Clinical trials are the final hurdle which potential new treatments for Parkinson’s disease (PD) must clear before getting approved to hit the shelves of your local pharmacy. Despite the critical role of these trials in drug development, doctors and scientists rarely receive formal training in designing and running them.
To help fill this gap, nearly a decade ago, the Parkinson’s Disease Foundation (PDF) collaborated with the Parkinson Study Group (PSG), the nation’s leading consortium of clinical trial investigators, to establish a new training fellowship called the Mentored Clinical Research Award. The fellowship provides up-and-coming scientists with the opportunity to conduct one year of patient-oriented research in Parkinson’s under the mentorship of an experienced investigator. The goal is to prepare scientists with the tools they need to turn laboratory findings into new treatments for people living with Parkinson’s.
The first recipient of this grant, back in 2004, was Wendy Galpern, M.D., Ph.D., a promising young scientist who had already earned her doctoral and medical degrees, and completed both a residency in neurology, and a clinical fellowship in movement disorders.
During her PDF-funded fellowship, while she was mentored by Anthony E. Lang, O.C., M.D., and Susan Fox, M.D., at Toronto Western Hospital, Dr. Galpern says, “I was able to design and implement a single-center clinical trial.” She investigated a drug originally designed for epilepsy, to see if it affected the involuntary movements called dyskinesias, which are experienced by some people with PD. While the trial was challenging — for example, it was difficult to recruit volunteers — the lessons learned were put to good use in future research.
The same lessons also guided Dr. Galpern’s next career move. As she describes it, “The PDF-PSG Fellowship gave me the opportunity to learn about the many aspects of clinical trials — including design, protocol development, regulatory requirements, informed consent, recruitment and involvement of participants, and data collection and analysis.”
After a year of immersion in these topics, she contemplated a clinical research career at an academic medical center. Instead, she chose a different, yet equally pivotal role: serving as a program director in the Office of Clinical Research at the National Institute of Neurological Disorders and Stroke (NINDS). In this capacity, she serves at the hub of not just a single clinical study of a treatment for Parkinson’s disease or another neurological disorder, but of many such studies — using her big-picture knowledge to shape successful research studies.
Coming full circle from her time as a mentee of Dr. Lang, Dr. Galpern guides doctors who are designing clinical trials. She helps them to target critical needs in PD therapeutics and to run well-thought-out trials. She helps them to refine their research questions and to develop plans for gathering and analyzing data that will help us to better understand the potential of new treatments for Parkinson’s disease.
She also serves on committees overseeing individual clinical trials supported by NINDS and organizes workshops for scientists on clinical trial design issues.
Looking back over a decade, she sees the opportunity that PDF gave her as,“reinforcing my interest in translating basic science findings into clinical investigations that will help to expedite new therapies for people with Parkinson’s disease.”
Is there anything more worthwhile?
Dr. Galpern’s research was funded via PDF’s Fellowship and Career Development Program. In FY2013, PDF is supporting this program with $1.38 million.