The wait is almost over for Deborah Cromer and her 12-year-old son, Kendric. This fall, the D.C. boy will become first of many to receive a groundbreaking gene therapy aimed at curing his sickle cell disease.
The disease is an inherited blood disorder that causes round red blood cells to mutate into crescent moon shapes. Those cells block blood flow, causing debilitating pain and severe damage to organs. The pain is sickle cell’s hallmark, with periodic episodes called crises.
“Sometimes they can be sharp and feel like something is biting me, like a shark,” Kendric told WTOP. “Sometimes it feels like I’m burning, or that I’m being hit by a four-wheeler.”
Doctors diagnosed Kendric when he was a newborn. Over the years, his crises have caused many emergency room visits and long-term hospital stays.
They have also cut into his attendance at school. But Kendric, who wants to be a geneticist, is an honor student.
Common activities for kids, like playing team sports and riding bikes with friends, come with consequences, his mother said.
“He stumbled over his own feet in January and ended up hospitalized,” Cromer said. “He didn’t skin anything. But the blow of the fall caused his body to go into a very severe pain crisis.”
Kendric was already part of a medical trial to find a cure for the disease, when the Food and Drug Administration approved two milestone treatments in December.
The treatment aims to genetically modify Kendric’s stem cells, causing them to produce more normal red blood cells. The reprogrammed healthy cells could help drive down the number of sickle cells, ending his crises and opening the door to a more “normal life.”
“My husband and I both marvel at the fact that we’re seeing this now,” Cromer said about the medical breakthrough. “I cannot wait to see, pain free, what type of kid he becomes.”
Kendric was tapped to get the treatment because he has a lengthy history of crises, many hospital visits and his insurance covers its hefty price tag, said Dr. Andrew Campbell, Kendric’s doctor and director of the Sickle Cell program at Children’s National Hospital.
Eligible patients also have to be 12 and older.
“It’s a game changer,” Campbell said of the therapy. “They don’t have to take medications anymore because they’re walking around, allowing the stem cell to do the job for them.”
Roughly 100,000 people in the U.S. currently have sickle cell disease, according to the Centers for Disease Control and Prevention. Most of them are Black.
Kendric is currently receiving initial trial therapies to prepare his body for this fall, when he’ll get the full treatment.
“The hope is that he will be functionally cured,” Campbell said.
As far as what a “normal life” looks like to Kendric, his expectations are what you would expect for a 12-year-old.
“It looks like me hanging out with my friends and being able to play games,” Kendric said. “Going to high school and college, that’s what [a normal life] looks like.”
Campbell and his team at Children’s National will only be able to give the cutting-edge therapy to 10 patients a year into the pilot program.
But other families eager for the therapy are already lining up.
“We have families who want this to happen,” Campbell said. “It is a win for science and it’s a win for our patients.”
Kendric’s mom encourages other parents with children who have sickle cell disease to take a second-look at the treatment if they’re apprehensive about it.
“It’s hard for us as parents to watch our children go through things,” she said. “I’ve cried almost every day since Kendric has been approved because I’m just so excited for what the future holds for our family.”
