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New sickle cell therapies come with side effects and a big price tag

STEVE INSKEEP, HOST:

There's new hope for the estimated 100,000 Americans who have sickle cell disease. This is an inherited blood cell disorder, and it can cause debilitating pain, even strokes. The Food and Drug Administration approved breakthrough gene editing therapies, although they cost millions of dollars. Colorado Public Radio's Elaine Tassy reports.

ELAINE TASSY, BYLINE: Like most people with sickle cell disease, Mia Hilton is Black. She's a 20-year-old esthetician in Denver, vivacious in long lashes, making jokes and very comfortable with her doctor, hematologist Chris McKinney with Children's Hospital Colorado.

CHRIS MCKINNEY: Can you lay down so I can feel your belly?

MIA HILTON: Yeah.

MCKINNEY: No belly pain today?

HILTON: Mmm-mmm.

MCKINNEY: OK.

TASSY: Hilton's sickle cell disease sometimes causes disabling leg pain. She's been hospitalized for a week at a time, and when she's given IV medication for the pain, the drugs sedate her to the point that she can't drive.

MCKINNEY: Have you heard about the recently approved gene therapies for patients with sickle cell disease?

HILTON: So I've heard a little bit about it, but not too-too much.

MCKINNEY: OK.

HILTON: Not too much. But yeah.

TASSY: For the last 30 years, Dr. McKinney has not been able to offer patients like Hilton any new treatments until now.

MCKINNEY: It's an exciting time (laughter) for sickle cell, and this has radically changed our conversations that we have with patients.

TASSY: Changed because in clinical trials, the new gene therapies have been very effective at stopping pain. Hilton sounds intrigued.

HILTON: I would be pretty much down to do it. Yeah, because I feel like in the long run it could really help because having sickle cell - it can really slow you down.

TASSY: Doctor McKinney tells Hilton there's significant side effects.

MCKINNEY: Side effects of chemotherapy include hair loss. It can also result in infertility.

HILTON: Personally, I don't want kids anyway, so that is a free form of birth control. I already have, like, 40 wigs in my closet, so it wouldn't be too different. Like, it would just be like, oh, new wig, oh, pink wig, red wig, you know?

TASSY: The new therapies are also very expensive. One is priced at $2 million, the other 3 million. The companies that make them say their price reflects the clinical and economic value of a patient's life and the one-time transformative therapy. Insurance companies will probably pay for them, says Dr. David Rind with the Institute for Clinical and Economic Review. It's a nonprofit that evaluates the cost effectiveness of medications.

DAVID RIND: It would be surprising and really inappropriate not to be covering these therapies, or at least the cheaper of the two therapies.

TASSY: A reason, Rind says, is because of sickle cell's unique historical legacy.

RIND: The reason that there is sickle cell in the United States in substantial numbers is because we brought Africans over as slaves. This is a population obligation in the United States to get this right.

TASSY: As her doctor prepares to start offering the therapies, Mia is still trying to decide if it's right for her, but she praises the availability of new treatments for people suffering with the disease.

HILTON: They don't have to worry about sickle cell reactions anymore because there's more medicines on the market, and I feel like it's making it more fair.

TASSY: For NPR News, I'm Elaine Tassy in Denver. 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.

Elaine Tassy