Is Gene Therapy The Key for Treating Sickle Cell Anemia?

According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle cell anemia is notorious for causing serious complications, like blood clots, kidney failure, and strokes. Regardless, these events do not have to end in death. Dr. Vichinsky blames insufficient infrastructure for monitoring adults with the disease, which he believes is linked to the fact that the disease tends to affect people of color. However, ongoing research with gene therapy could ultimately deliver on a possible cure for sickle cell anemia. 

About Sickle Cell Anemia

Sickle cell anemia is a genetic, inherited disorder of the blood. This disorder is characterized by an abnormality affecting hemoglobin, which is found in red blood cells and is responsible for carrying oxygen. The abnormality causes blood cells to lose their typical circular shape and instead become take on an elongated, sickle-like appearance. This is caused by a genetic mutation that may have arisen as a defense against malaria, although this benefit only occurs in people with sickle cell trait, not the disease. Symptoms begin to appear at around six months old and include swelling of the hands and feet, stroke, bacterial infections, and acute episodes of severe pain termed sickle cell crisis. Severity of disease varies, but these attacks can result in serious declines in health and organ damage. Treatment is mostly symptomatic, but bone marrow transplant has been curative in children. Life expectancy is between 40 and 60. To learn more about sickle cell anemia, click here.

A Cure Might be in Sight, But Can Patients Access It?

Proper monitoring could help save more sickle cell anemia patients, but gene therapy, which is currently in clinical trials, displays a lot of promise, as there is only a single gene that needs altering in order to treat the disease. Still, access to treatment is a major challenge for many patients, who are often black and low income. The approval of a breakthrough treatment doesn’t mean so much if the patients that need it can’t afford it.

About half of patients with sickle cell anemia must rely on Medicare and Medicaid in order to afford treatment. Even if gene therapy is successful, it is hard to say if insurers, particularly federally backed ones, will be able to cover it. Other concerns with gene therapy include the lack of long term monitoring data. Recruitment for trials is also challenging; black people are consistently underrepresented in trials even when they are disproportionately affected by a disease, often because black people are less likely to trust the medical field, largely due to the history of exploitation of black people in medical experiments.


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