The approval of CAR T-cell therapy for paediatric leukaemia marks an important shift in the blood cancer treatment paradigm, the President of the American Society of Haematology says.
Novartis’s therapy is approved to treat children and young adults with relapsed acute lymphoblastic leukaemia and will be marketed as tisagenlecleucel (Kymriah).
In response to the FDA approval ASH President Kenneth C. Anderson, MD, of the Dana-Farber Cancer Institute said: “We now have proof that it is possible to eradicate cancer by harnessing the power of a patient’s own immune system. This is a potentially curative therapy in patients whose leukaemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine.
However he added that the approval was only an option for a small population of children.
“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.
Earlier this year another CAR T-cell therapy company, Juno Therapeutics, brought a halt to its clinical trial after 5 patients died of brain swelling.