‘There’s nothing you can do. He will be dead in five years.”
That’s what a neurologist told Christine and Stephen McSherry in February 2001. The Pembroke couple had five young children, all 14 months apart, and their middle child, 5-year-old Jett, had just been diagnosed with Duchenne muscular dystrophy, a fatal neuromuscular disease that paralyzes boys.
“We had 40 children coming to our house for a birthday party that afternoon and I couldn’t breathe. It took every ounce of energy just to stand up,” Christine told me 18 months later, when we first met. After the party, she and her husband closed the door and spent the weekend “mourning the loss of a healthy child.”
And then they came out fighting.
I don’t know how people get up off the couch after they’re told there’s no hope, how they have the energy to breathe, never mind fight. But they did.
They created the Jett Foundation in 2001 and began raising money for research for a condition which affects one in 3,600 boys. They had a fund-raiser in their back yard, and local people and businesses heard what they were doing and donated. They have been raising money, $13 million to date, and awareness ever since.
From the beginning, Christine said, “Jett was born for a reason. This is the reason I became a nurse. And this is why God gave me the voice to speak.”
It’s her voice, a mother’s voice full of love and passion and conviction and facts, that has altered history. In February, Christine stood before the Food and Drug Administration and talked about a new drug, eteplirsen, produced by Cambridge-based Sarepta Therapeutic, which has stopped the progression of Duchenne’s in 12 boys.
She told how the drug worked. She told about a child who couldn’t walk, who two years later took her breath away when he ran past her. “Twelve lucky boys have been on this medication for over 120 weeks. There are no side effects. They are not getting worse, they are getting better. Their muscles are no longer wasting away.”
She voiced her frustration. “As the FDA sifts through paperwork, and more and more bureaucracy, our children are dying.”
The FDA listened and last month, in an unprecedented decision, announced that it will consider accelerated approval for eteplirsen. This is not a miracle drug for all kids with Duchenne, but for the 15 percent whose abnormality is on a specific part of the dystrophin gene, it may be.
The child who raced past Christine, 12-year-old Max McNarry, remains stable.
“He’s learning how to ride a bike. He’s a child whose quality of life has been resurrected,” said his mother, Jenn.
His 15-year-old brother, Austin, also has Duchenne, but is not on the drug. He’s in a wheelchair.
Jett McSherry has outlived his prognosis. The doctor was wrong; most boys with Duchenne’s live into their late teens or early 20s. But Jett is in a wheelchair, too, and is losing the ability to move his hands. This is what happens.
Jenn McNarry, also from Pembroke, has for the last two years worked with Christine and another mom, Mindy Leffler, of Bellevue, Wash., to get the FDA to understand this, to recognize that every day kids with Duchenne’s lose more muscle.
These women weren’t always knocking on congressmen’s doors, advocating and educating. They learned because of their kids.
Christine McSherry credits the Abigail Alliance, dedicated to getting wider access to drugs for life-threatening illnesses, for getting the FDA to listen. “That was the first step in this whole process.”
There have been countless steps. “We had to learn the laws and the terms and find the people to teach us. Lots of people helped us.”
Her job now, she says, is to make sure the FDA and the drug company stay on track. “This isn’t going to end here. There needs to be drugs and treatments for all the kids.”
A doctor told her, “There’s nothing you can do.”
She tells her kids there’s always something you can do. “You don’t have to take the road everyone else takes. You can make your own road.”
And go places where no one has gone before.
Visit jettfoundation.org for information about the Jett Foundation and how to help.
Beverly Beckham can be reached at firstname.lastname@example.org.