A proven method for curing sickle cell disease in adults is now poised to become available for children in the not-too-distant future thanks to investigators from Children’s National and a recent $550,000 grant by the Doris Duke Charitable Foundation.
Catalyzed through the foundation’s inaugural Sickle Cell Disease/Advancing Cures awards, which awarded about $6M to seven projects total this year, the multi-center research trial seeks to investigate the use of a chemotherapy-free bone marrow transplantation treatment approach in children living with the severe pain, risk of stroke, and other health issues related to this serious genetic disorder.
Led by Blood and Marrow Transplantation Specialist Allistair Abraham, MD, and Hematologist Robert Nickel, MD, the project won support for its potential to significantly improve the only current proven cure for sickle cell disease. With this generous grant from the Doris Duke Charitable Foundation, Dr. Abraham and Dr. Nickel are hoping to help children avoid the damaging effects of chemotherapy on their growing bodies, while still achieving a cure.
“This approach has proven to be effective for adults, so we are grateful for the opportunity to begin this important trial for children,” says Dr. Abraham. “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 will accelerate the timeline to the goal of a well-tolerated and safe cure for children with sickle cell disease.”
The three-year study is projected to begin in Dec. 2017 with the close collaboration of international experts, including the researcher who first helped pioneer the chemo-free approach in adults at the National Institutes of Health. The research is an important component of The Comprehensive Sickle Cell Disease Program at Children’s National. Among the largest in the country, the program treats more than 1,400 children and young adults with all types of sickle cell disease.
“We were excited to launch the Sickle Cell Disease/Advancing Cures grants competition this year for the very purpose of identifying projects with high potential for curing sickle cell disease. The Children’s National project led by Dr. Abraham and Dr. Nickel is a standout example of such a project and well-deserving of the award,” said Betsy Myers, program director for medical research at the Doris Duke Charitable Foundation. “The study’s ambitions are high—to heal children afflicted with the disease by developing an approach that adds significantly fewer side effects than current approaches—and we expect it will advance the field of sickle cell disease research further in even more unexpected and essential ways.”
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.
“We are very thankful for the Doris Duke Charitable Foundation for funding our study and making sickle cell disease a top priority,” says Dr. Nickel. “The outlook for children born with sickle cell disease today is much brighter thanks to advances from research funded by the Doris Duke Charitable Foundation and other philanthropic organizations.”
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