The Cure

by Geeta Anand

  A nurse hung a plastic bag filled with a clear liquid on an IV post to Megan’s right. Day-Salvatore nodded that they were ready to go. Megan sat propped up in bed, a pink stuffed dog named Nosey that Ed had given her tucked under her knees. “Who wants to press the button?” the doctor said. John pointed to Aileen.

  “Oh, no—you’re going to be the one, John,” Aileen said. John took a deep breath, looked around the room, and lifted his right hand to a switch and pressed the “on” button. He saw the switch open and the clear liquid begin dripping. He looked at Aileen, her eyes full of tears, and without a word pulled her into his arms and kissed her.

  Megan’s heart monitor began to beep and the parents realized that their little girl, overwhelmed by the emotion of the moment, was also starting to cry. As they rushed to either side of her, she lifted one arm and waved apprehensively, and soon everyone was laughing through their tears.

  “Now for our little redheaded stepchild next door,” Aileen said, still sniffling. She and John led the entourage into the next room and waited for a nurse to bring in Patrick’s bag of medicine. As a male nurse put a clear plastic bag on the IV pole to Patrick’s left, John joked, “Careful there. That bag cost $200 million,” his estimate of what Genzyme had spent developing the drug.11

  When Day-Salvatore signaled they were ready to go, Patrick lay in bed, not even registering the presence of the group, his eyes trained on a Sponge Bob cartoon on the television monitor overhead. In one hand, he clutched his favorite toy, a little stuffed Santa Claus. “He hates all the noise and attention,” Aileen explained to the group, bending down to kiss his forehead. “He’s pretending we’re not even here.” She stepped forward and pressed the button on his IV, beginning the infusion. Then she turned to kiss John again. As they hugged, Ed put the camera down and went over to embrace them.

  Later that day, as Ed and John stood chatting in the corridor outside the children’s rooms, Ed said, “You and Aileen look happy again, John. I’m really happy to see that.”

  John nodded, quiet for a few seconds, and said, “You know, I don’t really know how it happened, but somehow I stopped blaming Aileen. Not immediately, but very slowly, over time, we forgave each other. Never in a million years could I imagine myself with anyone else. Not after what we’ve been through.”

  “I have to say, I wasn’t sure today was ever going to happen, John,” Ed said. “This is really an incredible day.”

  “It’s different from how I had imagined it so many times over the years,” John said. “There are no bells, no trumpets, no bright lights. There are just two little sick kids who desperately need some Special Medicine. No more—no less. And that’s how it should be.”12

  The children’s first infusion lasted four hours. They both grew flushed and peed nonstop, but that was the only side effect. An hour into Megan’s infusion, the phone rang on the table beside her bed. Aileen picked up and passed the phone to Megan. Her kindergarten teacher, Mr. Fletcher, was on the line, with her eighteen classmates by his side, waiting to talk to her.

  “Oh, hi, Mr. Fletcher,” Megan said, holding the receiver. “Everything’s going fine here. Can I talk to Sophie? Okay, good, Sophie. Can you put Carly on the phone?” And down the line Megan went for the next half hour, talking to every classmate.

  When the infusions ended, the children were so energized they couldn’t sleep. Megan sat up and played with her Barbie dolls with Aileen until past midnight. Day-Salvatore said it was a good sign. It meant the experimental enzyme was metabolizing glycogen and turning it into energy. Aileen beamed, thinking that perhaps this was the first time since the children were born that they were experiencing a sugar rush, like any other kid who ate too much ice cream. The next day, the children stayed in the hospital to be sure there weren’t any side effects. They would need to return to St. Peter’s for an infusion every two weeks for the foreseeable future.

  For the next few weeks, the big question behind each child’s every movement was how well Special Medicine was working. In the trials thus far using Chen’s and Pharming’s version of the enzyme, it had worked on some children but not on others. It appeared to be more effective if the children produced at least a little bit of naturally active enzyme so that the body didn’t greet the infused enzyme as a foreign substance. That was a hopeful sign for Megan and Patrick, who both made a minuscule amount of active enzyme. But also in the trials, the children who received the enzyme therapy when they were only a few months old—before their muscles were substantially damaged—seemed to do far better than those who were treated as toddlers and older, which didn’t bode well for the Crowley children. It didn’t appear that muscles damaged beyond a certain point could regenerate. The first round of tests was scheduled for early April, three months after the first infusion.

  That winter and early spring, John accompanied Megan to school on some days and played nurse for Patrick on others, trying to make up for the lost time with them during the past three years. “You should come on a day when I have P.E.,” Megan said. “P.E.’s my favorite.”

  So the next Monday, a P.E. day, John followed Megan onto the handicapped school bus that picked her up at 8 A.M. in front of the house. She wore her new Nike sneakers and a pink sweat suit, even though she knew she wouldn’t be getting out of her wheelchair that day. “Wrong shoes,” she told her dad, frowning at his loafers.

  Megan’s kindergarten class was taught by a distinguished, grandfatherly, and very funny teacher named Mr. Fletcher. He’d been around the school so long and was so beloved that the parents and his colleagues just called him “Fletch.” He was a veteran teacher who swore every year would be his last. Sitting in a rocking chair in the front of the room, he spent the morning reviewing the days of the week with the children.

  “What comes after Tuesday?” he asked, looking around at the students seated in a circle around him.13

  John looked over at Megan, in the back row to his left, and saw she was staring out the window.

  “Hey, Gertrude,” Mr. Fletcher called, picking up on the name Sharon had called Meagan for so long no one remembered exactly why. “Why don’t you help us here?” Mr. Fletcher knew Sharon well since she usually accompanied Megan to school.

  Megan looked at him with a blank stare.

  “Did you hear the question?” he asked.

  “No,” she admitted, shaking her head.

  “I asked you what day comes after Tuesday. What’s the matter, Gertrude, are you deaf now, too?”

  “Oh, that’s easy. Wednesday,” Megan responded, oblivious to Mr. Fletcher’s sarcastic reference to her handicaps. John laughed, delighted. The wiseass in him liked the teacher’s sense of humor and the way he held Megan accountable.

  At P.E. time, John watched Steve Hennessey, the gym teacher who moonlighted as a guitar player at children’s parties, divide the students into two teams, wearing either red or blue mesh jerseys. John, standing a few feet behind Mr. Hennessey, couldn’t follow the rules, but the kids seemed to know what they were doing.14

  Mr. Hennessey blew the whistle, and Megan, with her blue teammates, raced across the gym. When a child tripped and fell in front of Megan’s wheelchair, John held his breath, waiting for the crash and the screams that would surely follow. But she swerved left and out of the way, careening into the path of some other kid, who screamed, “Watch out! Megan!” The whole group on Megan’s left leaped out of the way.

  Megan approached the wall at a sprint, brought her chair to an abrupt halt, holding her head with her left hand as she spun around, and accelerated back.

  “We won,” she yelled from the end of the blue line, pumping her left arm and looking at her dad.

  John waved, giving her a thumbs-up. Mr. Hennessey turned to John and smiled. “She’s a chip off the old block,” John said proudly.

  That night, as he and Aileen ate dinner, John said, “You know, watching Megan in P.E., I realized that she really doesn’t see herself as handicapped in any way. She thinks she’s just l
ike the rest of them.”

  “Are you kidding me? She doesn’t think she’s just like them,” Aileen laughed. “She thinks she’s a little superior!”

  In early April, John and Aileen sat with Dr. Day-Salvatore in a corner of the large, second-floor hospital room where the children were receiving their now-regular infusions, oblivious to the import of the day. Day-Salvatore held up an echocardiogram that showed Megan’s heart before the infusion—swollen to twice the normal size for a child her age. In the second picture, Megan’s heart had shrunk to nearly a normal size. Her liver, so swollen with glycogen that she had had an enormous Buddha belly, was inside the normal range, too. She’d lost eight inches from her waistline.15

  Day-Salvatore had painstakingly measured the strength of Megan’s skeletal muscles before her infusion and again last week. The scale ran from one to five, a one indicating a flicker of movement, and a five for normal strength. Before the infusion, Dr. Day-Salvatore had measured Megan’s neck muscle at a two. Now it was at a three. Megan’s shoulders had gone from a two and a half to a three. Even her very weak legs had shown a little improvement—her ankles had increased in strength from one to one and a half.

  “The results for Megan are very promising,” the doctor said.

  “This is great news, Dr. Day-Salvatore,” John said.

  “What about Patrick?” asked Aileen.

  The doctor hesitated and then said, “Unfortunately, Patrick’s results are less promising.” Patrick’s heart, like Megan’s, swollen to twice its size, had declined in size, but only slightly. His skeletal muscles had registered virtually no gain in strength. “He may just be slower to respond, so I’m not giving up hope yet,” the doctor said. “But we don’t have the same results for him that we have for Megan.”

  John and Aileen sat silently for a second, feeling both elated and deflated. The news was so good for Megan that they wanted to celebrate; but it was so disappointing for Patrick that they wanted to cry. And yet they were not surprised. Megan’s progress had been plain for everyone to see. As her liver shrunk, her belly had gone down in size daily—Sharon had measured the decline with a tape. Everyone had noticed that Megan could once again hold her head up and sit unassisted. She was making remarkable progress.

  Patrick, on the other hand, didn’t seem markedly different in his physical abilities. Everyone hoped that even though there was no outward difference, good things were happening inside to his heart and liver. But Day-Salvatore’s results made clear that was not the case—at least not yet. She had opened up the chilling possibility that their children were heading in different directions. One might live and one might die.

  Even the bad news for Patrick couldn’t contain John’s excitement over Megan’s good results. He carried the echocardiogram over to her bed to show her how much her heart size had declined. “See Megan, this is how big your heart was before Special Medicine,” he said, holding the picture in front of Chrysanthemum, the book she had been reading. “See here, this is how much smaller it is now.” He hugged her and said, “You know what this means? It means you’re gonna live to be an old lady.”

  Aileen went over to Patrick, who was pretend fighting with two little plastic action figures. She picked up his hand, kissed it, and said, “Now Patrick, you just tell that heart of yours to listen to what Special Medicine has to say, okay? We need to get Special Medicine working a little better in you.”

  For the rest of the spring and summer, Megan continued to gain strength. Aileen called John down to the kitchen one afternoon to show him that Megan could sit unassisted on a barstool. When Megan raised her hand to answer a question in class, Mr. Fletcher pointed out to Sharon that her arm shot straight up in the air where before she could only raise her hand shoulder high. “Next time I look around, Gertrude, you’re going to be touching the ceiling,” he said.16

  Taking a family picture one day, John saw through the digital camera lens that Megan’s cheeks were raised into a smile for the first time in two years. Aileen insisted she saw a difference in Patrick as well, not so much in physical strength, but in mood. He seemed more comfortable, more communicative; he now actively played with his toys more than passively watching TV.

  After five grueling years, John suddenly had no job, no schedule. His last day at Genzyme, December 19, had been a quiet affair—no big luncheons or speeches. A few colleagues stopped by to shake his hand and wish him well as he packed his pictures of Aileen and the kids and some mementos. Now, his tiny office forever behind him, he had more free time than he knew what to do with. He volunteered to teach Megan’s religious education class at St. Paul’s, signed up for Italian lessons, and began training for the Marine Corps Marathon. He indulged his passion for fine cars—adding a Mercedes E55 Sedan to the Jaguar convertible he had bought a few months earlier. He and Aileen took long weekends away in Tucson and Las Vegas. They even left the kids for a full week and flew to Ireland with Aileen’s parents and John’s Uncle Jim and Aunt Marie.

  And one bright July afternoon, as John washed the convertible in the driveway, Megan came whirring out in her wheelchair to watch.

  “I wanna go for a ride,” she said, “but first you have to finish cleaning your car.”

  It had been years since John had taken Megan out alone in her handicapped van, let alone a car. She had been too weak to sit up even in a toddler car seat, and there were too many things that could go wrong with a child on a ventilator and the only adult occupied with driving. John studied her for a long moment. Megan seemed so much stronger he thought it was worth a try.

  “Well, Princess, you arrived at just the right time,” he said, with an exaggerated bow. He ran to the basement, brought up her old car seat, and strapped it into the passenger side of the car. Then he lifted Megan out of her wheelchair, strapped her into the seat, and pulled the car around to the front doorway.

  When Aileen heard the horn blaring, she got up from the kitchen table where she was doing homework with little John while Patrick napped upstairs. She stepped out of the front door to find father and daughter in the convertible, smiling and waving. Her heart lurched to see them looking so happy and beautiful—her handsome husband with his youthful smile and thick, dark hair, and her spunky daughter with her naughty brown eyes and her penchant for pink. Aileen cupped her hands to her mouth and blew them a kiss.

  “Ready, Megs?” John asked.

  “Yeah!” his daughter said, motioning for him to turn up the sound on the CD player. Laughing, John amped up the volume and hit the accelerator. From the speakers, the Dixie Chicks sang out with exhilarating abandon.

  The car pulled out of the driveway and peeled down the street, the riotous notes of Megan’s newest favorite song, “Ready to Run,” dissolving behind them on the warm summer air.

  Afterword

  I first met the Crowleys on April 27, 2001, when I visited them to write a story on Novazyme for the Wall Street Journal. I arrived at their two-story brick colonial in Pennington, New Jersey, in the late morning. John, preppy and enthusiastic in a blue polo shirt and khakis, burst out the front door and ran to the curb to greet me. His personal exuberance was so great that it actually caught me a little off guard. He led me into the den, where Megan, four, and Patrick, three, sat on a couch watching Barney on TV. It was a scene out of any suburban household—except that both kids had tubes coming out of their necks connected to microwave-sized ventilators on the floor that were breathing for them.

  Aileen appeared for a minute, smiling politely if distantly, and disappeared upstairs with Patrick. Megan gestured toward the front door and said something in a muffled voice. “Her electric wheelchair has just arrived and she wants to go outside and try it out,” John translated. Sharon came in to attach Megan’s ventilator tube to a smaller portable one, and followed John and Megan outside. Her legs hung limply, swaying with the movement as he carried her.

  John fastened Megan into the seat belt of the bulky black wheelchair parked inside the garage. He explained the working
s of the joystick near her right hand. “When you want to go left, you push that way. To go right, this way,” he said, gesturing as he spoke. “For straight ahead, you push straight ahead.”

  Before he could finish, Megan took off. With John running behind—shouting, “I haven’t showed you how to stop yet!”—Megan sped down the sidewalk, her powderblue cotton dress billowing in the wind. Her fearlessness and zest for life were striking even then.

  My first story about the Crowleys and Novazyme appeared in the Wall Street Journal in July 2001. And over the next year and a half, I called John every month to ask about the children and how close they were to being treated. During that time, he sold Novazyme, became chief of the Pompe program at Genzyme, tried and failed to get his children into multiple clinical trials, and then finally resigned. In our interviews, his voice grew more strained, angry, and even desperate as the months went by.

  Finally, in January 2003, an ebullient John called to say that Megan and Patrick were going to get their Special Medicine the next week. He invited me to join them for the first infusion in St. Peter’s University Hospital in New Brunswick, New Jersey, a short train ride from Manhattan.

  That August, two years after my first piece appeared, I wrote a second, longer article about the children’s treatments and how they were growing stronger as a result. The story, which appeared on the front page of the Journal, also described John’s struggle against conflict-of-interest accusations and Genzyme’s own internal protocols to get the experimental medicine to his children.

  Readers wrote in for weeks thereafter, deeply moved and awestruck by the Crowleys’ struggle and their courage.

  The story is a “testament to the strong undying drive of devoted parents,” wrote one man. After reading the first few paragraphs, “I set my bag back in the trunk of my vehicle and proceeded to read the entire article leaning over my vehicle in the middle of the parking garage. As a doctoral student in the biomedical sciences with venture capital aspirations, your work touched a note on multiple bases beyond emotion,” said another.