Researchers led by Matthew Hsieh of the National Institute of Diabetes and Digestive and Kidney Diseases and the National Heart, Lung, and Blood Institute have reversed sickle cell disease in adults by utilizing stem cells from the patients’ siblings. The patients received a transplant that combined their own stem cells with those of a sibling, resulting in an increase in lung function and a decrease in the patients’ sickle cell count, as well as their dependency on immunosuppressant and pain relieving drugs.
Gene therapies utilizing stem cells are being developed that may lead to a cure for several genetic diseases. Currently, two clinical trials were announced to treat Cerebral Adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease.
Gamida Cell, a leading adult stem cell therapeutics company has completed enrollment for a Phase I/II trial for hematological malignancies utilizing their NiCord treatment. The treatment is a potential cure for blood cancers, sickle cell disease, thalassemia, severe autoimmune diseases and metabolic diseases. This trial is but one example of the increasing number of stem cell treatments moving out of the lab and entering the clinical testing phase.