New Gene Therapy May Reverse Effects of Sickle Cell Disease

Natalie Frank, Ph.D.

A recently published study in the New England Journal of Medicine, demonstrated that a gene therapy named LentiGlobin may provide a long lasting if not permanent cure for sickle cell disease
A new treatment for sickle cell disease may reverse the effects and provide a cure (CC BY 4.0

Sickle cell disease is a group of genetic blood disorders that impact millions of people around the world, with 100,000 of those in the U.S. It’s been estimated that 300,000 babies are born with one of these disorders annually.

These diseases involve the red blood cells which are responsible for transporting oxygen throughout the body. Normal red blood cells are disc shaped and flexible allowing them to easily more through the blood vessels.

Sickle cell disease causes the red blood cells to become misshapen, forming into crescent like sickle shapes. These cells lack flexibility and this prevents them from being able to move freely. At times, they can block blood flow (vaso-occlusions), resulting in an agonizing pain crisis, or possibly a stroke. Eye problems and infections are also common in those suffering from sickle cell disease.

The disease is most prevalent in areas with high rates of malaria as the genetic train protects against contracting that disease. However, as these areas often lack sufficient resources for treating sickle cell disease, the outcomes are usually poor, with most children who have the disease dying before the reach adulthood.

Now, a research study has determined that a new genetic treatment may reverse the effects the disease has on red blood cells. Subjects were required to have at least four vaso-occlusive events occurring in the two years prior to inclusion in the study. The treatment involved inserting a healthy beta-globin gene into the subject’s own stem cells after which they were transplanted back into the body by infusion.

Results indicated that a single treatment with led to continued production of unsickled hemoglobin in the majority of red blood cells. This was associated with decreased break down of red blood cells and the elimination of severe vaso-occlusions.

Markus Y. Mapara, a professor of medicine at Columbia University Vagelos College of Physicians and Surgeons who co-authored the study summed up the potential of this research, saying:

"You cannot overstate the potential impact of this new therapy. People with sickle cell disease live in constant fear of the next pain crisis. This treatment could give people with this disease their life back. We hope this therapy will also be successful in younger patients so they can grow up without experiencing pain crises and live longer."

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