Ryan Dunne is 9 years old and terminally ill. He has Duchenne Muscular Dystrophy, and it’s destroying all the muscles in his body. Soon, he won’t be able to walk. Eventually, he won’t be able to breathe.
Last year, Ryan and his family found hope. He took part in a 6-month clinical trial of a new drug, one of two that have shown great promise. Ryan’s parents told CBS4Denver’s investigative reporter Brian Maass that the drug worked wonders:
“When there was no hope, all of a sudden things were getting better,” said Ryan’s father, Chris.
“He walked further, had better stamina and energy and didn’t fall into bed saying, ‘I’m tired,’ “ said his mother. “And when he was pulled off of it he went downhill immediately. The drug is effective.”
The other drug markedly improved 100% of the kids who received it, and there were no side effects.
But the trial in which Ryan was enrolled is over. And there are years to go before the FDA eventually approves either of the drugs — if it ever does.
Thousands of boys suffer from the same debilitating, fatal disease, and the FDA has been asked to grant “accelerated approval” to the drugs that appear to be their life-savers. The FDA response? It says that it has an “evolving position on these drugs” and “has reached no conclusions.” In other words, don’t call us, we’ll call you.
In Colorado, Democrats and Republicans have joined forces in support of a bill (HB1281) that gives the terminally ill the right to use experimental or investigational drugs without FDA approval. The bill has all the safeguards you’d expect: no insurance coverage, no suing if things don’t work out, a doctor must sign off, and the drug maker must agree to provide the medicine. Today, HB1281 cleared its first hurdle when it passed out of committee on a 10-1 vote.
Opponents seem few and their arguments lame. On a recent newscast, I heard one lawmaker object that Colorado can’t enact a law that challenges the FDA’s authority — apparently not a fan of the growing 10th Amendment movement. Another worried that without FDA approval, a drug might not be safe. But to a terminally ill person, any side effect from a drug that seems to work is preferable to the “side effect” of waiting — death.
Similar “Right to Try” bills are under consideration in other states. If you’re in Colorado or one of those states, urge your state legislators to support such legislation. There is also an online petition urging the FDA to grant accelerated approval for one of the DMD drugs, eteplirsen. Please go to HelpRyanDunne.com for more information and to sign it.
UPDATE: The petition has surpassed the 100,000 signatures needed to get a response from the Obama administration. But 200,000 signatures might elicit a better response, so if you haven’t signed yet…
Nonetheless, some of the scores are interesting: