Data from Landmark MJFF Study Shows Impact of Promising Parkinson’s Therapy

Pharma giant Roche published trial results in October 2024, enabled by data from The Michael J. Fox Foundation's landmark Parkinson's Progression Markers Initiative (PPMI), that show promise for a new Parkinson's therapy.

The trial found significantly slower progression in the groups treated with prasinezumab (at least 40% slower), including even less progression in those on the therapy longer (i.e., those who got the antibody during the placebo-controlled trial). Another trial of the treatment is already getting started.

The research is possible, in part, because PPMI participant partners provide robust, vital data on disease progression. These contributions last year led to a breakthrough test for Parkinson's biology. Now PPMI data are acting as a proxy control, allowing a pharmaceutical company to continue developing this novel treatment to slow disease progression.

"PPMI is a study of how Parkinson's disease starts and changes over time," said Sohini Chowdhury, MJFF's chief program officer. "The contribution of this data to the Roche trial and the program's continued development is a true illustration of its intent and the impact of its generous volunteers."

Mixed Results from Early Testing

Prasinezumab is an antibody that binds to abnormal alpha-synuclein protein, which scientists believe may help slow cell death that leads to PD symptoms. Roche ran a one-year placebo-controlled trial. That is, some participants received prasinezumab and some received a placebo (a substance with no therapeutic effect).

That trial reported in 2022 no significant difference in a total sum of progression measures [Movement Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I + II + III]. Part I captures patient-reported non-motor experiences; Part II: patient-reported motor experiences; Part III: clinician-reported motor exam. Participants on prasinezumab, though, did show some slower progression on Part III alone.

The company then started an open-label extension study. All participants were given or could remain on prasinezumab, essentially removing an active control group. The study team turned to PPMI as a reference for expected progression rate.

Open-access Data Set to Speed Science

PPMI is MJFF's landmark study launched in 2010 and following more than 3,000 participants at 51 clinical sites in 12 countries and more than 40,000 online. Volunteers share data and biosamples over at least five years. All data is de-identified and shared with qualified scientists toward more discoveries. Researchers use this data - the most robust set in Parkinson's research - toward new measures of disease (such as the breakthrough alpha-synuclein test), better understanding of its clinical experience, and insight into risk factors. That all builds up to better care today and a tomorrow without Parkinson's disease.

Difference from Expected Progression Rate

In October 2024 in the journal Nature Medicine, Roche published results from their extension study. Scientists looked at MDS-UPDRS Parts II and III data from 271 prasinezumab-treated participants and 303 PPMI participants who, when enrolled, were diagnosed within the past two years, had only mild symptoms, and were not on PD medication. This matched the prasinezumab trial eligibility criteria.

They found significantly slower progression in the prasinezumab-treated groups (at least 40% slower), including even less progression in those on the therapy longer (i.e., those who got the antibody during the placebo-controlled trial).

Roche is further testing the impact of this therapy in another trial, PADOVA. That has enrolled more than 500 participants and is following those individuals now.

"I joined PPMI in 2021 to make a difference in the future of care for this disease," said Marty Acevedo, who was diagnosed in 2004. "I'm proud that my contributions have led to promise for this and other treatments and am hopeful for a day when we end Parkinson's."

PPMI is recruiting, especially people with risk factors of smell loss or acting out dreams. Visit michaeljfox.org/ppmi to learn more.

Interested in a treatment trial? Visit our online matching tool foxtrialfinder.org to find recruiting studies in your area.

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