Blood cancers

CAR-T therapy for refractory ALL wins expert support

Tuesday, 18 Jul 2017


An expert panel convened by the FDA has voted unanimously to recommend the approval of a novel immunotherapy that could potentially transform the survival prospects of young people with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL).  

If the FDA accepts its panel’s recommendation, CLT019 will be the first gene therapy to be approved in the US.

The 10 member panel’s decision to recommend the chimeric antigen receptor T cell (CAR-T) treatment CTL019, or tisagenlecleucel, is based on preliminary results of the global 25 centre ELIANA trial involving children with relapsed or refractory ALL. 

Overall 83% (52 of 63; 95% confidence interval [CI] = 71%–91%) patients achieved complete remission (CR) or CR with incomplete blood count recovery within 3 months of infusion.

The probability of survival was 89% at 6 months and 79% at 12 months.

Common side effects of CAR-T treatment includes seizures and delirium and were seen in the Novartis study.

Novartis plans to file for European approval this autumn but cost of the treatment remains a mystery, with speculated figures of around $ 30,000 or more per treatment.

A CAR-T treatment under study by Juno Therapeutics was suspended for the second time last year after five patients died from cerebral oedema.

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