Stem-Cell Gene Therapy for Sickle Cell Disease


Donald Kohn, a professor of pediatrics and microbiology, immunology and molecular genetics in the UCLA College of Letters and Science, and his colleagues, have successfully established the means to cure sickle-cell disease. This strategy uses hematopoietic (blood-producing) stem cells from the bone marrow of patients with sickle-cell disease in order to treat the disease itself.

This approach provides a revolutionary alternative to current treatments, since it creates self-renewing, normal blood cells by inserting a gene that abrogates the sickling properties into hematopoietic stem cells. With this technique, there is no need to identify a matched donor, and therefore, patients avoid the risk of their bodies rejecting donor cells.

During the clinical trial, the anti-sickling hematopoietic stem cells will be transplanted back into patients’ bone marrow to increase the population of “corrected” cells that make red blood cells that don’t sickle. Kohn will hopefully begin enrolling patients in the trial within three months. The first subject will be enrolled and observed for safety for six months. The second subject will then be enrolled and observed for safety for three months. If evaluations show that no problems have arisen, the study will continue with two more subjects and another evaluation, until a total of six subjects have been enrolled.

Sickle cell disease, which affects more than 90,000 individuals in the U.S., is seen primarily in people of sub-Saharan African descent. It is caused by an inherited mutation in the beta-globin gene that transforms normal-shaped red blood cells, which are round and pliable, into rigid, sickle-shaped cells. Normal red blood cells are able to pass easily through the tiniest blood vessels (capillaries) and carry oxygen to organs like the lungs, liver and kidneys. However, sickled cells get stuck in the capillaries, depriving the organs of oxygen, which can lead to organ dysfunction and failure.

Current treatments include transplanting patients with hematopoietic stem cells from a donor. This is a potential cure for the disease, but due to the serious risks of rejection, only a small number of patients have undergone this procedure, and it is usually restricted to children with severe symptoms.

“Patients with sickle-cell disease have had few therapeutic options,” Kohn said. “With this award, we will initiate a clinical trial that we hope will become a treatment for patients with this devastating disease.”

Finding for this work comes from new grants to researchers at UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, which total nearly $21 million.  These grants were announced Dec. 12 at a meeting of the California Institute of Regenerative Medicine (CIRM) Citizen’s Oversight Committee.  They are apart of the state agency’s Disease Team Therapy Development III initiative.

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mburatov

Professor of Biochemistry at Spring Arbor University (SAU) in Spring Arbor, MI. Have been at SAU since 1999. Author of The Stem Cell Epistles. Before that I was a postdoctoral research fellow at the University of Pennsylvania in Philadelphia, PA (1997-1999), and Sussex University, Falmer, UK (1994-1997). I studied Cell and Developmental Biology at UC Irvine (PhD 1994), and Microbiology at UC Davis (MA 1986, BS 1984).

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