The Michael J. Fox Foundation (MJFF) announces 57 grants totaling more than $34.5 million awarded in August and September. These supported projects aim to predict and define early Parkinson’s disease (PD) diagnoses and validate treatments.
Here we review some projects expanding our understanding of PD research, diagnostic tools and potential therapies. See full list of MJFF funded studies.
Disease Modifying Therapies
Early-onset PD diagnoses, linked to genetic mutations, have been on the rise. A group of scientists led by Deniz Kirik, MD, PhD, at Lund University studies the relationship between a promising target in this population, the gene that codes the parkin protein, and neuroinflammation in pathways causing PD. This pre-clinical study will determine whether increasing Parkin protein levels reduced brain inflammation and protects cells.
Another target for disease- modifying therapies, the Kv1.3 protein, appears in multiple MJFF-funded projects. This potassium channel is linked to the toxic environment in the brain that causes brain cell loss and motor symptoms. Here we list two studies with the same target:
-
Researchers at Muna Therapeutics aim to develop a novel therapy to reduce brain inflammation and ensure the function and survival of brain cells in PD by blocking the protein, Kv1.3. This pre-clinical study is driving towards novel therapeutics for PD.
-
Similarly, Ildiko Szabo, PhD, and his team at the University of Padua identifies Kv1.3 as a high- priority target. They explore different ways to manipulate and validate the protein as a potential drug candidate for motor symptoms.
Treatment for Symptoms
While there are medicines on the market to relieve motor symptoms, the effects wane over time, and long-term use in people with advanced stages of PD often leads to side effects such as dyskinesia. When motor symptoms become drug resistant, another way to control them is Deep Brain Stimulation (dbs), which is invasive and comes with unique challenges. Principal investigator Fabio Benfenati, MD, at The Italian Institute of Technology will lead a project to develop minimally invasive nano technology that can rescue nerve cells inactivated through dopamine cell loss, without some of the complications of dbs. If successful, this procedure can be done through a simple, intravenous injection to drive neuron activation in areas of the brain that are hard to reach, thereby restoring motor functions previously degraded or lost.
Tools to Predict and Define Disease
People with PD often suffer symptoms years before diagnosis, like REM sleep behavior disorder (RBD). Kelvin Luk, PhD, and his team at the University of Pennsylvania will explore a region in the brain called the sublaterodorsal tegmental nucleus (SLD) for its role in RBD and potential connection to the development of PD. This work could help scientists better understand early PD and the relationship between RBD and PD.
Constipation is another non-motor symptom of PD that severely impacts the quality of life. Using clinical and genetic data collected in our Foundation’s Fox Insight study, Rodolfo Savica, MD, PhD at the Mayo Clinic compares the prevalence of gastrointestinal symptoms in people with early-onset versus late-onset PD. Associations with constipation can be used as a predictive tool and identify possible subgroups of patients and differences in prognosis between early onset vs late onset PD.
Another team, led by Conor Fearon, MD, PhD, an MJFF Edmond J. Safra Fellowship alumni and the 2022 awardee of the Edmond J. Safra Movement Disorders Research Career Development Program at Mater Misericordiae University Hospitals, will examine an eye- tracking method aimed at diagnosing early-stage PD and atypical parkinsonism, while tracking their cognitive and motor functions. This two-year study measures the pupil changes in the eyes of participants with PD and atypical parkinsonism as they watch various short clips. It’s hypothesized that data from these eye movements can be used as a tool to track disease progression in clinical studies and could even predict the disease before it starts.
As it is today, the lack of tools to accurately diagnose and treat this disease is a huge barrier for patients. The Michael J. Fox Foundation continues to fund advances in technology and medicine to drive towards effective therapies that can prevent, slow or stop disease progression.
The Parkinson’s Progression Markers Initiative (PPMI) is our landmark study on a mission to stop the disease. It is open to anyone over age 18 in the United States. Whether you have Parkinson’s or not, join the study that could change everything.
Recently diagnosed with PD or live outside the U.S.? Connect with the PPMI team.