JAMA study shows bone marrow transplants can reverse sickle cell disease

Bone marrow transplants can reverse severe sickle cell disease in adults, according to new data published in the Journal of the American Medical Association.

The findings mean that some patients will be able to stop taking immunosuppressants for the rest of their lives. The Phase I and Phase II trial was carried out in 30 patients, aged 16 to 65. The transplant worked in 26 of 30 adults, and 15 of them stopped taking immunosuppressants one year later.

Approximately 100,000 Americans, primarily African Americans, have sickle cell disease, and worldwide, there are an estimated 300 000 affected infants born. A single nucleotide substitution causes the autosomal recessive disease: valine replaces glutamic acid in the sixth position of the beta-globin protein. Despite the discovery of the etiology of sickle cell disease more than 60 years ago, little progress has been achieved in the treatment or cure of this chronic disease.