The Michael J. Fox Foundation (MJFF) announces 57 grants that total more than $30 million awarded in October and November 2022. These supported projects aim to predict and define early Parkinson’s disease (PD) diagnoses as well as validate symptomatic and disease-modifying treatments.
Here we review some projects expanding our understanding of PD research, diagnostic tools and potential therapies. See full list of MJFF funded studies.
Addressing Drooling and Difficulty Swallowing
People with PD often experience motor symptoms such as difficulty swallowing and excessive drooling. To address these issues, Steve Xu, MD, from Siebel Health is developing an innovative digital therapy using a wearable sensor. This device vibrates silently on the upper chest of patients with trouble swallowing to monitor the frequency of swallowing. Coupled with oversight from speech-pathologists, this device can drive towards an increase of swallowing and less drooling. Modifications and further development are needed to improve the system and to obtain approval for clinical use.
Disease Modifying Therapies in the Pipeline
There are several therapies in the works to modify the experience and progression of Parkinson’s disease. Here we discuss some of the targeted therapies to slow disease progression and/or stop it.
One such example is gene therapy, which is used to combat gene mutations causing PD. Developed by Robert Klein, PhD, and his team at Addition Therapeutics, the project aims to identify gene mutations and safely introduce ‘normal versions’ of these genes back into the brain cells affected by PD, aiming to reverse the effects of the disease. This study will first apply the injection method in laboratory cells and then pre-clinical models for further optimization and to ensure safety.
Another method to stop the progression of PD is being tested by Hugo Vicente Miranda, PhD, and his lab at Universidade Nova de Lisbo. While high levels of the protein alpha-synuclein are associated with PD and can contribute to the development of the disease, it was recently discovered that increased insulin-degrading enzyme (IDE) in the pancreas prevents asyn clumping, which is what we see as PD progresses. IDE regulates the amount of insulin in a person and eliminates excess amounts of it. Miranda’s lab plans to test the validity of this novel mechanism in the brain. This can be helpful especially for vulnerable populations such as people with diabetes and the elderly, whose levels of aggregation-fighting IDE are reduced.
Mitochondria play an important role in preventing inflammation and protecting against PD. When mitochondria malfunction and can no longer properly do their job, there’s a likelihood of developing Parkinson’s.
Here we review two treatments against mitochondrial dysfunction:
Hyung Soon Park, PhD at Glaceum Incorporated investigates the benefits of an anti-obesity drug, HSG4112, to protect dopamine-producing neurons against mitochondrial dysfunction. This pre-clinical study will determine safe dosage before administering in people with PD, aiming to slow disease progression.
A mitochondrial inhibitor is being examined by Neil Miller, PhD, and Richard Rutter, PhD, at NRG Therapeutics Ltd. Their goal is to develop drugs that that block the opening in a mitochondrial membrane, which allows an overflow of calcium ions, leading to the death of dopamine-producing brain cells.
Improved Understanding of Parkinson’s
The process of diagnosing Parkinson’s can be lengthy and requires clinical expertise and evaluations. Some studies are developing improved and quicker diagnostic tools to detect early PD.
A team a Coeruleus Clinical Sciences, LLC, led by Jesse Cedarbaum, MD, and Sheng Luo, PhD, focuses on tracking the emergence of new symptoms in people with PD to assess disease progression versus standard methods, which includes assessment of old and new symptoms. The goal is to adopt the new method for future clinical trials.
Stella Sarraf, PhD, and her team from Amydis, Inc. aim to develop a new and simple way of diagnosing PD through an eye examination. This method requires a drug called retinal tracer to be injected into the bloodstream of PD patients. Once it is attached to misfolded alpha-synuclein clumps, it will emit a florescent light. While further testing is needed to determine safety and efficacy, if successful, it can be revolutionary for faster and more accurate diagnoses and the development of new medicines.
Studies have shown that the development of PD has been associated with abnormal cholesterol metabolism in the brain in conjunction with alpha-synuclein presence. A study led by Gabriele Kaminski Schierle, PhD, at the University of Cambridge investigates how interactions between alpha-synuclein and the structural mechanisms of cholesterol may contribute to the risk of developing PD. Results may pave the way for new preventative drugs and therapies.
The Michael J. Fox Foundation continues to fund advances in technology and medicine to drive toward effective therapies that can prevent, slow or stop disease progression.
You can be a part of that mission.
The Parkinson’s Progression Markers Initiative (PPMI) is our landmark study on a mission to stop the disease. It is open to anyone over age 18 in the United States. Whether you have Parkinson’s or not, join the study that could change everything.
Recently diagnosed with PD or live outside the U.S.? Connect with the PPMI team.