HA-1 TCR T-cell Immunotherapy for Acute Leukemia
Many children with leukemia are cured with chemotherapy alone but unfortunately some children have forms of leukemia that are resistant to standard chemotherapy. Children with chemotherapy-resistant leukemia can be cured by "hematopoietic stem cell transplantation" (HCT), including bone marrow, cord blood and similar forms of blood cell transplantation. HCT is successful in treating high-risk childhood leukemia most of the time but sadly about 25% of children and young adults who receive HCT relapse again afterwards. Chemotherapy and other standard treatments are generally ineffective in children who relapse after HCT and most (>90%) of these children die with an average survival of only 4 months. New therapies to prevent and treat relapse of leukemia in pediatric HCT recipients are desperately needed.
Dr. Bleakley is a pediatric oncologist who specializes in HCT and immunotherapy to treat leukemia. She has developed a form of "T cell" immunotherapy that targets a protein called "HA-1" which is found on the surface of leukemic cells in some patients. HA-1 T cell immunotherapy may be effective for treating and preventing relapse after HCT. Dr. Bleakley is proposing a clinical trial to test the feasibility, safety and potential effectiveness of HA-1 T cell immunotherapy in 10 children and young adults with early relapse of leukemia after HCT. If the trial is successful it will provide a new form of treatment for leukemia. The trial will also provide important information for the future development of other types of immunotherapy including vaccines to help many patients with high-risk leukemia.
"The ALSF Bio-therapeutics Impact Grant award provides us with an outstanding opportunity to move a novel type of T cell immunotherapy from our laboratory to the clinic. HA-1 TCR T cells recognize and kill several types of acute leukemia and lymphoma in laboratory studies and have the potential to prevent and treat relapse of leukemia following stem cell transplantation. The ALSF Bio-therapeutics Impact Grant will allow us to take the essential next steps of opening and conducting a phase I clinical trial of HA-1 TCR T cell immunotherapy for children who relapse after transplantation. At present children with post-transplant relapse have few if any curative treatment options available to them so the trial will address an area of critical need in pediatric oncology." 12/2014