On October 30, more than 300 leaders in Parkinson's disease (PD) research and business development gathered in NYC for The Michael J. Fox Foundation's 11th Annual Parkinson's Disease Therapeutics Conference. The event featured the latest in Parkinson's drug development and innovative research from the Foundation's portfolio.
David Standaert, MD, PhD, University of Alabama at Birmingham, set the stage by recognizing progress in the 200 years since James Parkinson first described his namesake disease in "An Essay on the Shaking Palsy." Dr. Standaert noted that we are closer than ever to realizing Dr. Parkinson's vision that we could one day slow or stop the disease. Read on to learn how the field is driving toward that goal.
Advancing Treatments to Affect Disease Course
The day's first presenter, Trent Martin Woodruff, PhD, The University of Queensland, discussed the potential role of brain inflammation and a specific protein complex (the inflammasome) in Parkinson's. His team developed a drug to inhibit this protein and limit inflammation, with the goal of slowing or stopping disease progression. Pre-clinical work on the drug has shown positive indications.
David Sulzer, PhD, Columbia University, expanded on the idea of immune system (the body's defense system) involvement in PD. He talked about how alpha-synuclein (sticky protein) clumps might activate an autoimmune response and harm cells. Blocking this response might be a way to impact Parkinson's progression and more work will investigate this approach.
Another route to potentially modify disease includes clearing out poorly functioning mitochondria (cells' energy powerhouses), which can build up and damage brain cells in PD. Paul Thompson, PhD, Mission Therapeutics, Inc., reported positive early results in developing a therapy that would clean up cells to prevent dopamine neuron loss.
Understanding and modifying mutations in a PD-associated gene, VPS35, is the focus for researchers led by Darren Moore, PhD at Van Andel Research Institute. Dr. Moore discussed the team's work in exploring the gene's normal function, its connection to alpha-synuclein, and ways that targeting this gene might lead to new therapies to slow or stop disease progression.
Progress in the Parkinson's Pipeline
Thomas Foltynie, PhD, MD, MRCP, shared results from an MJFF-funded Phase II trial evaluating the injectable type 2 diabetes drug exenatide in Parkinson's. Data showed potential benefit, but until exenatide's role in PD is conclusively evaluated, patients are urged not to add it to their Parkinson's medication regimen. Using a therapy developed for one condition to treat another, or repurposing, gives researchers an alternative route for drug development. Download MJFF's guide on repurposed therapies in PD to learn more.
Closing out the morning, Bernard Ravina, MD, MS, Voyager Therapeutics, reported interim results from an ongoing Phase Ib, open-label trial testing a gene therapy that helps the brain make dopamine from levodopa, the gold standard treatment for Parkinson's symptoms. The findings build on previous positive reports of the therapy's safety and feasibility in PD. Additional updates are expected in 2018.
Tracking and Measuring Disease
The afternoon began with updates in research on biomarkers, or ways to track and measure Parkinson's disease. Edilio Borroni, PhD, F. Hoffman-LaRoche, used specialized brain imaging (PET scans) to look at alpha-synuclein clumps in the brains of people with mid and late-stage PD. He was able to identify some alpha-synuclein, but more work remains to enhance pictures and improve detection.
Brit Mollenhauser, MD, Paracelsus-Elena-Klinik, and Andrew Singleton, PhD, NIH National Institute on Aging, were up next with analyses from MJFF's landmark PD biomarker study, the Parkinson's Progression Markers Initiative (PPMI). Dr. Mollenhauser examined markers that could indicate the presence or progression of disease. The results, which showed lower and stable levels of spinal fluid proteins in recently diagnosed people, may inform therapeutic development. Dr. Singleton spoke to the strength of PPMI's genetic cohort, which is helping investigators learn more about the genetic risk factors for Parkinson's.
Pathways to Promote Progress
The afternoon closed with updates on cutting-edge tools and science advancing disease understanding. D. James Surmeier, PhD, Feinberg School of Medicine at Northwestern University presented his work testing a combination therapy (blood pressure drug isradipine and PD medication rasagiline) for Parkinson's. Evidence suggests that, together, they might alter pathways associated with PD and potentially slow or stop the disease. Thomas Gasser, MD, PhD, University of Tuebingen, talked about how therapies targeting PD-linked genetic mutations might be beneficial for people with sporadic (cause unknown) Parkinson's. Dr. Gasser suggests that expanding the pool of genetic information available to researchers could uncover potential disease associations and deepen understanding of PD.
It was a full day featuring some of the best and most promising advances in Parkinson's research. We left with hope that the year ahead will speed critical breakthroughs for millions living with the disease.