Editor's Note: For more Foundation news and research updates, read the Spring/Summer 2018 edition of The Fox Focus, our biannual newsletter.
At The Michael J. Fox Foundation, we are investing tens of millions of dollars in genetic research. We frequently report back to you on the progress this science is helping us make toward breakthrough treatments, and even a cure, for Parkinson's disease. But how does a genetic finding become a direction to a new treatment?
A brief history of Parkinson's genetics: 25 years ago, scientific consensus held that little to no understanding of Parkinson's disease was to be found in our DNA. Ten years later, as the Human Genome Project was drawing to a close, this opinion had changed, with about 10 genetic changes discovered to be linked to PD. As I write this letter, more than 80 genetic associations have been implicated in Parkinson's. Every one of these gives the field something critical: a starting point to investigate cellular changes that mark disease -- and to search for new ways to stop them.
Certain genes play such a major role in PD onset and progression that they are drawing direct lines to new treatments. Today, experimental drugs are in human testing to target two of the most common genetic mutations linked to PD -- GBA and LRRK2. Scientists call these "precision medicine" therapies, because they precisely target and treat individuals with specific genetic mutations, and are working on finding more of them.
But our great hope is that precision medicine approaches eventually will benefit a much broader population of people with PD, including the great majority of patients -- those without known genetic mutations. Research undertaken with the help of mutation carriers provides tremendous insight into cellular function in everyone with the disease, offering clues researchers follow from genes to other dysfunction we may be able to target. This journey -- from a narrow to a broad population -- is not uncommon in biomedical research. Statins, for example, now taken by millions to reduce the risk of heart disease, were first tested and prescribed exclusively in people with a family history of high cholesterol.
With your support and participation, Parkinson's research is in the midst of a genetics revolution. I look forward to continuing to share news with you of the advances you are making possible. Together, we will change the course of Parkinson's history. Thank you for all you do.
17 New Genetic Links Identified
In fall 2017, scientists identified 17 new genetic changes linked to PD. These findings can help researchers plot the biology of Parkinson's and perhaps find new ways to stop it. Data for this and other genetic studies are coming from MJFF-led programs such as the Parkinson's Progression Markers Initiative (PPMI). Learn more at michaeljfox.org/ppmi.
18,500+ Enrolled in Fox Insight
The MJFF-led Fox Insight study, in a collaboration with genetic testing company 23andMe, is collecting data on Parkinson's genetics and daily life with disease. Scientists can use this information to discover new genetic links to PD and learn more about those they've already identified. Learn more at foxinsight.org.