SICK CHILDREN, BRAVE DAD
New York Post, September 24, 2006 -- THE CURE: HOW A FATHER RAISED $100 MILLION - AND BUCKED THE MEDICAL ESTABLISHMENT - IN A QUEST TO SAVE HIS CHILDREN BY GEETA ANAND REGAN BOOKS, 352 PAGES, $25.95
CAN you imagine what it would feel like to be CEO of a fledgling drug company whose goal is to produce a drug that could save your children's lives? Or how it would feel to be scrutinized by corporate officials because these same children supposedly represent a conflict of interest?
In "The Cure," Geeta Anand vividly explores this poignant true story and addresses the ethical questions that spring up throughout.
Infants Megan and Patrick Crowley are afflicted with Pompe's disease, a glycogen (stored sugar) storage disease which results in profound muscle weakness and degeneration as a result of an enzyme (acid maltase) deficiency. Their father, John, is a young lawyer and recent graduate of Harvard business school who responds to their fatal disease by throwing his considerable financial prowess and connections into finding a cure.
He forms a foundation which raises millions, and founds a company, Novazyme, that is ultimately bought by Genzyme (which already produces an effective enzyme replacement for a similar disease - Gaucher's) for $225 million. John is made the head of the Pompe research division.
Unfortunately, as his children grow weaker and closer to death, research targets only infants less than a year old, because they require less of the replacement drug, and the effects are likely to be more dramatic. By the time John manages to procure the new drug for his children, nearly four years have passed, and it appears they have lost much muscular function that can never be reversed, even as their lives are saved.
For those who believe that there is no such thing as corporate or drug company ethics, this book is an amazing wake-up call. The picture Anand draws of the biotechnology industry is inspiring, as company executives work together with devoted scientists toward a cure.
John Crowley introduces actual patients and their suffering parents to deliberately affect the company's approach. "John hated the disconnect he saw between the desperation of patients and the dispassion of clinical medicine. Now that he was in charge, John wanted to infuse the science and business of drug development with patients' life experience and their families' sense of urgency, the same sense of urgency that drove him."
"The Cure" explores human courage under the most trying circumstances because in the scientific realm, progress is frustratingly slow. Anand brings this contrast brilliantly to life, while the reader is left with a nagging awareness that Crowley's children might have fared better in a world less civilized. "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?"
Only in America can a man get rich trying to save his children, while having to rely on rules of professional restraint that almost cause him to miss his goal and lose his mind.
Marc Siegel, an associate professor of medicine at the NYU School of Medicine, is the author of "False Alarm: the Truth about the Epidemic of Fear."