The Michael J. Fox Foundation (MJFF), with input from experts in drug discovery, biotech investment and academic spaces, authored a paper for npj Parkinson's Disease outlining the in-depth selection process for its Targets to Therapies Initiative — showcasing MJFF’s robust approach to prioritizing new therapy targets outside of the most explored, like alpha synuclein and LRRK2.
The paper, called "A Community-Led Initiative to De-Risk and Advance Parkinson’s Disease Therapeutic Targets," is a detailed and in-depth description of the mechanics of the program, which used input from the Parkinson’s research community to determine a top list of 21 proteins – targets – for scientific exploration of their therapeutic potential.
The Targets to Therapies Initiative is a key component of MJFF’s efforts to improve and accelerate the drug development pipeline, bringing new treatment options for people with Parkinson’s that address both the progression of PD as well as the most challenging symptoms.
The paper emphasizes the structured, community-driven methodology used in Targets to Therapies to evaluate emerging therapeutic targets then make the knowledge gathered publicly available to facilitate drug development.
These targets stem from efforts across the field, including rich inputs from MJFF research program datasets and collaboration with the Aligning Science Across Parkinson’s (ASAP) initiative-supported programs including the Collaborative Research Network (CRN), the Parkinson’s Progression Markers Initiative (PPMI) and the Global Parkinson’s Genetics Program (GP2).
By focusing on fundamental knowledge gaps around these targets, the initiative aims to make developing therapeutics for them less risky, overcoming barriers that have historically hindered progress in PD therapeutics, such as a lack of detailed, well-characterized target profiles. Through this initiative, MJFF aims to build out comprehensive data profiles, tools, assays and biological data to validate approaches and a knowledge base to showcase findings. The Foundation also will establish networks of expert researchers with foundational knowledge and experience around each target.
Targets to Therapies will rely on three interconnected core pillars:
- Target Prioritization and Selection
- Target Validation and Toolkit Development
- Target Knowledge Base
“As we move forward, the Targets to Therapies initiative represents a pivotal step in advancing Parkinson’s research and therapeutic development,” said Shalini Padmanabhan, head of translational research at MJFF. “By fostering collaboration and maintaining a transparent, open-science approach, MJFF aims to address the unmet needs of PD patients through innovative and evidence-driven therapeutic strategies.”
MJFF encourages the global research community to engage with this initiative and contribute insights that will shape the future of PD therapeutics, both by participating in ongoing knowledge collection efforts and by offering relevant expertise. Together, we can build a path toward transformative progress for millions affected by Parkinson's disease.
Learn more about the Targets to Therapies Initiative and read the npj Parkinson's Disease paper.
If you would like to get in touch with the Targets to Therapies team, they can be reached at targets2therapies@michaeljfox.org.