This is a picture of Josh Hardy. You might recall seeing his face on TV or on the #savejosh hashtag on Twitter a few years back. Josh, who was 7 at the time, had undergone a bone marrow transplant for kidney cancer that left him hospitalized with a severe infection. He was dying.
There was an experimental drug called brincidofovir made by a a small company called Chimerix that his doctors thought might work. But the company declined their repeated requests to provide it. His parents, being smart and well-educated, rallied friends, who rallied their friends and their friends and so forth, until it seemed as though the entire Internet were behind them.
The ending was a good one: The company gave Josh the drug and it worked and he got to go home.
But not all cases like this go this well. A powerful report from STAT this week provides a heartbreaking reminder that the reason experimental drugs are not available for anyone to use is because they are just that — experimental. And there's as strong a chance that things may go wrong as right.
Read Full Article »