The Stream

Living with sickle cell anemia

What impact will gene therapy have on the disorder?

Doctors hope a recent medical breakthrough could lead to a cure for sickle cell anemia, an inherited condition that can cause crippling pain and shortened lifespans for the estimated quarter million babies born with it each year.

Earlier this month, doctors announced the results of an experimental gene therapy that is offering hope to those who suffer from the disease. A French teenager with the genetic condition achieved complete clinical remission after researchers in Paris injected his stem cells with an anti-sickling gene from his bone marrow. So far it seems to be working for the teen. Fifteen months since his last treatment, the patient is no longer dependent on medication, and his blood cells show no sign of the disease. Clinical studies have begun in other parts of the world to address the disease.

The disorder occurs when a child’s mother and father each pass on copies of a defective gene that helps make hemoglobin, a protein that carries oxygen in red blood cells. The defect causes red blood cells to collapse into a crescent shape, clogging blood vessels and preventing oxygen from flowing through the body. This can trigger serious problems like stroke and organ failure.

For most of the 20th century, sickle cell was a relatively invisible disease because it was so often misdiagnosed. Despite advances, race, poverty and stigma remain challenges in treating the blood condition most prevalent in people of African descent. It also affects people of Indian, Middle Eastern, Hispanic and Mediterranean heritage. In the United States, where the majority of people with sickle cell disease identify as black, patients seeking pain relief have been cast as drug seekers, and researchers have suggested racial discrimination affects the care sickle cell patients receive.

Doctors and patients join The Stream to discuss the progress and peril of sickle cell disease.

In this episode of The Stream, we speak with:

Jamie Wells @jamiewellsmd
Director of Medicine, American Council on Science and Health

Julie Kanter @jkw4444
Director, Sickle Cell research at University of South Carolina

Jenica Leah @JustJLeah
Children’s author

Bukola Bolarinwa @naijadaydreamer
President, Sickle Cell Aid Foundation

What do you think? Share your thoughts in the comments section below.