Sickle cell disease, which is caused by a mutated gene that results in red blood cells shaped like sickles, is a disease that primarily affects Black people. Given that, research surrounding a potential cure for the disease has been fairly modest. However, according to the New York Times, there have been advances in the science of gene therapy that could offer hope for the many people affected by the illness that can cause terrible pain, stroke, and an early death.

There are about six clinical trials that are either planned or already underway in which half of the participants who have shown signs of the disease no longer exhibit symptoms of the disease. The experimental treatments are in the early stages of testing, and most likely there will be a wait of at least three years before they are approved. But researchers are hopeful that the results of the early tests are indicative of a potential cure.

Currently, the only real relief for those who suffer from the disease is a dangerous and expensive bone marrow transplant operation, and though a gene therapy treatment that works would not be cheap, it would be safer than a bone marrow transplant.

In the newer trials, scientists are using stem cells removed from the patient’s bone marrow. The stem cells are genetically modified and then inserted back into the patient’s bloodstream, with the desired outcome being the creation of the new healthy red blood cells

In other trials, scientists are trying to block off the creation of adult hemoglobin, which would allow patients with sickle cell disease to create fetal hemoglobin which does not result in the blood cells being shaped like a sickle. Another trial manipulates genes with Crispr, a tool that allows scientists to cut out and paste in sections of genes to rewrite their function.

The cure that is perhaps the farthest along is a type of gene therapy which will produce fetal hemoglobin and it is in trials conducted by Bluebird Bio, a biotech company in Cambridge, Massachusetts. They are currently in talks to organize a larger study with the FDA, hoping to enroll 41 patients who will receive gene therapy. Bluebird would like to finish the study and have their treatment approved by 2022.

David A. Williams, chief scientific officer at Boston University told the New York Times, “Having tried for a number of years to raise philanthropic money (to study the disease), I can tell you it’s really hard.” Now, however, he believes “We are in uncharted territory” as it relates to a cure.