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Pitt, UPMC receive grant to treat sickle cell disease

Sickle cell disease is the result of one mutation in one gene. This slight deviation in the biological course has caused incredible pain for the people whom it afflicts, mostly people of African descent.

The mutation causes red blood cells to have an increased viscosity and an abnormal sickle- like shape. These factors make sickled cells more likely to clog the body’s blood vessels.

The disease, which affects approximately 100,000 Americans and 4.4 million worldwide, causes excruciating pain, strokes and often death. The only current remedy for sickle cell disease is a bone marrow transplant, an option that is rarely used due to its expense and danger.

But the University of Pittsburgh and UPMC are exploring a new option known as red cell exchange. Far from simple, the process replaces 70% of a patient’s blood with donor blood. But while the procedure has been around since the 1980s, it has never been subjected to rigorous research or trials in the treatment of sickle cell disease.

That’s what Pitt and UPMC are hoping to do with a new $ 19.2 million grant from the National Institutes of Health. The Pittsburgh researchers will led a seven- year clinical trial involving 150 patients at 22 medical sitesin the U. S., United Kingdom and France. Some patients will receive conventional methods of care while others will receive the red cell exchange. Dr. Mark Gladwin, Pitt Department of Medicine chair, told the Post- Gazette’s Kaisha Jantsch that he expects the red cell exchange to produce positive results, including improved health and organ function.

One patient, 60- year- old Gale Langhorn- Cox, has had her sickle cell disease treated with red cell exchange since 2013. She says the procedure has made her feel better and more energized.

There are concerns about red cell exchange, such as the potential for blood clots and infection. The procedure also requires a lot of blood, which, in turn, requires a lot of time and money.

But the potential to more effectively treat those with sickle cell disease is a worthy expenditure of time and money for Pitt, UPMC and NIH. Due to the disease’s concentration in minority communities, treatment for sickle cell disease has not often received adequate funding or attention.

Being at the forefront of treating such a pernicious and deadly disease will not only boost the reputations of the institutions involved, but will help heal those who have so often been overlooked.