Outlook

Duchenne duo

Friday, June 25, 2010

A kitchen table conversation about a frightening diagnosis has evolved into a fundraising organization with a global reach.

Paul Miller ’87 and Debra Miller founded CureDuchenne in 2003, after learning their son, Hawken, had a degenerative muscle disease called Duchenne muscular dystrophy.

Since then the couple’s Corona del Mar, Calif., non-profit has raised $5 million to build awareness about and fund research for the disease, paved the way for a nine-figure investment by a multinational pharmaceutical firm and garnered support from the sports and entertainment industries.

Historically, Duchenne muscular dystrophy has received a fraction of the research funding of other diseases. A bar graph on the cureduchenne.org Web site comparing National Institutes of Health grants across four diseases looks like a city skyline: Huntington’s chorea, cystic fibrosis and pediatric AI Ds are the skyscrapers and Duchenne muscular dystrophy is the threestory building.

In 2004, with just $10,000 in the CureDuchenne bank account, the Millers signed a contract to raise $1.3 million for Prosensa, a Dutch start-up, to help fund research on exon skipping, a method to skip over the genetic mutation that causes Duchenne muscular dystrophy.

In just two years the gamble paid off. CureDuchenne delivered $1.3 million and Prosensa’s pre-clinical work attracted venture capital. Then GlaxoSmithKline, the world’s fourth-largest pharmaceutical firm, committed $650 million and optioned four exon-skipping compounds. Clinical trials are underway for one compound and planned for another, said Debra.

CureDuchenne is funding other research projects including stem-cell therapies that would promote muscle regeneration and gene therapies that would force the body to produce dystrophin, the protein that enables muscle-tissue repair.

“Things don’t occur unless people can figure out how to make money out of it,” said Paul, a St. Cloud State management graduate and a Husky hockey player who played on the celebrated 1986-87 team that included Mike Brodzinski, Jeff Tollette ’86 and future Husky hockey coach Bob Motzko ’89.

Paul is president of Buena Vista Food Products, Azusa, Calif., which vends bakery products to K-12 schools. The Crookston native holds a master’s of business administration degree from Pepperdine University in Malibu. A recommendation letter from then President Brendan J. McDonald keyed his admission to the exclusive Malibu, Calif., school, Paul said.

In just seven years the Millers have leveraged their sales and marketing backgrounds to brand Duchenne muscular dystrophy and find big-name fundraisers.

“Our biggest enemy was ignorance of the disease,” recalls Debra. “Not a single national organization had Duchenne in its name.”

Now sports and entertainment luminaries – including actors Anne Heche and James Tupper, singer Mandy Moore and University of Texas football coach Mack Brown – are championing Duchenne.

Brown, who coached the Longhorns to a national title, is the latest celebrity advocate to join the fight for a cure. He and his wife, Sally, hosted a casino night May 1 in Austin. In attendance with their Heisman trophies were Longhorn legends Earl Campbell and Ricky Williams.

Celebrity poker tournaments, casino nights, mountain climbing and regattas are part of CureDuchenne’s “have fun” fundraising strategy, according to Debra.

Less fun is the constant monitoring and coaching necessary to keep Hawken healthy. The 13-year-old, who is still ambulatory, must follow a strict diet, restrict his activity and take two medications and more than 40 diet supplements each day, according to Paul.

On a recent trip home from a biannual visit to Cincinnati Children’s Hospital Medical Center, Hawken asked his parents: “Do I have a shortened lifespan?”

His parents, armed with hard-earned hope and knowledge, mastered their emotions and replied: “We can’t honestly say, Hawken. We don’t know.”

Duchenne Facts

  • One in 3,500 boys born worldwide each year is afflicted
  • Diagnosis typically occurs by age five
  • Most boys don’t survive their teens
  • Caused by inherited or spontaneous mutations in the dystrophin gene found on the X-chromosome
  • Progressive muscle damage typically results in death by heart failure or pulmonary failure
  • Find out more at cureduchenne.org

Sources: CureDuchenne.org and the Duchenne Muscular Dystrophy Center at UCLA (cdmd.ucla.edu).

- Jeff Wood '81 '87 '95

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