The Cure

by Geeta Anand

  “We at Novazyme are passionately driven by our mission to discover and develop drugs that profoundly enhance human life,” John began forcefully, vibrant in a navy suit and red tie.

  “This manufacturing plant is being dedicated for a brave young lady,” he said, looking down at a girl in a long-sleeved blue dress, reclining a few feet away in her wheelchair. The new facility, he said with gusto, would be called the Lindsey Paige Easton Biologics Manufacturing Facility. John had decided to name the new plant after the young lady who had moved and inspired him on her last visit.

  “Every day that we didn’t have our own manufacturing plant, every day that we don’t have enzyme, somebody in the world suffers greatly from one of these diseases,” John said. “We’ve dedicated this plant to you, Lindsey, so everybody knows, every time they enter this doorway, why it is we are doing what we do.” He pressed his lips together to compose himself, as Laurie Easton, standing beside her daughter, wiped away tears.

  John cut the ribbon to applause that continued for several minutes. From his position in the back of the crowd, Gus noticed the young employee to his right interrupt her clapping twice to wipe away tears. In the front of the room, near John and the sliced ribbon, Lindsey Easton, sat motionless except for her big eyes scanning the crowd from behind a pair of eyeglasses. Her face hadn’t changed and she didn’t seem to have shifted position since she appeared in front of the audience ten minutes earlier. Gus, uncomfortably aware of his ignorance, wondered if she could move at all.5

  Lunch followed, and Gus knew very well that it wasn’t coincidental that John had scheduled one of the Lunch ’N Learns he talked about so enthusiastically. Along with the other board members, Gus sat at the back of the second-floor conference room, uncertain what to expect. Other than the quick look at Lindsey that morning, he had never met a Pompe patient; in all the months he’d worked with John, it had never occurred to him to ask to meet one. That was the domain of doctors; his expertise was finance.

  The room was crammed with the company’s employees, most of them young and scrubbed looking. They fell silent as a gray-haired woman who looked to be in her mid-fifties walked in, leaning on a cane and clutching her husband’s arm. John introduced her as an Oklahoma resident, a mother and grandmother, who suffered from the adult-onset form of Pompe. The woman, speaking softly and matter-of-factly, said she was diagnosed with Pompe when she was forty years old. But for years before, she had felt something was wrong, though no doctor had been able to find the problem. As a child, she was always the slowest runner, the last one picked on the kickball team. After she married and had children, she noticed her legs feeling extra heavy one day and that she was getting winded just walking up a flight of stairs. She saw one specialist after another over five years until an endocrinologist finally diagnosed the disease.

  “I’ve gotten slowly weaker over the past fifteen years since I was diagnosed,” she said. “Now I can’t walk up stairs at all. But I still take time to enjoy life. I try to stay fit by walking every day. I’ve taken up painting. If no medicine comes along, I know I’ll keep getting weaker and in a few years I’ll need a wheelchair.

  “I can’t even begin to tell you what it means to have the hope of a treatment that might stop me from getting weaker. It makes me think about living instead of dying. I think again about growing old with my husband,” she said, looking lovingly at the man standing patiently beside her. “I think about seeing my grandchildren grow up. Before Novazyme, I didn’t let myself hope for a future—but now I find I can’t let go of it.”

  Looking at John smiling and thanking the woman for visiting, Gus thought—really thought—for the first time about what his life must be like. He wondered whether John’s children were mobile like this older woman or had to be pushed around in a wheelchair like Lindsey. If either of his own two children had the disease, what would he have done? He knew the answer before he finished the thought. There was absolutely no question in his mind. He would have set out, just like John, to drive science toward a cure.

  Gus returned from his visit to Oklahoma with a new respect and sympathy for John. He was driving his board crazy, but he had put together a real company filled with zealous young employees. Gus had even told John as much as the luncheon concluded. As he’d joined the end of the line for take-out Chinese food now spread over the conference table, John had come up behind him. “Gus,” he joked good-naturedly, “I’ll bet you’ve been wanting to try some Oklahoma Chinese cuisine your entire life.”

  Gus laughed, but was unable to shake the mental picture of this aggravating, exasperating man at home, pushing one of his children lying limply in a wheelchair. “John, what you’ve done here is pretty impressive,” he admitted. “You’ve established a remarkable corporate culture. It’s unlike any other biotech I’ve seen before.”

  Still, Gus was determined not to let these feelings cloud his decision on whether to invest more of HealthCare Ventures’s money in Novazyme. He kept reminding himself that the inspiring corporate culture aside, Canfield was not one inch closer to getting the scientific data they needed to move ahead.

  And then, as if reading his mind, John was on the phone to Gus, reporting exciting data from Dr. Barry Byrne, the University of Florida scientist who had been testing Canfield’s enzyme in sixteen mice with Pompe. “I’ll fax it over and you and your partners can take a look for yourselves,” John said.

  A few minutes later, Gus was standing by the fax machine reading that Byrne had treated eight mice with Canfield’s enzyme over several weeks, and compared the strength of their leg muscles with eight others that had received no medicine. Byrne reported the muscle strength of the eight treated mice had been restored to almost normal. He had measured the strength by running an electric current through samples of muscle from each of the mice. The stronger the muscle, the more it flexed when stimulated with the current.6

  It was far from the proof Gus and the other venture investors were seeking that Canfield’s enzyme worked better than Genzyme’s two experimental treatments—and Byrne hadn’t yet performed any confirmatory experiments, so it wasn’t necessarily even proof that Canfield’s enzyme worked at all. But Byrne’s results were the first indication from someone independent of Novazyme that Canfield’s approach held promise.

  “I still don’t know about Crowley, but maybe we should think more strongly about putting in more money to give Canfield time to generate some credible data,” Gus said, striding into his partner Mirabelli’s office to show him the data. Mirabelli shuffled through the papers, considering.

  “I think we’ve got Crowley more under control. He has heard us and brought on more guys with more experience,” Gus said, referring to Dr. Pedro Huertas and several other new hires. “Maybe Dennis is right. We give Novazyme a little more cash, and we sell it for a profit and get out.”

  “No point in throwing in the towel now,” Mirabelli agreed, handing back the papers.

  Back in his office, Gus got John on the phone. “Are you still on track to go into clinical trials in September?” he asked. In the venture world, certain milestones, like beginning a clinical trial, enhanced the value of the company—making it a good time for the original investors to sell.

  “Absolutely, we’re on the money,” came John’s unhesitating answer.

  Gus sighed. He didn’t really believe him, and despite the go-ahead from Mirabelli and his own urgings in that meeting, still struggled with whether it really was worth the risk to invest more money—and then an idea sprang to mind for protecting part of his next investment, if he was going to make it. He cleared his throat and phrased his words carefully.

  “John, I liked the data you sent over,” Gus began. “We’d like to be part of the B Round.” Then the part he knew John wouldn’t be so happy with: “You know, I’m going to talk to Dennis about investing in two stages. What I’m going to propose is that we give you $8 million now—and $8 million when you file your application to the FDA to begin clinical trials in September.
”

  “That sounds fair,” John said. But even from the other end of the line, Gus thought he could hear the enthusiasm draining.

  John hung up, closed the door, and sat silently in his satellite office in Princeton, watching a spring rain douse the cars in the parking lot.

  Gus had called his bluff. How could John argue against hitching half the investment to the start of clinical trials without admitting he would never be in trials by that fall?

  The people Gus had made him hire—from Huertas, the chief medical officer, to Bill Fallon, the new head of manufacturing—had convinced him that it was truly impossible to begin human trials in September. John still hoped he would be close, but he wasn’t yet ready to admit any expected delay. If he changed the timeline, he believed his investors would flee.

  What was he going to do? He now had seventy people employed, and he was spending more than a million dollars a month. Eight million would only get him to September. And if Novazyme wasn’t in clinical trials by that time, John was sure Gus and the others would fire his sorry ass on the spot.

  Where would Megan and Patrick be then?

  He stared at the rain silently funneling down the windows of the parked cars, turning the pavement a dark gray and slicking the lawn with flecks of silver.

  There was, he finally decided, only one way of doing what he needed to do—getting the company more money, saving himself from his investors’ wrath, and giving his kids a prayer of getting treated soon. It was a solution that had offered itself several times over the past few months, but one he hadn’t wanted to give in to. Now, he realized, it was the only way.

  He would take the money his venture capital investors were ready to offer and race the science as fast as he could over the next six months.

  And then he would sell Novazyme.

  18

  Making Memories

  Spring 2001

  Pennington, New Jersey; Orlando, Florida

  As Aileen watched the ordeal of the past few months, she had come to believe it was far from certain John would get their children treated in time. “I don’t know if I can make this work,” he had said to her repeatedly as he grappled with the funding, the science, and the venture investors. Seeing her husband’s struggle, Aileen rushed to play the role of comforter she had assumed in the marriage. Not a day had gone by in the past two years when John had not been completely focused on trying to cure the children. She decided to plan a vacation.

  John dismissed the idea at first, unable to conceive of how he could get away while trying to close the next round of financing with his wary investors. Only when Aileen pushed, saying, “The biggest tragedy would be if you spent all this time trying to save the kids that you didn’t get to enjoy them while they were here,” did he finally relent. Aileen promised that she would take care of the arrangements.

  Aileen remembered the “wish” Megan had been given by her mother’s friend Kathy Rosato and Kathy’s daughters at the first fund-raiser two years ago. The “wish” was from the nonprofit Make a Wish Foundation—dedicated to making the dreams of children with life-threatening illnesses come true. Aileen had accepted it graciously, but she had never intended to use it. She didn’t return calls from the Make a Wish volunteer, and she told John they should give the “wish” to another child who needed it more.

  “Our children aren’t dying,” she’d said firmly. “They’ll be getting an enzyme, and then they’ll be fine.”

  Now, two years later, Aileen’s resistance to using the “wish” had disappeared. Each time Chen’s clinical trials had gotten postponed, each time she overheard John talking about a new obstacle at Novazyme, she had let go a little bit more of the promise of a cure. With Sharon to help, she had resolved to embrace a new mission, which she would articulate when people asked when the enzyme therapy would be ready. Shrugging, she would answer, quite sweetly and truthfully, “I don’t know—I’m just trying to keep the kids as healthy and happy as I can.”

  One morning in early March, Aileen dug Megan’s “wish” from under a pile of take-out menus at the bottom of the kitchen drawer. The “wish” was a folded blue piece of paper emblazoned with a silver star attached to a Make a Wish volunteer’s card. Aileen called the volunteer and said her daughter was finally ready to use her wish, and she wanted to go to Disney World. When the volunteer called back, she had made all of the travel arrangements except for the flights. She wasn’t able to find a single commercial airline that would fly the children with a supply of oxygen in the cabin, which they needed in case of a respiratory emergency. It was against federal air regulations to carry oxygen in the cabin because it was flammable.

  Aileen, trying not to lean on her exhausted husband, realized she had no choice but to ask him for help. The only way to get the children to Disney World was to find a private plane to fly them there, she said. “Could you please ask Brad if his dad can help?” Lewis Campbell, the father of John’s friend Bradley was the chief executive of Textron who had helped underwrite the Boston fund-raiser. As she had expected, her lovably predictable John frothed and raged that he already had too much to do, then picked up the phone and dialed Brad. By the end of the day, Lewis Campbell had agreed to let the Crowley family use his firm’s corporate jet.1

  On the third Saturday in March, the Crowleys’ blue minivan rolled up beside Textron’s private jet at Trenton Mercer Airport, just a short ride from their house. Aileen could tell her husband was more excited about riding in a private plane than about the Disney visit. He jumped out of the car and ran up the steps to check out the cabin before returning to help load the kids, the luggage, and the medical equipment onto the small plane.

  Once everyone was on board, including Sharon and their regular night nurse, Yvonne, John, in his trademark navy shirt and khaki shorts, leaned back in his tan leather seat extra slowly. He puffed out his chest and marveled aloud, with boyish enthusiasm, that his seat could swivel, even though it would only move a few inches with three backup ventilators jamming the short aisle. The big bulky batteries of the ventilators were piled on top of the toilet in the rear, making it unusable.

  Soon John was striding into the cockpit to discuss the model and make of the plane with the pilots (it was a Cessna Citation 7) and the weather ahead (clear skies, good visibility with scattered low-level cloud cover). Out the window, he spotted some former Bristol-Myers colleagues getting on the next plane over and ran outside to talk to them—eager to be seen also traveling on a corporate jet. As she stood in the doorway watching him, Aileen overheard him say, deliberately casual, “I’m just taking the kids down for a quick trip to Disney World. We’ll chase you down there.”

  When John was back on board, the pilot said the flight would be delayed because he needed extra fuel to handle the weight of so much medical equipment. They were an hour behind schedule when the little plane finally charged into the air. So much for the big shot corporate executive.

  For the two-and-a-half-hour flight, Megan, who hadn’t been on a plane since she was old enough to remember, stared outside through her red Minnie Mouse sunglasses, fascinated by the clouds swirling by. Patrick cried and Aileen held him on her lap. John Jr. played his Game Boy quietly in the rear seat.

  “This is a nice way to travel,” John kept repeating, opening the cabinets and refrigerator several times to pull out snacks and drinks.

  When the plane landed, Aileen saw two Make a Wish volunteers and a dog parked nearby in a red pickup. They said they were there to drive John to the other end of the airport to rent a van. “Jump in back,” the driver said to John, leaving him no choice but to climb into the bed of the pickup, which was filled with hay. As he settled awkwardly onto a bale, Aileen pulled out her camera and clicked a picture, teasing, “Oooh, Mr. Big Man on his private plane—now look at you.”

  “Wave to Daddy, everyone,” Aileen shouted as the truck revved its engine and roared away.

  “No, look at Daddy’s hair!” Megan cried gleefully. “I’ve never seen Daddy’s
hair look like that before!” The pickup was accelerating onto the highway, and John was hunched over, grasping the sides, his carefully combed and gelled coiffure standing straight up into the streaming air.

  The Make a Wish Foundation arranged for families visiting Disney World to stay nearby in a fifty-acre village run by a nonprofit called Give Kids the World. The group was started by a successful hotelier who wanted to make sure children with life-threatening illnesses could visit Disney World. While Aileen checked in at the front desk, John led the kids and the nurses around the village. The “villas,” where the families stayed, were built around a village center that had a pool, rides, a fishpond, a movie complex, and an ice cream parlor. Clowns and jugglers performed everywhere.

  As they entered their two-bedroom villa, Aileen heard John joke, “Megs, why go to Disney World when we have all of this here?”

  “What? You have got to be kidding,” Megan retorted with a withering look. “That’s fine. You stay here. I’m going.”

  By now, Megan and John had become almost like a comedy duo. Of their three children, Aileen knew Megan was most like her husband: funny, confident, and happiest in the limelight.

  Aileen had promised her two youngest they could go to Disney World that day if they took naps, and she quickly settled Megan and Patrick, with their ventilators and IV poles, into one bedroom. John, Aileen, and John Jr. would share the other, while Sharon and Yvonne had reservations at a hotel nearby.

  The excitement of the journey had exhausted Megan and Patrick, who needed more rest than healthy children, and within minutes they were asleep. As John Jr. watched TV, John, Aileen and the nurses unpacked the luggage and medical supplies, including a dozen boxes of surgical tubes, medicines, and diapers shipped ahead. Because of muscle weakness, Megan, four, and Patrick, three, couldn’t control their bowel movements and still needed diapers.