Parkinson's Disease Research Agenda Implementation Review Meeting Cites Progress, Future Directions, and Common Themes
January 9 and 10th, 2002
Scientists, patient advocates, and representatives of nine National Institutes of Health (NIH) components gathered January 9-10 to review progress made in implementing the agency's Parkinson's Disease Research Agenda. In her welcoming remarks, NIH Director Dr. Ruth Kirschstein spoke of the "unprecedented push, unseen ever before," that the NIH has made in Parkinson's disease research since the document was written. Originally released in March 2000, the Agenda describes areas of significant scientific opportunity in Parkinson's research to be explored over the following five years. The January 2002 meeting reviewed progress made towards accomplishing the goals set forth in the Agenda, identified newly emerging areas of interest, and prioritized future research goals.
The general consensus of the attendees is that the NIH is doing a good job implementing the Agenda's initiatives. However, participants identified additional areas of interest too new to have been stressed in the original document, and at least one area, rehabilitation, where current knowledge of the field indicates that additional efforts would be worthwhile.
Two breakout groups-one on the basic mechanisms of Parkinson's disease, the other on its treatment-discussed goals believed to be obtainable over the next 3 years. At the closing plenary session, participants identified several common themes that reinforce these goals:
- Emphasize translational research. Commonly described as "bench to bedside and back," such research incorporates both basic and clinical components into one interactive program to the benefit of both. While such programs are traditionally run by one center or group, the possibility of a virtual center was also discussed, as was the possibility of expanding NIH Intramural efforts in this area. The NIH is already planning a program that may address many of these needs.
- Better understand the abnormal brain circuitry seen in Parkinson's disease. Although many factors that may contribute to the breakdown of dopamine neurons have been identified, the cellular targets and processes are not fully understood. Such knowledge would enable scientists to devise therapies to treat patients after these neurons have been depleted.
- Increase studies on non-motor aspects of Parkinson's disease. These symptoms, which include dementia, depression, sleep abnormalities, and speech and swallowing problems, often bother individuals with Parkinson's more than tremor and rigidity.
- Locate risk and susceptibility factors and identify physiologic, genetic and environmental biomarkers to aid in preclinical diagnosis, evaluate progression of the disease, assess therapies, and speed clinical trials while reducing their costs. Large population studies would help achieve these goals.
- Pursue research related to gene therapy. Studies to determine the best vectors for delivering gene therapy and encouraging sharing of vectors should be emphasized.
- Develop better animal models, particular for dyskinesias and non-motor symptoms.
Other significant issues targeted by the participants include:
- The need for clinical trials of neuroprotective agents and ways to reduce or eliminate dyskinesias.
- The development of surrogate measures, such as imaging and behavioral markers, to better assess non-motor symptoms, disease progression, and screen at-risk populations.
- Studies on the long-term effects of deep brain stimulation on both motor and non-motor symptoms, its mechanism of action, and its unintended consequences.
- Role of behavioral interventions such as voice therapy and exercise.
- Scientifically proven assessments of current treatments .
- Studies on how aberrations in the processing of the alpha-synuclein protein leads to the death of dopaminergic nerve cells.
- Development of assays to screen drugs.
- The need to draw young investigators and scientists from other fields into Parkinson's research.
- The development of resource banks to facilitate access to animal models and critical research tools.
"The feedback from all the participants was extremely gratifying," said Dr. Audrey S. Penn, Acting Director of the National Institute of Neurological Disorders and Stroke, which hosted the meeting, "but I was particularly encouraged by the positive feedback from the patient advocates."