“Steve, my daughter has juvenile rheumatoid arthritis,” he said. “It’s an IL-6 immunity event, just like this. There’s a drug for this. Do you think the pharmacy has any tocilizumab?”
“How is this going to work to help Emily?” Dr. Grupp wanted to know. “No one has ever used this drug on a cancer patient before. And IL-6 isn’t even made by T cells.”
“It’s not a widely used drug,” Dr. June said. “But it does work on this.”
In a situation like Emily’s, where the patient is critically ill and the doctors are not sure what is going on, they had to try something they wouldn’t try under normal circumstances. Taking a chance was better than doing nothing. Still, a number of things had to work out for Dr. Grupp to give the drug to Emily. First, he had to find out if the pharmacy had it. If they did not, it was likely that they couldn’t get it in time to save her. The distributor usually gets the drug to the hospital within twenty-four to forty-eight hours from when a doctor orders it, but they didn’t have that long. Dr. Grupp called the pharmacy, and he found out they had two vials.
Then he had to convince the pharmacist it was ethical and logical to give a leukemia patient a drug that is prescribed only for juvenile rheumatoid arthritis. Research hospital pharmacies are cautious about off-label uses for drugs. Dr. Grupp and the pharmacist discussed how he was going to use the drug with Emily. Thankfully, there was already a pediatric dose approved in the package insert. Dr. Grupp advised that he was going to follow those dosage instructions and the pharmacist signed off on allowing him to prescribe the drug. The next hurdle was the PICU staff.
Dr. Grupp decided to make his case in person. With the vials of tocilizumab in hand, Dr. Grupp walked into the PICU to talk to Dr. Berg about their idea. He knew if anyone along the way said no, they wouldn’t have anything left to try. He knew everyone he spoke with wanted the best for Emily, so what was the best? Did the drug pose another risk? Everyone wants what is safe, and safe and best sometimes don’t overlap. This was one of those moments. Basically, he decided, what he was trying to do was to convince other medical professionals that he and Dr. June weren’t nuts.
One of the reasons the barriers were falling, and falling quickly, was because it did not look like Emily would make it another twelve hours. A decision had to be made quickly. Dr. Berg and Dr. Grupp had a spirited conversation, with all of those values at play. Dr. Berg agreed that if Dr. Grupp had found something that gave Emily a chance to pull through, even if it was unconventional and perhaps risky, they had to give it a try. He signed off on the tocilizumab, with a grin of respect.
“You guys are cowboys—you know that, don’t you?” Dr. Berg said.
The top rung on the permission tree was me and Kari. Dr. Grupp came to us with the wind at his back. I didn’t know what was putting the spring in his step, but I sensed his fresh energy.
“We’re grasping at straws, but we have an idea,” Dr. Grupp started off.
By 8:00 p.m. the tocilizumab was in Emily.
Chapter 17
#WEBELIEVE
The doctors just rounded with us. Emily’s right lung is re-inflated and showing up on the X-ray. The doctor used the term “amazing” when describing her lungs from yesterday to today. He said you can’t get better until you stop getting worse. He said Emily is no longer getting worse and is starting toward getting better. He also said this is the reason why you never give up on a child’s chance of coming back.
—Kari’s journal
April 26, 2012
That evening when Emily received the tocilizumab, all we could do was pray that it worked, but we were not prepared for how quickly that happened. A few hours after it was administered, her fever disappeared. She’d suffered with a fever somewhere between 103 and 106 since Sunday, and suddenly it was gone. Although she was improving, she had a long distance to go before she was considered to be better.
The PICU doctors were concerned because, after days of lethal low blood pressure, a few hours after she received tocilizumab, her blood pressure started to climb into a dangerously high range. As a bone marrow transplant doctor, one of the things Dr. Grupp worried the most about with his patients was the dangerous combination of high blood pressure and low platelets, which is what Emily suddenly had. If left untreated, these two things combined can cause significant bleeding in the head. Dr. Grupp called the PICU to discuss with them immediately taking Emily off the three medicines that were supporting her blood pressure. Generally, doctors take a patient off a medication slowly because it’s safer, but in Emily’s unusual case, faster was safer. The doctors didn’t have as much experience in trying to take a patient off that blood-pressure-boosting medication so fast, as it usually takes a long time for a patient’s blood pressure to regulate. They didn’t know how long it would take for the drugs to work their way out of Emily’s system.
By dawn, less than twelve hours after the first dose of tocilizumab, Emily was off two of the three blood pressure medications. When Dr. Grupp came in the next morning, I could see he was as tired as we were, but I also saw relief. He had watched Emily’s breathtakingly quick steps to recovery on his laptop from home. Her miraculous progress was a huge contrast from the twelve
