On March 17, 2014 Bellicum Pharmaceuticals reported that the first clinical study is underway of a Chimeric Antigen Receptor (CAR) T Cell therapy that incorporates a safety switch to enable rapid elimination of the administered T cells if they threaten the life or health of the patient. Researchers from the National Cancer Institute (NCI) have begun treating pediatric patients with osteosarcoma and other non-neuroblastoma GD2-expressing solid tumors with a third generation CAR T cell therapy that incorporates Bellicum’s unique CaspaCIDe safety switch technology (Press release Bellicum Pharmaceuticals, MAR 17, 2014, View Source [SID:1234500805]).
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"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," said Kevin Slawin, M.D., Executive Chairman and Chief Medical Officer of Bellicum Pharmaceuticals. "We’re excited 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."
Bellicum’s CaspaCIDe technology consists of an inducible Caspase-9-based cell safety switch, and the small molecule activator, AP1903. CaspaCIDe is engineered into immunotherapy cells, in this case GD2-targeted third generation CAR T cells, before they are introduced to the patient. In the event of a serious or life-threatening toxicity caused by the administered T cells, AP1903 is infused to trigger rapid destruction and elimination of the CaspaCIDe-enabled cells.
CaspaCIDe technology has already demonstrated the ability to selectively eliminate harmful immune cells, resolving acute Graft versus Host Disease (GvHD) in stem cell transplant patients.1 Bellicum is currently evaluating its own lead CaspaCIDe-enabled product, BPX-501, together with AP1903, in clinical studies to improve transplant outcomes and reduce or eliminate GvHD following stem cell transplant in multiple indications, populations and transplant modalities. The collaboration with NCI represents Bellicum’s initial application of its expertise and technology to the clinical advancement of the CAR T cell field.