U.S. scientists published in The New England Journal of Medicine have succeeded in reversing the disease in 9 of 10 patients treated with a new procedure.
The preliminary study results show that the “mini” stem cells could reverse the severe sickle cell anemia in adults, according to a study by the NIH and Johns Hopkins University (USA), which is published today in The New England Journal of Medicine.
The phase I / II describes 10 patients with severe sickle cell disease who received intravenous transplants of hematopoietic stem cells, peripheral blood from healthy unrelated donors, which were adapted to tissue types of patients.
When the scientists used this procedure, 9 of the 10 patients showed normal red blood cells and recovery of organ damage caused by the disease.
Jonathan Powell explains, “the method of intravenous transplantation for sickle cell anemia, caused by a single mutation in the gene for hemoglobin, does not replace the defective gene but transplanted blood stem cells carrying the normal gene.
All patients in the study, from 16 to 45 years of age, were treated at the NIH with a “mini” and an immune suppressant drug, rapamycin.
Conventional transplantation methods using high doses of chemotherapy to suppress the immune system before injecting the transplanted cells, a process that has many side effects, including contracting serious bacterial infections that could kill patients. In the “mini” are used lower doses of medication and radiation to allow the arrival of the donated cells.
As pointed out by Powell, side effects were less and very mild compared with bone marrow transplantation. In 9 of the 10 patients, the donated cells now coexist with the patient.
The “mini” for sickle cell anemia patients were tested in nearly a decade ago and were not successful because the immune system from rejecting the transplanted cells affected. However, Powell indicated that the use of rapamycin promotes the coexistence of the donated cells to the patient.
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