Using Precision Medicine to Care for Patients with Movement Disorders
As Director of the Parkinson’s Disease and Movement Disorders Institute, Claire Henchcliffe, M.D., D.Phil., leads an outpatient program that specializes in the diagnosis and management of movement disorders including Parkinson’s disease. In addition to seeing patients, Dr. Henchcliffe’s multidisciplinary team of neuropsychologists, therapists and allied health professionals conduct a number of clinical trials available for patient participation.
The Institute is involved in several research studies to better understand Parkinson’s disease, as well as clinical pharmacotherapy trials that allow patients to try new therapeutics. A graduate of Columbia University’s College of Physicians and Surgeons (M.D.), and the University of Oxford (D.Phil), Dr. Henchcliffe is Vice Chair for Clinical Research in Neurology at the NewYork-Presbyterian Hospital/Weill Cornell Medical Center.
When did you become interested in studying movement disorders?
I grew up in the UK, where I trained as a neuroscience undergraduate at Oxford University. I was intrigued by neurogenetics and how the brain develops, so moved to UC Berkeley where I studied the genetics of how the nervous system develops in the fruit fly embryo. My research evolved when I moved to New York to attend medical school. It is here at WCM that my interest in Parkinson’s disease developed.
Over the past 16 years at WCM, I have built a clinical practice for patients with Parkinson’s disease and other movement disorders. I’ve developed a clinical trials initiative to create better treatments for our patients. Our group is now involved with a neurological clinical trials network called NeuroNEXT, jointly managed with Columbia University. We have also joined forces with those working on other neurologic disorders, including Alzheimer’s disease.
We would like to push the field forward, and are fortunate to have the opportunity to meet with experts in multiple disciplines to grow, develop, and test new therapeutics at WCM. Through our Clinical & Translational Science Center we established an active liaison team called the Trial Innovation Unit. I serve as Clinical Director of this hub, and we seek to be more efficient in the way we run clinical trials and bring new treatments to patients faster. Personally, I’ve expanded my ability to work with people outside of neurology through the Trial Innovation Unit.
What areas of research hold the most promise?
One emerging area is a regenerative medicine approach. The ‘incoordination’ and problems with motor control in Parkinson’s disease arise largely from cell loss that is quite geographically confined in the brain, an area where we can intervene and restore, regenerate, or rebuild the function of those damaged cells. One of my major interests is therefore in developing a cell therapy approach, with the capacity to restore what’s happened in the brain to someone with a neurological disorder such as Parkinson’s disease.
As we’re developing new treatments though, we need to keep in mind that genetics play an important role. Although only a minority of patients have an inherited form of Parkinson’s disease, the genetic contribution is actually extremely important. There is a lot of heterogeneity in Parkinson’s disease presentations and treatment response, and no one single patient is quite like another. Genetics likely accounts for much of this heterogeneity, and we are now entering an era in which we have tools to identify subtypes and to learn which patients will respond best to specific treatments. A great example is my colleague, Michael G. Kaplitt, M.D., Ph.D., who was the first to pioneer gene therapy in Parkinson’s disease, now proving the potential for specific gene therapy in individuals with an identified cause of Parkinson’s.
How does precision medicine fit into this new approach?
Precision medicine is the next step for our field. It’s so inspiring to learn how EIPM investigators blend research with clinical trials to benefit specific patients. I find it really important that a patient can present one way clinically or pathologically, but their genetics reveal that something else entirely is going on!
Typically, we group Parkinson’s patients on the basis of their coordination patterns in the clinic. However, if we take a precision medicine approach of digging deeper, we may discover people with a particular genetic form that allows us to intervene in a specific way. For example, we may discover that a patient has a high level of mitochondrial dysfunction and this could guide individualizing their treatment. Precision medicine may therefore be the key to unlocking the genetic mysteries of Parkinson’s. Our patients are eager to explore these possibilities.
Do patients discuss precision medicine with you?
Patients are learning more about the benefits of genetic testing and ask about precision medicine almost daily. They are interested in clinical application for themselves, but also how they can contribute to research for future patients.
Traditionally genetic testing is applied to specific Parkinson’s genes, but the information detected would not in the past necessarily alter a patient’s treatment. However, we are now at the point where we are starting to have new interventions and clinical trials specific to particular genetic forms of Parkinson’s.
While my passion remains with the clinical treatment of patients, I am dedicating more time to research. As we move toward gene therapy, cell therapy, and potentially into antibody-based therapy, we’re well-positioned to take advantage of the next generation of research and precision medicine to deliver new treatments to our patients. Now is a promising time for Parkinson’s disease, and I’m full of hope for a precision medicine approach in improving the future with better outcomes for our individual patients.
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