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Kendric Cromer, 12, left Children’s National Hospital in a wheelchair on Wednesday, wearing a T-shirt and cap printed with designs from the anime series “Naruto” and a black face mask. Staff lined the hallway, cheering and waving noisemakers. He had just become the first patient to receive a gene therapy for sickle cell since it was approved — a therapy that is expected to free him from the ravages of the disease.
After 44 days in the hospital, he was a bit dazed.
“I thought I would have sickle cell for the rest of my life,” he said. The disease had deprived him of his childhood, making everyday activities, like playing basketball or riding a bike, impossible because they could bring on searing pain, often resulting in hospitalizations.
But despite the celebratory atmosphere, Kendric and his parents are still shuddering over what they endured during his hospital stay.
Nothing, absolutely nothing — not all the discussions with doctors, not all of their reading and highlighting of texts, not the 13-page consent form that included organ damage and even death as possible outcomes — prepared them for what he would go through.
About 100,000 people in the United States have sickle cell disease. For the 20,000 or so with the most severe disease, gene therapy may be their only hope of living a normal life. The disease is caused by a mutation in hemoglobin genes that leads to crescent-shaped red blood cells, which tend to get stuck in blood vessels, causing episodes of excruciating pain. The blockages can damage organs, cause strokes and shorten lives.
Until recently, most saw no way out.
Then, last December, the Food and Drug Administration approved a $3.1 million sickle cell gene therapy by Bluebird Bio of Somerville, Mass., and a $2.2 million treatment by Vertex Pharmaceuticals of Boston. That potentially gives patients like Kendric, if their insurance will pay for the therapy, a path to a life that is not shadowed by the ravages of the disease.
But so far only a very small number of people have benefited. As of Sept. 24, Bluebird reports 10 patients who have started their treatments since F.D.A. approval, and Vertex in July reported 20.
Yet for all of the Cromer family’s luck — their insurer approved the treatment without too much difficulty, and he became Bluebird’s first commercial sickle cell patient — another story may get overlooked. Patients and their families are so desperate for the treatment that it can be difficult for them to take in how arduous it will be.
“Even though I say ‘this is a dangerous therapy, high risk, you could die,’ no one hears that,” said Dr. Akshay Sharma of St. Jude Children’s Research Hospital. “All they hear is ‘I could be cured.’”
Kendric’s treatment began on May 1 when doctors removed stem cells from his bone marrow and sent them to a facility in New Jersey, where technicians added new, healthy hemoglobin genes to the cells. Bluebird shipped Kendric’s cells back a few months later.
On Sept. 3, Kendric was admitted to Children’s National for the final phase of his treatment.
Two days later, he started intense chemotherapy to clear his bone marrow and make way for the treated cells to repopulate it. On Sept. 11, his doctors infused the treated stem cells back into his body. His medical team told Kendric and his parents that he would have to stay in the hospital for about a month.
Soon the side effects of the chemotherapy kicked in.
“It seemed like it happened overnight,” said Kendric’s mother, Deborah Cromer, who is a real estate agent.
Mucositis — a severe inflammation causing painful sores in his mouth and intestinal tract — came first, on Sept. 15.
The next day, “he woke up and his tongue was so swollen he could barely talk,” Mrs. Cromer said. “By the end of the day he was in a lot of pain,” a searing ache that radiated into his stomach.
He couldn’t eat because of his swollen tongue and the painful sores. He had to get nourishment intravenously. Talking was so difficult that he put bells around the neck of a little stuffed dog. “If I needed anything I would shake it,” he said. Then he would write a note describing what it was.
Kendric’s parents were tortured by his pain.
“I was watching him go through it and not able to do anything at all,” said his father, Keith Cromer, who works for the federal government.
Every joint ached. Kendric described it as an 8 or 9 on a 10-point pain scale. The only help were doses of morphine so high he was in a stupor.
On Sept. 30, Kendric’s hands and feet turned hot and hurt as if they had been burned, a very rare side effect. “It looked like he’d put his hands on a hot stove,” Mrs. Cromer said.
He had to tiptoe to go to the bathroom.
The burning spread over his body. Then his skin darkened and started to peel. And his hair fell out.
Slowly, though, he started to feel better. One day, the swelling in his tongue started going down. “By the end of the day I was able to talk again,” he said.
But his treatment is not over yet. Transfusions left him with excess iron in his blood. He will have to return for blood letting every two to four weeks to remove it. Excess iron can damage the liver or heart, said Dr. David Jacobsohn, chief of the Washington hospital’s division of blood and marrow transplantation.
And because the chemotherapy left him with limited ability to fight infections, he needs to get re-immunized with childhood vaccines and stay away from crowds for several months while his immune system recovers.
Other young people who had gene therapies for sickle cell while they were being tested in clinical trials had experiences similar to Kendric’s.
Alani Newton, 19 of New Orleans, refuses to talk about her treatment at the University of Alabama at Birmingham. She was discharged 10 months ago and has put her life with sickle cell and her treatment behind her the way some who return from the horrors of the battlefield will never discuss what they experienced.
Her mother, Jonata Newton, remembers it all too well, and the aftermath, when Alani came home from the hospital and experienced exhaustion and weakness for the two months after she was discharged, all too well. She describes how Alani’s skin turned almost black and her hair fell out.
“Mommy, I look like a monster,” she recalls her daughter saying.
But today, Alani is in college. “She’s happy, she’s healthy. She’s excited about school. She’s flourishing,” Mrs. Newton said. “Now Alani is a person. Before she wasn’t even living at all.”
Mrs. Newton, a high school teacher, said she lost her job when she took leave to care for Alani. “We were drowning in debt,” she said. She and her husband are now repairing the damage to their finances. She’s teaching again.
For Donte Johnson, 23, of Memphis, gene therapy at St. Jude in 2020 changed his life for the better. The whole experience of having sickle cell, with his many hospitalizations over the years, set his family back financially.
Caring for him took so much time that his mother, Angela Johnson, who lives in Memphis, could not hold a steady job.
She remembered standing on the steps of Donte’s school one day in desperate straits, near tears.
“I thought I had to file for bankruptcy because my car was repossessed and I was trying to keep my home,” she said. An elderly woman came up to her and told her, “You have to keep pressing on.”
Since Donte completed his therapy, Ms. Johnson has finished college and can keep a job. And she said she has “peace of mind because my son doesn’t have sickle cell.”
Kendric’s parents know their family will be changed by his therapy. They have faith they will get through this very tough period.
Learning to be a healthy teenager without sickle cell “will be a new challenge for him,” his father, Keith, said.
“These kids are walking miracles,” his mother, Deborah, said.
“We want Kendric to focus on that,” she added. “And not the bad stuff.”
So far, he is doing that.
“I haven’t felt all the amazingness yet,” he said. “But I am slowly starting to feel better.”
The Saturday night before his discharge, “Dancing with the Stars” came on the TV in his hospital room.
“I said, ‘Mommy, let’s dance,’” Kendric recalled.
And so they did. With sickle cell, such lively movement would have brought on a pain crisis that could have sent him to the hospital.
“I kept thinking it would happen,” Kendric said.
It never did.
“I felt great,” he added. “Apparently, I’m a good dancer.”
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