by Geeta Anand
“Yes, that’s Patrick back there. Our poor little redheaded ‘stepchild,’” Aileen said, joking about how often her easygoing baby was forgotten. “He’s so quiet and such a good baby. We also have an older boy, John, who’s home with Nana—that’s my mother.”
“How are the other two doing?” Mrs. Walter asked. “Any symptoms of—?”
“They’re both fine,” Aileen said quickly.
“That’s good, because we’ve talked on the phone to other families with the disease. Sometimes the siblings have it too,” Mrs. Walter said.
“Megan Crowley,” the receptionist called out. The couples exchanged phone numbers and promised to talk by phone in a few days.
Dr. Chen was as warm and deliberate in his exam as he had been on their last visit. He checked Megan’s gastrostomy and did what they now knew was the routine Pompe exam. He asked Megan to raise her hands. He put her on her stomach and she flipped over. She did everything he asked eagerly, eyes watching him. She didn’t seem measurably weaker. He made a few notes and was ready to say good-bye when his eyes fell on Patrick, who was sitting in Aileen’s lap.
“How’s the little boy?” he asked.
“He’s very strong,” John said.
“He eats very well, and look at his legs,” Aileen said, pulling up one of his pant legs to reveal a fat calf.
“While he’s here, if it’s okay, I’d like to do a blood test to rule out Pompe disease,” Chen said.
“I don’t think it’s necessary, Doctor,” John said, remembering how Megan had shrieked when she’d had blood taken. “Patrick’s so strong and so healthy—he’s very different from Megan, who was always weak.”
“Let’s test him, just for peace of mind,” Chen said, gently but firmly.
Aileen nodded, and John relented. She held Patrick as a nurse drew blood. John turned the conversation to the main reason he’d brought Megan back for a checkup.
“So, Doctor, how is the clinical study looking?” John asked.
The doctor frowned, and he shook his head.
“The CHO had a bad run,” he said.
“What’s that?” John asked, wondering if the doctor was using a Chinese word. Chen spoke with a strong Chinese accent.
“These are the cells that we grow the enzyme in,” Chen said. “They are originally taken from Chinese hamsters’ ovaries, so we call them CHO for short. Unfortunately, we’re having lots of trouble with them.”
John pressed for more details and found Chen to be a patient explainer of science. He told John that the enzyme was made in giant bioreactors by the CHO cells. John remembered reading about this on the Internet the night after Megan’s diagnosis, but he hadn’t understood how the process worked. These cells acted like little factories, churning out the missing Pompe enzyme, acid alpha-glucosidase. The process had been used for about a decade, since scientists had discovered that cells culled from the ovaries of this breed of hamsters not only multiplied quickly in a test tube, but also produced enzymes and other kinds of proteins that seemed to work effectively in humans.
Chen told John the manufacturing process was still very tricky—as much an art as a science. The cells had to be grown carefully in a sterile environment. The smallest mistake could contaminate the product and force scientists to begin the three-month-long process of producing protein all over again.
“That’s what had happened to the company in California making enzyme for us,” Chen said. “Bacteria got inside; maybe from the air handling, maybe from not being cleaned right. It happens all the time.” He cleared his throat and shrugged. “Either way, unfortunately, we can’t start without that enzyme. We won’t be going into trials anytime soon.”
Aileen looked down, quiet. The thought that things could go wrong when famous doctors at big-name hospitals were working on something had never occurred to her.
Two days later, the phone rang at Aileen’s parents’ house. It was Dr. Chen. Aileen, biting her lip, her face pale, stepped into her father’s study to take the call. Like the rest of her parents’ house, the walls of her father’s study were crowded with gold-framed family pictures. Aside from the big wooden desk in the center and a few books on the shelves, the rest of the study was all snapshots of the children and grandchildren.
As she picked up the phone, Aileen noticed her mother standing outside the door. Needing privacy, she felt the urge to hang up the phone and call the doctor back, but she knew she needed to know what the doctor had to say even more urgently.
Chen greeted Aileen in a quiet, concerned voice. He asked how the children were doing. “Fine, Dr. Chen,” she replied. Then, without fanfare, he told her he regretted to report that Patrick’s blood test had come back showing a high level of the same enzyme Megan’s did—CPK, or creatinine phosphokinase.
“But, Mrs. Crowley, I don’t want you to think too much until we do another blood test,” he continued. He didn’t have enough blood from the sample taken to be certain the results were accurate. He asked Aileen to take Patrick to a clinic nearby to have a second blood sample taken and sent to Duke for a confirmatory test.
“I’m really sorry to bring this bad and confusing news,” he said.
Aileen didn’t ask a single question. She thanked him, hung up, and put her head on the desk. Behind her, she felt her mother stoop to put her arms around her and hug her tightly, but Aileen pushed her away in defiant disbelief. How could this be happening? Chen had said the results weren’t reliable, but she knew that was just to cushion the blow. She knew they were. Her son Patrick had Pompe disease, too.
John was at his desk at Marakon in Stamford, Connecticut, when the phone rang.
“You’re not going to believe this,” Aileen said. It was how she began many conversations, prefacing what she had to say with a question or a dare for him to guess what the news might be. Her voice, normally so cheerful and sweet, sounded distant and rough, almost angry.
“Dr. Chen just called,” she said. “They got the bloodwork on Patrick. It looks like he has it too.”
John sat at his desk for a few minutes after hanging up. Everything had stopped. Everything had changed. He needed time—time to help his family. No more late nights at work. No more getting phone calls at his office two hours away from home. He picked up the phone and called the senior partner. They exchanged small talk, but John got quickly to the point. “For family reasons, I’ve decided Marakon is not the best place for me.”
“I understand,” came the measured response. John had told this partner and others about Megan’s dire prognosis to get the transfer to the Stamford office. “I’m disappointed, but I understand your family situation. When do you plan on leaving?”
“Two weeks,” John said without apology. He thought about telling this man the crushing news he’d just heard about Patrick, but he couldn’t.
Driving home that night, John remembered his mother saying, “God doesn’t give you more than you can handle.” Well, she couldn’t have been more wrong. She’d told him many times that because he’d experienced such a terrible loss as a child with the death of his father, he could count on the rest of his life being easy. It was unexplainable and inconceivable, John thought, that a loving God could ask any man, any family, to bear this death sentence that had just been handed down on a second child.
He walked in the front door and found Patrick alone in the living room on his mechanical swing. John pressed the stop button, picked up the baby, and held him high over his head. The baby opened his mouth and smiled, a fishing line of drool bobbing in the air above John’s face.
“You, little buddy, are not going to get sick,” John whispered. “No way you get sick.”
6
The Road to Power and Influence
Fall 1998
Rumson, New Jersey; Amsterdam, Netherlands; Geel, Belgium
During the summer of 1998, with Megan and Patrick strong, playful, and looking to everyone else like perfectly healthy kids, John and Aileen could consciously ignore the disease. Th
ere was so much to do. John focused on his job search, and, after a series of interviews, accepted a $120,000-a-year position as a director at Bristol-Myers. He was thrilled with the salary and the expectation of working eight to ten hours each day, seemingly a vacation compared to the demands of consulting. He liked the idea of running a business group, and the job at Bristol-Myers put him in a lead role in marketing neuroscience drugs. He and Aileen began to look at houses for sale close to Bristol-Myers’s headquarters in Princeton.
Aileen spent almost every minute of the day feeding, bathing, and playing with the children. She would leave one kid at home with her mom and take the other two to the neighborhood pool or the park, making sure they all got a chance to get out of the house each day. It was little John Jr., now three years old, who took most of her energy. He was unfocused and hyperactive. She couldn’t put a bowl of Cheerios in front of him and expect him to eat three consecutive spoonfuls. No sooner did she turn around to get a cup of juice than he had jumped up and was halfway up the stairs to his bedroom. If she left him alone in the bath for five seconds to get an extra towel out of the closet, she’d find him running through the house naked and wet. She wondered aloud to her mother how she had managed to take care of him and the two younger ones on her own before they left Walnut Creek, and told John she didn’t know how she was going to handle all three when they eventually moved out of her parents’ house. He took her worry as a call to action and immediately filled out an application for an au pair in a program he had seen advertised.
At night, John continued to search for information on Pompe disease in his father-in-law’s silent study, with only the silver-blue glow of the screen lighting his way. He reached into the dark portals of the Internet for any new hope. He regularly checked Randall House’s patient group website, which seemed to be updated frequently with news of the Pompe world.
One night, John found a new press release on the site. It was two pages long and announced a partnership between Pharming and Genzyme, the Cambridge, Massachusetts, company that sold a drug for the related Gaucher disease and was one of the most successful biotechnology firms in the world. Henri Termeer, the chief executive, was quoted as saying, “We are very excited about working with Pharming to develop this potentially lifesaving treatment.” The release also said that Pharming would be starting a clinical trial later that year.
The name Genzyme sounded familiar to John, and he realized that it was because the company had had a palatial brick manufacturing plant across the street from his Harvard dorm. Through Genzyme’s website, he found that it employed five thousand people and had a stock market value of about $2 billion.
“It can only be good news if a big company is interested in Pompe disease,” he said aloud in the dark room. He saw the deal as an endorsement of Pharming’s experimental Pompe medicine and a sign of hope for his children.
If Megan’s and Patrick’s lives were to be saved, they needed to get into the clinical trial that the press release spoke about. On the Harvard Business School website, he searched for fellow graduates working at Genzyme or Pharming, hoping for an opportunity to leverage his membership in the Ivy League elite to help his children. The network of graduates extended into almost every major corporation. Sure enough, he found fifteen graduates at Genzyme, but didn’t recognize any of the names. Pharming, far smaller with just about a hundred employees, had none.
John remembered that when he met Dr. Slonim in that hotel conference room, he had been introduced to a Pharming executive named Gerben Moolhuizen, and he had saved the man’s business card. John fished it out of his briefcase now, wondering if Moolhuizen was senior enough to be making decisions on whom to include in Pharming’s upcoming clinical trial. Megan and Patrick had to be in that trial. He needed to push their case with whoever was in charge at Pharming, even if that meant going to the Netherlands—a place he had never been to, much less thought about.
At his desk at work the next day, John called Slonim and asked what he thought of the idea of traveling to the Netherlands. “Sure, John, I think you should go over and see what’s going on for yourself,” Slonim said. “You never know what can come out of a trip. I went there myself last year, and look at the result—I’m going to be running Pharming’s trials in the United States.”
John hung up and pulled out a legal pad and began to draft a letter in longhand. “We met at the Inn at Great Neck where Dr. Slonim was examining my daughter, Megan,” he wrote. “I have since learned that my son Patrick also has Pompe disease, and I would like to learn as much as possible about the treatments being developed. I am interested in traveling to the Netherlands next week to visit with you and tour your facilities.” He put the pen down. This company was probably flooded with letters like his from desperate parents seeking to cozy up to company officials and get their child in the next trial. He needed to do something to distinguish himself.
Then he thought of Randall at the head of the conference table, with Moolhuizen and Slonim designing the clinical trial. What separated Randall from John and every other parent? The answer was obvious—Randall was the head of a patient group. He was a wealthy person who had personally funded a research conference for scientists working on Pompe disease.
Money, John thought. It always came down to money.
He picked up the pen, drew a line through the page he had written and flipped it over, beginning anew. “I am chairman of the recently formed Children’s Pompe Foundation,” he wrote. “Our goal is to raise $1 million over the next several months. I would like to learn more about your technology and planned clinical trials and discuss with you in detail the various ways in which the Children’s Pompe Foundation may advance the efforts of your company.”
It really wasn’t a lie, he told himself. He and his father-in-law had talked late one night over a bottle of wine about starting their own foundation to raise money for the disease. They’d even downloaded the requisite tax form from the Internet. He intended to start a foundation, and he would. He stared at a picture on his desk that Aileen had framed for him of the three children: Megan looking like Pebbles from the Flintstones, her hair in a ponytail on top of her head; chubby Patrick, his eyes wide and angelic; and John Jr. with his huge, manic grin. John had never known that he could love anything so much as he loved his children—as he had loved them since the day they were born. How far would he go to save them?
He stood up, tentatively, to take the letter to his secretary to type—and then a troubling thought pushed him back into his seat. Was it really right to be claiming to be head of a foundation that didn’t yet exist? Was that how he, John Crowley, son of a cop, formerly a star student at the Naval Academy, a lawyer by training, an executive at a big drug company, should comport himself?
And then he realized that he really didn’t care whether he was being completely forthright with Pharming. This was not about making sure you obeyed the rules so you advanced up the ranks at the Naval Academy. Slonim had painted a grim picture of how weak and sick the children would get over the next year unless there was some intervention. Nobody else was going to make sure Megan and Patrick were chosen when the clinical trial was ready to start. There was no one else to count on, no one who would save his kids. And without that experimental medicine, his children would die.
John walked briskly to his secretary’s desk and handed her the letter. In a few minutes, she was back, sliding the typewritten draft in front of him. John read it over and jotted a few corrections. “Here’s the phone number in the Netherlands—please fax this right away,” he told her.
The very next day, Moolhuizen was on the phone suggesting dates for a trip. John’s instinct had been correct. Drug companies pay attention to patient groups. They can help companies by paying for vital research, influencing politicians, and prevailing upon the FDA to approve a drug more quickly.
Before John left for the Netherlands, there was one more phone call he had to make: to the House family.
It wouldn’t be an easy conve
rsation. John knew that Randall felt his own foundation, AMDA, was doing everything possible to further research on Pompe. What he didn’t know was that Randall was very close to getting his own daughter, Tiffany, a place in Pharming’s upcoming trial. Having spent three years courting Pharming’s senior management, Randall was loath to let this brash new kid—in some ways, he thought, a younger version of himself—stride in and endanger all he’d done to save Tiffany.1
Now, on the phone, John described his plans for the Children’s Pompe Foundation—for all intents and purposes a competing organization. Even though he knew Randall would be unenthusiastic, he hadn’t anticipated the depth of his antipathy toward the idea.2
“It’s not a smart idea, and I don’t like it at all,” the older man responded. The AMDA was already leading the charge; for another organization to come along now would just muddy the waters—and possibly divert funding and attention away from the AMDA. “What you’re suggesting is a waste of time and money,” Randall told John, his voice cold. “It’s best just to take care of your kids.”
“We don’t want to compete with you,” John said, trying to assure him that they wouldn’t be working at cross-purposes. “We just want to raise money from our family and friends, and if our family is in the forefront, we can do a better job of that.”
“I think it’s a bad idea,” Randall repeated in a clipped tone.
After a few more back and forths, it became clear that neither man was going to bring the other around. “Well, Randall, I’m sorry you feel this way,” John said politely but defiantly as he brought the conversation to a close. “We each have to make our own choices, do what’s best for our families, and starting a foundation is what’s best for Megan and Patrick. So that’s what I’m going to do.”
John hung up, wondering if he’d made the right choice.
