New Parkinson’s Foundation pilot aims to help patients navigate trials
Goal is to increase enrollment in studies of genetic-focused treatments
The Parkinson’s Foundation has launched a pilot program it hopes will make it easier for Parkinson’s disease patients who carry specific disease-related genetic variants to enroll in clinical trials testing targeted experimental treatments.
The new program, dubbed PD Trial Navigator, is designed to help meet a central goal of the ongoing PD GENEration study and help match people with Parkinson’s to gene-targeted clinical trials. PD GENEration, launched in 2019, provides free genetic testing and counseling to individuals diagnosed with Parkinson’s.
“More than 25,000 people living with PD [Parkinson’s disease] have already participated in PD GENEration,” John L. Lehr, the president and CEO of Parkinson’s Foundation, said in a foundation press release announcing the new program.
“Once they learn their genetic link to Parkinson’s, they can become active participants in clinical trials, accelerating our understanding of this disease and achieving breakthrough discoveries,” Lehr said.
Eligible trials must be regulated and approved by the U.S. Food and Drug Administration and Institutional Review Boards, and be recruiting patients in the U.S.
Parkinson’s is caused by the loss of dopaminergic neurons, which are specialized nerve cells that produce a signaling molecule called dopamine that’s involved in motor control. Although the exact triggers for the loss of these neurons are not fully understood, in some cases, genetic mutations underlie the disease. Most commonly, these mutations are present in the GBA and LRKK2 genes.
Still, most people with Parkinson’s never undergo genetic testing to know if they carry a disease-causing mutation.
Parkinson’s patients can use pilot to see trials for which they’re eligible
PD GENEration study (NCT04057794, NCT04994015), essentially an observational study, offers free genetic testing and counseling, in English and Spanish, to people with Parkinson’s. Results from 8,301 participants in North America, shared last year, demonstrated that nearly 13% of patients tested carried a Parkinson’s-related mutation.
James Beck, PhD, executive vice president and chief scientific officer of the Parkinson’s Foundation, noted that, “through PD GENEration, we know that, while rare, more people than expected have a Parkinson’s-related genetic variant.”
According to Beck, “not only does this underscore the need for the widespread testing that PD GENEration offers but also means more people may be eligible for gene-targeted therapies than ever before.”
The Parkinson’s Foundation noted in the release that clinical trials testing therapies associated with specific genetic mutations experience challenges associated with funding and patient recruitment. Using the PD Trial Navigator, the foundation can guide PD GENEration participants to trials for which they are eligible.
Particularly, people with a confirmed genetic link to Parkinson’s, as tested in the PD GENEration study, may have access to precision medicine trials for GBA and LRKK2-associated Parkinson’s.
Through PD GENEration, we know that, while rare, more people than expected have a Parkinson’s-related genetic variant. … Not only does this underscore the need for the widespread testing that PD GENEration offers but also means more people may be eligible for gene-targeted therapies than ever before.
Daniel Rothschild took part in PD GENEration and now says he’ll make use of the PD Trial Navigator.
“I have participated in various research studies since my [Parkinson’s] diagnosis, but with the knowledge derived from PD GENEration, I can now target my efforts on research that focuses on my specific genetic mutation,” Rothschild said. “I am excited that my participation in this program, which was motivated by a desire to learn more about my own diagnosis, may now enable me to help others by participating in and accelerating research that focuses on this shared genetic abnormality.”
The foundation noted “continued support” from the Global Parkinson’s Genetics Program, an initiative of Aligning Science Across Parkinson’s.
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