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FDA Halts Off-the-Shelf Engineered T-Cell Trial

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(dimitrisvetsikas1969, Pixabay)

7 July 2020. A company designing gene-edited T-cells as cancer therapies stopped its clinical trial of a treatment for multiple myeloma when a patient in the trial died. Cellectis S.A. in New York and Paris says the Food and Drug Administration put a hold on the early-stage trial testing the safety of its engineered T-cell therapy.

Cellectis develops cancer treatments that harness the immune system by breaking down defenses tumors create to prevent the body’s immune system from fighting the disease. The company’s platform builds on recent developments that take T-cells, white blood cells from the immune system, and reprogram the cells through genetic engineering to find and kill cancer cells. The engineered T-cells become hunter cells, containing proteins known as chimeric antigen receptors that act like antibodies. These modified chimeric antigen receptor or CAR T-cells are infused into the patient, seeking out and binding to proteins associated with the cancer.

Most current CAR T-cell methods genetically engineer a patient’s own T-cells, then re-infuse the altered T-cells back into the individual. Cellectis’s process is designed to produce off-the-shelf CAR T-cell treatments, it calls Universal CAR T-cells, or UCARTs. These treatments use T-cells from healthy donors, rather than a patient’s own T-cells, then are genetically engineered to match the attributes of specific cancer types. In November 2019, Science & Enterprise reported on preclinical tests that show UCARTS accumulate on lymphoma cells in mice and generate cancer-killing proteins.

The clinical trial is testing Cellectis’s treatment for multiple myeloma code-named UCARTCS1. The early-stage study is enrolling 18 participants at three sites in the U.S. with relapsing or stubborn multiple myeloma, looking primarily for adverse effects among the patients. The company plans to test multiple dosage levels of the treatments, seeking the maximum tolerated dose.

Cellectis says a participant in the trial who had not responded to previous treatments for multiple myeloma suffered cardiac arrest after receiving UCARTCS1 and died. While the company says its investigation is continuing, the participant received one of the higher doses of the therapy. Cellectis says it earlier decided to expand the number of recipients at the treatment’s lower dose.

Carrie Brownstein, Cellectis’s chief medical officer, says the company is working with FDA to resolve the clinical hold, including changes to the design of the trial. “The safety of patients enrolled in our clinical trials is our utmost priority,” says Brownstein in a company statement, “and we at Cellectis remain committed to safely resuming the clinical development of UCART product candidate targeting CS1 for patients with multiple myeloma and unmet medical need.”

Cellectis has two other early-stage clinical trials underway testing UCARTs that are not affected by FDA’s hold, for acute myeloid leukemia and B-cell acute lymphoblastic leukemia. Both of the studies are testing UCARTs at various dosage levels.

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