The Cure

by Geeta Anand

  For one thing, according to Henri, John was supposed to be in charge of the Pompe disease program—but nobody reported to him. Landy reported to someone in the medical and regulatory affairs division. The woman who ran the core team in charge of manufacturing Chen’s enzyme reported to the head of the U.S. manufacturing division. And the scientists on each core team reported to the head of the science division. These divisions—medical affairs, manufacturing, marketing, and science—looked to John like silos without bridges, standing tall but having little to do with each other.4

  With no choice but to forge ahead, John told van Heek that it was time to announce he was taking over leadership of all the Pompe disease programs. He drafted a memo that van Heek sent around describing the formation of the new Pompe leadership team, which would oversee the company’s drug development programs for Pompe. The memo said that John would be the team’s chairman and that it would be comprised of eleven other members. There were eight people from Genzyme, including Landy and Mattaliano, the scientist trying to develop an enzyme that was easier to manufacture. Four came from Novazyme, including John; Tony McKinney; Bill Fallon, the manufacturing guru; and Julie Smith, a smart woman John had recruited from Bristol-Myers, who he’d put in charge of closing the Genzyme deal.

  Conspicuously absent was the name of William Canfield.

  It was hard to say who was more upset—Canfield or Landy. Canfield believed that John had done nothing less than betray him, and stopped returning his calls. John had promised that they would continue to work as a team after the acquisition, and nothing could have been a more blatant breach of that pledge than denying him a spot on the leadership team.5

  John hated to leave Canfield off the team, but concluded—after days of wrenching soul-searching—that he would be too divisive a presence. Every time Canfield was in the same room as the Genzyme scientists, the meeting would degenerate into a series of attacks and insults. It wasn’t always Canfield’s fault; sometimes the Genzyme scientists initiated the digs. But Canfield, dogmatic about his approach and often abrasive with those who disagreed with him, didn’t seem able to bite his tongue and make nice.6 John needed to build a coalition fast, and the only way he could expediently eliminate the tension was by removing one side from the argument.

  As for Landy, until he saw the memo, he had been certain he was the one running the show—and to his credit, the misunderstanding wasn’t his fault. Having led the clinical side of a major drug development effort at Serono, Landy had taken the job at Genzyme only because he had been promised a hugely expanded role. The senior managers who hired him had been unaware that Henri and van Heek had agreed to give John leadership of all the Pompe programs. Now, even as they apologized profusely, they told Landy they were powerless to change the situation. Henri and van Heek were in charge, and they were sticking with their commitment to John.7

  Determined not to let hurt feelings get in his way, John tried to move on. He scheduled a kickoff meeting of the Pompe leadership team for the week before Thanksgiving. To establish its significance, he didn’t hold the meeting in Genzyme’s offices but reserved a conference room at the trendy and expensive University Park Hotel on nearby Sidney Street.

  About twenty people were invited to attend the meeting, including the new twelve-member Pompe leadership team and a few others. As they settled around the U-shaped conference table at the hotel, John rose to give a welcoming speech.8

  “I am so happy you have all agreed to join my team,” he said. “I know we all realize the significance of what we’re doing, but before we get going here, I’d like to relate a very personal story. Last year, only a few months after I joined Novazyme, a family from Kansas appeared at our doorstep in Oklahoma. They came trying to save their tiny ‘floppy baby’ who was dying of Pompe disease. And they were begging for some of the treatment they had heard we were working on. We hadn’t even entered animal studies as yet.” Everyone in the room knew what that meant—the family had been turned away empty-handed. “The point,” John went on, “is that it was completely unrealistic for this family to expect any treatment, but also that this reflects the hopelessness that patients and families with Pompe struggle with every day. I think it’s important for the twenty of us here in this room to realize, truly, that we represent the best and only hope for these patients and their families.

  “The first order of business of the Pompe leadership team is to present Henri with a drug development strategy for Pompe disease within the next sixty days,” he said. Toward that effort, John wanted everyone to begin by participating in a simple but vital exercise. “I’d like us to talk about our vision for what the world will look like for Pompe patients five years from now. Let’s begin by each of us describing our vision.”

  John paused, waiting for someone to volunteer, but nobody raised a hand. The Genzyme employees leaned back in their seats and looked at the table. Determined not to be fazed, John forced a laugh, and said, “Well, I guess we’re all feeling a little shy today. Let me tell you my vision. I have a vision that five years from now we have a good treatment for the disease. We will completely understand the biology and the science around Pompe. And by then we’ll be researching wholly new ways to treat the disease that will make these enzymes obsolete.”

  Two Novazyme alumni, Tony McKinney and Julie Smith, raised their hands and added to that vision. “I would like to see us not only with a treatment for Pompe, but I would hope that there are also others on the market for some of the related diseases where patients have no hope today,” McKinney said.

  “My vision is that there would be newborn screening so Pompe babies would be identified as soon as they are born and treated immediately,” Julie added gamely.

  John looked around again for others who might speak, and found none.

  Hal Landy stared at his feet, fuming. “This kind of exercise was interesting when you were twenty-five—not when you’ve been in the workplace for twenty years,” he mumbled to the person beside him.

  “He’s like a car salesman,” scientist Mike O’Callaghan whispered to Mattaliano. O’Callaghan, a gaunt, white-haired New Zealander, was another of Genzyme’s top scientists.

  Shrugging, John barreled ahead into the PowerPoint presentation he had prepared, outlining the history of the development of treatments for Pompe disease. He began with the Dutch physician and researcher, J.C. Pompe, who made the link between the enlarged heart he saw in one baby he autopsied and the glycogen filling the muscle cells. John described early attempts at enzyme replacement therapy that failed. Enterprising doctors and researchers had purified the Pompe enzyme out of human placenta and infused it into a patient. Unfortunately, the patient showed little sign of improvement, which suggested to researchers that replacing the missing enzyme wasn’t an effective treatment for Pompe.

  Then, in the 1970s, researchers began to learn how enzymes got to the crucial sections of cells. They revealed that the sugars attached to the enzymes played a vital role in directing them to the lysosomes of cells. This body of research explained why the experiment with the placental Pompe enzyme had failed. That enzyme had already been excreted from the lysosomes, where the sugars needed to get inside had been trimmed down. So these placental enzymes, when infused, didn’t have the sugar chains needed to enter the lysosomes.

  O’Callaghan, shaking his head, pointed out to Mattaliano that the slides John was showing still bore the Novazyme logo.

  “How long does it take to change that to the Genzyme symbol?” he whispered. “It’s like the junior varsity has taken over.”

  John summarized the four drug development programs for Pompe that Genzyme now owned and then paused, trying to decide how to end the meeting. He had been wrestling with whether to show the team a segment about him that had appeared a few weeks earlier on the Today show. Was it tactically smart to remind people about his children when he needed to be seen as an objective leader interested in saving all patients?

  But things were going so badly, John decid
ed to take the risk. “And now, here’s a recent episode of the Today show that will give you a flavor of what my personal family’s hopes and challenges are,” John said. He hit the “play” button.

  “There’s been a lot of talk of heroes in the last several weeks,” host Katie Couric said, referring back to the terrorist attack that had shaken the nation two months earlier. Introducing the Crowleys, she said, “This is one father who is going the extra mile.”9

  The tape showed Megan, in a red polka dot dress, and Patrick, sucking his pinky finger, beautiful young children who looked almost like any other kids—except for the tubes coming out of their throats, indicating that they needed ventilators to breathe. The reporter, Anne Thompson, told the story of Megan and Patrick’s diagnoses and of John forming the Children’s Pompe Foundation, becoming CEO of Novazyme, and driving toward a treatment to save his kids.

  Then the reporter, camera honing in on John’s face, asked, “If Megan and Patrick die, will you have failed them?”

  Slowly, voice heavy with emotion, John responded, “I’d go back to the promise we made to them three and a half years ago that we would do everything we could—and I think we have.”

  “We wish the Crowleys our best,” Katie said, smiling into the camera—and the tape ended.

  “Well, that’s my family,” John said, a little awkwardly.

  When the lights came back on, several people were in tears. Landy swallowed several times, trying to clear the lump in his own throat. The tape had touched the father, pediatrician, and idealist in him. He had three sons himself, the youngest born with such a severe defect in his joints that he’d been in a body cast for the first few months of his life. The tape reminded Landy of when his wife, also a physician, was pregnant with that baby. A test had revealed that the baby might have severe birth defects. Doctors had raised the possibility of abortion. Landy and his wife had decided to continue with the pregnancy, believing that they, as doctors, were qualified to care for such a disabled child, if anyone was.

  The child, it turned out, wasn’t as disabled as the doctors had feared. But he had still needed a lot of extra care as a baby and young child. Seeing Megan and Patrick on the screen, beautiful and precious and horribly weak, Landy remembered the overwhelming love and helplessness he’d felt in the face of his own child’s situation.

  Now, looking at John standing quietly beside the television monitor, instead of seeing a power-grabbing, Ivy League know-it-all in a pin-striped suit, he saw a desperate father who loved his children and had committed all his enormous talent to one goal—finding a cure for their devastating disease. Landy had gone into clinical research for the very same reason—to help sick people. To think, he realized, that this guy standing before them, awkward and ill at ease, had been able to pull together a $137 million company in one year! If anybody was qualified to run the Pompe program at Genzyme, Landy thought, John certainly was. A turf war over leadership of the program suddenly seemed juvenile, and in that moment, Landy conceded it.10

  He was the first person in the room to rise. Picking up his papers, he walked over to John.

  “Wow,” he said quietly to the younger man, his face flushed. “That was—that’s very powerful. Thanks for sharing it with us.”

  22

  Tough Choices

  Winter 2001–Spring 2002

  Princeton, New Jersey; Cambridge, Massachusetts

  When John arrived home at 9 P.M. on Friday, the kids were already in bed. He tiptoed into their bedrooms and kissed them. John Jr. slept soundly, but the younger two stirred. Megan raised one arm and waved it with her pinky, pointer, and thumb extended, signing “I love you.”

  John had been away from home all week, staying at what was fast becoming his second home, the Charles Hotel in Cambridge. If he decided to remain at Genzyme after the one-year requirement of the acquisition agreement, the family would move to Boston. Otherwise, they would stay in New Jersey, where they were close to John and Aileen’s extended families and the network of nurses, speech therapists, physical therapists, and occupational therapists they had painstakingly recruited over the past four years.

  Downstairs, John heard Aileen in the kitchen. He found her pouring cream into a pot of meat sauce and knew she was making his Aunt Michele’s sinfully rich recipe. She poured the sauce over plates of bow-tie pasta and set them on the table.

  “How are you?” he asked, as they sat down.

  “Oh, I’m fine,” she said listlessly.

  “Honey, you don’t seem very fine at all,” he said. “Tell me about your day. How’d the little troopers do today?”

  “They’re fine—they’re fine now. They weren’t so fine a few hours ago.”

  “C’mon, Aileen—what happened? If you’re not going to tell me, who are you going to talk to?”

  “Nobody,” she said, her carefully controlled voice breaking a little. “That’s just it. I have nobody to talk to, John.”

  “Oh, Aileen,” he said, getting up and giving her a hug. She turned her face into his shirt and breathed deeply, leaning into his support and regaining her composure.

  When he sat back down, she told him she had taken Patrick upstairs to bed and had been undressing him when his ventilator started beeping. She checked the settings, and the vent seemed to be working fine, but the beeping continued. Patrick, distressed, pointed at his tracheotomy and cried, so she pulled the tube out of his throat to see if was clogged with a mucus plug, which happened at least once a week. It was plugged, so she’d suctioned it and tried to stick it back inside, but the hole in his throat seemed to have closed. It was one of those emergency situations that occurred with terrifying regularity every few months, literally bringing the children within minutes of death. It required skill, calm, and luck to overcome.

  As Patrick turned grayer, Aileen screamed for Sharon, who was downstairs with Megan. Sharon ran upstairs and after several attempts finally pushed the tube back in. By then Patrick had passed out, his lips blue. It took a minute or two for him to regain consciousness, with Aileen pumping the bag attached to his tracheotomy tubes to force air into his lungs.

  Megan, aware something was wrong with Patrick, was crying downstairs when Aileen found her. Aileen carried her up to bed and was reading her favorite “Beauty and the Beast” bedtime storybook when she pointed at her throat and signed she was having trouble breathing. Aileen checked the ventilator settings, which were fine, and then, thinking back to her son’s mishap minutes before, pulled her daughter’s tube, too. The airway was clear so she quickly stuck it back. In a few minutes, Megan felt better and fell asleep.

  “It’s nothing that hasn’t happened a hundred times before,” Aileen said, looking down at her plate. “It just got to me today. I don’t know why.”

  “Aileen, you know you do a great job with the kids. The best. You couldn’t do it any better,” John said, stroking her hand. “You know, I’ve always said you’re our best nurse. And certainly the best looking.”

  “I don’t want to be the kids’ nurse, John. I want to be their mother,” Aileen said, tears starting to fall again.

  “I know. I know this is really hard for you. I’ve been away too much. It’s hard for you to have the children’s lives in your hands alone, day after day. I know you’ve got Sharon, but you need me, too. I promise, we’re almost there,” he said, leaning in to look into her face. Aileen nodded, wiping her eyes.

  “I’ve almost accomplished everything I need to do at Genzyme. Then I’ll be here for you and the kids all we need,” he continued soothingly. “I think people at Genzyme finally get what we need to do. The Pompe leadership team is all set. They watched the Today show tape of us. I think they understand now what it’s like for a family to live with Pompe disease.”

  Aileen blew her nose on the table napkin and changed the topic. “Megan’s fifth birthday is coming up, John. I’ve been talking to her about what she wants to do. What do you think we should do?”

  “Aileen, we need to do something
big, really big,” John said, excitedly. “Think of it—nobody expected Megan to live until she was five. And the tough little girl has made it. We’ve got to do something big, real big. And we finally have the money.”

  It was one of the best immediate effects of the sale of Novazyme to Genzyme: The major players, including John, had made millions. Canfield, with the largest share of the company, earned about $30 million. The two big venture firms each exited with $28 million. And John, who owned less than 5 percent, mainly from stock options given to management, netted $6 million.1

  Suddenly, after struggling for ten years with credit card debt and the $140,000 in John’s student loans, John and Aileen were rich. They paid off the debts—a drop in the bucket of the $6 million. Then they splurged. John bought himself a Rolex Submariner watch, the same kind his dad had been issued as a Special Forces Marine. John had inherited the beaten-up watch when his father died and had tried many times to get it repaired, but it never worked. He bought a three-year-old Jaguar XK-8 on eBay, in British racing green. And he and Aileen made a down payment on an enormous new $2.7 million house in Princeton. It was unfinished and needed months of work before they could move in, but it gave everyone in the family something wildly exciting to think about. Megan talked constantly about how her new house was so big that she was going to be able to ride her electric wheelchair inside. A year ago, she had gotten the wheelchair, which she could operate herself using a little steering device, but she had only been able to use it outside. John Jr., focusing on the details, told everyone there were seven bedrooms, six bathrooms, and two kitchens. Aileen had been meeting with an interior decorator and had already picked out some big, comfy chairs and couches and floral draperies.