CAR T-cell therapy approved for pediatric, young adult B-cell precursor ALL
medwireNews: Tisagenlecleucel has been granted regular approval for the treatment of refractory or relapsed B-cell precursor acute lymphoblastic leukemia (ALL) in patients aged up to 25 years by the US FDA.
The chimeric antigen receptor (CAR) T-cell immunotherapy uses autologous T cells that are genetically modified to include a CAR protein that can identify and remove cells expressing CD19.
Between 2 and 14 days after completing lymphodepleting chemotherapy, patients are given a single infusion of 0.2–5.0 x 106 or 0.1–2.5 x 108 CAR-positive viable T cells per kg for patients below 50 kg and greater than 50 kg, respectively.
The approval is based on clinical trial data for 63 patients with relapsed or refractory pediatric precursor B-cell ALL, 35 of whom had previously received a hematopoietic stem cell transplant. A confirmed remission was achieved by 82.5% of the patients, including complete remission in 63%, and the median duration of remission in the group is not yet reached.
And approval was also given by the FDA for use of the interleukin-6 receptor antibody tocilizumab in patients who develop severe or life-threatening cytokine release syndrome during CAR T-cell therapy.
President of the American Society of Hematology, Kenneth Anderson (Dana-Farber Cancer Institute, Boston, Massachusetts, USA) commented in a press release that the CAR-T approval “marks an important shift in the blood cancer treatment paradigm.”
But noting that the development is applicable yet only to a small pediatric population, he added: “More research is needed to make this therapy more effective for a broader population, to reduce the severe side effects that patients experience during treatment, and ultimately to find a broader application beyond blood cancers.
“Continued research will also lead to improved manufacturing of large numbers of cells, which is necessary to make this therapy accessible to more patients.”
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