Some scientific breakthroughs, while exciting, require a calm, measured response. Most, make that all, demand repetition and duplication, as per the scientific method. But there are a select few that, even with all the caveats, are worthy of a “Joe Biden” response:
This is a big f***ing deal.
I’m going to go ahead and award this story five Joe Bidens. Researchers in France may have found an effective way to combat—and maybe even cure—sickle cell anemia using gene therapy.
Sickle cell anemia is a blood disease that disproportionately affects African Americans. It occurs when someone inherits an abnormal copy of a hemoglobin gene from each parent. The abnormal genes cause red blood blood cells to take on a crescent or “sickle” shape and become stickier than normal. As a result, sickle cells can block normal blood flow and cause a variety of painful symptoms and damaging organs.
Sickle cell anemia can be treated with certain cancer drugs, and in some patients a bone marrow transplant can effectively cure it. But a bone marrow transplant is risky, and the path to finding a donor is very narrow.
That’s what makes this study so exciting. Researchers at Necker Children’s Hospital in Paris took a sample of the patient’s stem cells from his bone marrow, then used chemo to clear out the remaining stem cells. The extracted stem cells—specifically, a gene called beta globin—were genetically modified to give them “anti-sickling” properties.
The newly altered cells were returned to the patient, and in the months that followed watched as his body grew new blood cells that bore the modified gene. As a result, the patient was effectively left with only one of the hemoglobin genes required for sickle cell anemia. In effect, he was cured.
Since then, the patient has been symptom free and hasn’t had to use his regular medications. There’s a pretty big “but” here: So far, the study has only looked at one teenaged patient over a 15-month follow up period. There’s no telling if the results will hold up throughout the patient’s life, or how it will play out once the study expands to additional patients.
But outside of risky bone marrow transplants, it’s the first really positive development in a long time. And even if we’re still a ways off from the day that sickle cell anemia becomes the type of disease people “used to get,” you can’t help but see this as an important leap forward to getting there.
And yes, that is a big f***ing deal.
