Vt. Mom Begs FDA: Save My Other Son

Good Morning America

Jenn McNary, a mother of six from Saxtons River, Vt., is desperate.

Both her boys have Duchenne muscular dystrophy, but only her 10-year-old Max has access to a wonder drug that appears to be reversing the symptoms of this deadly disease.

His 13-year-old brother Austin is languishing in a wheelchair while Max has been able to take the drug eteplirsen through a highly successful clinical trial.

After 60 weeks on an IV infusion, Max was able to participate in a three-mile Halloween walk.

"It's the first time ever -- he's never been able to walk that far. He's always gone with a wheelchair, even as a toddler," said McNary, 32. "He actually doesn't look like a Duchenne kid at all. And his balance is great."

"People all over the world are calling it a miracle," she said of the drug.

Now, McNary has written petitioned the Food and Drug Administration to give accelerated approval of the medication, the fastest way to help Austin and other boys with the disease.

Both boys, whose last name is Leclair, have the same gene mutation that the drug targets and will eventually kill them. Austin was diagnosed at 3 and Max at 3 months.

McNary and her husband Craig are also raising four other healthy children in a second marriage.

There is no cure for Duchenne muscular dystrophy. Until now, doctors have only been able to use steroids, which just temporarily delay the inevitable loss of muscle strength.

"My brother says he's doing it for me, that he's trying really hard," Austin told ABCNews.com in August. "That's why he wanted to do it."

Austin was not allowed to participate in the clinical trial because one of the inclusion criteria was that he be able to complete a six-minute walk.

"This has been a bitter-sweet journey for us," McNary wrote in a letter to the FDA this week. "As we watch Max get better, we also watch his older brother, Austin, 13, get worse. He suffers, silently, as his disease progresses."

Duchenne muscular dystrophy affects one in 3,500 male births, about 20,000 children in the United States and 300,000 worldwide, according to Cure Duchenne, one of three organizations that have funded the clinical trial.

The muscular disease strikes between the ages of 3 and 5 as boys progressively lose their ability to walk. Eventually, they are wheelchair bound, their upper body strength fails, and, like Austin, they eventually cannot raise their arms to feed themselves.

Later, their breathing is affected and they require tracheotomies and breathing assistance. Eventually, the heart and lungs fail.

Parents of children who were in the clinical trial of eteplirsen at Nationwide Children's Hospital in Columbus are calling it a "wonder drug."

According to McNary, all 12 children in the double-blind study received "some benefit" from the drug. It has no known side effects.

"Even two boys who stopped walking before taking it have stronger upper bodies and their hearts are strong," she said. "They have progressed to stable."

Muscular Dystrophy Drug Could Stabilize Disease

If this exon-skipping drug is approved, she estimates 15 percent of boys with Duchenne could be helped, those with the type that skips exon 51. As a class of drugs, they could help up to 85 percent of boys with the disease.

View photo

Austin Leclair dressed as a hot dog stand on Halloween. Meanwhile, on the new drug, Max walked three miles without a wheelchair.

Stock prices for its manufacturer, Sarepta Therapeutics. , have soared.

If Sarepta Therapeutics can get accelerated approval, the drug could be available in six to nine months, according to McNary. Otherwise, the wait could be four or five years -- too late for Austin.

"We are very encouraged by the data we have seen to date," said Chris Garabedian, president and CEO of Sarepta Therapeutics, which makes the drug and is pressing the FDA to take action.

"If we start using the drug earlier in patients, we might be able to stabilize whatever state they are in for a longer period of time," said Garabedian. "We are not going to end up creating Olympic athletes from this drug, but we are encouraged this could really halt or slow the progression."

McNary is reaching out to media and online petition sites to encourage as many people as possible to write letters of support to the FDA.

But as she waits approval, Austin gets weaker. In the last few months, he has lost all upper body control and must be lifted 100 percent of the time.

Just recently, he was diagnosed with sleep apnea and must go on a nighttime machine to keep his lungs inflated.

Austin keeps his spirits high, according to McNary.

On Halloween, he dressed his wheelchair up as a hot dog stand, carrying his dachshund in a cloth bun. And just recently, his father and uncle took him hunting. They held up the gun for Austin and he shot his first buck -- an eight-pointer.

McNary is convinced that if the FDA can move on approving the drug that has healed Max, it can also help Austin.

Until then, he's "hanging in there," she said. "He has a huge zest for life."

View Comments (6)

Recommended for You

  • Planned Parenthood Shooting Suspect Was Monitored by Security Cameras as Standoff Unfolded

    A man accused of killing three people -- including a police officer -- in a shooting at a Planned Parenthood clinic in Colorado Springs was monitored by security cameras as the dramatic standoff unfolded, an official said. After the suspected shooter, identified by Colorado Springs Police as…

    Good Morning America
  • University of Colorado Colorado Springs Plans Tribute to Officer Killed in Planned Parenthood Shooting

    The University of Colorado at Colorado Springs said today it would honor a campus police officer who was killed in a shooting at a Planned Parenthood clinic on Friday. Both the men's and women's basketball teams will observe a moment of silence in honor of Officer Garrett Swasey at their games…

    Good Morning America
  • Planned Parenthood Shooting Suspect Fired at Random, Witness Says

    A woman who was caught in the crossfire during a shooting at a Planned Parenthood clinic in Colorado Springs Friday said the suspect fired at random. Ketanya Craion was in an exam room when the shooting began Friday morning. Three people were killed and nine others were injured before police…

    Good Morning America
  • 3 Killed in Colorado Springs Shooting, 9 Victims in Hospital

    Two civilians and one police officer were killed at the Planned Parenthood building in Colorado Springs, Colo., Friday. The gunman has been detained, according to the Colorado Springs police department, and nine individuals, including five police officers, have been wounded. Federal law…

    Good Morning America
  • Colorado Springs Shooting: Man Hears Gunshots While Speaking to Sister at Planned Parenthood

    A woman inside the Planned Parenthood near a shooting in Colorado Springs Friday called her brother while shots were being fired, he said. Joan Motolilia spoke to ABC News and said that he got a call from his sister as the shooting was happening. Motolilia said his sister, whose name he did not…

    Good Morning America
  • Family of Belgian Terror Suspect Insists He Didn't Help Paris Attackers

    The family of wanted Belgian terror suspect Mohamed Abrini insisted he was innocent of helping the Paris attackers in an exclusive interview with ABC News. It was not clear when he allegedly traveled to Syria.

    Good Morning America
  • 'Rocky IV' Celebrates 30 Years: Here Are the Top 5 Lines From the Movie

    Widely regarded as one of the best "Rocky" films, the "Italian Stallion" is tasked with beating the unbeatable Russian Ivan Drago after Drago beats and kills his dear friend Apollo Creed. This is what Drago says after knocking out Creed in an exhibition match! Creed is bloodied and dies in Rocky's…

    Good Morning America
  • Black Friday Gets Off to a Roaring Start

    With major retailers including Walmart, Target and Macy's opening on Thanksgiving, some eager shoppers were able to get a jump start on their holiday to-do lists. The Black Friday frenzy was in full force at Macy's flagship Herald Square location in New York City where an estimated 15,000 people…

    Good Morning America
  • Chilling New Surveillance Video Shows Moments Leading Up to Shooting of Tulane Med Student

    New surveillance video shows a clearer picture of the moments leading up to the shooting of 25-year-old Tulane medical student Peter Gold as he stepped in to help a woman who was being dragged by a man on a New Orleans street last Friday. Peter Gold is seen stopping his car in the middle of the…

    Good Morning America
  • How the Kardashians and Jenners Celebrated Thanksgiving

    For Thanksgiving, Kylie Jenner shared a sweet photo of her entire "blended family" that includes Caitlyn, Kris and Kendall Jenner; Kourtney Kardashian and Scott Disick; Kim and Kanye West, and all the little kids like Penelope and North.Read: Khloe Kardashian Opens Up About Lamar Odom, Relationship…

    Good Morning America
  • Man Trains His Dog to Catch a Lobster

    Alex Schulze found a way to make man’s best friend even better. The co-founder of Devoted to the Ocean, an online retailer that donates a portion of its profits to ocean conservation, taught his black Labrador dog to catch lobsters from the ocean floor. Schulze posted a video of his dog, Lila, in…

    Good Morning America
  • Zach Galligan Shares Details of 'Gremlins 3'

    "It's not going to be a reboot," he told the audience in London, who had come out for a special screening of "Gremlins," followed by questions for Galligan, according to the BBC. "It will not be a remake in any way, shape, or form.

    Good Morning America
  • Mariah Carey on Her New Children’s Christmas Book, Directorial Debut and Holidays

    Her hit song, “All I Want for Christmas is You” is a classic, and now she’s made it into a children’s book. ABC News’ Amy Robach sat down with the superstar to talk about the book, her directorial debut and the holidays in the Carey home. “’All I Want for Christmas is You’is one of the all-time…

    Good Morning America
  • Best Shopping Deals for Black Friday

    Black Friday is now a season. Planning your shopping is key to deal hunting.The good news is that online deals are almost as good as the in-store deals, with a few notable door-buster exceptions. Even more interesting is the adoption of mobile-based deals that retailers are offering to entice you…

    Good Morning America
  • Your Black Friday Guide to Store Hours and the Best Deals

    There will be no rest for serious Black Friday shoppers. Just as the turkey, mashed potatoes and pumpkin pie feasts begin to settle, some stores will open their doors on Thanksgiving, marking the start of the 2015 holiday shopping season. Walmart kicks off the holiday shopping season on…

    Good Morning America
  • White House Lawn Locked Down Over Fence Jumper

    The North Lawn of the White House went on lockdown after a man identified as Joseph Caputo jumped over the fence and was taken into custody, authorities said. “Today, at approximately 2:45 p.m., Joseph Caputo scaled over the north fence line of the White House Complex gaining access to the North…

    Good Morning America