Max McNary was born with Duchenne muscular dystrophy, a progressive genetic mutation that causes muscle weakness throughout the body, and eventually results in pulmonary and cardiac failure. Most people with the disease do not live longer than their mid-20s. So when Max started taking an experimental drug that caused him to make remarkable improvements, it was like the miracle for which his mother, Jenn McNary, had been waiting.
She says the drug, called Eteplirsen, has changed Max in ways she'd never imagined possible. She told Today: “It really is a miracle drug. This is something that nobody ever expected and he looks like an almost normal 11-year-old.”
Incredible, although along with that amazing joy comes a huge dose of heartbreak, as McNary's other son, Austin, 14 -- who suffers from the same disease -- isn't eligible for the drug.
So as she watches one son heal, she's watching the other deteriorate -- all with the knowledge that if only Austin could get the drug, he too may be saved from the disease.
It's hard to imagine the anger and the complete disbelief she must feel that no one will step in and help her son because of rules, which deemed him too sick to participate in the trial. It's one thing to know that there's a drug out there that might help your sick child, but to actually see it working in one child and not to be able to provide it for the other has got to be excruciatingly painful. And for Austin too. He told Today: "It's really hard because I know I'm getting worse and he's getting better."
Until the Food and Drug Administration allows it, however, no one can receive the drug outside of this one clinical trial being tested at Nationwide Children's Hospital in Columbus, Ohio. Of course there have to be guidelines and limits on new drugs, but in a heartbreaking case like this that involves brothers, you'd think compassion would win out. So far, however, it has not, and Jenn is left fighting and hoping. She said without the drug, Austin will likely die in the next six or seven years.
She isn't giving up without a fight though and has asked the FDA for accelerated approval of the drug so that Austin, as well as others, may have access to the drug sooner. The FDA told NBC it's currently reviewing the application, and hopefully they will do so quickly for the sake of this family and others.
Can you imagine being in the position of this mother?
Image via Today