“From the beginning Emily had one of the roughest induction phases they had seen in years,” Whitehead said. “She started chemo on May 28 and by June 11 she’d developed necrotizing fasciitis in both legs. The surgeon said they might have to remove her left leg from the knee down.”
The surgery worked. But not before infection took hold and her wounds had to be repacked every 48 hours.
“Still, she was in remission and [we thought] maybe this is one of the toughest induction phases,” Whitehead said.
For awhile, until October 2011, Emma underwent chemotherapy and would be out playing again within hours of receiving chemo.
“She never complained through any of it. We explained from the beginning: ‘You have cancer. You are one of the strongest kids. Not all kids are strong enough but you will beat cancer.’ One day she was crying and said, ‘I’m not brave enough. I’m really scared all the time.’
“I said, ‘The biggest heroes in history were scared to death whenever they did something heroic. Don’t confuse being brave with being scared.’ From then on she didn’t complain. She said, ‘Just tell me when it’s going to hurt. Don’t lie to me,’ ” Whitehead recalled.
But the remission didn’t stick and more chemo didn’t help.
“The typical treatment is bone marrow transplant but she relapsed, got more intensive chemo, then relapsed again. From that time forward we could not get back into remission and couldn’t control it with chemo so bone marrow transplant was off the table,” Grupp said.
As Emma clung to life, doctors suggested the new treatment that had the mysterious name, Cart 19. The clinical trial, now called CTL019, uses immune T cells taken from a patient’s own blood. These T cells are genetically modified in the lab to express a protein that will bind to a target found on cancerous B cells. The engineered HIV is the agent that seems to make the process work, researchers found.
“The [HIV] virus has been engineered so it can’t cause disease anymore but retains its ability to reprogram the immune system so it will now attack cancer cells,’’ June said in a video that documents Emma’s story. “We call those modified immune cells serial killer cells. Each infused cell can kill more than 1,000 different tumor cells.”
Three adults were treated with this procedure before Emma. Two went into complete remission and have remained as such for 21/2 years. The Whiteheads decided to proceed.
“As we told her parents, Emma would be the first kid ever to get this and only the fourth person to get this,” Grupp said.
Since Emma’s procedure, 15 children have had the procedure.
“Of the first six patients we reported on in the literature, five went into complete remission, one of those had reoccurement of the disease. That’s what we are seeing in the study, an 80 percent short-term response rate. How that holds up in time we’re not in a position to say. But we’re very impressed by the short-term response rate. We will have to follow these kids,” Grupp said.
Whitehead hasn’t quite exhaled. “We’re thankful for every day but still nervous every day.”