Novartis highlights new CTL019 clinical data showing complete remissions in children and young adults with relapsed/refractory acute lymphoblastic leukemia - Seite 2

Additional highlights of the pediatric r/r ALL study include findings that patients have ongoing CR. Median follow-up was 6 months. Sustained remissions were achieved up to one year or more with 6-month event-free survival of 70% and overall survival of 75%, in most cases without further therapy[1]. The probability of six-month CTL019 persistence was 68%, which was accompanied by B cell aplasia, a pharmacodynamic marker of CTL019 persistence and function[2]. Persistence of CTL019 cells detected by flow cytometry and/or qPCR, and accompanied by B cell aplasia, continued for up to 30 months after infusion in patients with ongoing responses[1].

All responding patients developed cytokine release syndrome (CRS) at peak T cell expansion. Treatment for CRS was required for hemodynamic or respiratory instability in 33% of patients and CRS was managed with an IL-6 receptor antagonist, together with corticosteroids in five patients. These events were delayed, and few patients experienced infusional toxicities, including infusion-associated fever[1].

"Innovation in the cellular therapy field is accelerating right now. When we see the response patients have to CTL019 when they have few options left, it's incredibly inspiring," said Usman Azam, Global Head, Cell & Gene Therapies Unit, Novartis Pharmaceuticals. "Novartis will leverage our facility in Morris Plains, the first FDA-approved Good Manufacturing Practices quality site for a cell therapy, and the multi-center study for CTL019 in collaboration with the University of Pennsylvania, to broaden the reach of this therapy to additional patients in the clinical setting." 

Also included among the presentations at ASH is a study investigating CTL019 in the treatment of individuals with CD19+ B cell lymphomas (Abstract #3087; December 7, 6:00 PM - 8:00 PM) that reveals complete responses in patients with advanced, relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma[3]. In addition, data will be presented on three cases of refractory cytokine release syndrome (CRS) in adult patients with ALL (Abstract #2296, December 7, 6:00 PM - 8:00 PM)[4]. CRS is correlated with CTL019 proliferation and the severity of CRS is correlated with disease burden[1],[2],[4].