Twelve adult patients have been declared cured of sickle cell disease as part of a clinical trial at the University of Illinois Hospital & Health Sciences System (UI Health). The trial used a unique procedure for stem cell transplantation from healthy tissue matched from a sibling donor.
Stem cell transplants have been used for years as a means of possibly curing sickle cell disease. However, patients would first have to endure a taxing course of drugs to kill the cancer cells, otherwise known as chemotherapy. The new technique – first developed and performed at the National Institutes of Health – eliminates the need for chemotherapy to prepare the patient to receive the transplanted cells.
This offers the prospect of cure for tens of thousands of adults suffering from sickle cell disease – many of them African Americans. According to the National Heart, Lung & Blood Institute (NIH), about 1 in 13 African Americans is born with sickle cell trait; about 1 in every 365 of them is born with sickle cell 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. “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.”
The researchers transplanted 13 patients, 17 to 40 years of age, with a stem cell preparation from the blood of a tissue-matched sibling. Healthy sibling donor-candidates and patients were tested for human leukocyte antigen, a set of markers found on cells in the body.
Ten of these HLA markers must match between the donor and the recipient for the transplant to have the best chance of evading rejection. In a further advance of the NIH procedure, physicians at UI Health successfully transplanted two patients with cells from siblings who matched for HLA but had a different blood type.
In all 13 patients, the transplanted cells successfully took up residence in the marrow and produced healthy red blood cells.
Further research on this type of stem cell transplant is needed, but doctors are hopeful for what early trials show for adults.
“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.”