James Beck, PhD, on Laying Groundwork for Gene Therapy in Parkinson Disease

“Fundamentally, the issue with Parkinson disease (PD) is that we don't know what causes it. We don't know why it progresses. We don't know how to measure it objectively in a person with PD, we need biomarkers. There are lots of fundamental questions that remain unanswered. But we're making progress. The foundation is certainly funding a significant amount of basic research to understand the disease so we know what targets we should go for.”

The Parkinson’s Foundation has launched PDGENEration (NCT04057794) to increase the genetics knowledge base of people with Parkinson disease (PD). This remains an important unmet need to support the development of precision medicine and gene therapies as they grow in prominence and are starting to be explored and tested in more fields.

Genetic counseling and testing for people with PD needs to be a standard-of-care in the near-future world of genetic medicine treatment or participation in clinical trials. The genetic status of many patients with PD remains unknown since most are not genetically tested. Participation in PDGENEration is available at-home or in-person at sites across the US to people with PD.

CGTLive spoke with James Beck, PhD, chief scientific officer and senior vice president, Parkinson’s Foundation, to learn more about the potential of gene therapies in treating PD and further research needs that remain. He also discussed the need for clinicians to become more familiar with discussing the genetics of PD and genetic testing with their patients.

REFERENCE

Parkinson’s Foundation announces major expansion of PD GENErationstudy, increasing access to genetic testing and counseling across the U.S. News release. Parkinson’s Foundation. May 3, 2022. https://www.parkinson.org/about-us/press-release/Parkinsons-Foundation-expansion-PD-GENEration-study