Novel gene therapy success in B cell Acute Lymphoblastic Leukemia - Juno Therapeutics

The largest clinical study ever conducted to date of patients with advanced leukemia found that 88 percent achieved complete remissions after being treated with genetically modified versions of their own immune cells. The results were published today in Science Translational Medicine. Adult B cell acute lymphoblastic leukemia (B-ALL), a type of blood cancer that develops in B cells, is difficult to treat because the majority of patients relapse. Patients with relapsed B-ALL have few treatment options; only 30 percent respond to salvage chemotherapy. Without a successful bone marrow transplant, few have any hope of long-term survival. In the current study, 16 patients with relapsed B-ALL were given an infusion of their own genetically modified immune cells, called T cells. The cells were "reeducated" to recognize and destroy cancer cells that contain the protein CD19. While the overall complete response rate for all patients was 88 percent, even those with detectable disease prior to treatment had a complete response rate of 78 percent, far exceeding the complete response rate of salvage chemotherapy alone.

Investigators at Memorial Sloan Kettering Cancer Center together with The Fred Hutchinson Cancer Research Center and Seattle Children's Research Institute have been pioneering the use of genetically modified T cells to fight cancer. They have combined to form Juno Therapeutics, to develop therapies that re-engineer a patient's T cells with chimeric antigen receptors (CAR) to destroy cancer cells.

See-"Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia"- Marco L. Davila et al., Sci Transl Med 19 February 2014: Vol. 6 no. 224 pp. 224ra25 DOI:10.1126/scitranslmed.3008226