Date: 2013-12-07
Type of
information: Presentation of results at a congress
phase:
Announcement: presentation of results at the American Society of Hematology Annual Meeting, New Orleans, LA, 2013
Company: Novartis (Switzerland)
Product: CTL019 (tisagenlecleucel-T)
Action
mechanism: cell therapy/immunotherapy product/gene therapy/CAR-T cell therapy. CTL019 is an investigational, personalized T cell therapy, which was pioneered by Carl June and his team at Penn. In a CTL019 treatment cycle, immune cells (T cells) are drawn from a patient's blood. Then, using CAR technology, the T cells are reprogrammed to "hunt" cancer cells that express specific proteins, called CD19. When the T cells are re-introduced into the patient's blood, the cells proliferate and bind to the targeted cancer cells and destroy them. CD19 is associated with a number of B-cell malignancies including ALL, CLL, diffuse large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma.
Disease: acute lymphoblastic leukemia (ALL)
Therapeutic
area: Cancer - Oncology
Country:
Trial
details:
Latest
news:
- • On December 7, 2013, Novartis has highlighted new research from members of the faculty at the University of Pennsylvania's Perelman School of Medicine (Penn) on the investigational chimeric antigen receptor (CAR) therapy, CTL019. Several studies being presented at the American Society of Hematology (ASH) annual meeting add to the scientific understanding of CTL019 in the treatment of acute lymphoblastic leukemia (ALL) and build on earlier research findings.
- In a study evaluating CTL019 for the treatment of children and adults with relapsed/refractory ALL (abstract #67), pediatric patients (n=22) received a targeted T cell dose range of 107 to 108 cells/kg with a transduction efficiency (TE) of 11-45% and adult patients (n=5) received a target dose of 5x109 total cells split over three days with a TE of 6-31%. The study found that 19 of 22 pediatric patients with ALL (86%) experienced complete remissions and all five of the first adult ALL patients treated have thus far experienced complete remissions. There were no infusional toxicities greater than Grade 2, although five patients developed fevers within 24 hours of infusion and did not receive planned subsequent infusions of CTL019 cells.
Highlights of the presentations include findings that 19 of 22 pediatric patients with ALL (86%) experienced complete remissions. The first pediatric patient treated with the protocol remains in remission 20 months later. Five pediatric patients have relapsed, including one whose tests revealed new tumor cells that do not express the CD19 protein targeted by the reprogrammed cells. Additionally, all five of the first adult ALL patients treated to date have experienced complete remissions, the longest of which continues six months after treatment. One adult patient subsequently underwent a bone marrow transplant and remains in remission. Another adult patient relapsed after three months with disease that also tested negative for the CD19 protein (abstract #67 -Grupp S et al. T Cells Engineered With a Chimeric Antigen Receptor (CAR) Targeting CD19 (CTL019) Produce Significant In Vivo Proliferation, Complete Responses and Long-Term Persistence Without Gvhd in Children and Adults with Relapsed, Refractory ALL).
- Finally, a separate study (abstract #163) evaluating functional persistence, trafficking and bioactivity of CTL019 cells in patients treated with CTL019 determined that CTL019 holds the potential to effectively target CD19-positive malignancy. In all patients who achieved CR, in vivo expansion of CTL019 cells was observed followed by contraction and in all but one patient, ongoing stable persistence of engineered cells, elimination of tumor B cells and ongoing B cell aplasia in blood and marrow at all evaluated time points (minimum 3 months, maximum ongoing at 35 months).
- Novartis and Penn have an exclusive global agreement to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers. Novartis holds the worldwide rights to CARs developed through the collaboration for all cancer indications, including the lead program CTL019 (also known as CART19). This innovative collaboration has expanded to include multiple CART programs now in discovery and pre-clinical research phases for both hematological cancers and solid tumors.
Is
general: Yes
