by Geeta Anand
THE
CURE
THE
CURE
How a Father Raised $100 Million and
Bucked the Medical Establishment in a
Quest to Save His Children
GEETA ANAND
PENGUIN BOOKS
For the Crowleys,
who taught me how to live.
For my children, Tatyana and Aleka,
who taught me how to love.
For my husband, Greg,
whose support made this book possible.
Contents
Author’s Note
Prologue
1. Veritas
2. Trouble
3. Diagnosis
4. Hope
5. God Doesn’t Give You More Than You Can Handle
6. The Road to Power and Influence
7. Megan
8. The Conference
9. The Marriage
10. Sharon
11. Betting on Research
12. “Let’s Just Do It”
13. A Rocky Start
14. Failure Is Not an Option
15. Cowboys
16. Losing Support
17. Novazyme Time
18. Making Memories
19. The Bluff
20. The Deal
21. Genzyme
22. Tough Choices
23. The Mother of All Experiments
24. The Sibling Study
25. Plan B
26. “You Can Tell Megan”
27. Ready to Run
Afterword
Timeline of Major Events
Notes
Acknowledgments
Author’s Note
This is a true story. All the people and events depicted in this book are real. I researched it over the past five years, beginning with my articles for the Wall Street Journal in 2001 and 2003, and continuing in January 2004 when I started this book. Some of the material comes from my own observations, but most of it is based on my interviews with John and Aileen Crowley and several hundred others involved in the Crowleys’ lives and in the race to find a cure for Pompe disease. I also relied on scientific literature, corporate records of Novazyme and Genzyme, and newspaper and video clippings. Where I wasn’t present, the dialogue and scenes are primarily based on participants’ memories of what was said and how things unfolded. Where their memories diverged on important issues, or where I wasn’t able to interview a key player, I explained so in the endnotes.
Geeta Anand
Prologue
John Crowley’s hand shook as he hung up the phone in his wood-paneled study in Princeton, New Jersey, and looked up into the expectant green eyes of his wife, Aileen. She had been standing beside his desk for several minutes, listening intently. It was a Friday evening in October 2002.
“So what’s up?” she asked, her voice carefully neutral, trying not to show the hope he read in her expression.
John paused, absorbing the moment, and then his face broke into a massive smile. “You won’t believe it, Aileen,” he said, jumping up and walking around his desk to pull her into his arms. “The kids are going to get their Special Medicine. It’s finally time. They could start within two weeks! I’m going to Florida on Saturday to get everything set.”
Aileen started to respond, but she was interrupted by a screech from behind as they were drawn into a hug. The children’s nurse, Sharon, had overheard the conversation from the kitchen, and she ran into the room and threw her arms around the pair.
“Oh, Mister John. I’m so happy,” she whispered, squeezing them together tightly. John watched the lines of tension around Aileen’s mouth ease and her eyes slowly melt. The three of them stood together for a long time, crying, as even the hardened Aileen finally allowed herself to believe that the grueling four-year fight to save her children was almost over.
John was the first to pull away from the arms and tears. He walked through the kitchen into the adjoining playroom, where five-year-old Megan, in a pink flowered dress, sat in her electric wheelchair, slowly and methodically brushing a Barbie doll’s hair. Patrick, four years old, was already upstairs in bed, his ventilator steadily swishing in time with each forced breath. John pulled up a chair and sat beside Megan.
“Megs, you know Daddy’s been working on Special Medicine,” he said, using the term he had coined for the cure he had so desperately sought to halt the disease that had devastated the muscles of two of his children. Megan nodded her head ever so slightly and kept brushing.
“It’s taken a long time to get Special Medicine just right,” John continued, “but Megs, now we’re done making it, and I’ve found a special place to give it to you.”
At this, Megan looked up. “Where?” she asked, her straight brown hair swaying above her shoulders with her slight movement. She was a pretty girl with dark brown eyes and a porcelain complexion, a square face, and high cheekbones. A plastic tube sprouted from a hole in her neck and led to a pocket behind her wheelchair, where a ventilator hummed steadily, breathing for her. When she spoke, her voice was muffled, almost as if she were speaking from underwater. The weakness in her oropharyngeal muscles—the ones involved in speech1—made it difficult for her to enunciate, but her family and friends could understand exactly what she was saying.
“We’re going to give you Special Medicine in Florida, Megs,” John said.
His daughter dropped the doll and spun her wheelchair around in pure excitement so quickly he had to pull his foot back to avoid getting run over. Like many five-yearolds, Megan moved quickly and without regard to those around her. But unlike them, she drove a 400-pound electric wheelchair, and he knew she could inflict serious damage. She’d already broken her grandmother’s toe, gashed several walls in the house, and torn out a kitchen cabinet.
“Can I go to Disney World?” she asked in her distinct, slurring cadence, eyes imploring in an otherwise expressionless face. As always, John was awed by how much emotion burst from his daughter’s eyes. He wondered if this were true with everyone and he just hadn’t noticed, or if it were only so in children with Pompe disease who couldn’t move any other facial muscles. For them, the saying was genuinely true: their eyes really were the only window into their feelings.
“Yes, you can go to Disney World whenever you want,” he said, nodding vigorously.
“Yay!” Megan shouted, pumping her arms in the air as high as she could as she sped out of the playroom and into the kitchen, circling her mother and nurse, singing, “I’m going to Disney World.” John stood smiling, hands on his hips, relishing his daughter’s joy.
Four years ago, when their two youngest children were diagnosed with a disease they’d never heard of, John and Aileen Crowley had been told there was no treatment. Pompe disease, a rare genetic disorder—so rare that fewer than ten thousand people in the world are born with it— weakens the muscles of patients over time so that eventually they cannot walk, talk, or even breathe on their own. Babies and toddlers diagnosed with the degenerative disease usually don’t live past their second birthday.
But John couldn’t live without hope. He was a fighter, and he had never in his life accepted a negative outcome without a struggle. In the absence of any other options, he had simply made his own answers. The phone call he had taken earlier that day was from a doctor at the University of Florida in Gainesville, calling to say he had all of the internal hospital and university approvals to begin treating Megan and Patrick in a clinical trial—an experiment to test whether a proposed medicine works in human beings as well as it has in animal studies.
In such trials, patients are given different doses of a prospective medicine, and tests are administered to measure efficacy and side effects. Based on the results, the Food and Drug Administration (FDA) decides whether a new
medicine is safe and effective enough to be sold in the United States. For Megan, Patrick, and millions of others with imminently life-threatening and untreatable diseases, clinical trials offered the only hope.
After the kids were asleep that night, John sat back down in his study and called Continental Airlines to make a reservation on the first flight out the next day for Jacksonville, the nearest major airport to Gainesville. It was on Saturday of the weekend Aileen’s college roommate was getting married, but John believed he couldn’t afford to delay for a single day. He knew how easily things could go off track—he’d had his hopes dashed many times before. He needed to get to Florida immediately to make sure everything was ready for the trial.
John heard the television set click off upstairs and knew Aileen was going to sleep. Resisting the urge to slide into bed beside her, he put his head in his hands and sighed. He hadn’t told anybody—not even Aileen—about the enormity of the challenge that lay ahead.
Nobody knew that he had approached the Florida doctor without informing his company’s medical team, whose job it was to file clinical trial applications and coordinate human experiments. If he could get the Florida trial started, he was gambling that his colleagues would whine and protest that he’d gone behind their backs, but that in the end they wouldn’t stop him. “It’s better to ask forgiveness than permission,” he’d said often enough over the past four years.
It was one minute before midnight on October 4, 2002. Four hours remained before he needed to start the drive to Newark Airport. “It’s better to ask forgiveness than permission,” he repeated aloud to himself. That approach had propelled him through the first thirty-five years of his life. Would it work for him now, in the hour of his children’s greatest need?
1
Veritas
Summer 1997
Cambridge, Massachusetts
On a clear, brilliantly sunny afternoon in June 1997, John Crowley walked to the podium to deliver the Class Day address to his fellow Harvard Business School graduates. At five feet six inches tall, he stood ramrod straight in his navy suit, his dark hair closely cropped and his square face wreathed in a bright, eager expression. Eyes shining, he unleashed a crisp, white smile into the crowd.
John opened a folder containing his speech and paused, relishing the attention of nine hundred fellow graduates and a few thousand of their friends and family members. They filled the metal chairs arranged in hundreds of rows in front of him under a white tent. To his left stood Baker Library, and behind the audience the Charles River sparkled. Across the river, the green-topped cupola of Eliot House, a Harvard college dorm, poked out from behind the summer greenery.
The business school had developed a distinct, close-knit identity since moving in 1927 to its own campus of neo-Georgian buildings. Students spent many hours each day with one another in class, and many more hours together on group homework assignments at night. Friendships born here tended to live on as the students graduated to become a disproportionately large portion of the nation’s business and political elite. Many who came here were the sons and daughters of heads of state, ambassadors, and company chief executives; those who didn’t start off as part of the elite were likely to join it when they left. Of the nation’s Fortune 500 companies, some 15 percent of their top three officers came through this business school.
John’s family sat in the front few rows of the audience. His mother Barbara sat beside his stepfather Lou and half-brother Jason. In the next row, his six-month-old daughter Megan, a bottle in her mouth, looked up from the lap of his wife Aileen. Automatically, his eyes scanned the seats around her for their two-year-old son John Jr., before he remembered that they had decided to leave him at home with a baby-sitter. But the rest of his tight-knit family was there, including Aileen’s parents, Marty and Kathy, and her Uncle Charles and Aunt Jane.
“It is my great privilege and honor to share with you today the many experiences of the past two years and the hopes for the future of what is now and should always be the greatest class in the history of the Harvard Business School,” John began. “For those of you keeping count, that’s my first attempt to pander to the crowd,” he said, looking up and smiling as the audience laughed appreciatively.
“In the one and a half hours that I have to speak with you all today—scared you, didn’t I?—okay, in the next twenty minutes, I’ll do my best to capture what has been for so many of us such a powerful and moving experience both in learning and living.”
John’s mother nodded, thinking that in his opening, her son had expressed the awesomeness of the moment with enough humor to avoid being annoyingly grandiose. He had always exuded a boyish charm, and others had always seen him as the kind of guy who was almost too good to be true—but was true. It was a testament to the high esteem his classmates held him in that he’d been elected to be their Class Day speaker, their representative at this graduation event. He reminded her so much of his late father, a police officer, who had snared her with his wiseass sense of humor the night they’d been introduced by mutual friends at Oprandy’s, a New Jersey bar, in February 1966. When the bar closed, he and his brother had sat in her car for another hour, laughing as they regaled her with joke after joke, until her father drove up and knocked on the window, demanding to know why she wasn’t home. By April, they were engaged, and they rushed to marry in August because she was pregnant with John.1
As she did at every milestone in John’s life, Barbara thought of how thrilled his father would have been. She remembered the early morning in January more than twenty years earlier when she’d sat John, then seven, and his younger brother Joseph, four, side by side on her bed to tell them their father had died. Sergeant John Francis Crowley—after whom John was named—had been found dead at the end of the night shift, apparently of carbon monoxide poisoning caused by a defect in his police cruiser.
She’d left Joe at home and taken John to the funeral at the towering stone St. Cecelia’s Catholic Church in the town of Englewood, New Jersey, where she’d been married, her children had been baptized, and both sons would serve as altar boys. Thousands filled the twenty-five rows of dark wooden pews and spilled onto the street outside. Sergeant Crowley, the son of an Irish immigrant rubber factory worker, had grown up in the ground-floor apartment of a four-family brick house on Prospect Street, a few blocks from the church where he was being eulogized at age thirty-five. In stories in the local newspapers, friends and colleagues remembered him for his sense of humor and his pride in being a cop. Sergeant Crowley “was so proud to be a cop that nothing else was important to him,” Police Chief Thomas Ryan told one newspaper.
Little John Crowley had listened intently in the front row as the priest addressed the homily to him, telling him there was no way he could understand why God had taken his father from him so young, but that now it was his responsibility to help take care of his mother and his family. After his father’s coffin, draped in an American flag—Sergeant Crowley had also been a U.S. Marine—was carried down the twentytwo marble steps, John had instinctively saluted. Everyone assumed his mother had prompted him to do so, but in fact it was his father who had taught the boy the proper way to honor the departed. Now, as the boy saw the officers saluting the coffin, he did too. Then a lone bagpiper played “Amazing Grace.”
Sergeant Crowley loved the police force, but he was at least as devoted to his children. When his wife was in labor, he had rushed home to change into a suit so he’d be appropriately attired to receive his firstborn into the world. When the doctor came into the waiting room to tell him he had a son, Sergeant Crowley passed out in his excitement. It had been his dad, coming off the night shift, who drove John to his first day of kindergarten. As if it weren’t grand enough to arrive at school in a police cruiser, John had pushed the buttons so the lights and siren sounded as he got out of the car. John accompanied his dad on weekends when he made extra cash hauling people’s junk to the town dump. A former Special Forces Marine, Sergeant Crowley had filled h
is son’s imagination with stories of soldiers’ heroism and patriotism. He told John he was going to the Naval Academy when he grew up. John still had the picture his dad had taken holding him as a newborn, with his shotgun, baseball bat, football, Marine uniform, and a toy motorcycle cop arranged in front of his bassinet.
“Your father would want you to grow up strong like him,” John’s mother had told him and his brother Joe many times as they grew up.
John took on the responsibility of being her eldest son and confidant—more so than she had ever intended. By second grade, he was helping her keep track of their savings, adding up the numbers in her checkbook and balancing the columns. By ninth or tenth grade, he would emerge from his bedroom with charts he had drawn showing the progress of the family’s few shares of IBM and recommending new investments. Mother and son had developed a relationship so intense that even after he left home for college, they spoke almost every day on the phone.
Barbara and her late father, Frank Francis Valentino, a building superintendent, insisted her boys go to college, even though neither of their parents or grandparents had studied beyond high school. For each of her sons, she had saved $20,000 to help pay for college. Not only did John graduate from Georgetown University’s School of Foreign Service, but with a year and a half at the Naval Academy in between, he had gone on to earn a law degree from Notre Dame, and now an MBA from Harvard.
Watching him onstage today, Barbara thought for the hundredth time how differently children could turn out. Her father used to call John “governor,” even as a little boy, and then turn to her younger son Joe and say, “You’re a good boy—I wish I had the money to buy you a gas station.” Joe, who did not attend the graduation, had struggled since his father’s death, always seeming to be the one who ended up in trouble. The boys shared the same playfulness—in fact, Barbara privately thought Joe was far funnier, but the younger boy was a little reckless. He was now working as a police officer in Baltimore, but he carried two other sets of business cards, one saying he was Senator Ted Kennedy’s personal assistant, another claiming to be a talent scout for Paramount Studios. Which card he pulled out depended on the type of woman he was trying to impress. When it came to his brother, John spent half of his time worrying about Joe and the other half regaling his classmates with tales of Joe’s latest antics.2