Axicabtagene ciloleucel (Yescarta®) – Autologous Transplant CAR-T Cell Gene Therapy

Axicabtagene ciloleucel (axi-cel, Yescarta®) is a gene therapy modality used in the treatment of diffuse large B-cell lymphoma (DLBCL), a common subtype of non-Hodgkin lymphoma.  Axi-cel treatment involves isolation of patient T-lymphocytes, lentiviral transduction of the lymphocytes with an anti-CD19 chimeric antigen T-Cell receptor (CAR), and intravenous reinfusion of the resulting CAR-T cells into the patient.

Axicabtagene ciloleucel is the 2nd gene therapy product approved by the FDA after tisagenlecleucel (Kymriah®), a similar treatment for acute lymphoblastic leukemia.  The vast majority of patients exposed to axi-cel experience significant toxicity involving cytokine release syndrome and a resulting encephalopathy.



  1. Wishart DS, Feunang YD, Guo AC, Lo EJ, Marcu A, Grant JR, Sajed T, Johnson D, Li C, Sayeeda Z, Assempour N, Iynkkaran I, Liu Y, Maciejewski A, Gale N, Wilson A, Chin L, Cummings R, Le D, Pon A, Knox C, Wilson M. DrugBank 5.0: a major update to the DrugBank database for 2018. Nucleic Acids Res. 2017 Nov 8. doi: 10.1093/nar/gkx1037
  2. Epocrates Rx Online [Internet database]. San Mateo (CA): Epocrates, Inc. 2003. Retrieved at Web-based; continuous content updates. Accessed 2018 April 27.
  3. Neelapu SSLocke FLBartlett NL, et al. Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma. N Engl J Med 2017;377:25312544.