The FDA approved the first gene-editing treatment for human illness
SCOTT SIMON, HOST:
There's new hope this week for people who suffer from sickle cell disease. The Food and Drug Administration approved the first genetic treatments for the brutal blood disorder on Friday. The announcement marks the first time that a medical treatment that uses gene editing has become available in the U.S. NPR health correspondent Rob Stein joins us. Rob, thanks for being with us.
ROB STEIN, BYLINE: Oh, my pleasure, Scott.
SIMON: And, of course, sickle cell is a genetic disorder - causes red blood cells to become deformed, sickle-shaped. What are the effects of it?
STEIN: Yeah. It's a terrible disease. These misshapen, sickle-shaped cells can't nourish the body with oxygen like they're supposed to. And the misshapen cells cause unpredictable attacks of excruciating pain and serious health problems that usually cut patients' lives short. The only cure is a bone marrow transplant, and most patients just can't find a suitable donor for that.
SIMON: How do these genetic treatments work?
STEIN: So doctors remove cells from patients' bodies, genetically modify those cells in the lab and then infuse billions of the modified cells back into patients where the modified cells produce a healthy form of a protein sickle cell patients need called hemoglobin. That creates normal red blood cells and alleviates the symptoms. One of the new treatments uses a more conventional approach - a modified version of a virus, to ferry a gene into patients' cells. The other uses the gene editing technique known as CRISPR to edit a gene in the cells.
Scott, you might remember Victoria Gray of Forest, Miss. She was the first sickle cell patient to get the gene-editing treatment in 2019. NPR broke that story and had exclusive access to chronicle her experience. I talked with her about yesterday's approval.
VICTORIA GRAY: I've had a new beginning. I no longer have to go to the hospital because I'm in severe pain. I'm no longer tired, with lack of energy. And most of all, I no longer have to fear dying and leaving my kids behind without a mother. My life is limitless now. It is a real transformation.
STEIN: And, Scott, that's been the experience of most of the patients who've undergone both treatments so far.
SIMON: It's so wonderful to hear her say that. Are there negative impacts from any of these treatments?
STEIN: Well, you know, there are still lots of questions. You know, one of the questions is, will these treatments actually translate into a longer lifespan for patients? Could there be any long-term side effects that just haven't shown up yet? In fact, there is already some concern that the approach that uses the virus may increase the risk for blood cancer.
And another big question is, will patients be able to get these new treatments? They're expensive. One will cost $2.2 million per patient, the other 3.1 million. And they're very complicated and difficult to go through. So it will be hard to get for many patients, especially less affluent patients in this country and the millions of patients in Africa and Asia, where sickle cell is most common.
SIMON: All of this being noted, Rob, how significant a moment is this for gene editing?
STEIN: It's a big deal. You know, it's pretty remarkable how quickly gene editing went from being an experimental technique to something that is actually helping people. I talked about this with Jennifer Doudna from the University of California, Berkeley. She shared a Nobel Prize for helping discover CRISPR.
JENNIFER DOUDNA: It's only the beginning. It's an amazing time.
STEIN: Gene editing is already being tested for a long list of diseases, ranging from relatively rare genetic conditions like muscular dystrophy to more common health problems like cancer and heart disease.
SIMON: NPR health correspondent Rob Stein. Thanks so much.
STEIN: You bet, Scott.
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