Stem cell advance may help treat rare blood disorder

In an important advance in the stem cell field, researchers are reporting that they were able to use a patients’ own cells to create cells similar to those in the bone marrow and then use them to identify potential treatments for a rare blood disorder. The researchers obtained the so-called blood progenitor cells from the skin of two patients with Diamond-Blackfan anemia (DBA), a rare blood disorder in which the bone marrow cannot make enough oxygen-carrying red blood cells. They converted the skin cells into blood progenitor cells, which they then processed through a drug screening system.  The researchers identified one chemical in particular, called SMER28, during this screening process that showed promise in animal studies for producing red blood cells.  The finding could lead to new treatments for DBA, they say. The study, which appeared in Science Translational Medicine, was partly funded by NHLBI.