An Adult Stem Cell Makeover for Sickle Cell Anemia (Part 2)

Innovative advances in adult stem cells promise to revolutionize the treatment of numerous diseases. In this second post of a two-part series, I focus on the novel treatment of a patient with sickle cell anemia.

Since birth, Ijeuru Uzoma, 48, a Nigerian-born registered nurse in Bethesda, Maryland, had been plagued by the periodic “crises” of pain, anemia and fatigue that characterize sickle cell anemia, a genetic, blood-related disease particularly prevalent in people of African descent.

With sickle cell disease, a person’s red blood cells are abnormally shaped—like a “C” or a sickle instead of the normal disc shape—and as a result don’t move very easily in the blood vessels. That can block blood flow and lead to pain, infection and organ damage. In fact years of the disease had begun to wreak havoc on Uzoma’s kidneys. She was resigned to a future on dialysis. But during a particularly painful crisis in 2007, Dr. Oswald Castro (director of the Howard University Center for Sickle Cell Disease) told Uzoma about a study at the National Institutes of Health, using stem cells retrieved from the bone marrow of a closely matched relative.

Although transplants are standard for treating children with sickle cell disease, they haven’t succeeded so well in older patients, because their organs have been so damaged by the disease that the procedure is too risky. In the preliminary NIH study, doctors tweaked the regimen by using a less toxic dose of radiation and chemo to only partially destroy patients’ bone marrow. Then they infused patients with the stem cells. Uzoma got hers from her healthy older sister.

“With this study we were able to get bone marrow cells from a donor to start producing healthy blood in patients at levels high enough to make the sickle cell disease go away,” says John Tisdale, M.D., the principal investigator at the NIH’s Molecular and Clinical Hematology Branch. “It’s not completely gone, but it may be enough to keep them healthy.” In fact, the team was able to “cure” sickle cell disease in nine out of 10 patients in the study.

“Before the transplant, I was like a walking time bomb,” says Uzoma. “I didn’t know when I would be sick, when the pain would start. I was living in fear. Now I’ve been pain-free for two years and four months.”

Since she has some of her sister’s cells in her body, Uzoma must take immunosuppressant drugs like patients who undergo organ transplants, so that her body doesn’t reject the cells as “foreign.” But regular check-ups since her transplant show her blood is normal and, for the first time, Uzoma can enjoy visiting relatives in Chicago without the dread of having her pain return.

CONNECT THE DOTS

To read the first of this two-part series, and learn how a new stem cell treatment is helping Molly Foley battle the affects of scleroderma, visit here.

To learn more about sickle cell anemia, visit the American Sickle Cell Anemia Association or the National Institutes of Health’s U.S. National Library of Medicine.

Read more about exciting adult stem cell treatments at our blog post on turning adult wisdom teeth into stem cells, and another on reattaching teeth that have been knocked out with the aid of adult stem cells.

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