Last Thursday, The Michael J. Fox Foundation (MJFF) hosted a capacity crowd of leaders from industry, nonprofits and academia at our 12th annual Parkinson's Disease Therapeutics Conference, held in New York.
The conference kicked off with a welcome from MJFF CEO Todd Sherer, PhD, who spoke about the "incredible momentum" in Parkinson's research. He said: "Our ultimate goal is to develop new and better treatments for people with Parkinson's disease. That's why we are here -- to work collaboratively toward that goal."
In her opening remarks, this year's conference chair, Carole Ho, MD, of Denali Therapeutics thanked MJFF for "its amazing work supporting Parkinson's disease therapeutics with a patient focus." She went on to explain that the science of Parkinson's has evolved thanks in part to our deeper understanding of Parkinson's genetics, including GBA, PINK1 and LRRK2. Genetics were a focus for several presenters, including Dr. Ho's colleague Matthew Troyer, MD, who headlined the "Hot Topics in Parkinson's Disease Research" session.
Highlights from Dr. Troyer and other presenters follow:
- Matthew Troyer, MD, of Denali Therapeutics presented, for the first time, data from the company's Phase 1 clinical trial of LRRK2 inhibitor DNL201. The drug was found to be generally safe and well-tolerated in study volunteers, with target and pathway engagement. Denali's trials are the first-in-human studies of LRRK2 inhibitors, and the positive data it shared was featured in "Forbes." Based on the positive results, Denali plans to initiate trials in Parkinson's disease patients with and without LRRK2 mutations.
- Suzanne Pfeffer, PhD, from Stanford University presented research on how LRRK2 gene mutations affect our brains. She explained that LRRK2 modifies a type of Rab protein, which eventually leads to damage of brain cells. This finding provides important new targets for developing drugs to combat LRRK2 mutations and slow or stop Parkinson's disease.
- Two presenters shared progress made possible by MJFF's landmark Parkinson's Progression Markers Initiative (PPMI). David Craig, PhD, of the University of Southern California and his colleagues used PPMI samples to develop the largest RNA sequencing data set ever made available. This extraordinary resource includes more than 1 billion reads per sample. Dr. Craig explained that, when combined with genetic data sets from PPMI, this RNAseq data could help identify genetic drivers of Parkinson's and other diseases. Kenneth Marek, MD, of the Institute for Neurodegenerative Disorders, summarized the breadth of data available to researchers through PPMI and presented information on the clinical predictors of Parkinson's disease risk identified through the study, including REM sleep behavior disorder, smell loss and certain genetic mutations.
- Bastiaan Bloem, MD, PhD, from Radboud University shared his findings on the rapidly emerging field of wearable and digital measurement devices. In a series of studies, he and his colleagues demonstrated the feasibility and applicability of wearable sensors for Parkinson's. Wearable devices can address challenges of assessments done in doctor's offices -- which only capture a single moment of a complex, variable disease -- and provide a more complete picture of how patients experience Parkinson's.
MJFF investments are helping build a robust pipeline of drugs in development for Parkinson's disease. We look forward to sharing even more positive news at our conference next year.