Between 15 and 20 percent of the children treated for acute lymphoblastic leukemia (ALL) will suffer a relapse. The mortality rate of these patients ranges from 50 to 85 percent. Until recently, there were few options for treating cancer in relapsed ALL patients. However, a revolutionary treatment has been discovered in CAR T-cell therapy. 

CAR T-cell therapy, short for chimeric antigen receptor T-cell therapy, takes the patient's own T-cells and re-engineers them to express a receptor for CD19. CD19 is a protein typically present on the surface of B-cell malignancies, including several forms of leukemia and lymphoma such as ALL. The newly manufactured T-cells are then readministered into the patient to target CD19 and attack any residual leukemia cells. CAR T-cell therapy clinical trials have shown remission rates of up to 94 percent in severe forms of cancer. 

Innovative anticancer cellular therapies 

In November 2014, Cincinnati Children’s Cancer and Blood Diseases Institute (CBDI) was one of the nation’s first clinical trial sites to test Kymriah, the first CAR T-cell therapy for childhood ALL. This work helped lead to the FDA approval of Kymriah in 2017, and Cincinnati Children’s is one of the few sites approved for its use. Kymriah is a one-time treatment for B-cell acute lymphoblastic leukemia (B-ALL), and has shown an 83 percent remission rate after three months in clinical trials with patients who do not respond to standard treatments. In 2017, the Cancer Immunotherapy Center at the CBDI also instituted clinical trials with KTE-C19, an additional CD19-targeted CAR T-cell therapy using novel technology. These new, engineered anticancer cellular therapies are innovative and exciting, and a primary focus for our research.

AML breakthrough

Incorporating new therapeutic agents into the treatment of children and young adults with relapsed acute myeloid leukemia (AML) is another major focus of the CBDI’s Leukemia and Lymphoma Program. Michael Absalon, MD, PhD, is leading the development of CPX-351 for pediatric patients. He led the first pediatric cancer clinical trial of this new liposomal nanotechnology chemotherapy drug; the study has been performed entirely at Cincinnati Children’s. Based on this critical work, CPX-351 is now being studied nationally in the National Cancer Institute’s Children’s Oncology Group Consortium, with plans to incorporate it into the core therapy for all children with newly diagnosed AML across the country. Absalon presented his groundbreaking research at the Annual Meeting of the American Society for Clinical Oncology in Chicago, Ilinois.