The latest findings from an ongoing phase II study of CTL019, an investigational chimeric antigen receptor T cell ( CART ) therapy, further support its potential in the treatment of children and young adults with relapsed / refractory acute lymphoblastic leukemia ( r/r ALL ).
The study found that 55 of 59 patients ( 93% ) experienced complete remissions ( CR ) with CTL019. These results were presented at the 57th American Society of Hematology ( ASH ) Annual Meeting.
In the study, median follow up was 12 months, overall survival was 79% at 12 months ( 95% CI, 69-91% ) and relapse-free survival was 76% at six months ( 95% CI, 65-89% ) and 55% at 12 months ( 95% CI, 42-73% ). Results found that 18 patients had ongoing CR after 12 months of therapy.
Additionally, 52 of 59 ( 88% ) patients developed grade 1-4 cytokine release syndrome ( CRS ). CRS may occur after CTL019 infusion when the engineered cells become activated and multiply in the patient's body. During CRS, patients typically experience varying degrees of flu-like symptoms with high fevers, nausea, muscle pain, and in some cases, low blood pressure and breathing difficulties. Treatment for CRS was required for hemodynamic or respiratory instability in 27% of patients and was reversed in all cases with an IL-6 receptor antagonist.
Novartis and Penn have an exclusive global collaboration to research, develop and commercialize CART therapies for the investigational treatment of cancers.
In July 2014, the FDA ( Food and Drug Administration ) designated CTL019 as a Breakthrough Therapy for the treatment of pediatric and adult patients with r/r ALL under the Penn Investigational New Drug application ( IND ). Breakthrough Therapy designation is intended to expedite the development and review of drugs that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint.
Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019. ( Xagena )
Source: Novartis, 2014