The Cure

by Geeta Anand

  An hour later, John Jr. noticed someone missing in the usual Sunday lineup and asked, “Where’s the nurse?”

  “She quit,” John said, the words tumbling out before he realized he was lying in the presence of a rather outspoken witness.

  “Oh no, that’s not what happened,” Megan said, wagging her finger at him slyly. “Daddy told her, ‘Get the fuck out of here.’ ”

  John did a double-take, then laughed out loud. “You stop that, Megan,” he said, as seriously as he could, “before I tell Mommy what word you just used.”

  “Don’t tell Mommy,” she said, nervously. “Please, please, Daddy, I won’t ever say that word again.”

  By firing the nurse, John had ruined Aileen’s plans for the two of them to take John Jr. on a trip to her nephew’s sixth birthday party in Scranton, Pennsylvania. Now Aileen (a better nurse than John) had to stay home with Megan and Patrick while he went to the party with John Jr.

  But even backing out of the driveway proved too much for John that day. He heard a crunch and then a loud pop, jumping out of the car to find he’d driven over the portable TV set for the handicapped van. He kicked the back bumper, shouting, “I hate this car, I hate the nurses … I hate this disease.”

  Aileen came over from the doorway, where she had been waving good-bye, and wordlessly pulled John into her arms. They stood together, clinging to each other, until John Jr. climbed out of the backseat and flung himself at them, shouting, “Cool—group hug!”

  27

  Ready to Run

  Winter 2002–Spring 2003

  Cambridge, Massachusetts; New Brunswick, New

  Jersey; Princeton, New Jersey

  On Monday morning, Dr. Landy sat in a conference room in Genzyme’s headquarters in Cambridge with a dozen other members of the Pompe leadership team, waiting for John to show up. John was usually the first one there, ebullient, chatty, teasing McKinney or telling a funny story about his kids until the full team was assembled. They had read John’s letter that morning with relief and sadness. Landy wondered what shape John would be in. An uneasy silence prevailed.1

  When John finally appeared—a quarter hour late—he looked tired and drawn. He was dressed impeccably in his usual navy suit and red tie, scrubbed, every hair combed into place, but Landy could see plainly that his eyes were sunken, his posture was slumped and weary, his skin had broken out in acne. He went briskly to the podium, unsmiling and business-like, and then led the group through the usual round of updates from subcommittee chairmen on manufacturing and clinical trial planning. The bioreactors were working well. The clinical trial protocols were almost ready to be mailed to the FDA. Dr. Kishnani, who was running the clinical trials at Duke, was reporting very encouraging results in her eight-patient trial at the end of one year of therapy. John played a video she had sent that showed two one-year-old boys in diapers, sitting, standing, walking, and even climbing up stairs.2

  The news was almost all positive, but there were few questions and no laughs. Like Landy, most people in the group had come to like John and they were embarrassed by the Florida debacle. John made only one reference to what had happened, about an hour into the meeting. “We’ll deal with Megan and Patrick after we get the filings done on the two main trials,” he said crisply, echoing the e-mail from Friday night that they had all read by now.

  Landy approached John during a break and said, “Thanks for sending that e-mail. I think it was the right thing to do.” When the meeting was over, he asked John if he would come to his office and apologize to some members of his staff who were still upset. John hated the idea, but dutifully followed Landy around his department, thinking he had no choice if his children were to stand a chance of being treated. “I’m sorry if I let you down and put you in a difficult position,” John said to one person after another.

  Landy’s anger had subsided, and he felt mostly sorry for John. He hoped John’s apology would calm some of the younger people in his department who were still consumed by a self-righteous rage that John had gone behind their backs to set up the trial for his children. Perhaps, thought Landy, only those who had children and experienced firsthand the depth of a father’s love could appreciate why John Crowley had gone to Florida. The more he thought about it, the more he appreciated the restraint it took for John to wait as long as he had for a treatment. By this point, John had been at Genzyme almost thirteen months.

  That afternoon, determined to find a way to treat John’s kids, Landy called members of Genzyme’s sales team in New Jersey and asked if they would check with doctors and hospitals they worked with in the Princeton area to see if any might be willing to run the Sibling Study. Then he returned to his main job—trying to finish and submit the protocols for the two main clinical trials to the FDA for approval.

  As always, another crisis erupted, this one having nothing to do with John. A health minister from Spain was on the phone, begging Landy to send some of the extremely scarce experimental treatment to a baby dying of Pompe disease in Madrid. Desperate entreaties from parents were an almost daily experience for Landy and others at Genzyme. He politely told the minister there was barely enough experimental drug to run the upcoming clinical trials. “I’m very sorry to tell you Genzyme will not be able to provide drug for this child.”3

  But the Spanish health officials didn’t stop there. They called Henri; they reached out to van Heek; they beseeched John. Soon the U.S. ambassador to Spain was calling all of them. European governments not only were more lenient in their approval of the use of experimental treatments on dying patients, they were not above pressuring companies to make medicines available. European health officials held enormous sway over drug approval—and pricing—and the company couldn’t afford to alienate them. Genzyme’s leadership worried that the company would face retribution—that their drug approval would be held up or their pricing questioned—if they didn’t figure out a way to get drug to the child.4

  Van Heek told John to make the arrangements to send a shipment of the Pompe enzyme to Spain. Scarcely a month earlier, at van Heek’s instruction, John had coordinated another shipment, under pressure from the government of Italy. The Italian media had been carrying almost daily stories about a couple who had chained themselves to the gate of a hospital and were vowing to starve themselves until Genzyme produced some of its experimental medicine to save their two-year-old daughter Rosella. Pictures of the health minister presenting the Genzyme enzyme to Rosella appeared everywhere.5

  Back in September, John had been excited for Rosella and also for himself, believing Megan and Patrick would be next. But now there were no immediate plans for his children, and he was being asked to ship more of the scarce enzyme to someone else’s child.

  The Spanish press covered police escorting the Genzyme officials carrying the drug to Gregorio Maranon Hospital to save the baby. The irony consumed and depressed him. He could be a hero to a kid he’d never met thousands of miles away when he couldn’t do the same for his own children. Genzyme and the Children’s Hospital of Philadelphia were trying so hard to avoid being accused of favoritism—and yet here were other families using political influence to get to the front of the line. Influence peddling pervaded life. Would his children have been already treated if he’d gone on a hunger strike instead of starting a drug company? Had his position as head of the Pompe program at Genzyme become more of an obstacle than a stepping-stone to a treatment for his children?

  Days later, John was jolted out of his self-pity by an e-mail from a relative of Greg Assink saying his daughter Kelsey had died. She was nine years old and had the same nonclassical infantile form of Pompe disease as Megan and Patrick.

  John and Aileen decided to attend her funeral. Not only was Greg a long and ardent supporter, having flown to a fund-raiser carrying $20,000 in checks, and attended the Pompe Summit at Genzyme, but John and Aileen also considered him to be their friend. They were about the same age, and John and Greg had an easy understanding.

  Because of
John’s schedule, he and Aileen flew separately to Grand Rapids, Michigan. They met at the airport and drove together to the wake in Hudsonville, a suburb. As they neared the town, John told Aileen he had thought of a way to offer some hope to the Assink family. Greg and Deborah’s youngest, the baby they had named after the Crowleys’ Megan, had also been diagnosed with Pompe. “Think how thrilled they would be if they knew Genzyme planned to begin clinical trials early next year,” John said. “Think how thrilled they would be if they knew their Megan would definitely qualify.” Genzyme, like most drug companies, kept plans for trials quiet—particularly because the company hadn’t even submitted its application to the FDA. “I’m thinking of telling them about the trials and guaranteeing them a place for Megan,” John said.

  “Are you sure you should do that?” Aileen asked. She knew firsthand the agony of dashed expectations, and she had also learned how little John really controlled what happened at Genzyme.

  Before he could answer, the car pulled up outside the funeral home. Inside, they joined the end of a long receiving line. Hundreds of people were in attendance. As the line wound its way around the room, John and Aileen stared at Kelsey’s life documented in poster-sized pictures propped on easels.

  There she was, a blond, blue-eyed one-year-old, sitting up and looking almost normal except for the usual sign of Pompe—her tongue not quite fitting in her mouth. Just like Megan and Patrick.

  There she was as a five-year-old in a wheelchair, tubes coiling from the tracheotomy in her throat to the ventilator propped underneath. Just like Megan and Patrick.

  There was Kelsey on her Make a Wish trip to Disney World, surrounded by her parents, siblings, grandparents, and nurses. Just like Megan and Patrick.

  The parallels between the two families were unavoidable. John and Aileen couldn’t look at each other. They stood, holding hands, wiping away tears. They were thinking the same thing: unless their children got a drug soon, they would be at the front of a receiving line like this. It was only a matter of time—weeks or months.

  It took an hour for John and Aileen to reach the front of the line. Greg threw himself into John’s arms and wept. As they hugged, John whispered, “Please keep this quiet, Greg, but a study next year is going to be for kids under age three. It’ll begin in just about two months. And I promise you, your little Megan will be in the study.”6

  “Oh my God,” Greg burst out, immediately forgetting John’s urging to keep the news to himself. “Deb, Mom, Dad, everyone, this is John Crowley and this is Aileen Crowley. John has some wonderful news for us. The clinical trials are going to start in the next few months and our Megan will definitely qualify. We don’t know how to thank you.”

  On the drive back to the airport, Aileen asked, “How the hell are you going to guarantee Megan Assink a placement? Aren’t there always a million criteria for determining who qualifies? Isn’t this all up to Hal and those other people at Genzyme? You can’t even get your own kids treated.”

  “I don’t know how, Aileen, but I know I’m going to do it,” John said. “I can only imagine Hal’s horrified reaction if he ever knew. But you know what—it felt so good to be able to offer hope to that good and very decent family that I just don’t care what Hal or anyone else thinks.”

  A few days before Thanksgiving, Landy called John and asked him to send Megan’s medical records to a physician at Saint Peter’s University Hospital in New Brunswick, New Jersey, named Debra-Lynn Day-Salvatore.

  “And John, you need to stay out of it and let us do everything,” Landy said, his voice firm.

  John—being John—drove Megan’s medical records over to the hospital himself the next day. It was only about twenty miles from his home in Princeton. Carrying a big cardboard box, he wound his way through the corridors to Dr. Day-Salvatore’s office and knocked on the door.

  The door opened to reveal a tall, slender woman with the unconscious grace of a ballerina.

  “Hi, I’m John Crowley. I’m just dropping off my daughter’s medical records,” John said.7

  “I’m Dr. Day-Salvatore,” the woman answered, her voice just a little louder than a whisper. “Please, come on in.”

  John dropped the box beside the desk in Day-Salvatore’s tiny office. The office shelves were filled with books and pictures of her patients, some of whom John recognized as children with Pompe. She seemed as curious about him and his children as he was about her and her hospital. She asked not only about Megan’s and Patrick’s medical history, but also about their day-to-day lives.

  “Is Megan in school?”

  “She just started kindergarten this fall. She loves it and the kids love her,” John said. “She’s so popular. Our nickname for her is ‘The Mayor.’ She likes to be in charge, and she bosses everyone around. She has lots of friends. The kids seem to treat her just like any other kid, even though she’s in a wheelchair and has her nurse with her at all times, of course.”

  “I can see you’re very proud of your daughter,” Day-Salvatore said, chuckling kindly. “How about Patrick? I know he’s weaker. Does he go to school, too?”

  “He’s in preschool three days a week. Tollgate Grammar School. They have a handicapped program in Hopewell.”

  “I see,” Day-Salvatore said. She cleared her throat, looking sympathetically at him across the piles of papers on her desk. “I know you’ve had some real challenges getting your kids the drug.” She had heard from Landy about the debacle at Children’s Hospital.

  “Yes, it’s been really hard,” John said without elaborating. There was so much history involved with explaining to anyone the obstacles he’d overcome in his fight for his children—only to have more thrown his way—that these days it was just easier to agree. “I just want to get my babies better.”

  “I’d very much like to treat your children,” Day-Salvatore said, as John struggled to compose himself. “Our clinical trial review board is going to meet on December 19 to review your children’s protocol.”

  The next morning, John called van Heek to say he wanted to leave Genzyme a couple of weeks earlier than he’d planned. He told van Heek he wanted to be sure he was no longer a Genzyme employee when the St. Peter’s board met to review the clinical trial. Van Heek, who had known John intended to leave at the end of the year, understood completely. The two men then negotiated the terms of John’s severance package, the combination of benefits corporations often give senior management on leaving a job. John proposed a year’s salary and, most important, an extension of his family’s health benefits during that time. On joining Novazyme, he had held on to excellent health coverage at Bristol-Myers under the federal government’s COBRA program, which allowed people leaving jobs to continue their health insurance at the same level of coverage for one year, plus another eleven months if they were disabled. Before joining Genzyme, John had negotiated for comparable health benefits for his children, and now he was hoping to extend those for another nearly two and a half years—one year through the severance package, and another year and a half through COBRA. Van Heek eventually agreed. The quarter after John left, Genzyme claimed a charge of $4.2 million against John’s severance package—$4 million of that for the children’s future medical expenses, each estimated at nearly $1 million annually.8

  On December 24, a Christmas card containing several sheets of paper arrived at the Crowley house addressed to John. On the front was a picture of the Virgin Mary in blue, clasping a sweetly sleeping baby Jesus to her breast, smiling and shining with the promise of salvation. “You asked for something to put under your Christmas tree,” the note accompanying the card said. “I hope the enclosed approval letter will help fill that void.” It was signed, “Dr. Debra-Lynn Day-Salvatore.” Inside, the doctor had enclosed a copy of her hospital review board’s two-page letter approving the clinical trial for Megan and Patrick. Her hospital was willing to take the risk—the trial would begin on January 9.9

  When John had wiped the tears from his cheeks, he put the card and approval l
etter back in the envelope and propped them prominently on a branch of the tree.

  That night, after the children went to bed, John and Aileen sat down at their enormous new round dining-room table with Aileen’s parents, her brother Brian, cousin Kevin, and their wives Kim and Lisa. John stood up and made a toast.

  “Here’s to a year of good health for all,” he said. “And I have a little something to share.” John began to read from Day-Salvatore’s card. Before he finished, the relatives were crying and hugging John and Aileen.

  On January 9, 2003, Megan and Patrick, wearing matching baby blue hospital gowns printed with red clowns, lay in rooms next to one another in the pediatric intensive care unit at St. Peter’s. They had undergone surgery days earlier to have tubes inserted in their chests to make regular infusions possible. They had had biopsies performed on their legs to have a little muscle removed so the amount of glycogen could be measured and compared after they finished a year of treatment. The surgeries had been exhausting for them and for their surgeons. Megan’s muscles were so badly degenerated that it took the surgeon two hours to find a piece he could remove. Patrick’s blood pressure had fallen so low that he almost died during the grueling, three-hour procedure. Seeing Aileen waiting alone outside the operating room, the surgeon pointed his finger in her face and said, “This was a terrible thing to do to that baby. You better tell me this treatment is going to work.”

  At 9 A.M., Aileen, John, Sharon, and a dozen family members and friends crowded into Megan’s room, waiting expectantly. Megan was supposed to get her infusion first because she was the more communicative and could better report any immediate side effects. Aileen’s parents and John’s mother and stepfather had come, as had Aileen’s cousin Kevin, brother Brian, and their wives and children. John told them all that his father would have turned sixty-three today, had he been alive. John’s friend Ed held the video camera, poised to record the event they had awaited, prayed for, and dreamed of for five long years.10