"It's one of those really challenging things for me as a physician. "I took an oath not to harm. And I worry about that in the back of my head, because it's a new drug. And we don't even know what dose to start him with."[npr - listen]
Every once in a while, there's an experimental drug that's so promising it makes the race even more urgent. Patients and their families plead with pharmaceutical companies to get it before the Food and Drug Administration's approval.
The demand has been particularly high for a new class of drugs that harnesses the immune system to fight cancer.
"That's why I'm so desperate, contacting the drug companies," she explained. "I told them I understand the policy, I understand the regulation and I understand all the risks, but my child just has no time to wait."
Clinical trials were underway for several of the new immunotherapy drugs, but there were no trials available for children.
Pharmaceutical companies rarely offer clinical trials of new cancer drugs for children. Several pediatric cancer specialists said that's because of a lack of financial incentives for the drug companies, as well as the complexity of organizing trials for so few people, given the rarity of childhood cancer.
Only 1 in 285 children will be diagnosed with cancer before the age of 20, yet cancer is the second-leading cause of death for people in that age group.
The family's doctors advised Liu to "go home" with Joey to enjoy the rest of their time together.
"We can't just go home," she said. "For us, that means giving up. If that happened to me maybe I would make the decision we just go home, but it's my child. I can't just like do nothing and go home. I have to try everything."
Last spring with the help of a friend, Liu petitioned several pharmaceutical companies to gain access to one of a handful of immunotherapy drugs in development. The petition quickly received over 17,000 signatures, but was ineffective. And even if she could have gotten one of the drugs, their doctor, Dr. Joanne Lagmay, an oncologist at Shands Children's Hospital in Gainesville, Fla., was reluctant to give it to a child.
"It's one of those really challenging things for me as a physician," Lagmay said. "I took an oath not to harm. And I worry about that in the back of my head, because it's a new drug. And we don't even know what dose to start him with..."