An exciting new advancement in the treatment of sickle cell anemia was announced July 1, 2013 by researchers at UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.
In a study to be published in the Journal of Clinical Investigation, lab results demonstrate a foundation for using a patient’s own blood cells to create self-renewing normal blood cells by introducing a gene containing anti-sickling properties. The anti-sickling stem cells are transplanted back into the patient’s bone marrow, which then multiply, creating healthy red blood cells without sickling.
Dr. Donald Kohn, professor of pediatrics and of microbiology, immunology and molecular genetics led the study which uncovers this revolutionary alternative to current sickle cell disease treatment which does not rely on donor cells, thus avoiding the risk of rejection. More than 90,000 people in the U.S. have been diagnosed with sickle cell, mostly those of Sub-Saharan African descent. It is caused by an inherited mutation in the beta-globin gene which causes red blood cells to change from their normal shape, which is round and pliable, into rigid, sickle-shaped cells. Normal red blood cells pass easily through the tiniest blood vessels, or capillaries, carrying oxygen to the lungs, liver and kidneys. Sickled blood cells, due to their rigid structure, become stuck, unable to travel throughout the body.
The study was supported in part by a Disease Team I Award from the California Institute for Regenerative Medicine, California’s stem cell research agency, which was created by a voter initiative in 2004 to speed research that takes scientific discoveries from the laboratory to the patient bedside.
Find out how you can help in the fight against sickle cell disease by visiting the Sickle Cell Disease Foundation of California. Civic Duty is proud to be a supporter of The Sickle Cell Disease Foundation of California.