Children’s Healthcare of Atlanta and Emory University Awarded Nearly $10 Million For Sickle Cell Research

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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 many deaths in children with sickle cell disease.

Sickle Cell Disease is the most common single-gene disorder in the nation, Emory reports, 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 they 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, the school said.

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 cycle of more heme being released, and more lung damage occurring, sometimes until the patient dies.

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.”

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