The Cure

by Geeta Anand

  Mattaliano, in the interview in 2004, agreed the title was a dig at Canfield, but said Canfield had seemed supercilious about his approach in the past.

  Dialogue and details in this scene are based on recollections of Termeer, Landy, van Heek, John, McKinney, Canfield, Aileen, Dozier, Greg Assink, and Fallon in interviews in 2004 and 2005.

  Landy, in an interview in 2004, shared these emotions and recollected his actions and observations that day.

  Chapter 24

  Dialogue and details of this meeting are based on interviews with Landy, Smith, Fallon, McKinney, and John in 2004 and 2005.

  Landy, in an interview in 2004, described his feelings and thoughts after the meeting.

  Dialogue and details in this scene are based on recollections of Landy and John in interviews in 2004.

  A spokeswoman for the Children’s Hospital of Philadelphia said in an interview in 2003 that a hospital board questioned the fairness of a study that included as patients only the children of a senior official at Genzyme, the company sponsoring the study. The board was concerned that the conflict of interest could skew the trial, the spokeswoman said. She said Dr. Richard Finkel was unavailable for comment. Dr. Edward Kaye, a Genzyme official who had previously worked at CHOP, told me in an interview in 2004 that he had tried to intervene on John’s behalf with hospital officials. He said they told him they really wanted to help, but they couldn’t overlook the conflict of interest questions that arose because John, a senior Genzyme official, was the father of the children in the trial.

  Chapter 25

  Termeer, in interviews in 2004, recalled this conversation with his mother.

  Dialogue and details are based on recollections of John, van Heek, McKinney, and Landy in interviews in 2004, and Reuser in an interview in 2006.

  Dialogue and details are based on interviews with Byrne and Dr. Carolyn Spencer on April 21, 2004, and interviews with John.

  Details from Julie Berthy, interviewed on April 21, 2004.

  Dialogue and details in this scene are based on Landy and John’s recollections in interviews in 2004.

  Chapter 26

  Landy, in an interview in 2004, shared this perspective. Dialogue and details of the next scene based on interviews with Landy and John in 2004.

  Termeer, in interviews in 2004, recalled making this phone call to Landy, although Landy doesn’t recall the conversation, but he says it may have occurred and escaped his memory.

  Dialogue and details are based on John’s recollection; Termeer said he didn’t recall the details and said he couldn’t discuss internal legal matters anyway. A Genzyme spokesman said Tom DesRosier did not recall this specific conversation, but he and another Genzyme lawyer did have conversations with John from time to time “about situations in which his inexperience or questionable judgment led him to do something we would consider problematic.” The spokesman declined to allow me to speak to DesRosier, saying he didn’t recall the conversation I was referencing in this scene.

  Contents of John’s e-mail are based on his recollections of what he wrote; he didn’t keep a copy and Genzyme wasn’t able to provide me with one.

  Dialogue and details are based on an interview with van Heek in 2004 and interviews with John the same year.

  Chapter 27

  Dialogue and details in this scene and section based on recollections of John, Landy, and McKinney.

  Details on the video are based on recollections of Kishnani, in an interview in 2006, and John in 2004.

  Landy, in his interview in 2004, recollected receiving this entreaty and others.

  Information on entreaties from officials in other countries is based on interviews with Termeer, van Heek, Landy, and John in 2004.

  Detail from van Heek and John in interviews in 2004; Genzyme supplied copies of the articles in the Italian press about Rosella’s plight.

  Dialogue and details in this scene are based on interviews with Greg Assink in 2005 and 2006 and John and Aileen in 2004.

  Dialogue and details in this scene are based on the recollections of Dr. Debra-Lynn Day-Salvatore, in interviews in 2003 and 2004, and John.

  On March 5, 2003, Genzyme reported annual earnings for 2002, listing, among special expenditures: a $4.2 million severance charge associated with the departure of John Crowley, former chief executive officer of Novazyme Pharmaceuticals Inc. The charge primarily reflects the cost of medical insurance continuation for Mr. Crowley’s children, who both have Pompe disease. John provided the estimate of nearly $1 million annually for the medical care of each of his children.

  A copy of the card and review board letter was provided by John and Aileen.

  Dialogue and details are based on my observations, since I was present this day.

  Genzyme, counting the acquisition cost of Novazyme as well as Pharming’s and Chen’s enzymes, estimates the cost of developing the treatment, Myozyme, at $500 million by the end of 2005.

  Dialogue and details based on interviews with John and Ed in 2004.

  Dialogue and details in this scene are based on John’s recollections.

  Dialogue and details are based on interviews with John in 2006, and with Steve Hennessey on March 14, 2006.

  Details of test results are based on interviews with Dr. DebraLynn Day-Salvatore in 2003, and John in 2003 and 2004. Dialogue and details of the scene are based on recollections of Day-Salvatore and John and Aileen.

  Dialogue and details are based on recollections of Dozier in interviews in 2004.

  Afterword

  John wasn’t looking to sell the company to Genzyme because he believed any additional acquisitions of genetic disease companies by the bigger company risked being seen as anticompetitive. After the Novazyme acquisition, Genzyme faced a lengthy review by the Federal Trade Commission, which was concerned that the bigger company was buying up all potential competitors in violation of antittust laws. The commission eventually decided not to sanction Genzyme, but another acquisition might result in a different ruling.

  Kishnani presented these results at the American Society for Medical Genetics meeting in San Diego in March 2006.

  Results of these experiments were confirmed by Hung, Mattaliano, and O’Callaghan.

  Hung says he and others performed experiments in the years after the Mother of All Experiments and found that the Pharming enzyme worked better than initially realized. The Pharming enzyme did have some phosphate molecules on the carbohydrate chains, but the phosphate molecules were covered by other molecules. So in mouse experiments, the Pharming enzyme wasn’t well absorbed. But it turns out human beings have an uncovering enzyme on the cell surfaces, which exposes the phosphate in the carbohydrate chains, enabling the enzyme to be well absorbed. “We’re so arrogant as scientists,” he told me. “At any point in time, there’s so much that we don’t know.”

  Acknowledgments

  I want to thank John and Aileen for trusting me enough to share the full story of the struggle to save their children—not just the times they responded heroically, which are many, but also when they stumbled, compromised, or tried to escape. I want to also thank their children, Megan, Patrick, and John Jr., for allowing me to become part of their lives during the five years I spent reporting and writing this book and two earlier articles for the Wall Street Journal. Without the openness of the Crowleys and their wide network of family and friends, this book would not have been possible. I also want to acknowledge the vital cooperation of William Canfield, John’s partner at Novazyme Pharmaceuticals and now a scientist at Genzyme Corporation, as well as Henri Termeer, Genzyme’s longtime chief executive, and several other current and former officials at the company, in telling the story that is described here. Dr. Canfield and Mr. Termeer sat down repeatedly with me, and relived the journey they took with John, which tested the limits of their own courage, compassion, and resolve. Jan van Heek, Bo Piela, Bob Mattaliano, Mike O’Callaghan, Ed Kaye, and Frank Ollington gave generously of their time to make sure this book was accurate.
Hal Landy’s candor and thoughtfulness give this book a depth it would otherwise have lacked.

  I want to thank Randall, Marylyn, and Tiffany House for spending two days talking about their Herculean efforts to push Pompe research forward—and how their paths crossed with John, often fraught with tension. Given that they have often jousted with him, it is a testament to their graciousness that they spoke to me about their experiences. This book, which focuses on John’s story, does not do justice to the enormity of the Houses’ contribution toward advancing the development of a treatment for Pompe. Somebody needs to write their story, which is, in many ways, just as incredible.

  I am grateful also for the input of Deborah and Greg Assink, the parents of Megan, who is thriving, and her big sister Kelsey, who passed on in 2002. And I want to thank Laurie Easton for talking to me about her irrepressible daughter Lindsey and sharing her graduation speech.

  I need also to acknowledge the generosity of two other families who shared their stories after having lost children to the disease. Barry and Deb Koncel took time to tell the story of their son John’s participation in the first clinical trial of Dr. Chen’s enzyme, and Carolann Elmore talked with me many times about her son Niko’s participation in the same trial. Both John and Niko have since passed on, but their courage and their parents’ extraordinary dedication remain sources of inspiration to the doctors and nurses who met them along the way.

  I want to thank Tony McKinney, Hung Do, Bill Fallon, Julie Smith, Pedro Huertas, and others from the Novazyme team who helped shape this book. The venture investors, Gus Lawlor, Dennis Purcell, Steve Elms, Josh Phillips, and Dave Alberts, as well as the company’s lawyer, Doug Branch, spent many hours with me, and for that I am very grateful. Sherrill Neff and Stephen Roth, who used to run Neose, were invaluable in describing some of John’s and Novazyme’s biggest debacles. John Frick, whose firm didn’t invest, took time to explain his decision.

  Drs. Barry Byrne and Carolyn Spencer at the University of Florida were immensely helpful, as were the National Institutes of Health scientists Nina Raben and Paul Plotz, who allowed me to spend a day with them. Dr. Arnold Reuser and Dr. Rochelle Hirschhorn gave me insight into the scientific breakthroughs that made a treatment possible. I tremendously enjoyed visiting with Dr. Slonim and Dr. Martiniuk. This book would not have been possible had Duke University Medical Center’s Dr. Chen and Dr. Kishnani not spent hours explaining their research. The dedication of these physician researchers to their patients is nothing short of extraordinary.

  Sharon was not only a wonderful source of information but made the book writing process a whole lot more fun with her terrific sense of humor. I am also deeply indebted to Aileen’s parents, Marty and Kathy; her Uncle Charles and Aunt Sandra; her brothers, Brian and Marty; their wives, Kim and Kate; her cousin Kevin and his wife Lisa; and the many other relatives and friends who took time to talk about Aileen. John’s mother Barbara and brothers, Joe and Jason, deserve my gratitude as well, as do John’s best friend Ed and buddies Karl Palasz, Mike Ostergard, Andy Singer, Bradley Campbell, and John Gordon. Brian Markison, John’s former boss at Bristol-Myers, could recount verbatim his conversations with John back then.

  Much of this book was written in diners and coffee shops in Manhattan, as I strove to find a quiet place, away from my family. I want to thank Juan and Maria, the waiter and restaurant manager, respectively, at Euro Diner; the staff at the Starbucks at Third Avenue and Twenty-eighth Street and at Lexington Avenue and Fortieth Street; and Pete, the waiter at Frontier Diner on Third Avenue and Thirty-ninth Street. All of them were gracious when I stayed at my table for far longer than was profitable or proper, laboring over this book.

  I want to thank my dear friend and former Boston Globe editor, Sarah Snyder, who read initial drafts of each chapter, tore them to shreds, and recommended better structure. This book wouldn’t be as well organized if it weren’t for her. I want to thank Bob Dow, whom I first wrote about when covering the September 11 tragedy, in which he lost his partner, Ruth Ketler. Bob, an enormously talented creative writing professor at the University of Massachusetts in Amherst, read an early draft and helped me put more voice into the pages of this book. This book may never have come about were it not for Stephen Gendel, Novazyme’s publicist, who introduced me to the Crowleys in 2001 for the first story I did for the Wall Street Journal.

  I also want to thank my sister, Mona, my parents, Mohan and Mary Donna, and two of my friends, Deepika Mehra and Mary Silver, for reading early drafts and offering encouragement. Mary’s eleven-year-old daughter Natalie came up with the title. My college roommate, Ana, swept into New York City in March and April and took my kids to movies, bookstores, and ice cream parlors so I had time to make the final editing changes. My old friend and former Boston Globe colleague Kate Zernike, one of the best writers I know, polished my last draft. I also had better appreciate my husband Greg’s candid early readings, which were immensely helpful in improving the book. Greg took care of our children on many weekends over the two and a half years it took to write this book, and I am not exaggerating to say he never once complained. I couldn’t ask for more support than he gave me.

  I need to thank my Wall Street Journal editor, Elyse Tanouye, for encouraging me to write the first two stories in the newspaper that led to this book. She and the Journal’s top editors, Paul Steiger and Dan Hertzberg, deserve my gratitude for giving me eight months off to write the first draft. Ron Winslow, Carrie Dolan, and Amy Stevens, who edited the front-page piece on the Crowleys, asked smart questions that helped shape my thinking.

  Thank you to my book agent, Joelle Delbourgo, who was a constant source of encouragement. And thanks to my editors at Regan: Cal Morgan, whose wise letter after the first draft served as my guide in rewriting the book, and Alison Stoltzfus, who put her heart and soul into every page. A writer cannot ask for more than a passionate editor, and she was that and more.

  Geeta Anand is a senior writer for the Wall Street Journal. She was on the team that won the newspaper a Pulitzer Prize in 2002. In 2006, she received the most prestigious award for business reporting, the Gerald Loeb award. And in 2007, she was awarded the Victor Cohn Prize for Excellence in Medical Science Reporting. Formerly a political reporter for the Boston Globe, she now writes about health care, education, and environmental challenges in India. She currently lives in Mumbai, where she is a correspondent for the WSJ. A true story, The Cure is her first book, and inspired the Hollywood film Extraordinary Measures.

  John and Aileen Crowley were married in Indianapolis on August 18, 1990.

  John, twenty, with his best friend, Ed Devinney, during a day they snuck off from the U.S. Naval Academy in 1987.

  Aileen and John help their two-year-old son, John Jr., hold his newborn baby sister Megan for the first time.

  Moments after Megan nearly fell of his arms, John walks back to his seat at the Harvard Business School graduation, holding on to her, his son John Jr., their bears, and his diploma.

  Megan, while she could still smile, at nine months old in a portrait that adorns a hallway in the Crowley house.

  One of Aileen’s favorite pictures of Patrick, who was such a chubby, content baby that it was difficult to believe he was sick.

  Months before Megan was diagnosed, John, Aileen, Megan, and John Jr. enjoy a fall day in San Francisco, looking like the perfect family they would later envy.

  The Crowley kids baking cookies at home in Pennington, New Jersey. Megan’s fifty-pound ventilator is visible behind her on the kitchen counter.

  Dr. Y. T. Chen (right) and his protégé, Dr. Priya Kishnani (center), show off baby John Koncel, the first patient to receive the doctors’ enzyme replacement therapy, summer 1999. Barry Koncel, the baby’s father, looks on.

  The bird on the left is a normal, non-diseased quail, which tries to right itself when held upside down. The center quail has Pompe and hangs limply. The third quail, on the right, has been treated with Dr. Chen’s enzyme replacement therapy. After
treatment, the treated birds could flip over and one flew a short distance.

  Human muscle cells taken from a baby with Pompe disease before treatment and after twelve weeks of therapy. In the second picture, the glycogen pools have receded and the muscle cells have smoothed and grown.

  Megan accompanies her dad to a Children’s Pompe Foundation fundraiser at the Tavern on the Green in winter 2001.

  The visitor to Novazyme who most inspired John, Lindsey Paige Easton, returns for the dedication of the company’s manufacturing plant in her name.

  A page from Aileen’s 2001 Disney World trip scrapbook. John, after finishing a tough conversation with his venture investors on the only phone in their vacation apartment, hams it up for Aileen.

  Arms raised, Megan, with her brother Patrick, celebrates having finally arrived at Disney World on her family’s Make-A-Wish trip in spring 2001.

  Winnie the Pooh and Tigger pose with John, Aileen, Nana (Kathryn Holleran, standing at right), Megan, John Jr., and a nurse, Helen (sitting at left). Patrick stayed home with Sharon that day.

  Tony McKinney and John on a cruise for Novazyme employees in February 2001. The cruise was one of John’s team-building events.

  Dr. William Canfield, left, with John Crowley.

  Genzyme’s headquarters in Kendall Square when John worked for the company. Genzyme has since built a new headquarters nearby.