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The FDA is closer to approving a gene-editing treatment for sickle cell disease

JUANA SUMMERS, HOST:

The Food and Drug Administration took a crucial step today towards a historic decision - the approval of the first medical treatment that uses gene editing. A panel of independent FDA advisers paved the way for the treatment for patients suffering from sickle cell disease. NPR health correspondent Rob Stein joins us now. And, Rob, this sounds like an important moment for both patients and for the advancement of this new kind of medicine. Tell us, you've been covering this a long time, how significant is it?

ROB STEIN, BYLINE: Yeah, it's a big deal that's being closely watched by scientists, the biotech industry and patients, of course. And that's because gene editing is generating enormous excitement that it could lead to new treatments for many diseases by allowing scientists to manipulate DNA much more easily than ever before. And this is the first time a therapy that uses gene editing to treat a disease has gotten this far - getting formerly scrutinized by outside FDA advisers, which is typically the last step before getting approved. In this case, the treatment uses the gene editing technique known as CRISPR to treat sickle cell disease. And that's a terrible blood disorder affecting millions of people worldwide, including about a hundred thousand in the U.S.

SUMMERS: OK. Rob, how's it work?

STEIN: So scientists remove bone marrow cells from patients. They use CRISPR to edit a gene in those cells and then infuse billions of the modified cells back into their bodies. The edited cells pump out a protein that sickle cell patients need to make healthy red blood cells, called hemoglobin. The hope is that would alleviate the terrible attacks of excruciating pain that plague sickle cell patients and prevent the long list of complications that usually cut patients' lives short, allowing them to live full, normal lives.

SUMMERS: And today's meeting, how did things go?

STEIN: So it was a pretty unusual FDA advisory meeting. You know, typically, the FDA asks advisers to vote on whether a new treatment is safe and effective and should be approved. But in this case, the data looks quite clear. The treatment looks like it worked for virtually every sickle cell patient who's had their cells edited so far, completely transforming their lives. And it looks very safe.

The FDA scientists agreed, so the agency asked the advisers to focus on whether more research is needed to make sure the gene editing isn't accidentally causing genetic mutations that could cause complications in the long run. FDA scientist and independent researcher raised questions about that during today's meeting. But while many committee members agreed additional research could be helpful, many also expressed enthusiasm for the treatment and few concerns that theoretical risks would outweigh the clear benefits. Here's Scot Wolfe from the UMass Chan Medical School.

SCOT WOLFE: It's really exciting to see how many patients have been treated and how positive the results have been. We want to be careful to not let the perfect be the enemy of the good.

STEIN: The advisers also heard some pretty emotional testimony from sickle cell patients, including Victoria Gray. Victoria was the first sickle cell patient to get the treatment. And, Juana, you might remember NPR has had exclusive access to chronicle her experience. Here's some of what she told the committee.

(SOUNDBITE OF ARCHIVED RECORDING)

VICTORIA GRAY: I believe if you say yes to this treatment, that it's going to change the lives positively of many people who are suffering from diseases and disorders who now feel hopeless. But once it's come, they can feel hope again, just like I did.

STEIN: You know, all of Victoria's symptoms have disappeared since she got treated, enabling her to do things she could never do before, like work full-time and take care of herself and her kids.

SUMMERS: Wow. That's incredible. Rob, what happens next?

STEIN: The FDA has until December 8 to make a decision about CRISPR gene editing treatment for sickle cell. But there are still lots of questions. One big one is, will the patients who need it the most be able to get it? It's expected to be very pricey - millions per patient. And it's complicated. That will make it hard to make it widely available, especially in less-affluent countries where sickle cell is most common.

SUMMERS: NPR health correspondent Rob Stein. Rob, thank you.

STEIN: You bet. Transcript provided by NPR, Copyright NPR.

NPR transcripts are created on a rush deadline by an NPR contractor. This text may not be in its final form and may be updated or revised in the future. Accuracy and availability may vary. The authoritative record of NPR’s programming is the audio record.

Rob Stein is a correspondent and senior editor on NPR's science desk.
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