"If There's No Cure, I'll Build One" — The Father Who Founded a Pharmaceutical Company to Save His Children: The John Crowley Story

A Father's Worst Nightmare

In 1998, John F. Crowley and his wife Aileen were living a comfortable life in the suburbs. John, a Harvard Business School graduate, was working in strategic marketing at Bristol-Myers Squibb. They had three young children. Life seemed perfectly on track — until it wasn't.

Their daughter Megan, just 15 months old, was diagnosed with infantile Pompe disease, an extremely rare genetic disorder in which the body lacks the enzyme acid alpha-glucosidase (GAA), causing glycogen to accumulate in cells and progressively destroy muscle tissue — including the heart and respiratory muscles. The prognosis was devastating: most infantile-onset patients did not survive past age nine.

Then came the second blow. Their son Patrick received the same diagnosis.

There was no FDA-approved treatment. No drug in clinical trials. No pharmaceutical company with a serious program to develop one. The patient population was too small — roughly 1 in 40,000 births — to attract major investment.

That night, John made a decision that would change medical history.

"If there's no cure, I'll make one."

From Corporate Boardroom to Biotech Garage

John threw himself into the scientific literature with the same intensity he had once reserved for business cases at Harvard. Night after night, after his children were asleep — Megan on a ventilator, Patrick's muscles visibly weakening — he read biochemistry papers, emailed researchers around the world, and cold-called anyone remotely connected to glycogen storage disease research.

One scientist answered: Dr. William Canfield, a biochemist at the University of Oklahoma Health Sciences Center who had been working on recombinant GAA production but lacked the funding to move his research toward clinical application.

In 2000, John quit his stable corporate job and co-founded Novazyme Pharmaceuticals, a startup biotech company dedicated to developing an enzyme replacement therapy (ERT) for Pompe disease. He was 32 years old, had no medical degree, no laboratory experience, and two terminally ill children at home.

He pitched venture capitalists, recruited scientists, navigated FDA regulations, and built a company from scratch — all while racing against his children's biological clock.

The Ethical Tightrope

The greatest obstacle was not scientific — it was ethical. A CEO who had founded a company specifically to save his own children faced enormous conflict-of-interest scrutiny. Could his children be enrolled in clinical trials for a drug developed by his own company? Could he make objective decisions about resource allocation when his children's lives hung in the balance?

In 2001, Novazyme was acquired by Genzyme Corporation, one of the world's largest biotech firms specializing in rare diseases. John joined Genzyme as a senior executive to continue driving the Pompe program forward. But internally, the tension persisted. Colleagues questioned whether one executive's personal mission should consume disproportionate company resources.

John ultimately stepped back from direct oversight of the Pompe program so that Megan and Patrick could participate in clinical trials without ethical complications. He chose his children over his title.

"I'm not looking at this as a CEO. I'm looking at this as a father. But this drug must belong to every Pompe patient in the world, not just mine."

April 28, 2006 — FDA Approval

After years of research, clinical trials, setbacks, and breakthroughs, the FDA approved Myozyme (alglucosidase alfa) — the first-ever treatment for Pompe disease — on April 28, 2006.

Megan and Patrick had already been receiving the enzyme through clinical trials. Both children were wheelchair-bound and ventilator-dependent, but they were alive — years beyond what any doctor had predicted. Megan went on to graduate from high school. Patrick grew into adulthood. As of the early 2020s, both had surpassed their original life expectancy by more than 15 years.

A Story Told on the Big Screen

The story was first chronicled by Geeta Anand, a Wall Street Journal reporter whose Pulitzer Prize-winning coverage became the book The Cure: How a Father Raised $100 Million — and Bucked the Medical Establishment — in a Quest to Save His Children. In 2010, it was adapted into the film Extraordinary Measures, starring Brendan Fraser as John Crowley and Harrison Ford as a fictionalized version of the scientist.

John Crowley himself went on to serve as a senior executive at Amgen and later as CEO of several biotech firms, consistently championing orphan drug development and rare disease advocacy.

Beyond One Family

What makes John Crowley's story transcendent is that his personal desperation produced a universal gift. Myozyme and its successor, Lumizyme, have since been administered to Pompe patients across more than 70 countries. Hundreds of people who would have died in childhood are now living into adulthood.

Pompe disease was once a death sentence delivered in a doctor's office with an apologetic shrug. Today, because one father refused to accept "there is no treatment," it is a manageable condition for many patients.

John has said in interviews:

"What I wanted my children to see — more than anything — is that even in the most hopeless circumstances, you can act. You can fight. Despair is not the only option."

Megan and Patrick are still alive. And across the world, every dose of enzyme replacement therapy administered to a Pompe patient carries within it the echo of a father's impossible, magnificent love.

John Crowley's story is a testament to what happens when parental love meets relentless determination — and when one person decides that "impossible" is simply a problem that hasn't been solved yet.