New pain-free cure for sickle-cell anemia

A large team of Illinois researchers has announced the successful development of an original process of stem cell transplantation to treat sickle-cell anemia using healthy tissue from siblings.

According to a report in the journal Biology of Blood & Marrow Transplantation, the team was successfully able to cure twelve individuals with the condition, or 92 percent of the sickle-cell patients they treated.

Doctors have recognized for quite a while that bone marrow transplantation coming from a donor can cure sickle cell disease. However, few adults are treated because high-dose chemotherapy must be used to get rid of the patients’ own blood-forming cells to stop rejection of the transplanted cells. This also neutralizes patients' immune system leaving them exposed to infection.

Study author Damiano Rondelli, director of the blood and marrow transplant program at the University of Illinois Hospital & Health Sciences System, noted that the conventional treatment regimen also puts patients through quite an ordeal.

Living a higher-quality life

"Adults with sickle cell disease are now living on average until about age 50 with blood transfusions and drugs to help with pain crises, but their quality of life can be very low," Rondelli said in a news release. "Now, with this chemotherapy-free transplant, we are curing adults with sickle cell disease, and we see that their quality of life improves vastly within just one month of the transplant.”

“They are able to go back to school, go back to work, and can experience life without pain,” he added.

Sickle cell disease, a genetic condition, mainly affects people of African ancestry, including approximately 1 in every 500 African Americans born in the US. The condition causes red blood cells to be crescent-formed, like a sickle. These cells supply less oxygen to the body’s tissues than healthy cells, causing considerable pain and ultimately strokes or organ damage.

In the new treatment, patients are given immunosuppressive drugs prior to the transplant, as well as a very low dose of overall body irradiation, a regimen much less unpleasant and with fewer potentially severe side effects than chemotherapy. Next, stem cells from a healthy and tissue-matched sibling are transfused into the patient. Stem cells from the donor generate normal new blood cells in the patient, gradually in adequate quantity to get rid of symptoms. Oftentimes, sickle cells cannot be detected. Individuals must still take immunosuppressant drugs for a minimum of a year.

In 13 volunteers, the transplanted cells effectively established in the marrow and generated good red blood cells. One patient who didn't follow the post-transplant therapy program relapsed to the original sickle cell condition.

"Adults with sickle cell disease can be cured without chemotherapy -- the main barrier that has stood in the way for them for so long," Rondelli said. "Our data provide more support that this therapy is safe and effective and prevents patients from living shortened lives, condemned to pain and progressive complications."

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by Brett Smith
Source: http://www.redorbit.com