CRISPR Gene-Editing Tool: 1st Patient With Genetic Disorder Treated In U.S. : Shots - Health News Victoria Gray, 34, of Forest, Miss., has sickle cell disease. She is the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease.
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In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

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In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

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RACHEL MARTIN, HOST:

The first interview with someone who has volunteered for one of the most highly anticipated medical experiments in decades. The first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States. NPR health correspondent Rob Stein has our report.

(SOUNDBITE OF DOOR SLAMMING, CALL BOX BEEPING)

ROB STEIN, BYLINE: I went to the Sarah Cannon Research Institute in Nashville to meet Victoria Gray.

UNIDENTIFIED HOSPITAL EMPLOYEE #1: Victoria is in Room 7.

STEIN: Hello. I'm Rob Stein from NPR.

VICTORIA GRAY: Victoria Gray. Nice to meet y'all.

STEIN: Victoria is just turning 34. She lives in Forest, Miss., with her husband and four children.

GRAY: My twins, Jaden (ph) and Jadasia (ph), they are 11. Jamarius (ph) is 12. And my stepdaughter, Asia (ph), is 13.

STEIN: Wow. You've got your hands full (laughter).

GRAY: I do (laughter). And it's the right time to get healed.

STEIN: Victoria's been struggling with sickle cell disease all her life.

GRAY: It's horrible. When you can't walk or, you know, lift up a spoon and feed yourself, it gets real hard.

STEIN: Sickle cell affects millions of people around the world, including about 100,000 in the United States. Most, like Victoria, are African American.

(SOUNDBITE OF MEDICAL DEVICE BEEPING)

UNIDENTIFIED HOSPITAL EMPLOYEE #2: Victoria, this one is not going to stop today.

STEIN: A genetic defect deforms the red blood cells, making them sickle-shaped, hard and sticky, and they don't carry oxygen like they're supposed to, causing terrible bouts of pain.

GRAY: Sometimes it feels like lightning strikes in my chest, and real sharp pains all over. And it's the deep pain. You know? I can't touch it and make it better or do anything to make it better. Sometimes I will be just balled up and crying, not able to do anything for myself.

STEIN: That sounds just awful.

GRAY: Yes, it is.

STEIN: The sickle cells have already damaged Victoria's heart, made her weak, prone to infections, strokes and more. Many people with sickle cell can't work or finish school. Many don't make it past their forties.

GRAY: It's scary. And it affected my oldest son, you know, because he's older so he understands. So he's started acting out in school. And, like, his teacher told me, I believe Jamarius is acting up because he really believes you're going to die.

STEIN: I'm so sorry. So sorry.

Some patients can get bone marrow transplants, but for most, there's no cure.

GRAY: It was just my religion that kind of kept me going.

STEIN: But then Victoria heard about something new. Scientists were planning to use the gene-editing technique called CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

(SOUNDBITE OF KNOCKING ON DOOR)

HAYDAR FRANGOUL: Hey, Victoria. How are you doing today?

GRAY: I'm doing OK, Dr. Frangoul.

FRANGOUL: That's great.

STEIN: Dr. Haydar Frangoul is running the study at the Nashville hospital.

FRANGOUL: How was your last night?

GRAY: Last night, it went pretty well.

STEIN: CRISPR lets scientists make very precise changes in DNA. Doctors have started using CRISPR to try to treat cancer, mostly in China. Frangoul's sickle cell study is part of a new wave of studies trying to use CRISPR to treat other diseases around the world.

FRANGOUL: CRISPR technology has a lot of potential use in the future, not only in blood disorders - in other genetic disorders to help many other patients.

STEIN: Here's how CRISPR might help sickle cell patients like Victoria. Healthy red blood cells use a protein called hemoglobin to carry oxygen. Sickle cell is caused by defective hemoglobin. But babies, they're born with healthy fetal hemoglobin.

FRANGOUL: Once a baby is born, a switch will flip on. It's a gene that tells the red blood cell, the bone marrow cells that produce red cells, to stop fetal hemoglobin.

STEIN: So Frangoul will be using cells that scientists have edited using CRISPR to flip that fetal hemoglobin switch to turn the production of healthy fetal hemoglobin back on.

FRANGOUL: And this opens the door for many patients to potentially be treated and have their disease modified to become mild or cause them no long-term horrible, horrible side effects of sickle cell disease.

STEIN: Other doctors and scientists are also excited about studies like this. Laurie Zoloth is a bioethicist at the University of Chicago.

LAURIE ZOLOTH: This is an exciting moment in medicine. Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases. But no one knows.

STEIN: She worries the new CRISPR studies may not have gotten enough scrutiny by objective experts.

ZOLOTH: This is a brand-new technology. It seems to work really well in animals and really well in culture dishes. It's completely unknown how it works in actual human beings. So there's a lot of unknowns. It might make you sicker.

STEIN: Dr. Frangoul acknowledges there are always risks with experimental treatments. But he says the research will go very slowly and carefully with a lot of oversight by the Food and Drug Administration and others.

FRANGOUL: We are very cautious about how we do this trial in a very systematic way to monitor the patients carefully for any complications related to the therapy.

STEIN: For her part, Victoria says she understands that there may be risks. The treatment's not easy. It involves a grueling round of chemotherapy, weeks in the hospital, months of tests. And the study's just the first step that may only benefit other patients years from now. But she can't help but hope.

GRAY: Being able to wake up and not be in pain, and to just be tired because I've done something, (laughter), and just tired for no reason. And just to be outside and jump on the trampoline with my kids, and go to graduations and weddings, and see them grow up. That means the world to me.

STEIN: It'll probably be months before the first clues emerge about whether CRISPR-edited cells are safe and might be working for sickle cell disease. And it may be years before anyone really knows how well they might help patients like Victoria Gray.

GRAY: This gives me hope, if it gives me nothing else.

STEIN: Rob Stein, NPR News, Nashville.

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