Stem cell transplant for sickle cell patients
“A possible cure for a debilitating disease, it strikes one in 500 African Americans. Many live day-to-day on heavy painkillers. Now a new take on an old treatment may put an end to their suffering. Dr. Damiano Rondelli, University of Illinois Hospital Hematologist: “It’s a social issue. It’s not just a medical issue. The cost, the family involvement … it’s a really big deal.”
“It is sickle cell anemia, an inherited defect of the red blood cells. More rigid than normal round cells, the crescent – or sickle-shaped cells — get stuck in small vessels, blocked from delivering oxygen to organs, tissues and joints.
“Damage to lungs, kidneys, liver. The major symptom is pain.”Said Dr. Rondelli
Julius Means, sickle cell patient: “Some of my first memories having sickle cell, playing one day and the next day not being able to even walk.”
Diagnosed at eight-months-old, Julius has struggled with sickle cell disease for 25 years.
Julius Means: “In and out of hospitals all the time. The majority of the pain would hit me in my legs and my back. So being a young kid and not being able to play with the other kids was pretty awful.”
Beverly Means, mother: “It was mental torture. It was hard. Devastating to watch your kids and you can do nothing.”
Even doctors struggle — they have little to offer. Narcotic pain killers and blood transfusions help but offer only temporary relief. So when an NIH stem cell transplant study showed promise, University of Illinois Hospital’s Dr. Damiano Rondelli took notice. “They reported they could do transplant in sickle cell patients without using chemotherapy.”
In a stem cell transplant, doctors infuse healthy donor cells that gradually take over the recipient’s bone marrow to produce normal red blood cells. Traditionally, the process starts with high doses of chemotherapy to kill off a patient’s own cells and prep the body to accept the new ones – a practice deemed too dangerous for those with sickle cell who already have organ damage. But the NIH study used more tolerable and less toxic immunosuppressing drugs in place of chemo.
Dr. Rondelli: “The results were amazing. Because of our large patient population of sickle cell, we opened the trial here.”
That’s when the Means brothers jumped in. But they would need a highly compatible donor – a sibling. They turned to their big brother, Clifford. Doctors quickly tested his blood.
Beverly Means: “He was like, ‘You’re not going to believe it! He’s not only a match for Desmond but for Julius also.’ Ten for 10 perfect match for both brothers.”
Julius Means: “Wow, all this time we had the cure!”
Dr. Rondelli: “The cells in the blood, the red cells are completely from the donor. The sickle cell is gone.”
Sickle Cell Anemia Disease Description
“Sickle cell anemia is an inherited blood disorder characterized primarily by chronic anemia and periodic episodes of pain. The underlying problem involves hemoglobin, a component of red blood cells. Hemoglobin molecules in each red blood cell carry oxygen from the lungs to body organs and tissues and bring carbon dioxide back to the lungs. In sickle cell anemia, the hemoglobin is defective. After hemoglobin molecules give up their oxygen, some may cluster together and form long, rod-like structures. These structures cause red blood cells to become stiff and assume a sickle shape. Unlike normal red cells, which are usually smooth and donut-shaped, sickled red cells cannot squeeze through small blood vessels. Instead, they stack up and cause blockages that deprive organs and tissues of oxygen-carrying blood. This process produces periodic episodes of pain and ultimately can damage tissues and vital organs and lead to other serious medical problems. Normal red blood cells live about 120 days in the bloodstream, but sickled red cells die after about 10 to 20 days. Because they cannot be replaced fast enough, the blood is chronically short of red blood cells, a condition called anemia.”
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