The Center for International Blood and Marrow Transplant Research (CIBMTR) and Kite Pharma recently agreed to collaborate on tracking long-term outcomes of patients treated with Yescarta (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T-cell therapy.
Under the terms of this agreement, CIBMTR will collect and analyze long-term safety and effectiveness data for Yescarta, the first CAR T-cell therapy approved in the U.S. for the treatment of adults with different types of lymphoma.
CIBMTR collaborates with scientists around the world to advance stem cell transplants and cellular therapy. The research consortium stems from a collaboration agreement signed by the National Marrow Donor Program/Be The Match and the Medical College of Wisconsin.
“We are delighted to announce this partnership with Kite, a pioneer in the emerging cell therapy field,” Mary M. Horowitz, MD, chief scientific director of CIBMTR, said in a press release. “This collaboration will leverage CIBMTR’s deep experience collecting and analyzing data on both the short and long-term outcomes of patients receiving complex cellular therapies, initially gleaned during our work supporting the development of HCT, the first, and for many years the only, successful cellular therapy for cancer. We look forward to expanding into the field of targeted cellular therapy and to contributing to efforts to better understand the potential for these therapies to improve outcomes for patients with cancer.”
Not unlike other CAR T-cell therapies, Yescarta, developed by Kite, helps the patients’ own immune T-cells, which have been collected and genetically modified in a laboratory, recognize and fight lymphoma cells producing the surface protein CD19.
In October 2017, Yescarta was approved by the U.S. Food and Drug Administration as a treatment for relapsed or refractory large B-cell lymphoma (after a minimum of two lines of systemic therapy), diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), high grade B-cell lymphoma and DLBCL arising from follicular lymphoma, or transformed follicular lymphoma (TFL).
The approval was supported by data from the ZUMA-1 Phase 1/2 clinical trial (NCT02348216), which evaluated the effects of Yescarta in 101 patients with DLBCL, PMBCL, or TFL, most at an advanced stage of the disease.
About 72 percent of patients who received Yescarta responded to therapy and 51 percent achieved complete remission. After a median follow-up of 7.9 months, more than 50 percent of patients with complete responses were still disease-free.
While the gains are promising, cytokine release syndrome — a severe systemic immune reaction that is typically seen with CAR T therapies — was experienced by 13 percent of the study’s participants. One-third of participants also experienced severe neurological events caused by Yescarta.
These adverse events support CIBMTR and Kite’s joint initiative to evaluate the long-term safety and effectiveness of Yescarta.
The European Medicines Agency is currently reviewing a marketing authorization application requesting approval of Yescarta as a treatment for certain lymphomas in Europe. The decision is expected sometime in 2018.