Fall / Winter 2025 | Research 15
Gene Therapy Enters Clinical Trials
Researchers are testing a new gene therapy to determine whether it can slow or stop Parkinson’ s progression in people with GBA gene mutations.
GBA mutations— the most common genetic risk factor for PD— affect up to 15 percent of people with Parkinson’ s. These mutations decrease the activity of a protein called GCase. When GCase malfunctions, toxic lipids accumulate in the brain, resulting in clumps of alpha-synuclein that damage dopamine neurons. This new gene therapy, from Capsida Biotherapeutics, is designed to overcome the blood-brain barrier, which has limited previous approaches to supplementing GCase activity. The Phase I / II trial, which started in August, aims to enroll 31 participants aged 21 to 74 with a GBA mutation.
While this research focuses on GBA mutations, clinical trial findings may lead to discoveries relevant to the wider Parkinson’ s community.
Clinical Trial Participation, from the Comfort of Home
Volunteers are crucial for conducting clinical trials, and one Phase II study is making research participation simpler.
SUNRISE-PD is a study of bezisterim, a new drug designed to increase“ on” time for people with PD— as well as potentially slow symptom progression. Rather than relying on in-person study visits at a clinical trial site, the trial allows volunteers to participate from home.
Study nurses make home visits and complete assessments under the supervision of a neurologist who oversees the visit remotely via video. Enrollment is open to people diagnosed with PD within the last four years who have not started dopamine treatment. For information on participating in this and other clinical trials, visit michaeljfox. org / trial-finder.
Parkinson’ s Conference Champions Researcher Collaboration
MJFF’ s 17th Annual Parkinson’ s Disease Therapeutics Conference( PDTC), held October 16 in New York City, brought together more than 300 research and industry leaders to share advances in the MJFFfunded research pipeline.
New this year was a preconference Partnering Session, focused on showcasing meaningful collaborations between MJFF and industry groups. The session highlighted case studies across biomarker discovery, trial design and patient engagement to encourage deeper integration with Foundation programs.