Proceedings: cell therapies for Parkinson's disease from discovery to clinic

Abstract

In March 2013, the California Institute for Regenerative Medicine, in collaboration with the NIH Center for Regenerative Medicine, held a 2-day workshop on cell therapies for Parkinson's disease (PD), with the goals of reviewing the state of stem cell research for the treatment of PD and discussing and refining the approach and the appropriate patient populations in which to plan and conduct new clinical trials using stem cell-based therapies for PD. Workshop participants identified priorities for research, development, and funding; discussed existing resources and initiatives; and outlined a path to the clinic for a stem cell-based therapy for PD. A consensus emerged among participants that the development of cell replacement therapies for PD using stem cell-derived products could potentially offer substantial benefits to patients. As with all stem cell-based therapeutic approaches, however, there are many issues yet to be resolved regarding the safety, efficacy, and methodology of transplanting cell therapies into patients. Workshop participants agreed that designing an effective stem cell-based therapy for PD will require further research and development in several key areas. This paper summarizes the meeting.

Keywords: Cell therapies; Clinical trials; Experimental models; Parkinson’s disease.

Figure 1.

Coronal section of the human brain showing the relevant regions (left) and circuits (right) underlying the direct and indirect motor pathways under normal physiologic conditions. Neurons in the primary motor cortex synapse project to medium spiny neurons in the striatum, which, in turn, regulate motor activity via two mutually antagonistic circuits known as the direct and indirect pathways (reviewed in [3, 4]). Abbreviations: GPe, external globus pallidus; GPi, internal globus pallidus; SNc, substantia nigra pars compacta; SNr, substantia nigra pars reticulata; STN, subthalamic nucleus; STR, striatum.