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With the neuro tide rising, could a breakthrough for Parkinson’s be next?

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In 1991, Michael J. Fox was 29 years old filming “Doc Hollywood” when he noticed a tremor in his pinky finger. The nagging symptom led to a life-changing diagnosis, young-onset Parkinson’s disease, which is more commonly diagnosed after age 50.

Brian Fiske, chief scientific officer, Michael J. Fox Foundation for Parkinson’s Research

Brian Fiske, CSO, Michael J. Fox Foundation

Permission granted by Michael J. Fox Foundation


For years, he carefully hid the progressive brain disorder, until he no longer could. In 2000, he went public, becoming the face of Parkinson’s and founding the Michael J. Fox Foundation for Parkinson’s Research, which has since raised more than $1 billion dollars in its push toward a cure for this central nervous system disease. Fox’s diagnosis fortuitously came when Parkinson’s research was in need of a push, and it got one from his familiar face and star power.

“The genetics of Parkinson’s were really just getting off the ground,” said Brian Fiske, the foundation’s chief scientific officer. “There were a couple of genes that had been linked to the disease that were starting to point at mechanisms that might be relevant,” but scientists were still in the early stages of understanding.

“I think his coming on board brought the field together,” Fiske said. “Suddenly, people were coming out of the woodwork to study Parkinson’s.”

In two decades, the Michael J. Fox Foundation has contributed to many scientific leaps that have shed light on the genetics and science of this debilitating neurological disease, the second most common next to Alzheimer’s. Parkinson’s is unpredictable, thought to be driven by a complex interplay of genes and environmental factors, such as exposure to metals, solvents or pesticides or head injuries. Fueled by these new understandings, pharmaceutical companies are moving closer to disease-modifying drugs, although the bulk of them are still in early stage trials.

Some of these drug candidates are taking a page out of the Alzheimer’s playbook, targeting toxic clumps of proteins in the brain called alpha synuclein  a different type than the tau and amyloid-beta proteins that are the focus of Alzheimer’s research. These sticky proteins collect in the brains of people with Parkinson’s and are thought to cause damage.

“Clumping of this protein in the brain is associated with, we think, a huge percentage of cases of Parkinson’s disease,” Fiske said. Researchers hoped that targeting this protein will make a difference for patients, similar to what’s happening in the Alzheimer’s field with drugs, such as the recently approved Leqembi, an amyloid-beta targeting antibody.

Multiple companies are now testing alpha synuclein-targeting drugs, among them, MODAG’s small molecule drug Anle138b, Prothena/Roche’s Prasinezumab and an alpha-synuclein-targeting vaccine ACI-7104 from AC Immune in Switzerland, which is now in phase 2.

“Some of them are targeting alpha synuclein in a very direct way by getting rid of the clumps, and some of them are trying to target the mechanism for making the clumps in the first place — others are trying to target the downstream effects of the clumps,” Fiske said. “So, there’s a variety of different ways that companies are looking at this sort of alpha synuclein protein pathway linked to Parkinson’s disease.”

A biomarker breakthrough

The foundation announced a breakthrough in April — the discovery of a Parkinson’s biomarker that could speed progress. Researchers detected abnormal alpha synuclein protein in spinal fluid, using an α-synuclein seeding amplification assay. Researchers are now using this test to identify people with this abnormal protein to determine who might benefit the most from investigational drugs and to gauge therapeutic response. However, because the test is invasive, requiring a draw of spinal fluid, it’s not yet been useful as a screening test. The hope is that scientists will find a way to convert it into a more user-friendly diagnostic, Fiske said.

“Now that we have this measuring tool, this biomarker, paired with those (alpha synuclein-targeting) approaches, we think we can see some real momentum and excitement in coming years,” he said.

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