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Parkinson's Trial Recruiting GBA Mutation Carriers

Parkinson's Trial Recruiting GBA Mutation Carriers

A drug trial for people with a genetic form of Parkinson's disease (PD) is recruiting volunteers at more than 30 sites around the world. Study sponsor Sanofi Genzyme is looking for 243 volunteers with a mutation in the GBA gene for its Phase 2 MOVES-PD trial. GBA gene mutations are a common risk factor for Parkinson's disease.

We talked to Tanya Fischer, MD, PhD, Global Project Head of Neurology and Multiple Sclerosis at Sanofi Genzyme to learn more about the study.

Visit the study page on to find a study site.

What is the MOVES-PD trial?
Sanofi Genzyme is testing its drug GZ/SAR402671 for its impact on PD progression, as well as safety and tolerability. MOVES-PD plans to enroll at more than 50 sites worldwide and involves two phases. In the first, volunteers will be randomly assigned to take either the drug or a placebo for one year and will visit the clinic site every three months for an assessment. In the second phase, all participants will receive the study drug for two years (without placebo) and visit the clinic every six months.

What is GZ/SAR402671? How may this drug stop Parkinson's progression?
This drug lowers levels of glucosylceramide (GL-1), a fatty substance in the cell. Mutations in the GBA gene are associated with too much GL-1, which has been linked to build up of the protein alpha-synuclein. Alpha-synuclein is a major part of protein clumps called Lewy bodies, a hallmark of PD, which scientists believe may lead to the cell death that causes PD symptoms and progression.

Who is eligible for this trial?
People between ages 18 and 80 who know they carry a GBA mutation and who have shown Parkinson's symptoms for at least two years are eligible to participate in the MOVES-PD trial. It does not matter when you were diagnosed with PD, only that you know you have had symptoms for at least two years.

How can someone learn if they carry a GBA mutation?
Learning one's genetic carrier status is a personal decision. Talk to your doctor about genetic testing and counseling for Parkinson's-associated genetic mutations. It is important to understand what your status means for you and your family.

Says Fischer, "Before this trial and others that have since begun, people would ask 'Why should I be screened if it won't change my care?' Now we have these targeted potential therapies for genetic forms of PD in testing. I really feel we're at an exciting time in Parkinson's disease drug development."

Your doctor may be able to refer you for genetic testing and counseling. Research studies also provide these services to participants. The MJFF-led Parkinson's Progression Markers Initiative is screening people of Ashkenazi Jewish descent for GBA mutations, which are more common in that population. And MJFF's online Fox Insight study is testing anyone with Parkinson's disease through a collaboration with personal genetics company 23andMe.

Will this therapy work for people without a GBA mutation?
The Michael J. Fox Foundation is investing significant funds in learning more about the connections between genetic and so-called idiopathic (cause unknown) PD. To grow our existing portfolio in this area, MJFF -- in partnership with The Silverstein Foundation for Parkinson's with GBA -- is currently accepting funding applications for projects to investigate the GBA pathway in people without this mutation. This information could help understand the application of GBA therapies to a broader PD population.

Learn more about the MOVES-PD trial.

Learn more about other recruiting Parkinson's studies and be matched with studies looking for volunteers like you on MJFF's Fox Trial Finder website.

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