Safety and efficacy of anti-CD19 chimeric antigen receptor (CAR)-modified autologous T cells (CTL019) in advanced multiple myeloma.

8517 Background: CTL019, a 2nd-generation anti-CD19 CAR transduced via lentiviral vector, can induce regression of refractory B cell malignancies. Though multiple myeloma (MM) is reported to be CD19-negative, we hypothesized that CTL019 would exhibit efficacy in MM due to low-level CD19 expression on MM plasma cells (PC) or CD19 expression in drug-resistant, disease-propagating subsets of the MM clone. Here, we report initial results of an ongoing phase 1 study of CTL019 in patients with advanced MM. Methods: MM patients are eligible if they experienced disease progression within one year of a prior autologous stem cell transplantation (ASCT) and are medically fit to undergo second ASCT. Study therapy consists of 1-5x107CTL019 cells infused 12-14 days after high-dose melphalan + ASCT. Results: 4 subjects have been treated and have completed 30-220 days of follow-up. Median prior lines of therapy is 7.5 (range 3-10). 3/4 have unfavorable cytogenetics; 1/4 had PC leukemia. Adverse events have included hypog...