The Michael J. Fox Foundation (MJFF) devotes our donor-raised dollars to promising scientific efforts that will help bring new treatments and cures to people with Parkinson's. In this bimonthly report, we review some of the 46 grants totaling more than $21 million we awarded in June and July. See a full list of MJFF-funded studies.
Improved Approaches to Treat Symptoms
Some grants went to new approaches to ease hard-to-treat symptoms.
- Jay Alberts, PhD, at Cleveland Clinic is looking for a new way to treat freezing of gait. This symptom is hard to recreate in a medical clinic, which makes it more difficult to study. Alberts’ team will use virtual reality to trigger gait freezing then monitor brain activity. That information could lead to new ways to use deep brain stimulation (DBS) to target that brain activity and ease gait freezing.
- Oliver Rawashdeh, PhD, at the University of Queensland in Australia, with partner Biogen, will evaluate a drug in pre-clinical models that may help people with PD sleep better. The drug targets a protein that plays a role in the body’s biological clock. The team will also study if better sleep helps protect cells from other changes seen in PD such as inflammation.
- MapLight Therapeutics is studying a protein that may play a role in dyskinesia, uncontrollable movements. Dyskinesia arises from unbalanced brain activity in a region called the striatum. Activating a protein in the striatum may help restore balance and reduce dyskinesia.
Gene Profiling and Therapies for Genetic Targets
Studying gene changes tied to Parkinson’s can give scientists a starting point: what goes wrong in PD and how might we fix it to slow or stop disease? Those findings may apply to people with PD even without that same gene change. Some new projects are testing ways to target genetic pathways while others are further studying some PD-linked genes.
- Nicoletta Plotegher, PhD, at the University of Padua in Italy is testing nanobodies — antibody fragments — for their impact on improving GBA function. Mutations in GBA can cause PD, and dysfunction in this pathway is seen in people with PD without this mutation.
- Similarly, Mission Therapeutics is studying an approach to offset dysfunction in the PRKN pathway. Changes in the PRKN gene are linked to early-onset PD and impact mitochondria (the cell’s powerhouse).
- Sreeganga Chandra, PhD, at Yale University is studying the role of another PD-linked gene: auxilin. The auxilin protein plays a role in how cells communicate. Chandra’s team will study how disrupting auxilin function may contribute to PD.
- Edward Fon, MD, at McGill University in Canada will examine six lesser-studied Parkinson’s genes (part of the “dark genome”) to better understand their role in PD. This work could lead to treatment strategies against these targets.
New Ways to Understand and Measure Parkinson’s
Researchers also look for ways to better understand and measure PD. Tools to define, predict and monitor disease can help point to new therapeutic approaches and to ways to assess treatment impact.
- Miquel Vila, MD, PhD, at Vall d'Hebron Institute of Research in Spain is researching why some studies have shown more men develop PD. Men may build up a dark pigment (neuromelanin) faster, which could impact cell function. The team will use PD models to explore neuromelanin levels in males versus females, how those levels relate to PD, and the impact of modulating neuromelanin levels.
- Two projects capitalize on work that arose from a woman who could smell a distinct scent on people with Parkinson’s. That prompted MJFF-funded research that found that people with PD have differences in sebum (an oily substance on the skin). Now Marianna Cortese, MD, PhD, at Harvard University and her group will study sebum of people with PD risk factors. They will look for similar changes to understand if this sebum test may help predict Parkinson’s disease. And Paul Feinstein, PhD, at the City University of New York will work toward a test for the Parkinson’s “scent.”
- Four new projects are funded through the PIPETTE Consortium (Philanthropic Investments in PET TracErs), a partnership formed in 2017 by The Michael J. Fox Foundation and Rainwater Charitable Foundation. These projects seek to develop imaging tools (PET tracers) to visualize two key proteins in the living brain: tau and alpha-synuclein. Both are present in brain diseases such as Parkinson’s, Lewy body dementia and frontotemporal dementia.
We fund the most promising, cutting-edge projects — from using virtual reality to trigger gait freezing to measuring a dark pigment in cells of men — to discover the therapies and strategies that will improve daily life for people living with Parkinson’s today and achieve a tomorrow without the disease.
Interested in joining a study? Our landmark Parkinson’s Progression Markers Initiative (PPMI) is open to anyone over age 18 in the U.S. Take a short survey to get started. (Outside the US? Some international sites are recruiting.)