Hopeful new research for sickle cell disease

Researchers may have found a powerful new treatment for sickle cell disease. So far, the treatments have proven effective in mice.

LOS ANGELES, CA (Catholic Online) - Sickle cell disease is a debilitating condition which mostly affects African American, Hispanic, Mediterranean, and Middle Eastern men. The disease causes blood cells to form in a "sickle" shape that makes it difficult for them to carry oxygen throughout the body. The victim suffering from this condition can experience painful symptoms which include strokes, organ dysfunction, and even death.

Researchers have long known that sickle cell disease only develops after birth. Prior to birth, a baby uses a specific gene to make blood cells. After birth, a different gene is used. The new gene is triggered by a protein called BCL 11A. Researchers have found that by blocking the protein that causes the switch, they can get the body to start producing normal cells again.

The latest study, published in the journal Science, observed the blocking of production of BCL 11A protein in mice. Those mice with sickle cell disease showed prompt improvement as their bodies began to produce normal cells instead of the misshapen ones.

The discovery offers new hope for treatment of this debilitating condition in patients. Presently, the only functional treatment for the disease is a bone marrow transplant. Such transplants are risky, expensive, and are not assured of success. They also require a volunteer family member to have compatible bone marrow.

Scientists say that it will be several years before they are ready to start human trials, however the latest results look very promising and doctors intend to shortly follow-up this research with more.

Doctor Susan Shurin, the acting director of the National Institute of Health's National Heart, Lung, and Blood Institute which co-funded the study said, "This discovery provides an important new target for future therapies in people with sickle cell disease. More work is needed before it will be possible to test such therapies in people, but this study demonstrates that the approach works in principle."

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