WASHINGTON – Children’s National Health System will get greater than $550,000 in funding to guide a 3-year, multi-center trial which will practice a low intensity, chemotherapy-free transplantation method of cure patients with sickle cell disease utilizing a matched related donor. The grant was awarded with the Doris Duke Charitable Foundation’s inaugural Sickle Cell Disease/Evolving Cures Awards, which supplies funding to succeed curative methods for sickle cell disease.
The Children’s National project, brought by Allistair Abraham, M.D., bloodstream and marrow transplantation specialist, and Robert Nickel, M.D., hematologist, is among seven projects receiving roughly $six million total with the awards. The work won support because of its possibility to considerably enhance the only current proven remedy for sickle cell disease-hematopoietic cell transplantation. While transplantation utilizing a matched brother or sister donor today includes a high cure rate (more than 90 %) for sickle cell disease, traditional transplant approaches have numerous risks and negative effects both in rapid and lengthy term.
The research will examine if your chemotherapy-free approach can result in a effective transplant without leading to graft-versus-host disease (GVHD). GVHD is among the most difficult complications of the transplant, where the transplant immune cells attack the patient’s body. They anticipate this new transplant approach is going to be very well tolerated that patients’ quality of existence is going to be maintained and improved through the process, with the majority of the care administered inside a clinic setting.
“This approach has shown to be effective for adults with sickle cell disease, therefore we are grateful for that chance to do this important trial for kids because of the Doris Duke Charitable Foundation,” states Dr. Abraham, who together with Dr. Nickel also can serve as assistant professor of pediatrics in the George Washington College Med school and Health Sciences. “Children with sickle cell disease require innovative treatments, so we expect to locating more solutions that improve the caliber of existence of these patients.”
“Advancing strategy to sickle cell patients enough where they are able to live free from the condition is our main concern,Inches states Dr. Nickel. “This funding is crucial to the study and it’ll accelerate the timeline to offer the objective of a properly-tolerated and safe remedy for kids with sickle cell disease.”
Matthew Hsieh, M.D., who helped pioneer the work in the National Institute of Health in grown-ups, and Greg Guilcher, M.D., that has used this transplant approach in youngsters, are key collaborators around the project.
The research is forecasted to start in December 2018 and go on for 3 years. The Excellent Sickle Cell Disease Program at Children’s National is probably the largest in the united states, treating greater than 1,400 children and youthful adults with lots of different sickle cell disease. Children’s National also provides the biggest, very indepth bloodstream disorders team within the Washington, D.C., area.
Contact: Rebecca Porterfield Siddon [email protected] 301-244-6737
About Children’s National Health System
Children’s National Health System, located in Washington, D.C., continues to be serving the nation’s children since 1870. Children’s National is #1 for babies and rated in each and every niche evaluated by U.S. News & World Report including placement within the top ten for: Cancer (#7), Neurology and Neurosurgery (#9), Orthopedics (#9) and Nephrology (#10). Children’s National continues to be designated two occasions like a Magnet® hospital, a designation provided to hospitals that report the greatest standards of nursing and patient care delivery. This pediatric academic health system offers expert care via a convenient, community-based primary care network and niche outpatient centers. The place to find the Children’s Research Institute and also the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is among the nation’s top NIH-funded pediatric institutions. Children’s National is acknowledged for its expertise and innovation in pediatric care so that as a powerful voice for kids through advocacy in the local, regional and national levels. To learn more, visit ChildrensNational.org, or follow us on Facebook and Twitter.
Concerning the Doris Duke Charitable Foundation
The mission from the Doris Duke Charitable Foundation would be to improve the caliber of people’s lives through grants supporting the performing arts, ecological conservation, child well-being and scientific research, and thru upkeep from the cultural and ecological legacy of Doris Duke’s qualities. The foundation’s Scientific Research Program supports clinical research that advances the translation of biomedical breakthroughs into new preventions, diagnoses and coverings for human illnesses. To understand more about this program, visit world wide web.ddcf.org.