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First Person Treated for Sickle Cell Disease with CRISPR Is Doing Well

  • A Mississippi woman is doing well 1 year after undergoing an experimental treatment for sickle cell disease.
  • The treatment involves the gene-editing tool known as CRISPR.
  • Scientists say they hope this initial success will lead to more effective treatments for the disease.

Victoria Gray is a 34-year-old wife and mother from Forest, Mississippi.

She is also a pioneer and something of a medical marvel these days.

Gray is the first person in the United States to have her sickle cell disease treated with a gene-editing technique.

“I chose to participate in this trial because of hope — hope that it would change my life — and it has already in so many ways. I’m doing things for myself and for my family and just living life” Gray told Healthline.

Dr. Haydar Frangoul, the medical director of pediatric, hematology, and oncology at HCA Healthcare’s Sarah Cannon Research Institute Center and the Children’s Hospital at TriStar Centennial in Nashville, Tennessee, is treating Gray.

He says Gray first came to them to be evaluated for a bone marrow transplant.

“We said to her, ‘Oh, by the way, we are opening this clinical trial but nobody has yet enrolled in it. Nobody has been treated with it. In fact, we don’t even know if it works. It’s never been tried in humans,’” Frangoul told Healthline. “She basically jumped at the opportunity and said, ‘sign me up.’”

“That tells you a lot about how courageous Victoria is and how miserable she was with her disease. She was having a lot of complications,” Frangoul added.

Now one year later, he says her clinical trial is going so well he believes the procedure could eventually revolutionize the treatment for sickle cell disease.

“She is functioning as somebody who does not have sickle cell disease. I believe this is absolutely, totally transformative therapy,” Frangoul said.

The treatment

Gray’s treatment involves CRISPR, a tool that can be used to edit genes inside the cell.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.

In this clinical trial, the treatment is called CTX001. It uses CRISPR cas9 technology to edit a gene called BCL11A.

“Think of it as if you have a book with hundreds of pages and thousands of words. You’re trying to find that one word and fix it or change it,” said Frangoul.

“While a baby is still in the womb, it makes a type of blood called fetal hemoglobin, which can carry oxygen very effectively to help the fetus develop normally,” he explained.

“About 3 or 4 months after the baby is born, a gene called BCL11A turns on. That gene tells the cells to stop making fetal hemoglobin and start making adult hemoglobin,” he noted.

“What we are doing in this clinical trial is directing the CRISPR cas9 to go and turn that BCL11A gene off,” he added. “When you make high amounts of fetal hemoglobin, it will prevent the patient’s red blood cells from sickling.”

The journey

Gray began her treatment a year ago.

Frangoul says they use medication to mobilize the stem cells from the bone marrow and push them into the blood. Then researchers can collect the stem cells.

The cells get shipped to a lab to undergo the gene editing, then they’re frozen.

In the meantime, Gray was getting blood transfusions to keep her condition under control.

After a round of chemotherapy, the gene-edited cells are infused back into the body.

Prior to her treatment, Gray averaged seven hospitalizations a year and had multiple blood transfusions. Her hemoglobin was 7 (which is low compared with typical levels) and she had little energy.

But now a year later?

“She has not been hospitalized and her hemoglobin is 11 so she has more energy. She’s been able to spend time with her family. She is doing really well,” Frangoul said. “I am extremely excited about her progress.”

The next steps

Frangoul says they will continue to monitor Gray’s progress. They also plan to treat another 45 people in their clinical trial and see if they can replicate Gray’s results.

“Over time, if it is effective in adults, we are going to try to lower the age to work with adolescents,” he said. “If we can do it earlier in the course of the disease, maybe we can prevent some of the horrible complications related to sickle cell disease that happen during adulthood.”

There will likely be lots of interest.

According to the Centers for Disease Control and Prevention, sickle cell disease affectsTrusted Source millions of people around the world and an estimated 100,000 people in the United States. It occurs in 1 of every 365 Black American births.

“Going on family trips and to my children’s graduations are things I never thought I could plan for,” Gray said. “I hope that this treatment does for others living with sickle cell disease what it did for me, which is the gift of hope. And for people facing sickle cell disease, or any other genetic disorder, don’t give up.”

Dr. Francis CollinsTrusted Source, the director of the National Institutes of Health, told National Public Radio that he was impressed by the early success of the clinical trial.

“The progress we’ve seen for sickle cell disease, including for Victoria Gray and her involvement in this, made it clear that if this is starting to work, we need to get busy and figure out how to take it to the next level,” he said.

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