The wait
This is Chapter 7 of the eight-part series Lincoln’s Shot. Click here for the other chapters.
Their backyard always had been a jungle. Thick palmettos, thorny vines, tangled mounds of underbrush.
No one went out there, or even saw it.
Maggie Hoyle-Germann and Anthony DeLuna kept the sliding glass doors covered so the light wouldn’t bother Lincoln.
But on a sunny afternoon in October 2017, Maggie opened the curtains.
“What’s Daddy doing out there?” she asked Lincoln, who was propped up on his bed in the living room. “Is he working? Yes, he’s sweating.”
For almost four years, they had holed up in their Tampa home, held hostage by Lincoln’s illness, knowing every day he might die. He had a rare, terminal muscular disease called X-linked myotubular myopathy. No one had expected him to live to his second birthday.
So Maggie and Anthony hadn’t dared to picture what Lincoln might be like, what he might be able to do, someday.
But now that they had a date to get that shot on Nov. 6, now that the impossible seemed real, they began letting themselves dream: What if Lincoln could breathe on his own? Talk? Taste sausage gravy and biscuits?
They started making a life list — the opposite of a bucket list.
They wanted to take him camping, to see fireflies. Anthony wanted his son to sit beside him on the sofa and play Super Mario Bros. Maggie wanted to dance with her boy at his bar mitzvah.
“He’s going to want a swing set,” Anthony had told Maggie late one night. “With a big slide and a rock climbing wall. Kids like that stuff.”
The next day, Anthony went to Sam’s Club and bought a tiller, rake, clippers, weed killer, mulch. He hacked through thick stalks, tore down spiky vines, planted grass. In a couple of weeks, he tripled the open space in their yard.
“Look how much Daddy’s already cleared out!” Maggie said.
They didn’t tell Lincoln about Anthony’s plans. They wanted the playground to be a surprise.
How do you explain to a pre-schooler who has spent his entire life plugged into machines, lying limp on a bed, that soon he might be running around?
Lincoln had seen videos of kids playing on YouTube. He had seen boys batting baseballs on TV. He had never asked his parents why he couldn’t do those things, why he could barely move.
And they had never tried to explain. They didn’t want him to feel sorry for himself, or worry. “You have your mind,” Anthony kept telling him. “You’re smarter than them all.”
Still, Maggie and Anthony wanted to tell him things were about to change. Would he understand? How much hope should they give him?
Lincoln’s favorite movie then was the animated Sing. In it, a hefty hog named Gunter, who has a thick German accent, brags about “Piggy Power.” Each time he performs, Gunter flattens his front legs against his sides, points his hooves to the sky and wriggles his shoulders to show the super strength he is about to exhibit.
“You’re going to get some special medicine that will give you lots of Piggy Power,” Maggie told Lincoln just before Halloween. “You’re going to get super strong.”
In early November, Maggie texted Angelica Santiago-Townshend, wishing her son good luck. Jamie, who lived in Melbourne, was going to be the third boy in the country given the gene therapy, the first treated at the University of Florida.
“Jamie got his Piggy Power today,” Maggie told Lincoln that night. “Yours is coming soon!”
The next week, someone from UF called with bad news. The gene therapy had triggered an adverse reaction. Lincoln would have to wait to get his shot, probably until January.
Maggie collapsed, weeping.
They’d been hoping for so long, and the cure had been so close.
Another two months felt like forever.
Three boys with X-linked myotubular myopathy — including the Orlando toddler who had been waiting for his shot at UF — had died in the last three weeks.
She sent an email to Audentes, the bio-tech company sponsoring the clinical trial.
“You have to speed up,” she wrote. “Boys are dying.”
“This is the gamble of a life”
When Lincoln turned 4 on Dec. 14, no one celebrated.
Maggie asked her family to postpone the party until he got the shot, until he could blow out his own candles.
That night, as they were singing Lincoln to sleep, Maggie got an email from UF. Lincoln had a new date: Jan. 4.
The next day, his speech therapist made him a paper chain with 21 links.
Each morning, he ripped off a ring while Maggie counted down the days.
Another email came at the end of December. Doctors at UF were still trying to figure out what had gone wrong, why that first boy had gotten so sick. They wanted to make sure the next patient wouldn’t have the same reaction.
They wouldn’t give Maggie specifics. But from talking to Angelica, she knew that Jamie’s heart had swollen after the shot. His body was trying to reject the new protein. Doctors had put him on steroids and anti-rejection medication.
Now, two months later, he was home and doing better. He was starting to show signs that the shot was working.
Still, doctors said, Lincoln would have to wait. Again.
They gave him another date, less than a week away: Jan. 10.
Maggie’s family wanted to be there when Lincoln got his shot. But by the time her aunt and uncle flew in from Iowa, and her mother and stepfather drove down from the Panhandle, UF had cancelled for the third time.
This time, doctors wouldn’t say when the trial might resume.
“How can they keep doing this to us?” Maggie said.
Lincoln was getting more uncomfortable, having more trouble breathing. As his body grew, gravity was taking a toll on his bones and organs. He couldn’t bear being upright, in his stander or even stroller.
Maggie tore down the paper chain and stopped talking about Piggy Power. Anthony quit working on the playground.
On Jan. 10, the day Lincoln was supposed to get his shot, his family instead gathered around his bed in the living room. Maggie propped her cell phone on the bookshelf and clicked on the speaker. Then she hit record.
“Shhh!” she told everyone. “It’s about to start.”
That afternoon, Audentes was going to update investors. Through the company’s website, Lincoln’s family could listen to a livestream. Since UF wouldn’t tell them what was going on, they hoped the company CEO would.
Maggie’s sister, Katie, had moved out, to go to school in Fort Lauderdale, so Maggie had promised to text her an update.
“I’m so nervous,” Maggie whispered, snuggling on a sofa against her aunt. “It better be good news.”
Anthony and his mom were on another couch. She kept squeezing his knee.
They all sat, staring at the cell phone.
“We have a big 2018 coming up, and we’re really excited,” Matt Patterson announced on the webcast. “We are happy to report early data here.”
So far, he said, three boys had been dosed, ages 9 months, 2.5 and 4 years.
The baby had improved the most. He already was breathing on his own, able to swallow, eat and hold up his head. He could even sit up.
“If that doesn’t inspire you,” Patterson said, “I don’t know what would.”
Anthony’s mom gasped. Maggie ran to the bathroom to get tissues.
“It’s likely that we’ll continue to see positive results, even in those who have been living with this disease for some time,” Patterson said. “So we’re going to go ahead and treat three additional boys.”
Maggie started screaming. Anthony’s mom got up to kiss Lincoln. “Isn’t that wonderful?” asked Maggie’s aunt.
Anthony was still and silent. Soon, everyone was staring at him. “Did you hear that?” Maggie asked, coming over to sit in his lap. “I can’t wait for us to be able to live like a normal family.”
Anthony shook his head. “I won’t believe it until the needle is in his arm.”
They held hands around the dining room table that night, and while a nurse watched Lincoln, Anthony led his family in prayer: “Thank you for the news today. Please embolden us to stay firm and strong.”
He and Maggie sang a healing prayer in Hebrew. Then, over taco salad, the talk turned to science and God.
During their first years as a couple, Maggie and Anthony celebrated Christmas and Hanukkah but seldom went to church or synagogue.
Over the last year, though, they had found themselves searching for a spiritual buoy. They had started talking to the rabbi at Congregation Beth Am in Tampa, the synagogue Anthony’s great-grandparents had attended. They were trying to take the rabbi’s advice, to stop asking, “Why us?” and start thinking, “What now?”
He told them to celebrate their blessings: They had their son, and they had hope.
“But what is God’s role in all of this?” Maggie had asked Rabbi Jason Rosenberg.
The rabbi looked at his lap, then locked eyes with her. “I don’t believe God is a puppet master, controlling the world. Or that he sat up there in the high heavens, looked at you, and said, ‘I’m going to give their child a disease.’
“If that’s how God works, I need a new job.”
The rabbi continued: “One of the things that people say that makes me want to punch them in the face is, ‘God doesn’t give anyone more than they can handle.’ He does. All the time.”
And it’s okay to be angry at God, the rabbi told Maggie. Just be sure to be grateful.
“The fact that this gene therapy is even coming,” he said, “is a miracle.”
At dinner with her family, Maggie related some of the rabbi’s insight and wondered whether God had a hand in helping cure Lincoln. Could it really be a coincidence, she asked, all this science coming together, finding the carrier dog and millionaire investors, just as the government was approving ground-breaking trials?
“You know they’re talking about, one day, being able to treat MTM boys with the gene therapy before they’re even born, in utero,” Maggie told her family. “Eventually, they might even be able to cure carriers.”
Anthony laughed. “Wouldn’t that be the ultimate slap in the face of God?”
“If God had done his job, we wouldn’t have to be here messing with genetics,” said Maggie’s aunt and adoptive mother, Donna Germann. “We already buried three boys. That’s too many for one family.”
No one was trying to talk them out of getting Lincoln the shot or dwelling on the dangers of such a revolutionary treatment.
But on Maggie’s Facebook group, a debate was waging. A mom of two XLMTM boys who had lived to be college-age, long past the clinical trial cutoff of 5 years old, posted that gene therapy was morally wrong. God had made her boys the way they were.
Anthony’s mom, Ronda Clark, urged Anthony and Maggie to stop worrying. “Just let God take care of this,” she said. “He made Lincoln. Why don’t you let him just take over?”
“It’s not that easy,” Anthony said, his voice tight. “As long as he’s willing to hold on, we have to keep fighting for him.”
“Whatever happens,” Maggie said, “we have to be able to say we did everything we could.”
Maggie called UF a month later. They still didn’t have any news. A couple of weeks after that, she called again. Then emailed.
Finally, a doctor told her: the trial was on hold there.
He wouldn’t say why. Or what would happen next. He couldn’t tell her what was going on at other test sites.
Maggie’s despair turned to anger.
She asked Anthony, “Are we going to be left behind?”
The next week, on another Audentes webcast, Maggie learned that three more boys would be given the low dose in the coming weeks. Would those boys be chosen from other hospitals, in other cities?
Could Lincoln get the treatment somewhere else?
In late February, Lincoln started running a fever, and gagging on globs of mucus. Maggie and Anthony gave him antibiotics, to fight off what they thought was a cold.
A week later, his blood pressure dropped, his body bloated. His tongue went white. Maggie and Anthony, and a series of home health nurses, tried to stabilize him. But Lincoln was in too much distress. For the first time, they rushed him to the emergency room.
At St. Joseph’s Children’s Hospital, doctors told them that Lincoln’s liver was hemorrhaging. A black mass of blood was covering most of it. He needed emergency surgery.
The antibiotics could have wiped out his platelets, causing the bleed, doctors said. Something could have bruised his side, causing the organ to rupture. Or he could have some sort of liver disease.
As nurses wheeled Lincoln away on a gurney, as weak as he was, he reached out and picked up his red-rimmed mirror. Maggie and Anthony watched through tears as he stared at them behind him, getting smaller and smaller.
Lincoln’s liver was too soft to sew closed, too covered in blood to cauterize. Surgeons had to create a mesh casing to hold it together. No one thought he would survive.
Except Maggie.
In the hospital waiting room, she kicked chairs and flipped tables, shouting, over and over, begging her son. “You’re not allowed to die. Hold on. Fight. You can’t leave. You’re about to be cured.”
She reached out on Facebook, asking for prayers.
When Lincoln came through, she posted videos of him in his hospital bed, giving a weak thumbs up. He had a thick scar from his chest to his pelvis. One of her late-night livestreams got 7,000 views. Maggie told Anthony, “I don’t even know 500 people.”
After eight days, doctors finally discharged Lincoln. They wanted him back in a couple of months for a liver scan. If the bleeding had been caused by antibiotics, or a bruise, his liver would be healed by then.
But if the organ was diseased, doctors would need to figure out treatment — and remove him from the clinical trial.
March crawled by, then April. Lincoln went back to working with his therapists. A liver scan showed no sign of disease.
In May, Maggie learned that two more boys had gotten the shot, in Chicago and Los Angeles, both babies. That made a total of five patients. “I thought whatever safety concern they had, it was for the whole trial, all the sites, not just UF,” Maggie said to Anthony. “This is so unfair! Lincoln was supposed to be boy #4. They had his dose waiting in the freezer.”
Maggie worried that Lincoln was starting to focus on things he couldn’t do. He kept flexing his feet, saying he wanted to run, signing that he wanted to eat a granola bar.
June brought no news. July, only more questions.
No one at UF would tell Maggie and Anthony, officially, what was going on — or even if Gainesville was still a test site. One doctor ordered them not to contact the drug company, saying such interaction would jeopardize Lincoln’s enrollment in the trial.
Through other MTM moms, and some hushed conversations at the hospital, Maggie learned that UF wanted to change the protocol for the gene therapy — and add an anti-rejection drug so other boys wouldn’t have adverse reactions.
But that would mean going back to the Food and Drug Administration and starting the whole process over.
Audentes didn’t want to do that. Only one boy who had been treated had a bad response, and he was doing well now.
So the Internal Review Board at UF, which regulates clinical trials, and the biotech company, which sponsored the trial, were at an impasse.
Until they could work something out, UF wasn’t going to dose any more boys. It reported concerns to the FDA about “subject privacy and the management of adverse events.”
Lincoln was getting tangled in bureaucratic safety nets.
We’re working on it, doctors there assured Maggie. We’ll get back to you.
She couldn’t concentrate. She was constantly distracted, anxious, angry. She had to stop talking to Angelica because she couldn’t stand to hear how much Jamie was improving.
At the end of July, she emailed UF: “You’re failing Lincoln.”
She felt like she was failing him, too.
Click here for Chapter 8: The Answer
Senior news researcher Caryn Baird contributed to this story.
Contact Lane DeGregory at [email protected]. Follow @LaneDeGregory.