A 12-year-old boy who suffers debilitating pain from sickle cell disease has become the first patient in the United States to undergo a newly approved gene therapy.
Kendric Cromer is being treated at Children’s National Hospital in Washington. By next year, he will have a chance to become the first person cured of sickle cell disease using gene therapy.
News4 spoke with Kendric and his family about the treatment that could transform care for thousands of people.
His mother, Deb Cromer, was by her son’s side through every hospital stay and painful episode. She said she was more hopeful than ever.
“Seeing the light at the end of the tunnel and knowing that our child is the first to experience it outside of research – knowing that it’s safe and that the doctors here believe in it.” I would go to the ends of the earth to make sure he was cured. There’s nothing I wouldn’t do for my son, but it makes me proud,” she said.
Kendric spent his life in daily suffering.
“When I’m in pain, I can’t play sports with my friends, I can’t spend time with them, I can’t ride a bike. Most of the time it’s just too painful to deal with,” he said.
“Hearing your son scream in pain and say, “Mom, the 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 treatment that could cure the disease.
There are 100,000 people living with sickle cell disease in the United States, the majority of whom are African American and Latino.
Dr. Andrew Campbell specializes in treating genetic blood disorders 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.
This is how gene therapy treatment for sickle cell disease works
Kendric began the process of extracting his bone marrow stem cells a few weeks ago. These cells are now in a laboratory, where they will be genetically modified and then injected back into his body.
“The patient then receives chemotherapy, which allows the bone marrow to be kind of wiped out and grow to accept the modified stem cells benefiting from the gene therapy,” Campbell said. “So the idea is that you just walk around allowing your stem cells through gene therapy to work for you.”
The process will be both painful and expensive. Kendric’s insurance covers the cost. The Cromers hope this will finally bring some relief.
“Imagine taking care of a newborn for 12 years, because that’s kind of what it’s like,” Deb Cromer said.
The treatment will take a lot of work. Not all hospitals will have the capacity to provide it and those that do will be limited in the number of patients they can give it to.
Children’s National currently has 1,500 sickle cell patients. They will only be able to care for 10 patients a year, Campbell said.
News4 asked Kendric what the first thing he would do once he was no longer in pain.
“I go outside and do all kinds of stupid things with my friends,” he says, like a 12-year-old boy would.
He said he wanted to wrestle with his friends and cousins and learn to swim.
News Source : www.nbcwashington.com
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