CNN At first, Sandy Barker decided to behave nicely and sit silently in the audience as an official from the Food and Drug Administration extolled the virtues of a program to get experimental drugs to desperately ill patients.
Then she couldn't take it anymore. Barker's hand shot up.
"I've been sitting here for the past hour trying to be quiet, but I want to tell you what happened to my son," she said.
Barker looked down at a picture of Christian on her lap. She started to cry, but regained enough composure to describe how her son was diagnosed eight years ago with a rare form of leukemia when he was 13. A bone marrow transplant was supposed to help, but instead the donor's cells attacked Christian's body.
Christian's graft-versus-host disease was quickly getting worse. His life was on the line. Nothing was working.
The Barkers searched for studies he could join but found none. Christian's doctors desperately wanted to try an experimental drug, but first the FDA had to give its blessing.
The Barkers and their doctors begged the agency to allow Christian to use the medicine. By the time permission was given, more than three weeks had passed, and the graft-versus-host disease had moved to stage 4, the most severe stage.
Christian died two months later.
During a panel discussion at a conference on rare diseases, Barker says the FDA official noted it can be helpful to lobby one's congressman to get access to experimental drugs.[...]
Last month the parents of a 7-year-old boy did just that and made headlines around the world. Josh Hardy's parents took to Twitter and Facebook when the drug company Chimerix denied their request for an experimental antiviral drug to save Josh's life. After receiving death threats from "Josh's army" -- executives had to hire security guards -- Chimerix reversed its position and granted Josh and other patients like him access to the drug.
Now that he's had the medicine, the virus that nearly killed Josh is gone and he's been moved out of the intensive care unit.[...]
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