Pioneering Gene Therapy Freed Her of Sickle Cell. Is a Cure at Hand? – The New York Times

Posted: September 16, 2021 at 6:21 am

In January, his doctor, Alexis Thompson of Northwestern University, told him he no longer had sickle cell disease.

It is strange, Mr. Hubbard said, to think he has a future.

I am becoming more serious about life, he said. I didnt think I would have a life.

Helen, too, has been adjusting to life as a healthy person an alteration her pediatric hematologist, Dr. Alexander Ngwube of Phoenix Childrens Hospital, has observed in sickle cell patients cured with bone-marrow transplants.

Remember with sickle cell disease they are hospitalized a lot, he said. There are so many restrictions on their lives. They become depressed and when they are a little older they realize they have a life expectancy of 40 years. They start to think, What is the point of doing anything?

When they are cured, he said, it is almost like the world is theirs to play with.

When Dr. Esrick gave Helen the good news a year ago, Helen lay silent on the exam table, not daring to talk.

Helen and her family have since moved from Lawrence, Mass., to Mesa, Ariz. Ive followed her progress for more than two years. A lifetime of stoicism had taught her to keep her emotions to herself. Through a harrowing monthlong stay in the hospital for the gene therapy, she barely spoke to me. Even after it was completed, she tapped her mother on the arm and pointed when she wanted an ice cream cone as we walked through the Faneuil Hall Marketplace in Boston.

Freed of illness, shes become far more outgoing. On summer weekends, shes ventured to an arcade and a water park, and gone tubing.

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Pioneering Gene Therapy Freed Her of Sickle Cell. Is a Cure at Hand? - The New York Times

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