FDA halts trial after first CAR T-associated patient death
pharmafile | September 6, 2017 | News story | Research and Development | CAR T, CAR-T, Cancer, FDA, cellectis, leukaemia, patient death, pharma, pharmaceutical
French clinical-stage biopharma firm Cellectis has received an order from the FDA to halt its study of CAR T therapy UCART123 after a 78-year old patient died as a result of a toxic reaction to the treatment.
The man was enrolled in ABC123, the first of the company’s two studies into the treatment, which was investigating the effect of UCART123 in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN). He was first treated on 16 August after being given a preconditioning regimen involving fludarabine and cyclophosphamide, experiencing a grade 2 cytokine release syndrome (CRS) and grade 3 lung infection five days later.
Though these conditions improved with treatment of tocilizumab and antibiotics, three days later he suffered a grade 5 CRS event and grade 4 capillary leak syndrome which did not respond to tocilizumab or corticosteroids, resulting in his death a day later.
However, in the second of the two parallel studies – AML123, which investigated the treatment in acute myeloid leukaemia – a 58-year old woman survived similar complications even after being given the same dose of UCART123 and the same preconditioning regimen.
The company explained: “She experienced an initial grade 2 CRS at day 8, worsening to grade 3 at day 9 and resolving at day 11 with treatment management in an intensive care unit. She also experienced a grade 4 capillary leak syndrome at day 9, resolved at day 12.”
CAR T therapy works by extracting a patient’s immune T cells and genetically altering them to create a personalised treatment for disease. Cellectis’ UCART123 is a form of CAR T which modifies the patient’s T cells to target the CD123 antigen on the surface of cancer cells.
The company has said that it is, “working closely with the investigators and the FDA in order to resume the trials with an amended protocol, including a lowered dosing of UCART123.”
Matt Fellows
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