UCI Health tests novel stem cell therapy for advanced Parkinson’s disease

March 25, 2022
Claire Henchcliffe, MD, DPhil, UCI Health movement disorders expert and chair of the UCI School of Medicine's Department of Neurology
"It’s a one-shot deal that we hope would be able to provide consistent, life-long treatment,” UCI Health movement disorders specialist Dr. Claire Henchcliffe says of the first-in-human, UCI Health stem cell clinical trial for patients with advanced Parkinson's disease. 

UCI Health neuroscientists are testing a promising dopamine neuron stem-cell therapy to determine whether it reduces symptoms in patients with advanced Parkinson’s disease.

In a first-in-human clinical trial, researchers are assessing the safety of dopamine-producing stem cells that are surgically implanted in the brain of patients whose Parkinson’s disease has progressed to the point where existing medications no longer work to control their movements.

The treatment, MSK-DA01, consists of embryonic stem cells that have been grown and developed into neurons that produce dopamine, a naturally occurring brain chemical whose levels decreases in people with Parkinson’s disease.

Researchers hope to learn whether the signs and symptoms of Parkinson’s disease change in response to MSK-DA01 and how the treatment influences patients' quality of life.

“Dopamine is a neurotransmitter and when it’s lost, it causes the slowing, the lack of coordination, the stiffness and the tremors we see in Parkinson’s patients,” said Claire Henchcliffe, MD, DPhil, chair of the UCI School of Medicine's Department of Neurology and principal investigator for the trial.

“The hope is that these neurons will create connections with the patient’s nerve cells and deliver dopamine, relieving the symptoms of Parkinson’s disease.”

'A one-shot deal' 

Henchcliffe, an internationally recognized movement disorders specialist, developed the foundational study protocol for the MSK-DA01 trial before joining UCI Health.

In pre-clinical studies, researchers were able to isolate dopamine-producing stem cells to replace neurons that have been damaged. Transplanting these dopamine progenitor cells, taken from human embryonic stem cells, improved movement and coordination in animal studies.

“The reason we’re very hopeful is that in animal models, the movement symptoms — problems of slowing and coordination — were treated and sustained,” she said. “We now treat Parkinson’s patients with medications that help the brain produce dopamine, but these drugs lose efficacy over time. 

"With the stem cell transplant, it’s a one-shot deal that we hope would be able to provide consistent, life-long treatment.”

Establishing the treatment's safety and efficacy are the goals of the phase 1 clinical trial, which is sponsored by BlueRock Therapeutics.

Over the next two years, researchers will examine whether the implanted stem cells survive and whether they improve participants’ motor functions. The implantation surgery takes place at Memorial Sloan Kettering Cancer Center in New York City. Participants in the UCI Alpha Stem Cell Clinic trial are followed by the Parkinson's Disease and Movement Disorders Program team.

Previous clinical trials to restore dopamine function with cell transplantation showed promising but variable results, which Henchcliffe attributed to the limitations of earlier stem cells. “You couldn’t do quality control on the cells you were going to transplant. There simply weren’t enough.”

Differentiating stem cells

But rapid advances in stem cell technology over the last 20 years made this ground-breaking study possible, she said.

“We’ve figured out how to make embryonic stem cells that can be grown in the laboratory in almost unlimited quantities. Now researchers, including the people I worked with at Memorial Sloan Kettering, have found a way to differentiate those cells into dopamine-producing neurons.”

Henchcliffe is excited that this cell therapy trial, the culmination of decades of research efforts, is available to UCI Health patients.

For more information, please email the UCI Alpha Stem Cell Clinic at stemcell@uci.edu or call 949‐824‐3990.

About Parkinson’s Disease

Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor functions is caused by the loss of dopamine-producing neurons. At diagnosis, it is estimated that most Parkinson's patients already have lost 60% to 80% of their dopamine-producing neurons. Parkinson's disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with one million living in the United States. The MSK-DA01 therapy is being clinically evaluated in a subset of patients with advanced Parkinson’s disease whose regular medications are no longer fully effective.

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