Treating Sickle Cell At The Source

More and more, pharmaceutical and medical researchers are finding ways to treat sickle-cell anemia. A genetic condition affecting 90,000 Americans, sickle-cell anemia is found primarily in people of African descent. The red blood cells of people with sickle-cell anemia are rigid and misshapen, impeding circulation. In addition, the red blood cells are fragile, needing to be replenished after less than three weeks, as opposed to the usual lifespan of up to four months. Due to these factors, sickle-cell patients are prone to infection, and experience pain in the chest and joints.

Singer T-Boz was diagnosed with sickle-cell anemia as a child.

Singer T-Boz was diagnosed with sickle-cell anemia as a child.

Because sickle-cell is caused by a genetic problem with the red blood cells, it is a lifelong disease, with effects starting in infancy, and causing slowed growth and delayed puberty as well as symptoms in adulthood. It has been considered incurable, but a woman in Chicago who was treated with a stem cell transplant showed no symptoms of the disease—and her red blood cells were found to be normal after the transplant, meaning the procedure completely eliminated the patient’s disease. The woman is one of the first people to be successfully cured by this technique, which requires a healthy donor who is genetically a sibling of the patient. The stem cells gradually take over the task of making red blood cells from the patient’s own cells, and makes normal red blood cells.

For patients who are not eligible for the stem cell procedure, there are other options. Some researchers are exploring the possibility of gene therapy that repairs the red-blood-cell-making mechanism in the patient’s own cells. Rather than relying on donor bone marrow to manufacture healthy cells, the flaw that causes the patient to produce sickled cells is repaired. This technique is only in preliminary stages, but clinical trials are expected to begin in 2014.

Already in trials are two medications that may help alleviate the symptoms of sickle-cell disease. The drug regadenoson is currently used to diagnose heart disease. It has anti-inflammatory properties, and researchers are hoping that it is can improve blood flow in sickle-cell patients. The other medication being tested, referred to as SelG1, prevents blood cells from sticking together and keeps blood vessels from becoming blocked.

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