by Geeta Anand
With the sick children resting, Sharon lowered herself onto the couch in the den to rest. John and John Jr. were coming through the door again, carrying the same Nerf ball. Sharon looked at the Nerf ball with “Notre Dame” written on it and wondered if he went to that college. His sweatshirt said “Navy” on it, so she had been thinking he was in the Navy. But before she could ask, he was back out the front door. Aileen had made John Jr. lunch. She stuck him in a high chair and plopped a bowl of macaroni and cheese in front of him. Sharon stopped herself from asking what a four-and-a-half-year-old boy was doing in a high chair. Sensing Sharon’s silent question, Aileen said, “I can’t get him to sit still for a second. This is the only way I can get him to eat.”
Sharon watched the little boy lift the fork to his mouth and set it down before he took a bite.
“How long is Sharon staying?” he asked.
“Until tomorrow, John, now eat your food,” Aileen said.
The little boy picked up the fork again, but before he could put the macaroni in his mouth, he saw his father out the window in the backyard trying to fix the swing.
“Daddy, Daddy,” he shouted, trying to get out of the chair. Aileen groaned, pulled a chair up to his high chair and began to feed him herself. Sharon had seen enough children with attention deficit hyperactivity disorder to diagnose one, but for the second time during her visit, she stopped herself from saying anything.
A few hours later, Megan and Patrick were awake, and Sharon got up to help Aileen give them breathing treatments—pounding on their backs, suctioning their breathing tubes, and inserting medicine in them to ease the airflow. Then Aileen and Sharon put the children and their ventilators in wheelchairs and pushed them to a nearby park. As they walked, Aileen’s eyes brightened and her voice grew animated. Sharon noticed that she smiled much more often and chatted easily with the parents of neighborhood children they passed along the way. At length, she pulled up at the edge of a pond, stopping Megan’s wheelchair on the muddy bank just a foot from the water’s edge.
“C’mon, Sharon, let’s feed the ducks,” Aileen said excitedly, giggling as she whipped out a loaf of bread hidden on the little shelf under Megan’s wheelchair, on top of the ventilator. Sharon stopped Patrick’s wheelchair beside Megan’s and joined Aileen, tearing apart slices of bread and flinging chunks into the water. Soon, a dozen ducks were quacking and feasting at their feet.
“Give me some,” Megan piped up. “I want to throw it myself.” Patrick sat quietly beside her, content to watch as Megan went through one slice of bread after another.
Sharon was impressed with Aileen’s attitude and told her so. “You know, Miss Aileen, I’m so glad to see you take the kids out to do stuff. I’ve taken care of handicapped kids for the past twenty years and I’ve seen too many parents who won’t let them go anywhere. If I take care of your kids, I need to know that I can take them out every day to the park and the beach and to movies and picnics. I’m not going to treat them like they’re dying.”
“I don’t have a problem with that,” Aileen said. “I want them to have as normal a life as possible.”
She paused, and then she added, “Of course, we don’t think they’re going to be like this forever. We’re waiting for a clinical trial to start that will get them the enzyme they’re missing and make them all better.”
Sharon started to ask Aileen when this trial was starting, but Patrick was now crying, pointing in the direction of home. “I think he’s had enough,” Aileen said. “It’s all right, honey, we’re going to go home now,” she soothed. She started pushing Patrick’s wheelchair toward the park entrance, and he quieted down.
Sharon had only touched the handles of Megan’s chair to follow her mother when the little girl let out a scream of protest. “I want to stay—just five more minutes,” she wailed.
“I can’t, sugar, your brother is tired and we have to go home,” Sharon said.
As Sharon pushed, Megan cried and twisted, tears streaming down her face. Aileen seemed unfazed, even when the beepers on the ventilator began to wail.
“I’m sorry—Megan does this every time we leave. She never wants to go home,” she said apologetically.
Sharon thought Megan seemed a little spoiled, but she really liked her spunk. “It’s always better when the kids want to do more than to have them sitting around refusing to go anywhere,” she replied, looking at Aileen with a big smile.
They returned home, gave the children sponge baths and more medicine to help with the breathing, and read a few books before settling them in bed. By 8 P.M., the children were finally asleep. Aileen asked Sharon if she wanted a drink.
“Miss Aileen, I would love a glass of wine,” Sharon said, more tired than she could ever remember feeling. Aileen sliced some cheese over a plate of chips to make nachos—dinner. John joined them, and they ate together, standing at the bar in the kitchen.
“Did Aileen tell you about the clinical trials we’re waiting for?” John asked as soon as he joined them. He handed Sharon copies of the Pharming and Duke press releases. She had been curious about the experimental medicine ever since Aileen mentioned it in the park.
Sharon skimmed the documents and asked, “When do you think these trials might start?”
“We’re hoping any day,” John said.
“That’s what they’ve been saying for a full year,” Aileen said.
“If these doctors don’t get started soon, we’ll find other doctors who will,” John said. “We’ve started a foundation of our own. We’re going to raise money so we can fund the trials if nobody else will.”
“Well, good luck to you, Mr. John,” Sharon said. “Nothing would make me happier than to see these babies cured.
“Now, Mister John,” she said, changing the subject to indulge her own curiosity. “I saw you throwing a Notre Dame Nerf ball with your son. Did you go to school there?”
“Yup. For law school.”
“Wow. I ain’t never met anyone who went to Notre Dame,” Sharon said, playing up to him.
“Go ahead, hon. Give her the list of schools,” Aileen prompted John, knowing that he was dying to list the litany of prestigious schools he had attended.
“Well, I went to Georgetown and the Naval Academy undergrad, Notre Dame for law school, and then Harvard Business School,” John said.
“That’s more schools than my whole family combined,” Sharon said, impressed but also recognizing he liked the opportunity to show off—and giving it to him.
“Mine too,” John said. “Lots of great schools. And I’ve got the student loans to prove it!”
“One other thing, and it’s not about schools, it’s about George Washington,” Sharon said, looking from John to Aileen. “I saw a sign coming in from the airport about ‘Washington’s Crossing.’ Is that the spot where George Washington crossed that river?”
“Funny you should ask, Sharon,” John beamed. “Our home is just a few miles from the very spot where George Washington crossed the Delaware River from Pennsylvania into New Jersey to launch a desperate attack on the British garrison at Trenton.”
Sharon had no idea what she had just done. John loved American history. He continued on for ten minutes, delivering a soliloquy about Princeton’s place in American revolutionary history. “And on January 3, 1777, all of this culminated in the battle of Princeton.”
When he finally stopped, Sharon, who had listened to this recitation of American history politely if dispassionately, smiled at him and simply stated, “That’s great, Mister John. Truly fascinating. But you know, a simple ‘yes’ or ‘no’ would have sufficed.”
John laughed with his boyish smile, liking her wit and sarcasm. He knew he’d been had on this one.
“Now tell us about you, Sharon,” he said. “How did a good Southern woman like you end up in New Hampshire?”
“My husband Eddie and I raised our girls in a small town in Arkansas, where I worked as a home care nurse taking care of handicapped children. I took care of
the first baby in Arkansas ever to be sent home from a hospital on a ventilator. I even ran my own nursing agency. But then Eddie—he had managed a men’s clothing store in Arkansas for twenty years, and then he got an offer to be assistant manager of a new Wal-Mart in New Hampshire for a whole lot of money. We sold the store and moved north.”
“How do you like it up there?” Aileen asked.
“We love it,” Sharon said. “Our girls both followed us up there with their husbands and children. We all live together in a big old house with nine bedrooms.”
“Do you really think you could commute back and forth between here and New Hampshire?” Aileen asked.
“I don’t see why not,” Sharon said. “I drive two and a half hours each way to go to a hospital in Massachusetts where I work weekends. On weekdays I do the night shift for a child in Maine, and I work days for a family in New Hampshire. I am already what they call a traveling nurse.”
By the time they went to bed, it was past midnight. They’d been talking and laughing for three hours, and the three of them had emptied two bottles of wine.
When John climbed into bed that night, Aileen said, “I like her, orange outfit or no orange outfit. That woman knows what she’s doing with the kids.”
“She’s a wiseass,” John replied. “She’ll fit in with the family just fine.”
It was one of the few things they had agreed on in a long time.
On Monday morning, the headhunter called to tell Sharon the Crowleys wanted to offer her the job.
“I’m fifty-four years old. Maybe I’m too old for a job like that,” Sharon said. She’d been thinking about it the whole day. “I’m pretty much wiped out from my weekend with that family.” She wasn’t sure whether to accept. She had liked Aileen right off, finding her eager for help and easy to get along with, but John struck her as a bit full of himself with his Notre Dame Nerf ball, his Navy sweatshirt, and all of his colleges and degrees. She couldn’t believe he’d had the audacity to deliver a lecture on American history in response to her innocent question about a road sign. But under that cocky exterior, she was certain he was just as desperate as Aileen, or he wouldn’t be paying a headhunter thousands of dollars to find her.
“Why don’t you at least negotiate salary and see what you think,” the headhunter pressed.
“Listen, have you ever taken care of two handicapped children on vents at the same time, mister?” she retorted. “This family bought a house that isn’t handicapped-accessible—they are so convinced their kids are going to get saved by a miracle medicine. And I just spent my weekend taking the kids and the equipment up and down and up and down the steps.”
“Well, think about it, Sharon,” the headhunter said. “All I can say is this family really liked you, and they definitely really need you.”
A few days later, rested, and having gotten the go-ahead from her family, Sharon called back to say she would talk salary. If she could make the same amount of money taking care of one family, life would be easier than driving to three different homes in three different states, as she’d been doing each week. And like it or not, she knew the truth about herself—she bored easily and relished a new challenge. Those two children were going to get sicker quickly, unless they got into the clinical trial their parents kept talking about. Keeping them alive—let alone healthy and happy—would be a challenge unlike any she’d ever experienced.
So Sharon made what she thought was a fair proposal. “I need a salary of $70,000, and a travel stipend of $800 a month to help pay for the plane fare. And most important, I must have complete control over the nurses. I’ve been in situations in the past where nurses try to go around me and ask the family for a day off. I need total control of nursing.”
The headhunter called back the same day to say the Crowleys had agreed to everything. They had just one question, he said. “Can you start in two weeks?”
11
Betting on Research
Spring–Winter 1999
Pennington, New Jersey; New York, New York;
Durham, North Carolina; Boston, Massachusetts
With life at home stabilized, at least for now, John threw himself back to the task at hand: figuring out which scientist was most likely to succeed with a drug.
Money from family, friends, and Harvard Business School classmates had poured in—more than $60,000 in the first few months of 1999. But who to give it to? There was no one “expert” to ask. Randall was backing Pharming, but Moolhuizen, the head of the Pompe program at the Dutch company, had told John there was no plan to begin a U.S. trial soon. Chen had been delayed several times, and John couldn’t pin him down in telephone conversations on the question of when his trial would start.
Slonim suggested that John meet his friend Frank Martiniuk, the research associate at New York University who had been among the presenters at Randall’s research conference at the NIH. Martiniuk had worked for years under one of the prominent Pompe researchers, Rochelle Hirschhorn, and the two had only recently parted ways. Slonim considered Martiniuk to be the cowboy of the Pompe research community—brilliant but a little undisciplined.1
At Martiniuk’s suggestion, the two men met at East Bay Diner across from Bellevue Hospital Center in Manhattan, where he had an office and lab. Tall and lean, with wavy gray hair, Martiniuk was the son of a New Jersey farmer turned truck driver. He had a hardened New Jersey accent and a down-to-earth manner. “Call me Frank,” he said, smiling widely as they sat down in a small booth in the corner.2
John got right to the point, telling Martiniuk candidly about Megan and Patrick, the Children’s Pompe Foundation, and his desire to fund researchers who could produce the fastest results. “We’re looking for a scientist who can move quickly into a clinical trial,” John said. “We need to beat science—and we need to beat time.”
“You’ve found the right guy then, John,” Martiniuk said, his Jersey accent lengthening the name so that it rhymed with yawn. “I’ve produced some enzyme in CHO cells in my lab. I’ve treated some mice with Pompe disease. And I have to tell you, John, the results are spectacular.” His results were so new he hadn’t had time to confirm them or perform the analysis to present them at Randall’s conference.
“Can I see the mice?” John asked, leaning forward.
“I would be honored,” Martiniuk said. “Let’s call it a date. How about a week from today?”
John, Aileen, and John’s mother Barbara drove in together to Bellevue Hospital for the tour. John wanted Aileen, in particular, to accompany him so she wouldn’t lose hope for the children. He had noticed the distant look on her face when he was telling Sharon about the Pharming and Chen trials, and he knew the delays had dampened her faith that their children would ever get better.
Martiniuk met them at the front gates of the hospital and led them through a labyrinth of crowded corridors to an elevator that took them to the sixth floor. The group chatted nonstop with Martiniuk, discovering that they had a lot in common. He was animated, talking fast and loud, peppering almost every sentence with the words “you know.” He said he lived in Union City, which Barbara rushed to tell him was only about six or seven miles from the house where John was raised and where she still lived.
“John, where’d you go to high school?” Martiniuk asked.3
“Bergen Catholic,” John replied.
“I’m the JV basketball coach there!” Martiniuk exclaimed.
“What’s a smart scientist like you doing coaching basketball?” Barbara interjected.
“I played, you know, semipro ball and I love the sport,” Martiniuk said, stopping in front of a tan door with a sign that said “Radioactivity—Keep Out,” which he said was his trick for keeping out unwanted visitors without having to lock the door. John thought that was very unacademic, and he liked it.
Martiniuk pushed open the door and an appalling, putrid stench emerged, offering the first clue of what lay inside. John followed him into a dark room so narrow and crammed with equipment, plants,
and papers that the group had to walk single file. Old magazines, tomato plants, antique-looking bottles of chemical reagents, and a few cages of mice lined the shelves.
John turned to Aileen, who was following close behind. She raised her eyebrows skeptically and gave a slight shake of her head. John shrugged his shoulders a fraction of an inch in return. This looked nothing like the labs they had seen at Duke University or Bristol-Myers.
“This mouse has been getting enzyme for, you know, three to four weeks. Look at him go. He’s like an athlete, an Olympic athlete,” Martiniuk said, pointing to a sleek brown mouse in a cage. The mouse was scampering energetically through a maze of half-chewed cardboard pieces. “And see those two sick-looking mice in that cage,” he continued, pointing to a cage on the shelf below with two animals with shabby, unkempt coats, awake but unmoving. “These are untreated mice with Pompe disease. Don’t they look terrible?”
“Dr. Martiniuk, are you telling me that this healthy mouse over here looked like those sick ones over there before you gave them your enzyme?” Barbara asked.
“You better believe it,” he said, with a wide grin. “I’ll give these sick ones the enzyme tonight, and you come back here in eight weeks and they’ll be just like these strong mice over here.”
“Do you want to see the enzyme?” he asked, opening a seven-foot-tall refrigerator with glass doors to pull out a vial of colorless liquid.
“I make it myself,” he said, “right over there.” He pointed to a hood in the corner of the room. The hood contained a small glass vial with a tiny mechanical arm that was spinning the liquid inside the vial. There were scores of apparently unrelated beakers and vials under the same hood.
