Last month, CEO, of The Michael J. Fox Foundation (MJFF), Todd Sherer, PhD, led a panel at the Precision Medicine Leaders Summit called “The Power of Prediction — New Models for Neurodegenerative Disease.” Though precision medicine approaches are increasingly common in cancer, they are in early stages for brain diseases such as Parkinson’s and Alzheimer’s.
MJFF is working to usher in the era of precision medicine in Parkinson’s. This approach has the potential to help every patient by tailoring treatments based on each person’s unique disease. As panelist Melissa Nirenberg, MD, PhD, of the New York Stem Cell Foundation pointed out: “No two people with Parkinson’s are the same.” The task now is to deepen our understanding of all the factors that can contribute to a person’s disease and then, as Todd added, “convert that understanding into smarter therapies.”
Lawrence Severt, MD, PhD, of Allergan, elaborated on the point. While researchers used to believe that there were no genetic factors involved in Parkinson’s, Severt said: “We now know that’s definitely not true.” Research has demonstrated that several genes — including LRKK2 and GBA — affect Parkinson’s risk. Tests have been developed to check for mutations in these genes, and MJFF is funding the testing of potential therapies for people who carry those mutations. But this is just the beginning of what precision medicine could do.
In other diseases such as certain cancers and cystic fibrosis, precision medicine began similarly, with drugs designed for a small subset of patients. Researchers built on those successes to develop drugs to benefit more and more people. The goal in Parkinson’s is to create tests that can tell doctors exactly what factors are driving each patient’s disease, and then develop a therapy or combination of therapies that will help in all of those cases.
The Michael J. Fox Foundation is working to increase understanding of Parkinson’s disease through landmark projects including the Parkinson’s Progression Markers Initiative and Fox Insight. These programs are helping to map the biological causes of Parkinson’s and establish how these differ across distinct groups of people. With this information, researchers can develop diagnostic tools and precision medicine approaches to help ever more of the estimated five million living with Parkinson’s worldwide.