UCI opens first natural killer cell clinical trial for recurrent glioblastoma

Investigative CYNK-001 therapy shows anti-tumor activity

The CYNK-001 therapy ”enlists the patient’s own immune system in the fight against a terrible disease that has a very low five-year survival rate,” says Dr. Daniela A. Bota, the clinical trial's principal investigator and medical director of the UCI Alpha Clinic.

Irvine, Calif. — UCI Health and the UCI Alpha Clinic have opened the first clinical trial to assess the safety and effectiveness of CYNK-001, a natural killer cell therapy, in patients with recurrent glioblastoma (GBM), the deadliest form of brain cancer.

Natural killer (NK) cells, a type of white blood cell, are an integral component of the body’s immune system. They contain an enzyme that can identify and target the recurrent tumor while the body’s adaptive immune response generates T cells, another type of white blood cell, to attack it.

“Patients with recurrent glioblastoma have very few treatment options,” said Daniela A. Bota, MD, PhD, the trial’s primary investigator, director of the UCI Health Comprehensive Brain Tumor Program and vice dean of clinical research for the UCI School of Medicine. “We are pleased to offer these patients access to a promising investigative therapy, which enlists the patient’s own immune system in the fight against a terrible disease that has a very low five-year survival rate.”

CYNK-001 is a cryopreserved, allogeneic, off-the-shelf, NK cell therapy developed from placental hematopoietic stem cells grown in a laboratory as a potential treatment for various hematologic cancers, solid tumors and infectious diseases, according to trial sponsor Celularity Inc.

Glioblastoma (GBM), the most aggressive type of cancer originating in the brain, arises in glial cells, which normally support the health of the nerve cells within the brain. There is no known method of preventing this cancer or its relapse, which can occur within months of initial treatment. Frontline therapy usually involves surgery followed with chemotherapy and radiation therapy.

At the 2019 Society for Neuro-Oncology annual meeting, Celularity presented results of CYNK-001 studies showing anti-tumor activity against GBM while sparing healthy cells in mouse models. In March 2021, the U.S. Food and Drug Administration granted fast-track approval to use the therapy to treat recurrent GBM, the company announced.

In a previous UCI Alpha Clinic trial, the investigational CYNK-001 therapy was used to treat COVID-19 patients.

For more information about the CYNK-001 clinical trial for patients with recurrent GBM and other stem cell clinical trials, contact the UCI Alpha Clinic at stemcell@uci.edu or call 949-824-3990.

The UCI Alpha Clinic is the clinical trials arm of the UCI Sue & Bill Gross Stem Cell Research Center and part of a network of the leading medical centers funded by the California Institute for Regenerative Medicine. It specializes in delivering leading-edge stem cell clinical trials and gene therapy to patients, and it seeks to accelerate the development of new treatments through partnerships with patients, medical providers and clinical trial sponsors. Visit stemcell.uci.edu to learn more about UCI clinical stem cell trials and regenerative medicine research.

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