The Cure: How a Father Raised $100 Million--And Bucked the Medical Establishment--In a Quest to Save His Children

354 Pages·2006·1.65 MB·English

by  Geeta Anand

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 ..........................................................................................................v
Prologue ....................................................................................................................1
1. VERITAS..............................................................................................................7
2. TROUBLE ..........................................................................................................15
3. DIAGNOSIS.......................................................................................................23
4. HOPE.................................................................................................................27
5. GOD DOESN’T GIVE YOU MORE THAN YOU CAN HANDLE.........................41
6. THE ROAD TO POWER AND INFLUENCE .......................................................53
7. MEGAN .............................................................................................................63
8. THE CONFERENCE...........................................................................................77
9. THE MARRIAGE................................................................................................85
10. SHARON..........................................................................................................107
11. BETTING ON RESEARCH ...............................................................................117
12. “LET’S JUST DO IT” ......................................................................................131
13. A ROCKY START.............................................................................................143
14. FAILURE IS NOT AN OPTION.........................................................................157
15. COWBOYS.......................................................................................................161
16. LOSING SUPPORT..........................................................................................177
17. NOVAZYME TIME............................................................................................185
18. MAKING MEMORIES......................................................................................199
iv Contents
19. THE BLUFF......................................................................................................209
20. THE DEAL .......................................................................................................223
21. GENZYME .......................................................................................................233
22. TOUGH CHOICES ...........................................................................................245
23. THE MOTHER OF ALL EXPERIMENTS ..........................................................257
24. THE SIBLING STUDY .....................................................................................269
25. PLAN B............................................................................................................283
26. “YOU CAN TELL MEGAN” .............................................................................291
27. READY TO RUN ..............................................................................................297
After word...............................................................................................................313
Timeline of Major Events ....................................................................................324
Acknowledgments.................................................................................................327
Notes .....................................................................................................................331
About the Author
Credits
Cover
Copyright
About the Publisher
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 in-
terview 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 walk-
ing 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 to-
gether 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,
2 The Cure
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 contin-
ued, “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 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-year-olds, Megan moved quickly and without regard to
those around her. But unlike them, she drove a 400-pound electric wheel-
chair, 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 ca-
dence, eyes imploring in an otherwise expressionless face. As always, John
was awed by how much emotion burst from his daughter’s eyes. He won-
dered 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, nod-
ding vigorously.
“Yea,” Megan shouted, pumping her arms in the air as high as she
Prologue 3
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 smil-
ing, 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 that
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 ab-
sence of any other options, he had simply made his own answers: He quit
his job as a marketing executive, raised $27 million from venture capital in-
vestors, and built a biotechnology company around promising science for
Pompe disease. Two years later, he made what was considered by the busi-
ness community to be an unimaginably successful sale of the firm to Gen-
zyme Corporation, one of the world’s largest biotechnology companies,
knowing it had the money and know-how to get a drug developed faster
than did his own still youthful company. At his insistence, as part of the
deal he was named head of Genzyme’s program to develop a medicine for
Pompe disease.
His intent in making this a condition of the sale had been twofold:
first, he truly believed that the urgency he felt as a Pompe parent would
help speed a treatment to all Pompe sufferers. Second, he wanted to use
his proximity to the drug’s development to guarantee Megan and Patrick
placement in the first clinical trials—the experiments to test whether a
proposed medicine works in human beings as well as it has in animal stud-
ies. In these trials, patients are given different doses of a prospective med-
icine, 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.