But then Cunningham told the Tinaglia family that Sam was eligible for a CAR T-cell therapy clinical trial at Children’s Hospital of Philadelphia (CHOP). "He called it a groundbreaking procedure, that had shown incredible success rates in kids like Sam, who had recurrent leukemia," Suzie said. CAR, or chimeric antigen receptor, T-cell therapy is a type of cancer immunotherapy that involves genetically engineering a patient’s disease-fighting T-cells, rewiring them to seek out, recognize and attack cancer cells. Scientists refer to CAR T as a "living drug."
"CAR T-cell therapy is filling a gap in the goal we have of curing every child with acute lymphoblastic leukemia," Cunningham said. "We've been looking for a treatment like this for more than 30 years."
Cunningham referred Sam to CHOP and the Tinaglias arrived in Philadelphia – anxious but hopeful — in the fall of 2015.
"I’m obviously very happy, because my cancer, hopefully, is gone forever. It’s amazing," said Sam.
Sam’s T-cells were collected through apheresis, a non-invasive process (similar to donating blood). In a laboratory, scientists modified the cells, supercharging them to seek out a protein found on leukemia cells. After the CAR T-cells multiplied into the millions, they were returned to Sam’s blood stream where they began fighting the cancer.
Soon after the infusion, Sam experienced some of the serious side effects associated with the treatment. Cytokine-release syndrome (CRS) brought on high fevers, fluctuating blood pressure, temporary memory loss and seizures. Also known as a cytokine storm, CRS is the result of T-cell activation, so it was a positive sign that the attacker T-cells were performing.
"We went into it expecting that something like this could happen," Suzie said, "but we also knew it meant the treatment was working. And we needed this treatment to work." Following a week’s stay in a pediatric intensive care unit, Sam recovered.
"Nothing difficult came after the storm," he said. “Once it was over, I was done with cancer treatment. No more chemotherapy. No more medications. The CAR T-cells really did the job. They’ve been working ever since.
"I’m obviously very happy, because my cancer, hopefully, is gone forever. It’s amazing."