Sickle cell disease is a slow, vicious killer.
But those years are a lifetime of pain, as abnormal, crescent-shaped haemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage.
Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.
Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from “sickling.” In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped haemoglobin.
To reiterate, the paper is a case study of just one patient.
Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported.