Claire Henchcliffe, MD, DPhil

“Right now we’re very optimistic—it’s not a cure and there are obvious caveats&mdash;but we’re really excited to take the first step and get this into a first-in-human trial and start learning.”

In Parkinson disease, despite having a gold standard therapy for treatment in levodopa, patients often experience off periods where their symptoms return as a result of the medication wear off. Although treatments exist to help maximize levodopa and minimize that off time, it remains a challenge.

Claire Henchcliffe, MD, DPhil, and her colleagues would like to change that. They are conducting a first-in-human, open-label study using MSK-DA01, a cellular therapy which aims to replenish the dopaminergic cell loss that occurs in Parkinson. While the therapy still has a long way to go, reaching human clinical trials is a milestone.

To explain more about the planned trial of these cells, the associate professor of neurology and neuroscience at Weill Cornell Medicine sat with 

 in an interview. Henchcliffe spoke to the specifics of the trial as well as the investigators’ goals. 

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The associate professor of neurology and neuroscience at Weill Cornell Medicine spoke to the specifics of the trial as well as the investigators’ goals.

Claire Henchcliffe, MD, DPhil: MSK-DA01 Cellular Therapy for Parkinson Disease

“Although we have very good treatments for people’s motor symptoms, even some good treatments for the nonmoter symptoms, there’s still a long way  to go.”

The vast majority of motor symptoms that patients with Parkinson disease experience are the result of a lack of dopamine inputs into the striatum. This is why the mainstays of treatment, such as levodopa, work to replace this lack.

However, even when the medications are working properly, over a period of time these medications wear off, and patients will develop dyskinesias, which truly limits the impact these otherwise efficacious therapies can have.

Claire Henchcliffe, MD, DPhil, an associate professor of neurology and neuroscience at Weill Cornell Medicine, along with many others, believes that because the loss of this particular cell type appears to be geographically limited to one area of the brain, that these cells could be replenished with either autologous cells or allogeneic cells.

At the American Neurological Association’s 143rd annual meeting in Atlanta, Georgia, Henchcliffe sat with 

 to discuss this work, and what she and her colleagues believe could be addressed by a successful effort.

The associate professor of neurology and neuroscience at Weill Cornell Medicine discussed this work, and what she and her colleagues believe could be addressed by a successful effort.

Claire Henchcliffe, MD, DPhil: Dopaminergic Cell Replenishment in Parkinson Disease

“We’re really in the position now where it should be possible to grow an unlimited cell source and be able to differentiate those cells into something that is, for all intents and purposes,  an authentic, adult, mid-brain, substantia nigra, dopamine-producing neuron.”

Parkinson disease, while currently only managed symptomatically, mainly with treatment regimens including levodopa, has had a rich history of stem cell-derived therapeutic development. With prospective treatments and research dating back to the 1980s, the work being done now is far from the groundbreaking ceremony in this area.

Claire Henchcliffe, MD, DPhil, is currently involved in the study of cell-based therapies for the condition. Although, even though she’s a member of the newer class of research, she is very aware of the stepping stones that were laid before her and her colleagues by research groups in the 1980s and 1990s.

The associate professor of neurology and neuroscience at Weill Cornell Medicine, along with her colleagues in research, have utilized these older trials and scientific inquiries to better inform the current approaches to stem cell therapy. Coupled with the advances made in stem cell production, this base knowledge has provided invaluable information for new developments.

At the American Neurological Association’s 143rd Annual Meeting in Atlanta, Georgia, Henchcliffe sat with 

 to discuss the progress that has been made in this area, and where things are headed going forward.

Henchcliffe discussed the progress that has been made in this area, and where things are headed going forward.

Claire Henchcliffe, MD, DPhil

Articles

Claire Henchcliffe, MD, DPhil: MSK-DA01 Cellular Therapy for Parkinson Disease

Claire Henchcliffe, MD, DPhil: Dopaminergic Cell Replenishment in Parkinson Disease

Claire Henchcliffe, MD, DPhil: The History of Stem Cell Therapies for Parkinson Disease