The Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and Emory University Awarded Almost $10 Million for Critical Blood Disorder Research
Grant to Develop Treatment for Lethal Complication of Sickle Cell Disease
(ATLANTA, October 22, 2013) The Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and Emory University have received a grant of almost $10 million to target lethal lung damage that causes the many deaths in children with sickle cell disease.
Sickle Cell Disease is the most common single-gene disorder in the nation, affecting about 100,000 Americans. In addition, millions of people worldwide have sickle cell disease, making it a significant global health problem.
The grant, awarded by the National Heart Lung and Blood Institute, was given to Emory University and the Aflac Cancer Center of Children's. It will provide about $2 million in funding each year over five years, fostering bench-to-bedside research to find treatment that could stem a complication of sickle cell disease called “acute chest syndrome.” Acute chest syndrome damages the lungs, causing them to fill with fluid and sometimes resulting in respiratory failure.
“If we can prevent children from having acute chest syndrome, we can reduce a lot of the misery that they experience – and a real threat to their very existence,” said Dr. Clinton H. Joiner, director of Hematology in the Aflac Cancer Center and professor of pediatrics with Emory University School of Medicine. “We would make important progress in stopping serious illness, suffering and death. We do not have a silver bullet yet, but this would be significant progress.”
Specifically, the grant calls for Emory and the Aflac Cancer Center to accomplish two key goals:
- Find a drug or a biological agent that would protect, stop or stem lung damage in sickle cell patients.
- Develop and encourage researchers to find innovative therapies for sickle cell and its complications.
The Aflac Cancer Center and Emory in Atlanta were a frontrunner for the grant because it already treats the largest population of sickle cell patients in the nation and is one of the few institutions focused on combating acute chest syndrome. But Emory won the grant because of its compelling research that focused on a receptor called the Toll-like receptor #4 or TLR-4. This receptor is tied to the often-deadly process that occurs in sickle cell patients, in which heme, released from hemoglobin when sickle red blood cells are fragmented in the circulation, triggers a toxic inflammatory response by TLR-4 that damages the lungs. This creates a vicious cycle of more heme being released, and more lung damage occurring, sometimes until the patient dies.
The grant has a two-pronged objective: to prove that the TLR-4 receptor is indeed the key to acute chest syndrome; and to find a drug – preferably one already developed for other purposes – or a biological agent that could block the TLR-4 from performing its ultimately deadly task.
In earlier work, the team found that mutant mice that had no TLR-4 receptors did not develop acute lung injuries when challenged by heme – even after receiving blood transplants from mice with TLR-4. This indicates that the mice were protected – and that the TLR-4 on endothelial cells that line the blood vessels is critical in mediating the response to heme. “The study was elegant in its proof,” Joiner said.
In addition, the grant will also foster research to detect genetic variation in patients that predispose them to the development of acute chest syndrome.
“Sickle cell is quite variable from one person to the next,” Joiner said. “With some kids, sickle cell is a chronic illness – but they’re less affected. And other kids – they are really sick – and they hurt every day of their lives – and we want to find out why there’s such a big difference and come up with some customized treatments.”
About Children’s Healthcare of Atlanta
Children’s Healthcare of Atlanta has been 100 percent dedicated to kids for more than 100 years. A not-for-profit organization, Children’s is dedicated to making kids better today and healthier tomorrow. Our specialized care helps children get better faster and live healthier lives. Managing more than 870,000 patient visits annually at three hospitals and 27 neighborhood locations, Children’s is the largest healthcare provider for children in Georgia and one of the largest pediatric clinical care providers in the country. Children’s offers access to more than 60 pediatric specialties and programs and is ranked among the top children’s hospitals in the country by U.S. News & World Report. With generous philanthropic and volunteer support since 1915, Children’s has impacted the lives of children in Georgia, the United States and throughout the world. Visit www.choa.org for more information.
Aflac Cancer and Blood Disorders Center of Children’s
The Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta is a national leader among childhood cancer, hematology, and blood and marrow transplant programs, serving children and young adults. Recognized as one of the top 10 childhood cancer centers in the country by U.S. News & World Report, the Aflac Cancer Center cares for more than 400 newly diagnosed cancer patients each year and follows more than 2,500 patients with sickle cell disease, hemophilia and other blood disorders. The Center has a well-developed clinical research office and a robust Phase I/II clinical trials infrastructure. Visit aflaccancercenter.org or call 404-785-1112 or 888-785-1112 for more information.
Tags: General News
, Sickle Cell Disease
, Cancer and Blood Disorders
Published: Tuesday, October 22, 2013