News Feature | March 19, 2014

First Cancer Trial Using CAR T Cells With Safety Switch Begins

By Estel Grace Masangkay

Bellicum Pharmaceuticals, Inc. announced the launch of the first clinical cancer study of Chimeric Antigen Receptor (CAR) T cell therapy that uses a safety switch to trigger rapid elimination of administered T cells if they endanger the life or health of the patient.

Kevin Slawin, M.D., Executive Chairman and Chief Medical Officer of Bellicum Pharmaceutical, said “CAR T cell therapy has recently shown dramatic results including apparent cures in some of the deadliest of cancers, but the treatment can also cause life-threatening toxicities, and has resulted in some patient deaths. Experts in the field have talked about the critical need for an effective safety mechanism to allow the development of more potent products within this new class of immunotherapy.”

The company said that researchers from the National Cancer Institute (NSI) have started treating pediatric patients with osteosarcoma and other non-neuroblastoma GD2-expressing solid tumors with third generation CAR T cell therapy that integrates Bellicum’s novel CaspaCIDe safety switch technology.

An estimated one-fourth of cancer diagnoses in pediatric patients are non-CNS solid tumors, which represent about one-third of all deaths due to childhood cancer. Only about <20% of patients with metastatic osteosarcoma or who have unresectable disease survive. Likewise, patients with recurrent osteosarcoma have survival rates of about 20 to 30% even with resection with or without chemotherapy. Many patients suffer lifelong toxicities associated with cancer therapy, presenting an unmet medical need to reduce toxicity and improve survival for patients with pediatric solid tumors.

CaspaCIDe safety switch technology includes Caspase-9-based cell safety switch together with the small molecule activator AP1903. In cases of serious or life threatening toxicity event caused by the administered T cells, AP1903 is administered to enable rapid destruction and elimination of the CaspaCIDe–enabled cells.

The company said the inclusion of safety switch in third generation CAR T cell therapy allows for safer evaluation of the therapy in patients. “We're excited to be the first to make this a reality by incorporating our unique safety switch into this third generation CAR T cell therapy, thereby potentially giving these patients, and their families and physicians, an added layer of safety,” said Slawin.