WASHINGTON - Children's National Health System will receive more than $550,000 in funding to lead a three-year, multi-center trial that will study a low intensity, chemotherapy-free transplantation approach to cure patients with sickle cell disease using a matched related donor. The grant was awarded through the Doris Duke Charitable Foundation's inaugural Sickle Cell Disease/Advancing Cures Awards, which provides funding to advance curative approaches for sickle cell disease.
The Children's National project, led by Allistair Abraham, M.D., blood and marrow transplantation specialist, and Robert Nickel, M.D., hematologist, is one of seven projects receiving approximately $6 million total through the awards. The project won support for its potential to significantly improve the only current proven cure for sickle cell disease- hematopoietic cell transplantation. While transplantation using a matched sibling donor today has a high cure rate (greater than 90 percent) for sickle cell disease, traditional transplant approaches have many risks and side effects in both the short and long term.
The study will examine if a chemotherapy-free approach can lead to a successful transplant without resulting in graft-versus-host disease (GVHD). GVHD is one of the most challenging complications of a transplant, in which the transplant immune cells attack the patient's body. The researchers anticipate that this new transplant approach will be so well tolerated that patients' quality of life will be maintained and improved throughout the process, with most of the care administered in a clinic setting.
"This approach has proven to be effective for adults with sickle cell disease, so we are grateful for the opportunity to begin this important trial for children thanks to the Doris Duke Charitable Foundation," says Dr. Abraham, who along with Dr. Nickel also serves as assistant professor of pediatrics at the George Washington University School of Medicine and Health Sciences. "Children with sickle cell disease are in need of innovative treatments, and we look forward to finding more solutions that improve the quality of life for these patients."
"Advancing treatment for sickle cell patients to the point where they can live free of the disease is our top priority," says Dr. Nickel. "This funding is critical to our study and it will accelerate the timeline to achieve the goal of a well-tolerated and safe cure for children with sickle cell disease."
Matthew Hsieh, M.D., who helped pioneer this work at the National Institute of Health in adults, and Greg Guilcher, M.D., who has used this transplant approach in children, are key collaborators on the project.
The study is projected to begin in December 2018 and continue for three years. The Comprehensive Sickle Cell Disease Program at Children's National is among the largest in the country, treating more than 1,400 children and young adults with all types of sickle cell disease. Children's National also offers the largest, most comprehensive blood disorders team in the Washington, D.C., area.
About Children's National Health System
Children's National Health System, based in Washington, D.C., has been serving the nation's children since 1870. Children's National is #1 for babies and ranked in every specialty evaluated by U.S. News & World Report including placement in the top 10 for: Cancer (#7), Neurology and Neurosurgery (#9), Orthopedics (#9) and Nephrology (#10). Children's National has been designated two times as a Magnet® hospital, a designation given to hospitals that demonstrate the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers. Home to the Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is one of the nation's top NIH-funded pediatric institutions. Children's National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.
About the Doris Duke Charitable Foundation
The mission of the Doris Duke Charitable Foundation is to improve the quality of people's lives through grants supporting the performing arts, environmental conservation, child well-being and medical research, and through preservation of the cultural and environmental legacy of Doris Duke's properties. The foundation's Medical Research Program supports clinical research that advances the translation of biomedical discoveries into new preventions, diagnoses and treatments for human diseases. To learn more about the program, visit http://www.ddcf.org.