Tuesday, January 19, 2010

A father's fight to save his kids

Movies and biochemistry aren't words that usually turn up in the same sentence. That makes Extraordinary Measures, which opens nationwide Jan. 22, a rarity: Among other things, the movie deals with the gains that can occur when science, business and academia join forces to conduct research.

Hollywood being Hollywood, Extraordinary Measures is being billed as an inspirational story about one man's fight to save his children. Fair enough, I suppose: After all, the story focuses on John Crowley, a father who learned that his two children had Pompe disease, a rare neuromuscular disorder that's considered a form of muscular dystrophy. Crowley quit a good marketing job and set out on his own, forming a company dedicated to finding a drug that could save his children's lives. For Crowley, business literally became a matter of life and death.

Crowley's story initially was told by Geeta Anand in a Wall Street Journal article which the author later expanded into a book called The Cure: How a Father Raised $100 Million -- and Bucked the Medical Establishment -- in a Quest to Save His Children."

Inspired by Anand's book, Extraordinary Measures joins Crowley's story to the efforts of an ornery research scientist played by Harrison Ford. Ford's Dr. Stonehill is a composite character who represents a variety of researchers with whom Crowley, played by Brendan Fraser, worked over the course of three years.

Today, the 42-year-old Crowley runs Amicus Therapeutics Inc., a company that seeks cures for rare genetic diseases. He recently visited Denver to talk about the movie:

Q. Are you satisfied with the way the movie condensed real events?

A. They did a beautiful job of telling the story in a dramatic fashion, holding true to many of the events in life and holding 100 percent true to our family life and spirit.
Q. I don't think it's giving anything away to say that you succeed in finding a drug that has kept your kids alive. Megan was 15 months old when diagnosed and Patrick was five months old when you learned he had Pompe disease. How are the kids today?

A. Megan just turned 13. Patrick will be 12. They’re 15 months apart. Our oldest son, John, is 15. (John does not have the disease). They're in sixth, seventh and eighth grades in Princeton public schools in New Jersey. The disease never affects the mind. They’re incredibly smart, precocious little kids. Megan’s a straight-A student.

The medicine we discovered and helped to make fixed their hearts, which is the most life threatening aspect of the disease. That saved their lives. Now, the work that I do – and that many other companies and universities do – is geared toward pushing innovation and finding the next best treatment to continue, extend and improve their lives -- to make them as strong and healthy as possible.

Q. I've read that Pompe shows up in something like one out of 40,000 births. How do we justify costly research into this and other equally rare diseases?

A. Research into rare disease is incredibly important for a lot of reasons. First, you have to remember that there are 7,000 rare diseases. Taken together, they affect more than 30 million people in the United States.

Second, these are such debilitating and often-fatal disorders. If there were a 1,000 kids stuck in a bus hanging off a cliff, we would move heaven and earth to save them. We have to think about what it means in terms of people, most of whom are the weakest and most vulnerable in society. I think we -- as a society -- have and will continue to make choices on the side of helping people who fall into those categories.

But also, by understanding and treating these diseases, we’re opening up new ways of understanding much more prevalent disorders. (He talks about how his company invested $75 million in trying to develop a drug to treat Gaucher disease, which has stricken perhaps 5,000 people in the entire world. Thus far, the effort has failed, but it turned up important links between those who carry the gene for Gaucher, a genetic disorder, and those who contract Parkinson's disease.

Q. Science is painstaking and often slow moving, and it's certainly not easy to raise large sums of money. There must have been times when you were tempted to throw in the towel.

A. There were many, many times – after traveling the country and the world and after being away for five or 10 days – when I got tired and frustrated. Science isn’t easy, and a lot of times, I thought I didn’t want to be away from home. If the kids weren’t going to live a long time, I didn’t want to miss all that.

Q. But you kept going.

A. I wasn’t the best CEO. There were better, smarter people who could do that. But what I lacked in experience and money, we made up for in determination and passion. If I did nothing else I tried to hire the very best people and tried to get them the resources they needed.

Q. How do you see the relationship between science and business?

A. What happened in this case is something very uniquely American. You had private industries – small companies, start-ups, Big Pharma, biotech companies, university researchers, government researchers, government regulators, patient groups and philanthropists coming together. Without any one of those, this may not have happened or certainly wouldn’t have happened as quickly as it did. That’s very uniquely American. You don’t see that anywhere else in the world. Academic research is a great strategic advantage for us. We wouldn’t have drug development today without it. But at the end of the day, it’s private and public companies in the bio-tech and pharmaceutical industry that translate ideas into medicine.

Tomorrow: Brendan Fraser talks about playing John Crowley.

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