Blood & Marrow Stem Cell Transplantation: Research Milestones

Our physicians have been at the forefront of advances in hematopoietic (blood-forming) stem cell transplantation since the procedure was developed decades ago. Advances in therapy achieved at Memorial Sloan Kettering include milestones that have improved outcomes for patients everywhere.

Allogeneic Transplantation

  • Performed first successful transplant in the world from an unrelated donor, in a child with severe combined immune deficiency.
  • Performed first successful transplant using marrow from an HLA (human leukocyte antigen) half-matched parent whose tissue type partially matched the patient’s.
  • Developed novel molecular methods for tissue typing, permitting more-accurate identification of potential donors.
  • Developed novel molecular methods for the early detection of opportunistic infections, particularly from viruses, leading to earlier treatment and improved outcomes.
  • Introduced the use of specific immune T cells to treat life-threatening viral infections.
  • Introduced effective treatments for cytomegalovirus pneumonia, a major complication of allogeneic marrow transplants.
  • Introduced immune cell (T cell) depletion to prevent graft-versus-host disease (GVHD) — the most frequent complication after marrow transplantation. Many centers around the world now use this method.
  • Introduced hyperfractionated total body irradiation, a method of delivering radiation therapy before transplant to increase the antileukemia and antitumor effects of radiotherapy, while decreasing short-term and long-term side effects of radiation on growth and endocrine functions.
  • Introduced escalating doses of immune cells for treatment of relapses in patients transplanted for chronic myelogenous leukemia, an approach that eradicates leukemia cells while radically reducing the risk of severe GVHD.
  • Developed novel therapies for steroid-refractory GVHD based on laboratory studies implicating certain types of dendritic cells in causing GVHD.
  • Introduced novel therapies for mismatched related transplants using new preventive measures against GVHD without impairing immune reconstitution.
  • Developed effective monitoring of immune reconstitution to guide both preventive and therapeutic interventions against opportunistic infections.

Autologous Transplantation

  • Developed a treatment, called ICE, that combines three chemotherapeutic agents — ifosfamide, carboplatin, and etoposide — to precede transplantation in patients with aggressive lymphoma. ICE enables physicians to harvest more stem cells for transplantation and can significantly improve the tumor response rate and reduce toxicity.
  • Developed prognostic models for both non-Hodgkin lymphoma and Hodgkin’s disease that help physicians predict which patients can benefit from autologous transplantation and which patients will require newer, more intensive therapies.
  • Introduced hyperfractionated total body irradiation, a method of delivering radiation therapy before transplant to increase the antitumor effects of radiotherapy and decrease short-term and long-term side effects of radiation on growth and other endocrine function.
  • Developed novel molecular methods for the early detection of opportunistic infections, particularly from viruses, leading to earlier treatment and improved outcomes.