Clinical Trials

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.

In studies of adult patients with CLL, 15 of 32 adult patients (47%) responded to the therapy, with seven of those experiencing a complete remission of their disease (abstract #4162 and #873). These results are derived from a completed pilot study of 14 CLL patients (abstract #4162) and results to date of the first 18 patients in a Phase II, dose-optimization trial (abstract #873)[2],[3]. In the pilot study of CTL019 for the treatment of adult patients (n=14; median age=67) with relapsed/refractory CLL (abstract #4162), the target dose of cells administered to patients was 5x109 mononuclear cells with an expected TE of 10-40% (total CTL019 dose 5x108 - 2x109 total cells)[2]. The Phase II dose optimization study (abstract #873) of two doses of CTL019 cells in patients with relapsed/refractory CLL (n=27; median age=63 years) was conducted to better define an optimal CTL019 cell dose following results from the initial pilot study. In this ongoing trial, patients were randomly assigned to receive either 5x108 vs. 5x107 transduced CTL019 cells. Results thus far from 18 patients in the study are being presented. A preliminary analysis through July 15, 2013 showed that in 10 adult patients, 20% of patients (n=2) achieved a complete response (CR) and 20% a partial response, for an overall response rate of 40% (n=4).

• 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 chronic lymphocytic leukemia (CLL) and build on earlier research findings. In studies of adult patients with CLL, 15 of 32 adult patients (47%) responded to the therapy, with seven of those experiencing a complete remission of their disease (abstract #4162 and #873). These results are derived from a completed pilot study of 14 CLL patients (abstract #4162) and results to date of the first 18 patients in a Phase II, dose-optimization trial (abstract #873). Separately, investigators observed in vivo expansion of CTL019 cells in all patients who achieved a complete response. This was followed by contraction and, in all but one patient, ongoing stable persistence of the reprogrammed T cells (abstract #163). 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.