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Hematopoietic Cell Transplantation: From a Curative Concept to Cure

This article was published in December 2008 as part of the special ASH anniversary brochure, 50 Years in Hematology: Research That Revolutionized Patient Care.

In 1868, two investigators from Prussia and Italy were the first to report that bone marrow generates blood cells in mammals. This observation was the starting point of intense efforts to replace malfunctioning marrow through the transplantation of blood-forming (hematopoietic) cells in humans. However, it took an entire century to develop reliable techniques to identify suitable donors, design potent antibiotics, and establish essential supportive care methods to successfully perform clinical hematopoietic cell transplantation (HCT) procedures. This important accomplishment would not have been possible without decades of  intense pre-clinical research in animals, especially to understand issues related to graft rejection and the prevention of graft-versus-host disease (GVHD), a serious reaction caused by the donor's immune cells attacking the recipient's vital organs. The research team at the Fred Hutchinson Cancer Research Center in Seattle, under the leadership of Dr. E. Donnall Thomas (Nobel Laureate in Medicine and Physiology, 1990), deserves the credit for the major contributions to our knowledge in the field of HCT - although countless investigators around the globe have also made many essential research observations.

In 1968, three patients in the United States and the Netherlands suffering from severe combined immunodeficiency syndromes, in which the ability of the body's immune system to fight infections is compromised, were successfully transplanted and cured by HCT. Since then, the number of HCT procedures has risen to 50,000-60,000 per year, and the cumulative number of patients treated worldwide has now reached 800,000. The majority of HCT recipients are patients with leukemia, lymphoma, myeloma, myelodysplasia, bone marrow failure conditions, severe red blood cell disorders such as sickle cell disease or thalassemia, and certain solid tumors.