MJFF assembling experts toward research on new Parkinson’s targets
5 genes, proteins to be studied to ID promising treatment opportunities
The Michael J. Fox Foundation (MJFF) has brought together top experts from around the world to speed up research on five lesser-known biological targets in Parkinson’s disease in an effort to identify promising treatment opportunities. Seven more will be explored in a future phase.
This effort is part of the foundation’s Targets to Therapies Initiative, which seeks to bridge the gap between basic research and clinical development by fostering collaboration among scientists, industry researchers, and clinicians toward new Parkinson’s treatments.
With “ten of millions of dollars” in funding, the initiative will support research strategies to deepen the scientific understanding of these novel molecular targets, making them more attractive for drug developers to invest in and develop as potential targeted therapies.
“The Michael J. Fox Foundation exists to increase the number of available treatments for people and families with Parkinson’s. Expanding the number of well-characterized, druggable targets is an important step in that direction,” Shalini Padmanabhan, PhD, MJFF’s senior vice president of discovery and translational research, said in a foundation press release. “Ultimately, the knowledge gives us a field-wide view of emerging targets so that we can prioritize those that give us the best chance at new therapies.”
A neurodegenerative disorder, Parkinson’s disease, which features the gradual loss of dopamine-producing neurons in the brain, remains without a cure as current treatments are primarily focused on managing symptoms rather than modifying the disease’s progression. Dopamine is a chemical messenger involved in motor control.
Exploring new treatment targets
While several druggable targets have been well-characterized and are being explored as possible treatments, the MJFF’s initiative explores additional biological targets that could offer new ways to slow the disease’s progression and aid symptom management.
Launched in 2024, the Target to Therapies Initiative began with a global survey of the scientific community, which generated a list of more than 280 possible targets.
This included data from MJFF research programs and collaborations with the Aligning Science Across Parkinson’s initiative, such as the Collaborative Research Network, the Parkinson’s Progression Markers Initiatives, and the Global Parkinson’s Genetics Program.
Five high-priority targets were chosen for immediate exploration, with seven more set for future investigation.
“It’s not just about finding druggable targets. It’s about finding the most promising druggable targets. We are looking to make advances that lead to new therapies. With the collective might of the scientific community, we can achieve that goal,” said Stacie Weninger, PhD, who sits on the Targets to Therapies Initiative governance committee. “That’s what The Michael J. Fox Foundation has done here — assemble many of the best minds in [Parkinson’s] research around the pressing need for new ways to treat Parkinson’s.”
One target is TMEM175, a protein involved in clearing out cellular waste. Malfunctions in TMEM15 may lead to the toxic buildup of substances in nerve cells, potentially worsening Parkinson’s.
Another important target is ATP13A2, a gene linked to a rare, early-onset form Parkinson’s disease. Understanding ATP13A2 could reveal insights into broader disease mechanisms.
MCOLN1 (also known as TRPML1) is a gene that codes for a protein involved in cellular processes, particularly in lysosomal function. The gene has been chosen as a potential target since activating it may help remove excess alpha-synuclein, a misfolded protein that accumulates in the brains of those with Parkinson’s, contributing to disease progression.
Researchers are also looking at OGA, a protein that, when inhibited, might protect dopamine neurons from degeneration, and NOD2, a protein being investigated for its role in neuroinflammation. Researchers want to determine if blocking the interaction between this protein and alpha-synuclein could reduce brain inflammation and slow Parkinson’s progression.
By the end of 2025, the Targets to Therapies Initiative will launch a public knowledge base, making research findings on the targets accessible to scientists and drug developers. This may help accelerate the development of new Parkinson’s treatments by reducing barriers to research and investments.
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