Local mother in DC fighting for approval for Duchenne drug

BOSTON — An experimental treatment for Duchenne Muscular Dystrophy is being developed locally, but there has been a major setback for families who want that drug approved.

FOX25’s Heather Hegedus spoke with a local mother who's in the nation's capital making her case.

Christine McSherry said she believes her son is alive, thanks to this experimental drug that's being developed in Cambridge.

Monday night she was there as an FDA panel delivered some very disappointing news.

Still, Christine and other parents say it's not over yet.

More than a thousand people packed a room in Maryland for 11 hours to push a deeply skeptical FDA to accelerate approval of a new drug for the treatment of Duchenne.

“We expect you to recognize the safety and effectiveness of this drug,” one parent at the hearing said.

Duchenne gradually weakens muscles in boys resulting in a cruel decline and death by the mid-twenties.

But after three years on the drug Eteplirsen, which is manufactured by Cambridge biotech company Sarepta Therapeutics, ten of 12 boys in a clinical trial were able to continue walking.

“Andrew has experienced zero negative side effects on Eteplirsen,” Jodi Nichols, a parent, said.

Kingston's Christine McSherry Skyped with FOX25 from Capitol Hill Tuesday and said that after being on the drug for a little over a year her 20-year-old son Jett has gained skills that he had lost.

“Jett is able to open a bag of chips, a bottle of water, he's feeding himself much better now, he's able to get his computer off his desk,” she said.

But despite countless compelling stories like that, the FDA's advisory committee picked apart the drug maker's study and voted the drug has not shown to be effective in helping patients walk again.

The vote is non-binding, but it does carry weight in the FDA's final decision whether to approve the drug.

McSherry says she remains hopeful and families made a strong case that the FDA will take into consideration.

“The ball's still in the air. Right? So the community came and did their part and now we wait until May 26,” she said.

May 26 is when the FDA's final decision is expected to be made.

FOX25 also reached out to the Muscular Dystrophy Association who tells us they too remain enthusiastic that some of those families’ stories will resonate with the FDA, and lead to approval.

But for now, the agonizing wait continues for thousands of young patients including McSherry’s son whose time could literally be running out.

Cox Media Group