Research presented at the American Society of Hematology (ASH) annual meeting highlighted the potential of Novartis' experimental personalised cellular therapy CTL019 in both adults and children with acute lymphoblastic leukaemia (ALL) and chronic lymphocytic leukaemia (CLL) that has failed to respond to standard therapies. Carl June, a professor of immunotherapy at the University of Pennsylvania, which licensed the chimaeric antigen receptor (CAR) technology used in the treatment to the company, said "this cell therapy is showing a very promising remission rate in both adult and children with advanced leukaemia where there is no therapy that works."
Results presented at last year's ASH annual meeting showed that nine of 12 patients with CLL who received infusions of CTL019, which consist of a patient's own T-cells modified to target cells expressing CD19, responded to the therapy. The latest data included a pilot study of 14 adult patients and an ongoing Phase II trial that has enrolled 18 adult subjects. Results demonstrated that across the 32 adults with CLL, 47 percent responded to CTL019, with seven experiencing a complete remission of their disease.
Two of the three first patients who received treatment in 2010 for CLL are still in remission, and the genetically modified cells are still circulating. The duration that the cells have lived suggests the treatment is durable, June said.
In a separate study, results demonstrated that 19 of 22 paediatric patients with ALL experienced complete remissions and all five of the first adult ALL patients treated have so far experienced complete remissions. Novartis noted that the first paediatric patient treated with CTL019 remains in remission 20 months later. In the study, five paediatric patients have relapsed, including one whose tests revealed new tumour cells that do not express the CD19 protein. According to Novartis, the longest complete remission in the adult patients continues six months after treatment. Results also showed that one adult patient subsequently underwent a bone marrow transplant and remains in remission, while another adult patient relapsed after three months with disease that also tested negative for the CD19 protein.
June said the results in ALL are particularly encouraging, because it’s a fast-progressing disease and particularly fatal. He indicated that other cancer types, including multiple myeloma and solid tumours may also respond to the procedure. June noted that multicenter trials of CTL019 will start in the US next year, with international studies also beginning in 2014. "We are committed, through our collaboration with Penn, to expand the clinical trials of CTL019 to include as many patients as soon as possible," commented Herve Hoppenot, president of Novartis oncology unit.
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