The president salutes OKC biotech, sort of

Published: Sunday, March 12, 2017 By: Stephen Prescott, M.D.

In his first address to a joint session of Congress last month, President Donald Trump highlighted a young woman he'd invited to the speech. Her name was Megan Crowley.

“Megan was diagnosed with Pompe disease, a rare and serious illness, when she was 15 months old,” the President said. “She was not expected to live past 5.”

But, he explained, Megan's father “fought with everything he had to save the life of his precious child. He founded a company to look for a cure and helped develop the drug that saved Megan's life.” He then introduced Megan, now 20 years old and a sophomore at Notre Dame, who received a big round of applause.

It was a heartwarming moment. Still, one of the people most responsible for that moment went unrecognized.

His name is Dr. William Canfield. He was a scientist at the University of Oklahoma Health Sciences Center, and his research led to the drug that saved Megan's life.

In his lab, Dr. Canfield made an important breakthrough about how to deliver a missing enzyme into the bodies of people afflicted with a rare genetic disorder called Pompe disease. Pompe is a form of muscular dystrophy that can strike at birth or later in life. For children (like Megan Crowley and her brother) born with the condition, they start to develop muscle weakness shortly after birth, and they typically died in childhood of heart failure.

Canfield founded an Oklahoma City biotechnology company, Novazyme, to develop that discovery. Thanks to the Presbyterian Health Foundation's commitment to providing best-in-class, subsidized laboratory space to biotech startups, Novazyme was able to set up operations in the research park that bore the foundation's name. (It's since sold the facility to OU.)

Under Canfield's leadership, Novazyme made rapid progress developing an experimental therapy to treat Pompe. John Crowley, Megan's father, eventually left his job in management at big drug company to join Novazyme as its CEO in an effort to speed the quest to find an effective treatment for Pompe — and save his children's lives.

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