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$23M toward Novel Therapies and Technological Advances to Develop Treatments 

The Michael J. Fox Foundation (MJFF) announces 75 grants totaling more than $23 million awarded in June and July. These supported projects are driving toward earlier diagnosis and new treatments for people with Parkinson’s disease (PD).  

Here we review some projects expanding our understanding of PD research, diagnostic tools and potential drug candidates. See full list of MJFF funded studies.   

Therapies Targeting Brain Inflammation 

Brain inflammation remains a key target for scientists studying PD. People with Parkinson’s have inflammation in the brain, which may contribute to cell loss and symptoms. MJFF continues to back researchers as they search for new ways of addressing brain inflammation and its effects.  

Scientists at the Scripps Research Institute are testing the drug PDM608, which is a potential anti-inflammatory candidate to prevent cell loss. PDM608 is a long-acting version of a drug called GM-CSF, which showed promising results and a reduction in symptoms such as tremors and gait challenges. However, GM-CSF had side effects such as body aches and required frequent injections. Researchers hope PDM608 will avoid some of these pitfalls and provide a more viable treatment option. Scripps will test PDM608 with volunteers to ensure safety and tolerability.  

Another novel treatment approach originates with bone morphogenetic proteins (BMP), naturally secreted proteins that play critical roles in nervous system developments. Scientists at the Ben-Gurion University of the Negev in Israel aim to use BMPs to prevent cell loss from brain inflammation. Read more on this potential disease-modifying therapy here. 

Parkinson’s Pathology & Assessing Progression 

The most common practices to diagnose PD and to assess progression require evaluations by a doctor. Whilst effective, these methods do have limitations. MJFF is supporting work toward faster, more objective tests of Parkinson’s disease and efforts to partner with true experts: people living with PD.  

Researchers at the California Institute of Technology will test blood-based markers of PD activity. The team will look at bacterial DNA sequences found in blood, part of what is called the blood microbiome. The researchers hypothesize that distinct microbial patterns can help identify people with PD and potentially even aid in assessing the severity of progression. The goal is to create an unbiased biological measure of PD that would help standardize diagnosis as well potentially reveal new information about the underpinnings of PD. 

Meanwhile, a team led by researchers at Rush University will review reports from study participants to determine which motor and non-motor symptoms impact the quality of life of patients and care partners the most. Understanding the unique course of the disease for each person can impact patient care and drug development, making it possible to deliver effective and individualized therapies for people with PD. 

Technology to Monitor Disease Activity  

Many grants fund studies to understand the underlying pathologies of PD and how to detect and track those changes. Of particular value are technological advances that push the field of Parkinson’s research forward, both in terms of the types of data collected and what can be done with that data.  

Researchers at the University of Liverpool will engineer the Parkin gene so that the protein it encodes, which is frequently mutated in Parkinson’s disease, glows fluorescent. When mutated, the Parkin protein can no longer work as efficiently removing damaged parts of cells in the brain, an observed deficiency in Parkinson’s patients. The researchers aim to generate a fluorescent sensor to monitor the newly glowing protein’s activity. If successful, the engineered Parkin gene’s cell lines and fluorescent sensor can be standardized for use in the research community to enable drug development, specifically for treatments that would help replicate the function of fully functioning Parkin protein.  Read more here.  

Meanwhile, a team at Koneksa Health aims to identify and quantify disease progression using digital measures from a wrist-worn wearable device. The team will employ machine learning to analyze data on physical activity, gait, sleep and cardiac activity, allowing them to explore how these wearable devices can provide more fine-tuned clinical signals to physicians. Generating highly specific data from wearables enables more specific research into the individualized progression of PD and more personalized approaches to medicine. 

Research or business development professional interested in learning more about the latest in PD drug development? Register and join our 2022 Parkinson’s Disease Therapeutics Conference in October.  

Help contribute to research on Parkinson’s biology and experience. Join PPMI and get started today. The online portion is open to anyone over the age of 18 years in the United States. View a list of international recruiting sites if you are outside of the U.S. 

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