People with Huntington’s wanted for first trial of novel stem cell therapy
First participant reports no side effects from cells implanted into brain
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An early-stage clinical trial designed to test a novel stem cell therapy — one designed to replace lost or damaged nerve cells in the brain — in people with Huntington’s disease is now recruiting adults with the genetic condition at its single site in California.
The Phase 1/2 trial, dubbed REGEN4HD (NCT07451613), is mainly evaluating the safety of the treatment candidate, called hNSC-01, which is implanted directly into the brain.
It aims to enroll 21 participants, ages 18 to 65, with early-stage Huntington’s. All participants will receive the experimental stem cell therapy and will be followed for up to one year to evaluate its safety and preliminary effectiveness. Recruitment is underway at the University of California, Irvine (UCI).
The study is the first to test the therapy in humans.
“This clinical trial represents the culmination of many years of preclinical and translational research, pivotal safety studies, discussions with the FDA [U.S. Food and Drug Administration], and support from the California Institute for Regenerative Medicine,” Leslie Thompson, PhD, the trial’s sponsor at UCI, said in a press release from the International Society for Stem Cell Research.
“Reaching the point of launching the first clinical trial of a pluripotent stem cell-derived product for Huntington’s disease is an important milestone for the field,” Thompson said. Pluripotent stem cells can grow into many other types of cells, making them useful for developing a variety of treatments.
Thompson noted that “the Huntington’s disease families and the close partnership between patients and scientists have been a constant source of inspiration.”
The first trial participant was dosed last month, and the second patient is expected to receive the cell therapy this month, according to an earlier UCI Health press release announcing the start of patient dosing.
“The first patient intervention went very well,” said Ravi Rajmohan, MD, PhD, the trial’s principal investigator and a neurologist at UCI Health, the university’s clinical enterprise, adding that, “to date, they haven’t reported any serious adverse events.”
“This trial may help us move one step closer to a future with available treatments that could potentially slow the progression of Huntington’s disease,” Rajmohan said.
Trial testing safety, early effectiveness of treatment with stem cells
Huntington’s is characterized by progressive damage to the brain, caused by the loss of nerve cells. hNSC-01 aims to deliver neural progenitor cells, or stem cells that are able to grow into many types of brain cells, including new nerve cells.
The therapy, which uses pluripotent stem cells derived from human embryos, essentially aims to replace lost or damaged nerve cells in order to restore normal brain signaling. It is also designed to release signaling molecules to support brain cell health. As such, hNSC-01 is expected to help slow Huntington’s progression.
In animal models, the therapy showed promise, “with no negative outcomes, supporting [its development],” according to the research team. Now, it’s being tested in people with the disease.
The study’s Phase 1b dose-escalation phase will evaluate increasing doses of hNSC-01 in 12 participants. Based on the findings, an optimal dose will be selected for further evaluation in nine additional participants in the study’s Phase 2a dose-expansion phase.
The therapeutic cells are implanted directly into the striatum, a brain region profoundly affected by Huntington’s, via a surgical procedure that lasts about six hours and is done under general anesthesia.
The trial’s main goal is to determine the therapy’s safety, while secondary goals are meant to identify potential signs of effectiveness. These include changes in validated measures of motor and cognitive function, behaviors, and functional capacity, as well as in brain scans and levels of a nerve damage marker in bodily fluids.
“The initial goal of this study is to establish safety,” Thompson said. “If future studies ultimately demonstrate that a one-time cell therapy approach can slow disease progression, it could have meaningful implications for people living with Huntington’s disease. However, those questions can only be answered through careful clinical evaluation.”
The trial is being funded by a $12 million grant from the California Institute for Regenerative Medicine (CIRM), a state agency dedicated to advancing new treatments for serious diseases.
“In addition to CIRM, these advances are possible because of the broader Huntington’s disease research community supported by federal and foundation funding,” Thompson stated in the earlier press release. “We are so grateful to our patients and their incredible families for their bravery to provide hope for others with very few options.”
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