MJFF Resources to Accelerate PD Research

This program seeks to advance the development of high-performance, quantitative alpha-synuclein seed amplification assays (aSyn-SAAs), addressing the critical need for less invasive, highly sensitive and quantitative biomarkers in Parkinson’s disease (PD) clinical trials.

This program seeks to fund clinical testing on personalized approaches for understanding, assessing and improving gait in Parkinson’s disease (PD) to address the wide variances of gait challenges across the continuum of the disease. The program aims to establish standardized assessment and tailor treatments to individual symptoms that improves gait and motor-cognitive functions.

This program seeks to advance therapeutic development through pre-clinical and/or clinical testing of approaches addressing unmet needs of people with Parkinson’s disease (PD). The program is set up to benefit therapeutics with clear potential to prevent, stop, or delay disease progression or to reduce the burden of daily symptoms.

MJFF's new target validation initiative, "Targets To Therapies" (T2T), seeks to increase confidence in targets prior to therapeutic development and speed the translation of promising biology into and across therapeutic pipelines. To see our exciting progress, please view T2T's poster presentation here at the conference today. Additionally, please review results from an RFI sent to the global research community earlier in 2024 with the purpose of sourcing emerging target recommendations to be evaluated within T2T.

The LRRK2 Investigative Therapeutics Exchange (LITE) program aims to support new approaches to targeting LRRK2 with therapies, while also supporting the development of LRRK2-relevant clinical biomarkers to measure progress.

In light of the breakthrough biomarker test that was validated in 2023, the alpha-synuclein seeding amplification assay (aSyn SAA), MJFF is investing heavily in validating quantitative biomarkers for PD. 

In 2010, The Michael J. Fox Foundation and a core group of academic scientists and industry partners launched the Parkinson’s Progression Markers Initiative (PPMI) toward critically needed biological markers of Parkinson’s onset and progression. PPMI has since engaged thousands of partners — more sites; scientists and clinicians; industry experts; philanthropic partners; and, most importantly, study volunteers — to build a cornerstone of Parkinson’s research. Analysis from its open-access data set and available biosample library has deepened understanding of disease and informed design of dozens of therapeutic trials.

The FDA issued a “Letter of Support” in late Summer 2024, following multi-stakeholder collaboration amongst The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and the Critical Path Institute (C-Path), with both organizations supporting use of the biomarker for clinical trials and advancing new drugs for Parkinson’s disease (PD).  

In the letter, the FDA emphasizes that their support stems from significant evidence collected as part of MJFF’s flagship Parkinson’s Progression Markers Initiative (PPMI), which first validated the αSyn-SAA biomarker in April 2023.  This is only the second Letter of Support issued for PD markers. 

Parkinson's disease and dementia with Lewy bodies are currently defined by their clinical features, with α-synuclein pathology as the gold standard to establish the definitive diagnosis. We propose that, given biomarker advances enabling accurate detection of pathological α-synuclein (ie, misfolded and aggregated) in CSF using the seed amplification assay, it is time to redefine Parkinson's disease and dementia with Lewy bodies as neuronal α-synuclein disease rather than as clinical syndromes. This major shift from a clinical to a biological definition of Parkinson's disease and dementia with Lewy bodies takes advantage of the availability of tools to assess the gold standard for diagnosis of neuronal α-synuclein (n-αsyn) in human beings during life. 

The Path to Prevention (P2P) platform trial is designed to catalyze clinical development for prodromal PD. P2P capitalizes on PPMI study infrastructure, expertise, and ongoing clinical and biomarker data collection to de-risk industry investments in pioneering therapies for individuals at risk of PD diagnosis.

This report tracks therapies currently in clinical development for Parkinson’s disease as of late March 2024. It includes brief rationale and progress in key pipelines of therapeutic interest along with detailed listings of specific therapies, current development status and information about MJFF support (funding and/or non-funding connections).

The Parkinson’s Disease Research Exchange (PDRx) is a global network of scientists from academic labs, pharma/biotech, CROs and investors focused on assessing the current state of Parkinson's disease research, identifying key challenges, and how to translate findings into improved therapies. In Fall 2024, MJFF launched the PDRx online platform to enhance networking and facilitate open discussion.

Join hundreds of scientists for the 2nd GBA1 Meeting, returning to Montreal from June 5th-7th, 2025-a golden opportunity to collaborate and advance GBA1 research and treatment.

Topics of discussion will include all aspects of GBA1 research, from basic science to clinical trials. The meeting will serve as a unique platform to collectively advance the understanding and treatment of GBA1-associated neurodegenerative diseases.

The Parkinson's Disease Therapeutics Conference (PDTC) is The Michael J. Fox Foundation's annual scientific conference and the only one in the world focused exclusively on Parkinson's disease drug development. The 17th Annual PD Therapeutics Conference is scheduled to be held on October 16, 2024, in New York City.