The search for more effective and less toxic therapies for cancer has occupied scientists for decades. Recently approved treatments involving the genetic modification of a patient’s own T cells to better target cancer cells are an important advance in cancer treatment, thereby sparing patients less effective therapies while offering a meaningful chance at remission. Chimeric antigen receptor (CAR) T cells involve the ex vivo transduction of T cells so they express a receptor on their surface that both is specific for a tumor antigen and contains T-cell activation domains such that engagement of the receptor by tumor antigen results in activation of the T cell against the tumor cell. The 2 approved CAR T-cell products include axicabtagene ciloleucel, manufactured by Kite Pharma, and tisagenlecleucel, a Novartis product. Both therapies target CD19 on the surface of B lymphocytes and are approved as single infusions for the treatment of relapsed and refractory B-cell malignancies, including B-cell acute lymphoblastic leukemia (B-ALL) in children and young adults (tisagenlecleucel) and aggressive B-cell non-Hodgkin lymphoma (B-NHL) (both tisagenlecleucel and axicabtagene ciloleucel). Even though these cell therapies produce durable remissions in some patients with no existing effective options, they are priced at a 1-time cost of $475 000 for B-ALL and $373 000 for B-NHL. Countries around the world are grappling with the cost of these treatments.
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Jacobson C, Emmert A, Rosenthal MB. CAR T-Cell Therapy: A Microcosm for the Challenges Ahead in Medicare. JAMA. Published online July 29, 2019322(10):923–924. doi:10.1001/jama.2019.10194
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