A Phase I/II Study Assessing BPX-501 T Cells Given After Partially Mismatched Related T Cell-Depleted Stem Cell Transplantation in Patients with Hematologic Cancers

Full Title

A Phase 1-2 Dose Escalation Study Evaluating Safety and Feasibility of BPX-501 T Cells after Partially Mismatched, Related, T Cell-Depleted HSCT


About 60 percent of patients who have a stem cell transplant experience a complication called graft-versus-host disease (GVHD), in which T cells (white blood cells) from the donor attack tissues of the recipient. Symptoms of GVHD include rash, digestive problems, weight loss, and liver problems. Removing T cells from the stem cell donation can reduce the risk of GVHD, but it also increases the amount of time it takes for the recipient’s immune system to recover after the transplant.

In this study, researchers are seeking to find the best dose of specially modified T cells that can be given to patients with hematologic cancers who are receiving a stem cell transplant from a partially matched relative. The T cells from the donor are removed, sent to a laboratory, and given a gene that will make them react to an investigational drug called AP1903, which can turn off their ability to cause GVHD.

The modified T cells, now called BPX-501, are given to the patient with the stem cell transplant. If the patient develops GVHD, he/she can receive AP1903, which will activate a “turn-off” switch and make the BPX-501 T cells stop working. This could be a novel approach to enhancing immune system recovery after stem cell transplantation while reducing the risks associated with GVHD.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have acute leukemia, myelodysplastic syndromes, or non-Hodgkin lymphoma and be scheduled to receive a stem cell transplant from a partially mismatched donor.
  • Patients should have had chemotherapy within three months of entering the study.
  • This study is open to patients ages 18 to 65. Donors may be between the ages of 18 and 70.

For more information and to inquire about eligibility for this study, please contact Dr. Miguel-Angel Perales at 212-639-8682.