This past weekend, I attended the 2011 American Society for Hematology (ASH) meeting in San Diego, where two NHLBI-supported studies were featured at the plenary session. A report on the first successful gene therapy treatment of hemophilia represents a major scientific advance in the field. Hemophilia has long been one of the disorders thought most likely to be correctible with gene therapy, but previous approaches to deliver the gene have been disappointing. This approach uses AAV8 to target gene delivery to the liver. The second NHLBI study highlighted at the plenary session was a report from the Bone Marrow Transplant Consortium demonstrating a higher incidence of overall chronic graft-versus-host disease (GVHD) and more common chronic extensive GVHD when allogeneic bone marrow transplant was performed with donor cells obtained from peripheral blood than from bone marrow. There were no survival differences according to graft sources. These articles were published simultaneously in the New England Journal of Medicine and gathered significant attention in the press.
ASH sponsored a press conference entitled "Assessing Therapeutic Strategies and Improving Quality of Life for Patients with Sickle Cell Disease," which I moderated. The panel discussed findings from two clinical trials presented at ASH that showed further benefit of hydroxyurea and one demonstrating pre-operative transfusions for pediatric patients with sickle cell disease.
Sickle cell disease causes progressive organ damage beginning in childhood. Prevention of organ damage is critical to managing this condition throughout life, and interventions must be safe for very young children over years of follow-up. Follow-up of patients in the NHLBI's Pediatric Hydroxyurea Phase III Clinical Trial (Baby HUG) is providing evidence that hydroxyurea is effective at preventing some of the complications of sickle cell disease. Another report from Baby HUG indicates that DNA repair seems to occur as well in children receiving hydroxyurea as in those who are not, which is very encouraging for long-term safety.
A study from the United Kingdom addresses another significant clinical question: whether pre-operative blood transfusions can prevent post-operative complications when patients with sickle cell disease have abdominal and airway surgery. The study was terminated early because patients who received transfusions had only about one-third as many serious complications as those who did not receive transfusions. While further work is needed to define the optimal use of transfusions, this is valuable information for patients and clinicians. Studies like these continue to build necessary evidence to improve the lives of patients with sickle cell disease.
NHLBI investigator Dr. George Daley gave an outstanding lecture in honor of E. Donnell Thomas, "Hematopoietic, Embryonic, and Induced Pluripotent Stem Cells: Diseases, Myths, and Medicine." He showed how early work on the molecular biology of chronic myelogenous leukemia (CML) led to his work on hematopoietic stem cells and subsequently to work on embryonic and induced pluripotent stem cells. Dr. Daley’s tour de force painted a vivid picture of the phenomenal contributions of many scientists and laid the groundwork for the Ernest Beutler lectures given by Dr. Janet Rowley, the mother of cytogenetics in leukemia, and Dr. Brian Druker, developer of tyrosine kinase inhibitors for leukemia.
Many other NHLBI investigators presented excellent work at ASH, demonstrating what an exciting time this is for hematology research.
Related NHLBI press releases:
Related NHLBI Health Topics: