On November 29, the Parkinson’s Action Network (PAN) and Vanderbilt University co-hosted a briefing – “Partnering Against Parkinson’s: How Academia, Government, Patient Organizations, and Industry are Working Together for New Treatments” – in the Rayburn House Office Building for Capitol Hill staff and members of the biomedical research community.
At the briefing, panelists discussed the innovative collaboration between Vanderbilt University, The Michael J. Fox Foundation for Parkinson’s Research (MJFF), and Bristol-Myers Squibb (BMS) to develop a promising new treatment for Parkinson’s disease. Representatives from each organization discussed how their arrangement could become a model for future public/private partnerships to develop new treatments and cures for diseases like Parkinson’s.
From L to R: MJFF CEO Todd Sherer, Ph.D.; Vanderbilt Center for Neuroscience Drug Discovery Director Jeff Conn, Ph.D.; PAN CEO Amy Comstock Rick; Vanderbilt School of Medicine Vice Chancellor for Health Affairs & Dean Jeff Balser, M.D., Ph.D.; and Bristol-Myers Squibb Neuroscience Executive Director Charlie Albright, Ph.D.
Jeff Conn, Ph.D. , Director of the Vanderbilt Center for Neuroscience Drug Discovery , explained that early NIH-funded research at Emory University identified a neurotransmitter receptor in Parkinson’s disease and an agonist that could dramatically decrease the presentation of Parkinson’s symptoms. However, pharmaceutical companies still believed that the research was too preliminary to bring to market. This promising discovery risked never being developed into a drug that could help patients.
Todd Sherer, Ph.D. , CEO of The Michael J. Fox Foundation for Parkinson’s Research , described the foundation’s role in providing Vanderbilt with a total of $4.6 million in grants to conduct “translational” research about this compound, collecting additional data and bringing it closer to the stage where a pharmaceutical company would feel confident enough in the findings to invest heavily.
Charlie Albright, Ph.D. , Executive Director of Neuroscience at Bristol-Myers Squibb , then discussed BMS’s role in this partnership. BMS is currently funding the compound through Phase I clinical trials. Dr. Albright emphasized that the role of MJFF in this process was critical, and he urged Congress to fund NIH at high levels to address the tremendous unmet need in translational research so that promising research ideas do not sit idle.
“This briefing illustrated a well-known fact in the research community: too many promising discoveries that are funded by NIH never make it to clinical development,” said PAN CEO Amy Comstock Rick. “That is why PAN played a key role in the creation of a new center at NIH – the National Center for Advancing Translational Sciences  (NCATS).” NCATS brings together NIH’s translational activities and expertise into one Center, aimed at supporting and strengthening translational research; fostering collaboration between federal agencies, the private sector, and non-profits; and supporting and training current and future translational research investigators.
During an engaging question and answer session, the panelists agreed that sequestration  would have a devastating effect on science. Dr. Sherer stated that the number of people diagnosed with Parkinson’s each year is expected to grow as the population ages, so now is not the time to decrease our investment in promising research. As an example, NIH-funded basic research could provide more information about the complexities of the human brain, allowing researchers to model brain disorders more accurately. As more is learned about the brain, it will reduce the risk for pharmaceutical companies in investing in new neurological treatments for diseases.
PAN looks forward to hosting more educational briefings in the future as part of its efforts to inform policymakers about the importance of federal research funding and strong policies for the 500,000 – 1.5 million Americans living with Parkinson’s disease.