by Geeta Anand
Although they’d been talking on the phone for months, Greg hadn’t met John in person until the evening of the dinner. “I feel like I’m meeting my twin,” Greg gushed when Aileen led him over to John, who was bidding in the silent auction. The two men shook hands and hugged. They looked nothing alike—Greg, balding, blue-eyed and blond, stood half a foot taller—but with their boyish smiles, they exuded the same unabashed enthusiasm. Greg handed John a thick wad of checks. “This is everything we’ve collected from our friends and relatives—close to $20,000,” he said with justifiable pride.10
John kept Greg by his side for much of the evening, introducing him to his and Aileen’s extended families, the Bristol-Myers executives, and the Pompe researchers who were among the three hundred and fifty people in attendance. Martiniuk, whose JV basketball team had a game that night, didn’t come, but Drs. Slonim and Chen were gaily socializing, as was Dr. William Canfield, the naysaying researcher from Oklahoma whom John had first met at Randall’s conference. Canfield had made a trip to meet John in New Jersey earlier in the year, looking for funding for a new company he had started, and although John hadn’t given him any money, he had invited him to the fund-raiser.
“Aileen, I think we’re finally where we need to be,” John whispered to her, choking up as they stood up from the head table—he in a black tuxedo, she in a purple taffeta gown—welcoming the guests. “This is amazing. All of these people and they’re here for us, for the kids.”
Then he played the video showing him and Aileen at home with Megan and Patrick, who sat on either side of the couch in the den, their ventilators beside them. Aileen described the children’s healthy infancy, their devastating diagnoses, and their rapid weakening to the point that they now needed ventilators to breathe. The camera closed in on John carrying Megan, a floppy white bow in her long brown hair, with Aileen’s melodic voice filling the room. “On most nights, Megan always dances with her daddy before she goes to bed,” Aileen said. Then the camera focused on Aileen’s wide-eyed, cherubic face, seeming to radiate hope, as she blinked, nodded her head, and said, “I just believe, I know in my heart that one day she’ll be dancing with him at her wedding. I just know we need to keep pushing the science, and one day they will be cured. They’re going to be better, one day, soon.”
By the end of the night, the Children’s Pompe Foundation had taken in another $150,000.
Greg went home from the event feeling even more taken with the Crowleys than he had been from their conversations over the phone. “They’re just like us,” he told his wife. “Every aunt and uncle and cousin and grandparent was there supporting them.”
The single biggest donation that John and Aileen received that year, however, was not one they had personally solicited. Like most of John’s friends, his childhood buddy Tom Westdyk had been helping raise money, and his wife had mentioned the Crowleys’ mission to a neighbor who ran a family foundation. The neighbor, moved by the story, invited John and Aileen to her office and told them her foundation would donate $250,000 on the condition the gift remain anonymous. The Crowleys thanked her gratefully.
As 1999 drew near a close, John had money to give out and influence to exert. For the first time, it felt like he had some ability to control the direction his children’s lives were taking, and some ability to meld the areas he was good at—business and management—into the world of medicine and disease in which he’d previously been so helpless. He liked being in this position, and he didn’t waste any time. He wrote several other checks: one for $100,000 to Chen, another for $60,000 to Martiniuk, and a final one for $100,000 of his $500,000 commitment to Slonim.
All that was needed now, John thought, was for another clinical trial to begin.
12
“Let’s Just Do It”
Winter 1999–Spring 2000
Princeton, New Jersey; Durham, North Carolina;
Oklahoma City, Oklahoma
In the fall of 1999, Dr. Chen called John at his office to ask for his help reviewing a manufacturing contract. John had evolved into more than just a benefactor to the Duke doctor. With his legal background and his job at a big drug company, he had become a trusted adviser to both Chen and his Taiwanese corporate partner, Synpac Pharmaceuticals. They had previously told John that their biggest challenge was manufacturing the enzyme. John used his connections at Bristol-Myers to try to help. First, he tried to broker a deal for Bristol-Myers to buy Chen’s research program, bringing the Duke scientist and his Synpac partner to a meeting with Bristol’s top drug development team. While the company officials wanted to help John, they could not legitimize investing millions in such a risky project in a disease they knew almost nothing about. So John had settled for the next best thing—getting Bristol-Myers to pull a biotechnology drug manufacturing plant off the selling block and sign a contract to produce Chen’s enzyme there.1
Today, Chen spoke hurriedly, as if he wanted the call with John to be over as quickly as possible. John, as always, pressed for details on the progress of the clinical trial.
“How are the babies doing?” he asked. He knew two additional babies had joined the trial.
“Not doing so good,” Chen said.
“What? Why?”
“There could be many reasons,” the doctor said tersely.
“What is your hunch?” John pressed.
“Antibodies,” came the cryptic reply.
“What are they?”
“John Koncel’s body seems to be viewing the enzyme therapy as a foreign substance,” Chen said. “His body’s immune system is making antibodies to knock off the enzyme. So the enzyme is not having much effect anymore.”
“What about the other baby?”
Chen said the second baby in the trial was still doing well, but he hadn’t been receiving the therapy as long. He couldn’t predict if the other baby would develop the problem as well.
John hung up and sat silently, trying to absorb the blow. He felt like he was floating in a cold sea, and the life preserver he had been reaching for had been violently ripped away. He was supposed to travel to Duke’s business school the next week to recruit employees for Bristol-Myers, and he decided that he would drive over to Children’s Hospital and find out more.
The day John arrived at Duke, baby John Koncel was receiving one of his weekly infusions. By now, John was not only a major benefactor of Chen’s, but he was also such a frequent visitor at the hospital that a nurse led him right to baby John’s room. The baby was in the same room as before, this time alone, lying in his crib. John walked over and peered inside at the eight-month-old baby, seeing immediately that the little face that had been so alert the last time he visited looked pale and tired. The baby had a tube in his nose for feeding, which meant he was having difficulty swallowing.2
“Oh, you poor little guy,” John said, choked up, stroking the baby’s head.
“He was doing so well before,” the nurse said.
“I saw him myself,” John nodded.
“He got even stronger than that. We even let his mom take him home to an apartment nearby…” She gestured unhappily at the baby’s limp arms.
“How long has this been going on?” John asked.
“A couple of weeks.”
He shook his head regretfully. “And how are his parents doing?”
“They’re incredibly strong,” the nurse said. “His mother is by his side constantly. She must have stepped away for lunch, but other than mealtimes, she never leaves. His father flies out here from Chicago all the time. I don’t know how they do it.”
They stood by the crib in silence, listening to the labored sound of the baby’s breathing. His nostrils flared with each breath, a sign of the effort it took for each gulp of air.
“I hate to say this,” John said, “but he looks and sounds a lot like my own son Patrick did right before we had to put him on a ventilator.”
A few days later, John dialed Martiniuk, trying to get an update on his research.
�
��He’s in the lab,” his assistant said, promising he would call back.
A week later, John called Martiniuk again. Again, his assistant said he was in his lab, and again she promised that he would return the call.
Frustrated, John realized he had no idea what Martiniuk was doing with the money from the Children’s Pompe Foundation. In the weeks after he’d agreed to fund Martiniuk back in April 1999, John had tried to broker a deal between the scientist and a biotech company in California called BioMarin Pharmaceutical, which was interested in developing his research into a treatment. John had concluded, rightly, that scientists weren’t experienced in turning research into medicine and could move much faster with the funding and expertise of a drug company behind them. BioMarin was developing a treatment for a related disease, also caused by a defect in an enzyme—but a different one—responsible for processing waste material inside cells. John thought it would be a perfect partnership. He flew Martiniuk and Slonim with him to California to meet with BioMarin. Soon company executives had flown east to follow up. It looked like a match made in drug industry heaven, until BioMarin sat down to make a deal with lawyers for New York University Medical Center and failed to reach an agreement. Martiniuk was disappointed, but John was crushed.3
Never one to dwell on failures, John’s attention had shifted quickly to Chen, who had called soon after with news of FDA approval to start his trial. Chen had consumed almost all of John’s time and attention—and hope—that summer and fall. Now he realized he had no idea where Martiniuk stood with his research. With Chen’s trial in trouble, John wanted and needed to know about Martiniuk’s progress. Almost a year had gone by since he’d given him the first check, and he didn’t even know whether Martiniuk was close to starting the clinical trial for his children.
When no call came in from Martiniuk the next week, John dialed again. This time, the scientist picked up.
“Frank, it’s John Crowley. I was just wondering how the research is going.”4
“Things are going very well, John,” Martiniuk said. “It’s been very busy. I’m in the middle of a very important experiment now. And I have two grant applications due next week. I’ll give you a call after that, I promise.”
“I was just wondering if you could send me a progress report or something on your research, Frank,” John said.
“Things are going very well, John, very well,” Martiniuk repeated.
“Well, how close are you to filing the IND?” John asked, referring to the application to the FDA to begin a clinical trial. “A year’s almost up and I was wondering if we were close.”
“The research is going great. We’re testing the enzyme in older mice to see if we can reverse the defect, and they’ve been able to recover. We’ve reversed the defect! They’re running on the wheel. I’m doing studies now looking at how low we can dose them. But we need to do more work to file with the FDA. We’re going to need more money and more time to get there.”5
That night, John came home and headed right into his study. Aileen knew something was wrong when he didn’t come upstairs first to help her put the kids to bed. After she had put the kids to sleep, she found him on the phone with Dr. Slonim. From the kitchen, as she boiled a pot of pasta for the two of them, she listened to him talking, his frustration making his voice louder and shriller.
“I’m getting worried, Alf,” he was saying, now so close to Slonim that he called him by his nickname. “Chen’s trial isn’t going so well. Baby John has built up antibodies. And I’ve no idea what’s going on with Martiniuk. He says he’s getting great results, but he doesn’t seem close to filing his stuff with the FDA to begin a trial for the kids. And now he wants more money.”
Whatever Slonim was saying seemed to have a calming effect on John. John always trusted Slonim’s advice.
“I appreciate that, Alf,” John said before hanging up. “I’d just like to find out how far he is from filing the IND.”
“Having trouble with Martiniuk?” Aileen asked when John emerged from his study.
“I’m pissed that he doesn’t seem anywhere near ready to get papers to the FDA to start a trial,” John said. “It’s almost impossible to reach him. Alf is going to call and get me an update.”
“I didn’t know about baby John,” Aileen said quietly, as she set the dining table.
“I didn’t want to worry you,” John said. “It’s not all terrible, it’s just that the enzyme isn’t working on baby John right now because his body’s immune system is making things called antibodies to fight the enzyme. Chen is trying different strategies for getting rid of the antibodies. Hopefully, one of them will work.”
Aileen was quiet. Ever since she had seen baby John, she had been counting on Chen’s enzyme to save her children. The disappointment she felt now was too enormous to absorb. Instead of trying, she changed the subject.
“I’ve been thinking about what we should do for Megan’s birthday,” she said. “Let’s take her on a special trip out—just the three of us. We’ll take her to that cute little toy store at the mall and let her pick out whatever she wants. And then we can go to Friday’s with her for dinner.”
“What’s Megan going to wear?” John asked, giving in to Aileen’s need for diversion. This was one way he had adapted since deciding to come back to her that night nearly a year ago. People had their own ways of coping, and he tried not to be so judgmental about hers.
“I found the perfect dress at Baby Gap,” Aileen enthused. “It’s blue with little flowers.”
“It sounds beautiful, honey,” he said, kissing the top of her head gently. Aileen gave him a brilliant smile.
That night, after Aileen had gone to bed, John sat at his desk in his study, thinking. He needed another backup. There was only one more horse to bet on—and it was a long shot.
He had held off investing in Dr. Canfield’s new company, even after meeting twice with him over the past year to discuss his research. John and Marty, Aileen’s dad, had even flown out to Oklahoma City for a day to see Canfield’s operation firsthand. But John had continued to hold off, in part because he had been so convinced that Chen’s research was the answer, and Canfield was just a skeptic, insisting the Duke scientist’s enzyme treatment would never work because it didn’t have the right attachments of carbohydrates and phosphates. But also—and perhaps more crucially—Canfield was so far behind Chen and Pharming that John really hoped he was wrong. Canfield was still testing his potential drug in test tubes, where Chen and Pharming were in human clinical trials. Megan and Patrick didn’t have the time to wait for him to test his treatment in animals and advance into human clinical trials.
But with Chen’s success in doubt and Martiniuk’s progress uncertain, John decided it was time to reach out to Canfield. He picked up the phone and called Oklahoma City, counting on the time difference to find Canfield still in his office.
“Dr. Canfield, I’m calling with some really good news,” John said. “We just got two hundred and fifty grand from a family foundation. We’d like to invest it in your company.”6
Canfield, who had been struggling to get his company off the ground, didn’t say anything. He seemed stunned at his sudden good fortune, and it took him a few moments to respond. Then he bubbled over with enthusiasm. “John, that’s the best news I’ve heard in a long time! Shall I come out there? Do you want to come here? How shall we proceed?”
John told him he would fax over an agreement shortly and, once it was signed, a check would quickly follow.
The agreement arrived at Canfield’s office a few days later. At his desk, he reviewed the typewritten pages. In exchange for its investment in his company, the Children’s Pompe Foundation wanted a seat on Canfield’s company board, 2 percent ownership, and a commitment that the first clinical trial would test the therapy in patients with Megan and Patrick’s nonclassical form of Pompe disease. Canfield had known the foundation might seek a board seat and a small amount of equity, but he had never imagined it might try to influe
nce the direction of his research and drug development. He slammed the pages onto the table and picked up the phone.
“I knew it was too good to be true,” Canfield fumed to his lawyer, Doug Branch. “I could never agree to tie my hands in that way.”7
That same day, Canfield called to tell John he couldn’t agree with the investment contract. “I want to treat your children,” he said quietly, “but I cannot accept these terms. It would be unethical.”
John backed down immediately. “Fine. We’ll take that provision out, Doctor,” he said. “We trust you to design the best trial possible for all children.”
“It was worth a try,” he told Aileen’s dad Marty later. “But we’ll have to find another way to work with him.”8
By January, Canfield had the $250,000 in the bank, and the Children’s Pompe Foundation owned a board seat and a small stake in Canfield’s nascent company. And Canfield was finding his new investors to be a fountain of assistance in many ways besides financial. He needed to find a chief executive to run the company, and John and Marty agreed to help. When a search firm turned up a potential candidate for CEO, Marty agreed to interview the guy. He called Canfield back, confirming the scientist’s impression that the candidate wasn’t up to snuff. “He doesn’t have the fire in the belly—let’s keep looking.”
After another couple of weeks went by and no better candidate emerged, Canfield began to call, downcast.
“I don’t think I can get the right candidate to Oklahoma to work for a small startup,” he said to John in a late-night phone call.9
“You may be right, Bill,” John said, now on a first-name basis with Canfield. “Hell, maybe I should come there and run the company for you.”