Trial of cell-based therapy CK08031 finishes dosing 1st patient group

VA hospital in Houston now will enroll veterans with ALS in early study

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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An early-stage clinical trial evaluating CK0803, Cellenkos‘ regulatory T-cell-based therapy for people with amyotrophic lateral sclerosis (ALS), has completed dosing its first patient group, the company announced in a press release.

The six patients, all adults, were treated at the Columbia University Irving Medical Center, in New York City, in the Phase 1 run-in period of the REGALS Phase 1/1b clinical trial (NCT05695521), which focused on safety. The trial now will enroll another 60 adults with ALS in its randomized Phase 1b part to continue to examine the treatment’s safety and efficacy.

The participants to date have been recruited at the single New York study site, but a U.S. Department of Veteran Affairs (VA) hospital in Houston has announced it will recruit 10 patients for the clinical trial, slated to span about six months.

The Michael E. DeBakey Veteran Affairs Medical Center is the first and only VA hospital in the U.S. to join REGALS — and provide military veterans the opportunity to receive the cell-based therapy, which is designed to reduce neuroinflammation and balance the activity of the immune system.

“We are thrilled that our veterans will get the opportunity to receive such a cutting edge therapeutic that could be life altering,” said James Orengo, MD, PhD, principal investigator of the study at the DeBakey VA Medical Center.

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“The incidence of ALS is elevated in veterans and novel therapies are desperately needed,” Orengo said.

Indeed, it’s estimated that more than 16% of all ALS patients are veterans, who are twice as likely to develop the disease as are individuals in the general population.

Scientists have suggested that this increased prevalence likely is related to increased exposure to chemicals, strenuous physical conditions, and trauma during military service.

“We made a conscious decision to make this novel cell therapy available to the veterans and remain committed to provide our continued support to those who have served our country,” said Tara Sadeghi, Cellenkos’ chief operating officer, noting that “there is an urgent need for novel treatments for all forms of ALS.”

Altogether, the REGALS trial will involve a total of 66 ALS patients with disease onset in the previous five years. All will receive the cell-based therapy through intravenous or into-the-vein infusions. The first four treatment doses are given weekly, followed by monthly infusions for the next five months.

We made a conscious decision to make this novel cell therapy available to the veterans and remain committed to provide our continued support to those who have served our country.

In the Phase 1 part of the trial, which is now complete, researchers assessed the treatment’s safety and efficacy. The main goals of the Phase 1b part are to determine the incidence and severity of side effects as well as changes in a combined assessment of function and survival.

Secondary efficacy measures include changes in the rate of disease progression, quality of life, lung function, and muscle strength.

For its part, the Houston VA clinic is “excited to be one of the first sites [to test this] novel T regulatory cells therapy for ALS,” Barbara W. Trautner, the center’s deputy associate chief of staff for clinical research, said in the VA announcement.

“We hope this is the first of many trials that we can open to develop new therapies for veterans with neurodegenerative disorders,” Trautner said, noting that the center’s neurologists “jumped at the chance to offer this to our veterans.”

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Cell-based therapy aims to reduce inflammation in ALS

People with ALS generally have high levels of inflammatory T-cells, which aid in the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movements. Conversely, immunosuppressive regulatory T-cells, known as Tregs, usually are found at lower levels than are normal in these patients, and are also less effective.

Tregs are immune cells that help suppress excessive inflammation and maintain the immune system’s healthy functioning. As such, they’re believed to help reduce some of the inflammation that causes damage in ALS.

CK0803 is a cell-based therapy that uses Tregs isolated from healthy umbilical cord blood, without the need to match patient and donor. These cells are then enriched and differentiated to target a given condition using the company’s proprietary CRANE technology.

The technology enables the production of several CK0803 doses from a single umbilical cord blood unit, and the final product can be frozen and stored for long periods — making treatments readily available for patients when needed.

“We are very excited to open this first cell therapy trial for ALS in the VA,” Orengo, also a professor of neurology at Baylor College of Medicine, in Houston, said in the Veteran Affairs announcement.

“We know that neuroinflammation fuels the ALS disease process. We believe that this new treatment, which involves calming the immune system in the central nervous system, holds great promise in halting this disease,” Orengo said.