What is the role of gene therapy in the treatment of acute lymphoblastic leukemia (ALL) relapse?

Updated: Jul 02, 2021
  • Author: Karen Seiter, MD; Chief Editor: Matthew C Foster, MD  more...
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The first autologous chimeric antigen receptor (CAR) T-cell therapy, tisagenlecleucel (Kymriah), was approved in August 2017 for use in patients aged 25 years or younger with B-cell precursor ALL that is refractory or in second or later relapse. [38] Autologous T-cells are collected from peripheral blood and genetically engineered to express a CAR that targets a specific molecule on cancer cells. The modified T-cells are then expanded and reinfused into the patient, 2-14 days after completing lymphodepletion with conditioning chemotherapy. [39]

Because of the risk of potentially fatal adverse effects, use of tisagenlecleucel is limited to hospitals and clinics with special certification in risk evaluation and mitigation. [38]

Tisagenlecleucel IV infusion is based on patient weight, as follows [39] :

  • ≤50 kg: 0.2-5 x 10 6 CAR-positive viable T cells/kg
  • > 50 kg: 0.1-2.5 x 10 8 CAR-positive viable T cells/kg

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