Interim data from Bellicum Pharmaceuticals’ adjunct T cell therapy trial of BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies (PIDs) shows high rates of disease-free survival and overall survival.
BPX-501 comprises of engineered donor T cells which incorporate Bellicum’s CaspaCIDe® safety switch. This facilitates the elimination of alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. Intended for administration after an allogeneic hematopoietic stem cell transplant (HSCT), the therapy enhances immune reconstitution and viral infection control.
Data from the ongoing BP-004 trial of BPX-501 conducted at various transplant centers in the US and Europe suggests that BPX-501 T cells contributes to a durable anti-leukemic effect in patients with AML. 38 pediatric AML patients in their first (n=13) or second (n=25) complete response underwent a haplo-HSCT followed by treatment with BPX-501. Treatment with BPX-501 resulted in 91.5% relapse-free survival and 97.3% overall survival in AML patients, with a median follow-up of one year. This is the highest one-year rate reported thus far in pediatric AML patients.
Dr Neena Kapoor, Director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and an investigator in the BP-004 trial commented: “The recurrence of cancer is one of the most serious risks to AML patients receiving a stem cell transplant. These impressive results in children with AML suggest that BPX-501 may be effectively reducing or eradicating residual cancer cells following a haplo-transplant procedure.
Of the 59 pediatric PID patients who underwent haplo-HSCT and treatment with BPX-501, disease-free survival and overall survival was reported at 88.1% and 88.6%, respectively, with a median follow-up of one year.
Bellicum is now working with the investigators and the US FDA to develop a protocol for a potential US registration study in pediatric patients. Pending regulatory clearances, the company expects to initiate the study by the end of 2018.
Source: Bellicum touts promising T-cell therapy data, but U.S. program remains on hold; Press Release