New researchers boost UPMC's sickle cell fight


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UPMC, in a push to achieve advances in sickle cell research and treatment, has recruited three national leaders in the disease, the medical center announced this week.

"It is pretty unprecedented to have this many people focused on sickle cell come together in one place, so it is very exciting," said Mark Gladwin, director of the Pittsburgh Heart, Lung, Blood and Vascular Medicine Institute.

Solomon Ofori-Acquah from Emory University in Atlanta and Gregory Kato from the National Heart, Lung and Blood Institute at the National Institutes of Health are already here. Laura DeCastro will come to Pittsburgh from Duke University in January.

The will help UPMC in "creating a critical mass" to make progress against the disease, Dr. Gladwin said.

The new hires, together with existing researchers and doctors at UPMC, represent an "impressive scope of experience," said Dr. Kato, that extends "from bench to bedside."

"This is a real moment of renaissance for sickle cell research, and it's an opportunity for UPMC to try to take the lead in national sickle cell research," he said.

The announcement comes about a year after UPMC joined with the Ryan Clark Cure League, founded by the Steelers safety who carries the sickle cell trait, to raise awareness of the disorder.

Sickle cell disease, a genetic disorder that affects about 100,000 Americans, causes normally round red blood cells to become hard, sticky and sickle-shaped, which can block the flow of blood throughout the body, causing pain and various severe complications. There is just one FDA-approved drug for sickle cell treatment and the sole cure, through a bone marrow transplant, works for only a small minority of patients.

It is most common among people with ancestors from sub-Saharan Africa, Spanish-speaking regions in the Western Hemisphere, Saudi Arabia, India and Mediterranean countries. One out of every 500 African-Americans carries the trait, and in the Pittsburgh region, about 300 families are affected by the disease, said Andrea Williams of Beltzhoover, executive director of the Children's Sickle Cell Foundation and mother of Jonathan, a 13-year-old with sickle cell disease.

Mr. Ofori-Acquah, originally from Ghana, where the disease is far more prevelant than in the United States, will develop partnerships between UPMC and research sites in Africa. That will increase the number of patients enrolled in clinical trials, which can speed up the development of potential treatments. Much of Mr. Ofori-Acquah's research is focused on acute chest syndrome, a complication of sickle cell disease, with the goal of developing a treatment to manage the lung ailment.

Dr. Kato, who previously worked with Dr. Gladwin at NIH, has focused his research on identifying biomarkers that can help doctors detect what complications sickle cell carriers are likely to develop, which can allow for more personalized medical care.

Dr. DeCastro, according to the UPMC news release, has focused her research on developing treatments for sickle cell disease and exploring its impact on organ damage. She's also developed and coordinated clinical care for adult sickle cell patients and at UPMC will help direct the adult sickle cell clinical programs.

Ms. Williams, whose son's sickle cell disease meant frequent hospitalizations when he was younger, believes UPMC's increased focus will mean "we're going to see great things happen."

"As a parent, it means the world to me to have a number of researchers working on this," she said.

Kaitlynn Riely: kriely@post-gazette.com or 412-263-1707.

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This story originally appeared in The Pittsburgh Press. To log in or subscribe, go to: http://press.post-gazette.com/ First Published September 20, 2013 7:45 PM


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