Patients’ cells provide possible treatment for blood disorder
Researchers at Boston Children’s Hospital’s Stem Cell Research Program were able, for the first time, to use patients’ own cells to create cells similar to those in bone marrow, and then use them to identify potential treatments for a blood disorder. The work was published today by Science Translational Medicine. The team derived the so-called blood progenitor cells from two patients with Diamond-Blackfan anemia (DBA), a rare, severe blood disorder in which the bone marrow cannot make enough oxygen-carrying red blood cells. The researchers first converted some of the patients’ skin cells into induced pluripotent stem (iPS) cells. They then got the iPS cells to make blood progenitor cells, which they loaded into a high-throughput drug screening system. Testing a library of 1,440 chemicals, the team found several that showed promise in a dish. One compound, SMER28, was able to get live mice and zebrafish to start churning out red blood...