Pursuing Genetic Targets in Parkinson Disease: The Next Big Step


The chief scientific officer of the Parkinson’s Foundation spoke about the developing interest in genetic forms of Parkinson, the potential to target therapies in that direction, and how a genetic treatment is the next big stage for the field.

Dr James Beck

James Beck, PhD, chief scientific officer of the Parkinson Foundation

James Beck, PhD

Parkinson disease is a complex condition to treat, with numerous symptoms which require additional medications and cooperation between different specialties. Despite not having a complete understanding of its cause, research continues, and for some patients, the conversation has shifted toward genetic origins.

James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, and others have noticed this trend in thinking and, mirroring what has been done in oncology, have imagined the use of a genetically focused therapy for the treatment of Parkinson disease.

To further discuss this possibility and what the basis for this type of treatment would be, NeurologyLive® spoke with Beck, who detailed the process through which this goal may ultimately be achieved.

NeurologyLive®: Is there anything being developed for Parkinson disease or that’s in the pipeline but isn’t getting enough attention?

James Beck, PhD: One of the really interesting things that is occurring in the realm of Parkinson disease is a focus on genetic forms of the disease. Parkinson disease is a movement disorder where the cause of it remains unknown for the vast majority of people. However, for about 10% of people with Parkinson disease, it’s a known genetic cause. That, in 1 of their genes, there is something that can lead to the development of Parkinson disease. The pharmaceutical industry is focusing on these genetic forms of Parkinson for next-generation therapies.

This is kind of what you’ve seen in the cancer field of precision medicine where identifying a specific mutation can lead to a specific drug to target that mutation and, in the case of cancer, try to seek remission of the cancer. For Parkinson disease, gene-targeted therapies might be able to slow or stop the progression of Parkinson—and maybe prevent it altogether, if utilized in the right population and assuming these drugs are successful. That’s where the future lies when it comes to Parkinson disease treatments.

I think many neurologists who care for those living with Parkinson aren’t necessarily thinking about what it means to have a genetic form of Parkinson. Primarily because really right up until about now, knowing someone’s genetic status for their disease—that is, do they have a genetic form of Parkinson disease or idiopathic form of Parkinson disease—doesn’t impact their care. But again, that is about to change as drug companies work to develop and test new therapies that target these forms of Parkinson disease.

Do you believe that will be the next big step in Parkinson?

I think genetic targets are the next big step. But how do you get to that goal? It’s a big step and you can’t just go at it all at once. The issue here is because the physician community, and even the patient community, hasn’t been focused on genetic treatment. We’re not ready as a community to begin to really trial these investigative new drugs that are going to be coming out, and that’s a real problem.

A better way to put it is: How do you enroll someone in a genetic trial or a trial targeting genetic forms of Parkinson when they don’t walk around with a big stamp on their forehead that says they have a genetic form of Parkinson? What you have to do is have genetic testing on a wide scale so individuals can find out, their doctors can find out, and they can make the affirmative decisions to enroll in clinical trials as they come online.

Recognizing that our community is not ready, our foundation is going to be offering free genetic testing and counseling for up to 15,000 people with Parkinson disease through our centers of excellence and another group called the Parkinson’s Penny Group that will allow us to reach a wide swath of the United Sates as a part of this. It’s something that we will probably be looking to team up with community neurologists who are within the realm of a center of excellence to allow their patients to go in to get tested, so they can identify if their patients have genetic forms of Parkinson.

Is there anything you’d like to add?

I think that one of the things that the Parkinson’s Foundation provides is its educational resources, as these can help both the patient and the clinician. Our website has tremendous information about many aspects of Parkinson disease, and this can be useful for neurologists as they need to provide a good resource of information to their patients to help them better manage their own disease, and referring people to our website Parkinson.org would be a very useful tool. We also have a help line, which again, can be in adjunct to the efforts of a neurologist caring for people with Parkinson. Patients have a limited time frame to talk about the disease and talk about a continuum of support to learn about their disease and best manage it. That number is 1-800-4PDINFO.

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