Seeking a sickle cell cure: 12-year-old in DC is 1st patient in US to get new gene therapy

A 12-year-old boy who suffers debilitating pain because of sickle cell disease has become the first patient in the U.S. to undergo a newly approved gene therapy.

Kendric Cromer is being treated at Children’s National Hospital in D.C. By this time next year, he stands a chance at becoming the first person cured of sickle cell with gene therapy.

News4 spoke with Kendric and his family about the treatment that could transform care for thousands of people.

His mother, Deb Cromer, has been by her son’s side for every hospital stay and painful episode. She said she’s more hopeful than ever.

“To see the light at the end of the tunnel and know that our child is the first to experience it outside of research – to know that it's safe and the doctors here believe in it. I would go to the end of the earth to make sure he was cured. There's nothing I wouldn't do for my son, but this makes me proud,” she said.

Kendric has spent his life in daily pain.

“When I have pain, I can't play sports with my friends, can't hang out with them, can't ride my bike. It’s just too much pain for me to handle most of the time,” he said.

“To hear your son screaming in pain, saying, ‘Mommy, sharks are biting me!’ is one of the most haunting feelings,” his mother said.

But thanks to a newly approved gene therapy, Kendric is the first patient to undergo a treatment that could cure the disease.

In the U.S., 100,000 people live with sickle cell disease, the majority of whom are African American and Latino.

Dr. Andrew Campbell specializes in treating the genetic blood disorder and is Kendric’s doctor at Children’s National.

“Pain is the hallmark of this condition. But over time, it can affect different organs,” he said.

Here’s how the gene therapy treatment for sickle cell disease works

Kendric began the process of extracting his bone marrow stem cells a few weeks ago. Those cells are now in a lab, where they will be genetically modified and then infused back into his body.

“The patient then gets some chemotherapy and that allows the bone marrow to kind of be wiped out and expand to accept the modified stem cells that have the gene therapy,” Campbell said. “So, the idea is that you're just walking around allowing your stem cells with the gene therapy to work for you.”

The process will be both painful and costly. Kendric’s insurance is covering the costs. The Cromers are hopeful that it will finally provide some relief.

“Imagine caring for a newborn for 12 years, because that’s kind of what it’s like,” Deb Cromer said.

The treatment will be labor intensive. Not every hospital will have the ability to provide it and those that do will be limited in the number of patients to whom they can give it.

Children’s National currently has 1,500 patients with sickle cell. They will be able to serve just 10 patients a year, Campbell said.

News4 asked Kendric what will be the first thing he’ll do once he’s no longer in pain.

“I’ll go outside and do a whole bunch of stupid stuff with my friends,” he said, as a 12-year-old boy would.

He said he wants to wrestle with his friends and cousins and learn how to swim.