C W; Starr, P A; Larson, P S et al. (2009) Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73:1662-9|