Illustration of red blood cells affected by sickle cell anaemia. Sickle cell anaemia is an inherited blood disease in which the red blood cells contain an abnormal form of haemoglobin (blood's oxygen-carrying pigment) that causes the blood cells to become sickle-shaped, rather than round. Sickle cells cannot move through small blood vessels as easily as normal cells and so can cause blockages. This prevents oxygen from reaching the tissues, causing severe pain and organ damage. Image Credit: Nemes Laszlo/Science Photo Library via Getty Images
Health USANew – and Cheaper – Treatment Against Sickle Cell Disease
Two independent papers praised the new bone marrow transplant method for curing sickle cell disease, which is more accessible and less costly than recently approved gene therapies.
“A common misconception in the medical field is that transplantation for sickle cell disease requires a perfect matched donor… which this trial and other studies have shown aren’t true,” explains Dr. Robert Brodsky of The Johns Hopkins University School of Medicine.
Sickle cell disease, a genetic blood disorder, changes the shape and function of red blood cells, causing severe pain, organ damage, reduced quality of life, and increased mortality. It has been reported that where gene therapies for sickle cell disease cost between $2 and $3 million, the bone marrow transplant method costs less than $500,000. As for hospitalization, bone marrow transplants require eight days on average instead of six to eight weeks for gene therapy. Another paper analyzed data from 42 young severe sickle cell disease patients who underwent the “reduced-intensity haploidentical bone marrow transplant”. Two years after the procedure, 95% were alive, and 88% were cured, not experiencing disease-related events. Finding an appropriate donor is much easier since the new method doesn’t require marrow from a specifically matched donor to avoid triggering an immune response. “These results are every bit as good or better than what you see with gene therapy,” says Dr. Richard Jones, also of Johns Hopkins.
