Children's National Health System Experts to Present Data on Novel Approach to Cell Therapy for Cancer Patients

ORLANDO, Fla. – Emerging data from Children’s National Health System are providing hope that a new approach to cell therapy may effectively harness the cancer-killing potential of the natural immune system as a treatment for patients with blood and bone marrow cancers for whom stem cell transplantation has not worked. These patients typically have few treatment options and experience very high mortality rates. 

Initial results from the Phase 1 RESOLVE trial, a multi-institutional, Phase 1 dose-ranging study co-led by Kirsten Williams, M.D., and Catherine Bollard, M.D., M.B.Ch.B., Chief of the Division of Allergy and Immunology and Director of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National, showed that the majority (78 percent) of patients responded to multi tumor-associated antigen specific lymphocytes (TAA-L) treatment, and 44 percent of patients achieved complete remission with limited toxicity. The trial included patients diagnosed with one of four tumors who were treated with TAA-L upon relapse of disease post stem cell transplant. 

“These initial findings suggest that non-genetically engineered antigen-specific lymphocytes can be isolated, expanded and adoptively transferred to severely ill patients with active disease and positively impact tumor regression,” says Dr. Bollard. “We are encouraged by the promise of these data, which support similar efforts to exploit the immunotherapeutic potential of the natural T-cell repertoire.” 

The data, presented at the BMT Tandem meeting in Orlando, Florida, was a collaboration between investigators at Children’s National and researchers at Johns Hopkins University and Baylor College of Medicine who are testing a novel approach to treating cancer. Their approach includes introducing TAA-L – a natural T-cell subtype from the immune system – to high-risk patients with advanced hematologic or blood cancers including acute myeloid leukemia (AML)/ Myelodysplastic Syndromes (MDS), B-cell acute lymphoblastic leukemia (ALL) and Hodgkin lymphoma. The trial evaluates the safety and efficacy of both donor and patient-derived TAA-L as a novel treatment for patients with AML/MDS, B-cell ALL or Hodgkin lymphoma who are in active disease relapse pre- or post-allogeneic hematopoietic stem cell transplantation (HSCT).

Patients with refractory and relapsed AML, MDS, ALL and Hodgkin lymphoma often have extremely poor clinical outcomes. For patients with these malignancies who relapse after allogeneic stem cell transplantation, the prognosis is even more dismal. Sadly, one-year mortality rates for this population approach 90 percent.  

The results include patients receiving adoptively transferred TAA-L manufactured cells across all dosing cohorts allowed per protocol (dose levels one through four), 10 patients in total. Preliminary observations conclude that ex vivo manufactured TAA-L, composed primarily of central effector memory T-cells, can be successfully isolated and expanded to clinically relevant numbers, cryopreserved, and safely infused to patients after relapse of disease. There were no cases of graft-versus-host disease or other autoimmune-mediated toxicity reported, and there have been no observations of Cytokine Release Syndrome or neurotoxicity associated with TAA-L treatment. 

The RESOLVE trial has been and/or is funded by a National Institutes of Health grant (P01CA015396), the Leukemia Lymphoma Society, Ben’s Run and Hyundai Hope on Wheels. The BMT Tandem meeting is the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.