Clinical-stage biopharmaceutical company, Cellectis, has received a notice from the US Food and Drug Administration (FDA) that a clinical hold has been placed on its ongoing Phase I studies evaluating the CAR-T therapy (UCART123) after a fatality.
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The cell therapy was being evaluated for the treatment of acute myeloid leukaemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). The fatality, leading to the hold on the clinical trials, occurred in the first BPDCN treated patient, who was a 78-year old male. The patient experienced cytokine release syndrome and capillary leak syndrome, eventually leading to death nine days after administration of UCART123.
The patient initially treated in the AML trial (a 58-year old female) also experienced these complications but these were resolved after 12 days. No patients treated in the Cellectis trials of UCART123 have experienced graft versus host disease.
In a statement from Cellectis, the company has revealed that it is working closely with investigators and the FDA to resume the trials with an amended protocol, which includes a lower dose of the cell therapy.
This fatality in a CAR-T cell therapy trial comes just days after the FDA approved the first ever treatment of its kind from Novartis and is anticipated to OK another form of this treatment from Kite soon, reported Endpoints News.