The Washington PostDemocracy Dies in Darkness

‘How do you tell a kid he’s not going to grow up?’

"You wake up in the morning, and you think your kid is fine, and you go to bed that night, and you know they're dying," Mindy Leffler says, describing what it felt like to receive a diagnosis that her son Aidan has Duchenne Muscular Dystrophy (DMD), a debilitating and ultimately fatal rare disease that overwhelmingly strikes young boys.

The Lefflers, who live near Seattle, are one of four families featured in a new documentary that focuses on the very human struggle behind the disease and the ongoing push to get the Food and Drug Administration to speed up potential treatments for boys with DMD before their time runs out. The film, called "To The Edge of the Sky," is a project by producers Todd and Jedd Wider, whose documentaries include the Oscar-winning "Taxi to the Dark Side."

The Widers, with the support of the families involved in the documentary, have launched a Kickstarter campaign to raise funds they say are necessary to accelerate the post-production and editing process and distribute it widely.

At the heart of the film lie tough questions that go beyond just Duchenne and apply to a range of fatal diseases that currently have no cure: How should federal regulators balance their obligation to ensure the safety and effectiveness of new drugs with getting promising treatments to terminally ill patients as quickly as possible? How much data is enough data to make that call? How much risk is acceptable, particularly when the alternative is death?

The FDA has long wrestled with such difficult questions. But never has the spotlight been as intense, the pressure to move rapidly as constant, as in recent years, when patients and their families can harness the organizing power of the Internet to make their voices heard.

Patients with ALS, for example, also have publicly lobbied the agency to speed potential therapies to market, in hopes of saving thousands of sufferers who otherwise will die. Numerous states have passed "right to try" laws that, at least in theory, grant terminally ill patients access to drugs not yet approved by the FDA.

[Right-to-try laws spur debate over dying patients access to experimental drugs]

But of all the battles over access to experimental drugs for dying patients, one of the most passionate and most poignant involves Duchenne. The devastating disease leaves most patients wheelchair-bound by their teens and dead in their 20s. It affects an estimate 15,000 young males in the United States, and currently no cure exists.

In recent years, families of boys with Duchenne have waged an unrelenting campaign to convince, cajole, pressure and persuade the FDA to fast track approval of promising new therapies before another generation of children with the disease runs out of time. In particular, they have advocated for a drug called eteplirsen, produced by Massachusetts-based Sarepta Therapeutics, which appears to have slowed the progression of Duchenne in a dozen boys who received the treatment as part of a small clinical trial over the past several years. The results of the trial have given hope to families desperate for a treatment, even as regulators have expressed a desire for more data before making a decision.

The story has included many twists and turns, from face-to-face meetings with the FDA, to Facebook campaigns and White House petitions, to the abrupt recent firing of the company's outspoken chief executive. At the same time, other potential treatments for the disease also are in the pipeline.

But ultimately, "To the Edge of the Sky" is about the struggle of families thrust into a heartbreaking situation, and their determined race against time.

"We wanted to show what parents experience when a child is stricken with a deadly illness, and the efforts that they will take, and the lengths they will go to, to save their children," Jedd Wider says in the film's trailer.

"These families were all living normal lives. When they got these diagnoses, their worlds were transformed; they were transformed into activists," adds Todd Wider.

One of those activists is Jenn McNary, whose younger son, Max, was one of the dozen boys in the original trial for the Sarepta drug. Her older son, Austin, also has Duchenne but did not qualify for the trial. McNary told The Washington Post last year that Max had flourished during his time on the drug, even as his older brother was confined to a wheelchair and had begun to lose the ability to feed himself.

"I really thought both of my boys had a death sentence, and I was preparing for that," McNary says in the film. "[But] I could have the first child in history to survive this disease, and I could have the last kid to die from it -- living in the same house."