#United States #New York #Parkinson's Disease #Michael J. Fox #Treatment Targets

MJFF's New Frontier in Parkinson's Disease Research

The Michael J. Fox Foundation for Parkinson's Research (MJFF) has taken a significant step forward in the quest for effective treatments for Parkinson's disease with the launch of its Targets to Therapies (T2T) Initiative. Announced on December 8, 2025, this new program aims to validate emerging therapeutic targets, a process crucial for the development of new treatments for those living with Parkinson's.

Background on the T2T Initiative

The T2T Initiative is a multi-year collaborative effort designed in conjunction with top scientists from academic institutions, the private sector, venture capitalists, and global Parkinson's disease networks. The initiative seeks to mitigate the risks and uncertainties that have historically impeded the progress in developing effective therapies for Parkinson's disease. Over the past two years, MJFF has worked alongside a global network of scientific and clinical advisors to critically evaluate over 280 nominated targets, employing a structured prioritization framework that draws upon substantial contributions from MJFF’s research programs and partnerships like the Aligning Science Across Parkinson's (ASAP).

From this extensive evaluation, 21 priority targets emerged, laying the groundwork for the validation efforts to follow. This marks a transition from merely identifying potential targets to actively validating their therapeutic viability.

Funding and Early Validation Efforts

MJFF has committed an initial $7.5 million in early validation grants within the T2T framework, focusing on three multidisciplinary validation teams. These teams will focus on high-priority targets, including NOD2, OGA, and key endolysosomal mechanisms such as TRPML1, TMEM175, and ATP13A2. Each of these targets is linked to crucial biological processes that play a role in Parkinson's disease, such as the removal of toxic proteins and maintaining neurological health.

This proactive shift towards validation addresses one of the primary bottlenecks in therapeutic development—many promising targets still lack sufficient early mechanistic evidence, essential tools, and experimental models to make significant advances.

Expanding Research Collaborations

According to Dr. Gaia Skibinski, Director of Translational Therapeutics at MJFF, this initiative is designed to broaden the pathways towards developing new treatments for Parkinson's disease. The transition from identifying promising targets to building the necessary evidence for their validation is critical for expanding the pool of viable drug candidates in the pipeline. The T2T initiative also aims to provide a transparent and shared view of the therapeutic landscape through its T2T Target Explorer platform.

Through this digital open-science resource, approximately 59 high-potential target profiles will be made available, showcasing where knowledge gaps exist and outlining proposed validation approaches. The platform is poised to serve as a hub for curated evidence, expert assessments, and dynamic validation plans, encouraging collaboration among researchers, pharmaceutical companies, and other stakeholders.

The Path Ahead

The initiative reflects MJFF’s commitment to transforming Parkinson's research into practical therapies for patients. The collaborative nature of T2T emphasizes the necessity of open data access and pooling of expertise across industry and academia—essential components in speeding up the development of effective treatments.

As contracting for a number of validation projects progresses towards completion by year-end, a surge of interest is anticipated from academic and industry teams eager to engage with high-priority targets. The launch of additional validation teams in 2026 promises to enhance the foundation's investment in translational research, ultimately broadening the landscape of druggable biology for Parkinson's disease.

This collective effort is vital for unlocking the next generation of therapeutics, aiming to move from potential options to real solutions for the millions impacted by Parkinson's globally.