Aidan Leffler is doing well with his latest treatment for Duchenne Muscular Dystrophy, but Mindy Leffler continues to fight for better drugs to save her son’s life. That work had been done mostly behind-the-scenes, until a documentary film crew came knocking.
DMD is a rare form of muscular dystrophy that develops in young boys, and is 100-percent fatal. Most who suffer from DMD don’t live to see 20.
Mindy Leffler has been campaigning with other parents across the nation to convince the Food and Drug Administration to clear new, more effective drugs that are stalled by the agency’s bureaucratic process for testing. Given the rarity of DMD — meaning a small field for drug testing — parents fighting for their children’s lives argue such drugs should be made available more quickly.
“It’s remarkable, it’s so crystal clear that it’s working,” Leffler said of the drugs. “At this point, we’re just hoping they will take a new drug application from the company without asking for another round of study information.”
The Lefflers and three other families are featured in “To the Edge of the Sky,” a documentary by filmmakers Todd and Jedd Wider, who are raising funds through Kickstarter to complete the film and raise awareness about DMD.
“These parents and these families have been with this disease for almost the entirety of their sons’ lives,” said Jedd Wider, who was inspired to make the documentary when he heard a speech by a father with a son suffering from DMD. “They are fully aware as to the drugs that are attempting to work their way through the FDA approval process.”
“The impetus on these parents is to do all they can on god’s earth to save their children,” said Todd Wider. “Each set of parents is heroic, in their own way, and they’re very compelling to watch on the screen.”
The Widers say Mindy Leffler’s intelligence and determination to save her 11-year-old son, who has lived with DMD since he was two, encouraged them to add the family to the documentary. Leffler said the film crew interviewed and followed her and Aidan last year over two weekends.
At that time, Aidan was in worse shape, having recently broken his femur — his legs frequently collapsing under him. Leffler said Aidan is doing better with his newest treatment, running around as much as he can and dreaming of being a soccer player.
“He was pissed off,” Leffler said of the documentary crew’s presence. “Aidan wants to be normal so badly, so anything that’s a reminder to Aidan that he’s not normal — right now at this stage in his life —he hates it. He just wants to be a soccer player so badly. He’s really cooperative because he’s such an awesome kid.”
Leffler said she knows the challenges she’s facing, trying to convince the FDA that new drug treatments for DMD need to be approved more quickly, as time is of the essence.
“I don’t like being criticized for what I’m doing, especially something that’s so personal,” Leffler said. “I’m on Team Aidan.”
The directors of “Taxi to the Dark Side” and “Semper Fi: Always Faithful” are about $48,000 into their $125,000 Kickstarter campaign, hoping it will allow them to expedite post-production and get the film out faster. The Widers also hope it will make the FDA take DMD and the potentially life-saving drugs waiting to come to market more seriously.
“Some boys are not aware at all as to the consequences of the disease itself and what’s in store for them in the near future, if a drug is not approved or a drug is not found,” said Jedd Wider. “As filmmakers, it’s very emotional. It’s one of the most emotional things that we’ve done.”