The pediatrician’s hands were trembling when he gave John and Aileen Crowley the devastating news about their 1-year-old daughter. Megan, he said, had an incurable genetic condition called Pompe disease. It would progressively weaken her until she died. There was no treatment.
“I’m so sorry,” he said, adding that the Crowleys’ son, Patrick, only weeks old, should also be tested.
As it turned out, Patrick had also inherited Pompe disease from his parents, both of whom appeared to carry recessive genes for the inevitably fatal condition.
The Crowleys refused to give up.
More Top Picks Samsung Galaxy Watch Review
In “The Cure” (Regan, 343 pages, $33.50), Wall Street Journal reporter Geeta Anand chronicles the desperate efforts of John Crowley to find a cure for his children, while he and Aileen struggled to cope with the pain the disease was bringing them, their children and their marriage.
With $36,000 in savings, they started a family foundation to fight Pompe disease. They raised more money from friends, family, the parents of other victims and their friends and families.
They invested in a small biotech firm with a long shot at hope. Crowley, who had a new master’s degree in business administration from Harvard University, left his job with a big drug company to become the startup’s CEO.
In August 2001, less than four years after Megan was diagnosed, Novazyme Pharmaceuticals Inc., the company Crowley was running, was sold to a competitor for $137.5 million.
And while work continues on perfecting the drug for Pompe disease, Megan and Patrick are among kids who already have received the “Special Medicine.”