Chemotherapy-free stem cell transplant shows promise for sickle cell disease

Published on March 6, 2013 at 6:12 AM · No Comments

Two brothers have been cured of their sickle cell disease at the University of Illinois Hospital & Health Sciences System using a relatively uncommon type of stem cell transplant that is performed without chemotherapy.

Their transplants were possible thanks to a third brother who was a match for both, against long odds.

Julius and Desmond Means never imagined life without sickle cell. The brothers, ages 25 and 19, have spent their lives in and out of hospitals, each suffering from different complications of the disease.

Growing up, they tired easily and couldn't keep up with their friends. As they grew older, the disease caused bone damage and affected Julius's lungs. Desmond's organs were also being damaged, and he was jaundiced.

For either young man to receive a transplant, a healthy sibling who is a compatible donor was needed. An acceptable match requires that at least eight of 10 known human leukocyte antigen (H.L.A.) genes be identical between donor and recipient.

Julius and Desmond's healthy older brother Clifford, 27, matched 10 of 10 H.L.A. genes with both of them -- an occurrence of "extremely low" likelihood, according to Dr. Damiano Rondelli, director of stem-cell transplantation at UI Health. The men's mother, Beverly Means, also noted the good fortune.

"I had won the lottery of health," she said.

In preparation for the transplant, Clifford was given medication to increase the number of stems cells released from the bone marrow into the bloodstream. His blood was then processed through a machine that collects white cells, including stem cells. The stem cells were frozen until the transplants.

Last May, Julius received his transplant at UI Hospital. He was given medication to suppress his immune system and one small dose of total body radiation right before the transplant. Then the frozen bags of stem cells were thawed and hung by IV pole for infusion into him.

Then in September, Desmond received his stem cell transplant.

Now several months since the transplants, both Julius and Desmond no longer have sickle cell disease. Their bone marrow is producing healthy red blood cells.

"Being cured, I feel like we can do anything," says Julius, who composed a rap about his transplant while recovering in the hospital. The brothers are pursuing their interests in rap music and dance and plan to attend college.

The chemotherapy-free stem cell transplant is a new procedure and is much better-tolerated by patients with aggressive sickle cell disease, who often have underlying organ damage and other complications.

UI Health is the first center in the Chicago area to offer this treatment, and one other patient has been successfully transplanted here. The efficacy and safety of the pre-transplant medication regimen are currently being studied at UI Health.

About 30 adults have received chemotherapy-free stem cell transplants for sickle cell disease at the National Institutes of Health in Bethesda, Md. Approximately 85 percent have been cured.

Sickle cell affects as many as 100,000 people in the U.S., predominantly of African ancestry. The disease is an inherited defect that causes the red blood cells to become crescent-shaped. These cells can obstruct small blood vessels, depriving the body's tissues of oxygen. The disease can cause extreme pain and damage the lungs, heart, kidneys and liver. Stroke and other complications can be life-threatening.

Source:

University of Illinois Hospital & Health Sciences System

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