A Dad Lowered the Minimum to $5,000 to Start a Drug Company That Saved His Kids' Hearts in 12 Weeks

In June 1997, John Crowley walked across the stage at his Harvard Business School graduation with a child in each arm. John, his son, was two and a half. Megan, his daughter, was six months old. Tucked in with them was a teddy bear in a school sweater and a diploma.

Then Megan almost fell. The audience gasped, and Crowley caught her. He thought she had slipped. Only later did he understand that she had not slipped at all. She had lacked the strength to hold on.

Over the next six months, after the family moved to California for his new job, the Crowleys consulted one specialist after another and moved through blood work, imaging, and a deep muscle biopsy. The diagnosis was Pompe disease, a rare genetic muscle condition that devastates the heart, the breathing muscles, and the skeletal muscles while leaving the mind intact. A doctor told them Megan would not live a couple more years. When Megan was diagnosed, Patrick, their newborn son, was seven days old, so the Crowleys had him tested and learned he also had the disease. Less than a year after the diagnosis, both Megan and Patrick were in wheelchairs and on ventilators.

This is the story of what a father with no science background did next. Crowley described himself as "the desperate dad and the accidental entrepreneur." He started Novazyme, the company, with $30,000 in seed capital from a small group of backers in Oklahoma City. Nobody else wanted to run a startup to chase a treatment, so the job fell to him.

To raise a $1 million angel round, he cut his $250,000 minimum investment to $5,000. He collected checks from 37 individuals, the largest for $75,000. Those early backers earned about 12 times their money in 18 months, before the company was acquired by Genzyme, a larger drug company.

That speed matters because developing a drug usually takes about a decade, and 90 percent of drugs that enter human trials never become commercial products. Crowley says there were a thousand reasons his company should not have succeeded, and that luck and timing played a part.

For his family, the result arrived just in time. By the time the medicine reached them, the left side of each child's heart was three times its normal size, and they had only months to live. Within six infusions, over 12 weeks, their hearts shrank to normal size and remodeled. For the better part of a year, they grew stronger, especially Megan.

Then, at about a year, the improvement stopped. The slow decline of the disease resumed. The medicine had saved their lives, and the lives of other children with Pompe, but it was not a cure, and Crowley realized he had to start the search for a better treatment.