MJFF is Assembling Experts to Research New Parkinson’s Targets

The Michael J. Fox Foundation (MJFF) has assembled top experts worldwide to accelerate research on five lesser-known biological targets in Parkinson’s disease, aiming to uncover potential treatment options. An additional seven targets will be explored in a later phase. This initiative is part of MJFF’s Targets to Therapies Initiative, which bridges basic research and clinical development by fostering collaboration among scientists, industry researchers, and clinicians. With tens of millions of dollars in funding, the program aims to enhance scientific understanding of these molecular targets, making them more attractive for drug development. Shalini Padmanabhan, PhD, MJFF’s senior vice president of discovery and translational research, emphasized that increasing the number of well-characterized, druggable targets is key to advancing Parkinson’s treatments. Parkinson’s disease, a neurodegenerative disorder marked by the gradual loss of dopamine-producing neurons, currently has no cure, and existing treatments primarily manage symptoms rather than slowing disease progression. While some druggable targets have been well-studied, MJFF is focusing on additional biological targets that may offer new approaches to slow disease progression and improve symptom management. The initiative, launched in 2024, began with a global survey identifying over 280 potential targets, informed by MJFF research programs and collaborations like the Aligning Science Across Parkinson’s initiative. Five high-priority targets were selected for immediate research: 1) TMEM175, a protein involved in clearing cellular waste, which may prevent toxic buildup in nerve cells. 2) ATP13A2, a gene linked to early-onset Parkinson’s, which could offer insights into broader disease mechanisms. 3) MCOLN1 (TRPML1), a gene involved in lysosomal function, which may help remove excess alpha-synuclein, a misfolded protein associated with Parkinson’s progression. 4) OGA, a protein that, when inhibited, might protect dopamine neurons from degeneration. 5) NOD2, a protein linked to neuroinflammation, which researchers hope to target to reduce inflammation and slow disease progression. Stacie Weninger, PhD, a member of the initiative’s governance committee, highlighted the foundation’s focus on identifying the most promising targets to drive new therapies forward. By the end of 2025, MJFF will launch a public knowledge base to share findings, aiming to reduce research barriers and accelerate drug development for Parkinson’s.