In what’s being hailed as a virtual miracle, University of Illinois Hospital & Health Sciences System physicians have replicated a treatment pioneered at the National Institute of Health and have been able to cure 12 patients of sickle cell disease.
The process involves transplanting stem cells from a healthy, tissue-matched sibling, and it completely eliminates the use of chemotherapy in the treatment of the disease. An excellent and detailed article on the U of I website explores the ground-breaking treatment:
About 90 percent of the approximately 450 patients who have received stem cell transplants for sickle cell disease have been children. Chemotherapy has been considered too risky for adult patients, who are often more weakened than children by the disease.
“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,” says Dr. Damiano Rondelli, chief of hematology/oncology and director of the blood and marrow transplant program at UI Health, and corresponding author on the paper.
“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,” said Rondelli, who is also the Michael Reese Professor of Hematology in the UIC College of Medicine. “They are able to go back to school, go back to work, and can experience life without pain.”
Sickle cell disease is inherited. It primarily affects people of African descent, including about one in every 500 African Americans born in the U.S. The defect causes the oxygen-carrying red blood cells to be crescent shaped, like a sickle. The misshapen cells deliver less oxygen to the body’s tissues, causing severe pain and eventually stroke or organ damage.
Doctors have known for some time that bone marrow transplantation from a healthy donor can cure sickle cell disease. But few adults were transplanted because high-dose chemotherapy was needed to kill off the patients’ own blood-forming cells — and their entire immune system, to prevent rejection of the transplanted cells, leaving patients open to infection.
In the new procedure, patients receive immunosuppressive drugs just before the transplant, along with a very low dose of total body irradiation — a treatment much less harsh and with fewer potentially serious side effects than chemotherapy. Next, donor cells from a healthy and tissue-matched sibling are transfused into the patient. Stem cells from the donor produce healthy new blood cells in the patient, eventually in sufficient quantity to eliminate symptoms. In many cases, sickle cells can no longer be detected. Patients must continue to take immunosuppressant drugs for at least a year.
Replication of results is an important part of the scientific method, and it’s always great to see replication that makes such a difference in peoples’ lives.
A great video included in the UI article chronicles the journey to a cure for one family:
Learn more from the excellent and somewhat jaw-dropping article on the UI website.
Source: News.UIC.edu – “UI Health validates cure for sickle cell in adults“