Kaitlyn Johnson, a 7-year-old from outside Dallas who loves cheerleading, her science classes, and the color pink, and Justin Pritikin, a 17-year-old high school senior in New Jersey looking forward to starting college, have never met. But they've got one important thing in common: they were both among the courageous participants in clinical trials of a completely new way to treat cancer.
The medicine is called Kymriah, and is among a new wave of therapies that use a patient's own cells as medicine. It was approved by the Food and Drug Administration on Aug. 30 and heralded as a historic event in the annals of cancer treatment.
Kaitlyn's and Justin's are both stories of hope. But they also illustrate the early nature of understanding of this new technology, and of how society will pay for it.
The technology is CAR-T, for chimeric antigen-receptor T-cell therapy. It uses viruses to deliver genetic material to patients' immune cells, equipping them with homing devices to track down markers on cancer cells.
"That is, in this way, a living therapy," said Dr. Jay Bradner, president of the Novartis Institutes for BioMedical Research. Novartis markets Kymriah. "This growth of T-cells in the patient will allow these serial killers to take out millions of cancer cells."
The FDA cleared the medicine to treat patients up to 25 years old with acute lymphoblastic leukemia for whom other therapies have stopped working. It's a small number of patients, about 600 a year in the U.S., but a group with few other options.
The agency called the approval a "historic action… ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases."
Earlier that same week, biotechnology giant Gilead spent $11.9 billion to acquire Novartis's CAR-T competitor, Kite Pharma. Shares of Juno Therapeutics, third in line with CAR-T therapies in development, soared on news of the deal. CAR-T is the new darling of the drug industry.
It's a moment years in the making. Years that Kaitlyn and Justin contributed to. But here is where their stories diverge.
For Kaitlyn, the journey to CAR-T started in late 2011. She was just 18 months old when her parents, James and Mandy, noticed she had strange bruises. Then a fever. A bewildering trip to the hospital revealed the worst.
"I don't even remember the doctor's name," Mandy recalled last week. "I was like, 'So, does my daughter have cancer?' And she was like 'Has nobody told you yet? Yes, she does.'"
Then started the two and a half years of punishing chemotherapy that's the standard of care for acute lymphoblastic leukemia, the most common form of childhood cancer. For more than 80 percent of kids, that regimen — though grueling to get through — works.
Kaitlyn was among the unlucky few for whom it didn't. Just a few months after she finished treatment, the cancer came back.