March 20, 1998 – Walnut Creek, California: John and Aileen Crowley were on top of the world. Thirty-year-old John had graduated from Georgetown University then received a Juris Doctor degree from Notre Dame Law School before earning an MBA from Harvard Business School. He was earning a nice salary with a top financial consulting firm in San Francisco. More importantly, the young couple had two beautiful children, Megan, age 15 months and Patrick, a week old newborn. Then the doctor called.
John and Aileen had consulted a physician to determine why Megan wasn’t walking. After receiving the test results the doctor phoned the couple with the bad news. “I am sorry,” he began, “Megan has a rare genetic disorder called Pompe Disease. Most children die from respiratory failure or cardiac arrest by age two. There is no cure. You should enjoy the time that you have with her.” Then further tests revealed that Patrick also had the disease. When Megan almost died from pneumonia, John promised her he would find a special medicine to cure her.
July 27, 1998, Princeton, New Jersey: John quit his job and moved the family to New Jersey where he joined Bristol-Myers Squibb a large pharmaceutical company that was researching cures for diseases like Pompe. As the new marketing director in the research division, he could learn more about genetic disorders.
A year later John learned about Novazyme, a small four-person company in Oklahoma City, Oklahoma, that focused exclusively on Pompe research. He convinced a venture capital group to invest $8 million in the company and then quit his job with Bristol-Myers and joined Novazyme. He would not let the research fail; his children’s lives depended on it.
June 2001, Boston, Massachusetts: Novazyme had yet to come up with a cure but John, now CEO, had quickly grown the company and sold it for $225 million to Genzyme, another biotech enterprise with the stipulation that he would join Genzyme as Senior VP and head up their Pompe research.
By this time, Megan and Patrick were both wheelchair-bound and dependent on ventilators to breath. Both had hearts twice the size of normal. Time was running out. Doctors predicted the children could not live much beyond age five and Megan would be five in a month. John’s urgency increased and he drove the research team relentlessly as they evaluated four enzymes that might provide a cure.
On a Friday night in October 2002,John got the call that Megan and Patrick had been accepted to a clinical trial involving 12 children using one of the Genzyme drugs. On January 9, 2003, John and Aileen pushed the button to start the children’s enzyme drip at St. Peters University Hospital in New Brunswick, New Jersey. The children received the drug every other week.
After the first test in April both children’s hearts had reduced to normal size and their neck and shoulder muscles had grown marginally stronger. Genzyme invested $200 million in Megan and Patrick’s special medicine, Myozyme. The drug has helped hundreds of children with Pompe survive.
February 26, 2018, Washington, D.C. – President’s State of the Union address: John, Aileen, 20-year-old Megan and 19-year-old Patrick were recognized in the balcony of Congress by President Donald Trump as he lobbied the Food and Drug Administration to spend more money on research for genetic diseases.
Today, still in her wheelchair and supported by a round-the-clock nurse, Megan is a straight ‘A’ student at Notre Dame. Patrick, also in a wheelchair, continues to live at home. Two decades after he quit his job to discover Megan’s special medicine, John is the CEO of Amicus Therapeutics, a biotech company. He saved his children’s lives – now his mission is to cure them.
“Impossible is just a big word thrown around by small men who find it easier to live in the world they’ve been given than to explore the power they have to change it. Impossible is not a fact. It’s an opinion. It’s a dare.” Muhammad Ali