Findings from phase I/II of the clinical trial are published online in the journal Biology of Blood & Marrow Transplantation.
Stem cell transplants have been used for years as a means of possibly curing sickle cell disease. However, before the stem cell transplant could be completed patients would have to endure a taxing course of drugs to kill the cancer cells, otherwise known as chemotherapy.
The more traditional form of stem cell transplant uses chemotherapy to destroy the patient’s own bone marrow, which shuts down their immune system and makes them vulnerable to infections.
The new technique – first developed and performed at the National Institutes of Health campus in Maryland – eliminates the need for chemotherapy to prepare the patient to receive the transplanted cells and offers the prospect of cure for tens of thousands of adults suffering from sickle cell disease – many of them Black Americans.
According to the National Heart, Lung & Blood Institute (NIH), about 1 in 13 African American babies is born with sickle cell trait. About 1 in every 365 black children is born with sickle cell disease.
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.
The chemo-free transplant performed by UI is described in a news release:
In the reported trial, 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. One patient who failed to follow the post-transplant therapy regimen reverted to the original sickle cell condition.
In Thomas’ case, her sister was a match and following the transplantation she no longer required blood transfusions.
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.”
To learn more about ongoing sickle cell transplant trials at NIH (a participant in a trial will not be charged for a procedure) call 1-800-411-1222 or visit the NIH clinical trials registry at www.clinicaltrials.gov and search under ‘sickle cell disease.”
article by Gwendolyn Harris via blackdoctor.org