CAR-T has been used to treat certain blood cancers for two decades and got its first Food and Drug Administration approval in 2017. In recent years, doctors have been exploring the treatment in solid tumors, including those in the brain, where patients have few options.
Gavin enrolled in Vitanza’s trial before his third birthday, after he had already completed his radiation treatment.
Doctors started by extracting T cells from Gavin’s blood. Those cells were then modified in a lab to go after the tumor target, called B7-H3. About a month later, he started getting infusions of the T cells, delivered directly into his cerebrospinal fluid. Once reintroduced back into the body, the T cells replicate, creating a surge of cancer-fighting immune cells.
The results of Vitanza’s early-stage clinical trial were published in Nature Medicine in January. In addition to Gavin, 20 children and young adults with DIPG got CAR-T therapy every two to four weeks. The median survival was about 20 months — nearly double the expected prognosis. Three patients, however, are still alive 3 ½ to 4 ½ years after starting their treatment.
Gavin is one of those patients. Now 6 years old, he’s lived four times longer than doctors initially predicted.
“It’s the biggest miracle that I could ever ask for, just to have time,” Ashlee said.
Gavin is still getting CAR-T infusions every two to three weeks. Sometimes, for about 12 hours after the infusions, he has headaches, nausea and vomiting. Other times, he’s ready to run around on the soccer field.
Dr. Mark Souweidane, director of pediatric neurosurgery at Weill Cornell Medicine and NewYork-Presbyterian Hospital, who wasn’t involved with the trial, said the results were encouraging, but added that it’s not “out of the realm of the norm” to have some patients survive longer.
“There are going to be outliers. You will get 5%-10% of kids who live beyond two years,” he said.
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