It should have been an occasion for celebration, for elation, for deep satisfaction over achieving what had often seemed an impossible goal.
And Christine McSherry did feel a sense of joy. For 15 years, the Pembroke mother has waged an unceasing crusade to find a cure for the ghastly muscle-wasting disease that afflicts her son. While she battled, she watched desperately as Jett, her middle child, gradually lost his ability to walk, to use the bathroom without assistance, even to feed himself.
Then, last week, a surprise and controversial development: Overriding its own advisers, the Food and Drug Administration approved an experimental medication to treat the rare fatal illness, Duchenne muscular dystrophy, and authorized its immediate sale.
McSherry’s relentlessness — her fund-raising, her testimony before Congress, her appeals to federal regulators — had paid off. Now Jett, 20, along with a few thousand other boys, may have a chance at a longer life.
But that victory came at a bittersweet price.
As McSherry rejoices in what she and others accomplished, she is also mourning what was damaged along the way. Her marriage. Her relationships with some of her other four children. She feels profound regret that, in her quest to rescue one family member, her ties with the rest of her family frayed.
“I self-admittedly buried myself in my work because it was the only place I could feel useful,” said McSherry, 50. “I missed birthday parties and football games and field trips and parents’ weekends at college, all the everyday things parents do. I didn’t have time to give my marriage any attention. But when your son is diagnosed at age 5 and you’re told he’ll only live till he’s 20, that’s 15 years to get your job done.
“The irony,” she added, “is you go into it trying to save your son’s life, and you end up not only losing your life, but also losing that time you would have had with your dying child and your other kids.”
Among families of children with crippling diseases, especially uncommon ones with few or no remedies, McSherry’s story is painfully familiar.
“Very often, when it’s a progressive disease, there’s such a terrible sense of urgency that the families feel like it’s all on their shoulders, and they feel responsible for making a treatment come available to save their loved one’s life,” said Mary Dunkle of the Connecticut-based National Organization for Rare Disorders.
“I’ve seen it draw families together, and they become incredibly close with a sense of mission and focused on their goals,” she added, “and I’ve seen it tear families apart.”
Stephanie McSherry, 22, who will graduate from the University of Alabama in December, sums up her mother’s campaign this way: “The personal toll it took on our family was outrageous.”
“I don’t want to trash my mom, because I love her and she loves me and I know that,” said Stephanie. “But it was like a personal sacrifice for me and the rest of us when we needed our mom for a lot [of] stupid little problems and she wasn’t there because she didn’t have the time.
“I’m proud of everything she’s done,” she added, “but it was like we had to give her up for the greater good.”
“She’s very angry at me,” Christine McSherry said of Stephanie. “Very, very angry.”
McSherry had five children, the oldest just 7 years old, when Jett, her third-born, was diagnosed with Duchenne in 2001. Her seemingly healthy little boy was at his 5-year physical when the pediatrician noticed something odd: Jett pushed his hands off his knees to hoist himself into standing position, an indicator of a weakening torso.
Blood work was ordered, and the doctor’s suspicion was confirmed: Duchenne, the form of muscular dystrophy most likely to strike children, usually boys. Duchenne patients are typically in wheelchairs by age 12 and die by age 25.
Since Jett’s diagnosis, Christine McSherry has been on an intensifying mission to keep him alive. She created a nonprofit organization, the Jett Foundation, to raise money for research and advocacy.
She funded camps for other victims, provided services to them and their families, organized educational roundtables. She cofounded an umbrella group, the Duchenne Alliance, to coordinate similar efforts worldwide.
McSherry’s endeavor went from aggressive to obsessive about four years ago, when she learned of an experimental drug trial involving 12 boys with Duchenne who had seen improvements from the medication.
Newly energized, she began flying regularly to Washington, D.C., for meetings on Capitol Hill, to California for talks with pharmaceutical companies, to London to speak with British parliamentary officials. She became fixated on spreading the message that children were dying horrific deaths, a drug existed to possibly help them, and it should be made widely available.
“Everything I did, whether it was a social event or a family event, ended up being about this work,” McSherry said, “and the cost of that became very high.”
Her husband, Steve, essentially became a single parent for long stretches while also working full-time, taxing their relationship.
“He is resentful of the amount of time I spent away,” Christine McSherry said. “But there was no time to throw a divorce in there, no time to reach out to an attorney, no time for marriage counseling. It was like: ‘We should get divorced.’ ‘I agree.’ ‘Do you have time?’ ‘Nope.’ ‘Can we talk about it after the drug is approved?’ ‘Okay.’”
McSherry also says the couple have been reluctant to split up because they didn’t want Jett to lose the one thing he’d been able to count on his whole life: his mom and dad.
Steve McSherry declined to comment for this story, saying, “I’m not that comfortable talking about anything.”
Jackson McSherry, 19, recalls his mother’s life becoming “so busy and so complicated” that he couldn’t keep up with her whereabouts.
“She was traveling so often it got to the point where it wasn’t strange for her to be gone one day and back the next and me not even knowing she left,” said Jackson, a sophomore at the University of Missouri.
Now that a Duchenne drug has been approved, he added, “maybe she’ll be able to relax and be more of herself without always having to work.”
In approving the drug — called eteplirsen, it’s made by Cambridge-based Sarepta Therapeutics — the FDA overruled its own staff and advisers, who concluded there was not enough evidence it worked. Even if it does, it’s expected to help only 13 percent of the estimated 20,000 people in the United States with Duchenne.
But advocates like Christine McSherry begged the FDA to approve it, arguing it was their only hope.
Jordan McSherry, 23, the oldest sibling, works for the Jett Foundation, a job that’s given her more time with her mother than her siblings have had in recent years, and a better sense of why her mother acted as she did.
“When you find out a drug can help your kid and there’s no other option, you do whatever you have to do to sacrifice. It doesn’t matter how many planes you have to get on,” she said. “We lended her out to an entire community when they needed her.”
Jett himself, who Christine McSherry says has seen subtle but noteworthy benefits from the drug through a different clinical trial he entered two years ago, is also gentle on his mom.
“I didn’t feel like I lost her; it just felt like she worked a lot,” said Jett, now in his third year at Bridgewater State University, “and I understood how important the work she did was.”
The family’s youngest child, Tabor, 18, a freshman at Curry College, declined through his sister Jordan to be interviewed.
Stephanie McSherry, the University of Alabama senior, is her mother’s toughest critic, still smarting from years of long absences, distracted phone conversations, and unanswered texts.
“But I’m not angry any more,” she said. Now that a drug is available, “I’m just relieved and happy that I have my mom back. At least hopefully.”
Her crusade partly won, Christine McSherry is still reconciling her gains with the losses — hers and her family’s.
“For my kids, their mom was just gone, and I’m sure they’ll have some resentment for the rest of their lives,” she said. “But at the end of the day I think burying their brother would have been far worse than losing their mother.
“I can’t get those years back,” she added. “All I can do going forward is be the best mom I can be for my kids, and be there for them as much as I can.’”Sacha Pfeiffer can be reached at firstname.lastname@example.org. Follow her on Twitter @SachaPfeiffer.