Debra Miller’s son has Duchenne muscular dystrophy. She has a mother’s will to find a cure—a resolve that has propelled certain Duchenne treatments to new levels of development in the past decade.
There still is no cure or treatment for the inherited genetic disease, which causes progressive muscle degeneration, leaving patients wheelchair-bound by their mid-teens. Almost all are boys or young men, and most don’t survive their mid-20s.
Miller’s son, Hawken, was diagnosed with Duchenne in November 2002.
Miller launched Newport Beach-based CureDuchenne the following October. The nonprofit organization has become a force in propelling significant advancements in Duchenne therapy, earning Miller and the organization recognition as one of the five honored at the Business Journal’s 21st annual Women in Business Awards on June 23 at the Hotel Irvine (see related stories, pages 1, 4, 9 and 12).
Venture Philanthropy
CureDuchenne employs a “venture philanthropy” model to find and fund third-party companies developing Duchenne drugs and treatments.
“My head was telling me there has to be a plan to this,” Miller said. “So we set out to develop a nonprofit that would operate like a business.”
Miller, who had prior experience working for technology publications, said she received solid entrepreneurial training while managing the southwest region for PC Magazine.
Miller left PC Magazine to work at a publishing startup and later became an at-home day trader so she could spend more time with her young son, who hadn’t yet been diagnosed with Duchenne.
CureDuchenne’s first significant investment was in a company called Prosensa. CureDuchenne agreed to invest $1.3 million in 2004 when it had only about $10,000 in the bank.
“It was scary,” Miller said. “But we just knew that we had to get this money. Our son’s life was basically at stake.”
CureDuchenne raised the capital from donors in Orange County including Vinny Smith, who was chief executive of Quest Software at the time and later sold the company to Dell Inc. for $2.4 billion.
Prosensa sold to BioMarin Pharmaceutical Inc. in January for approximately $840 million.
The $6 million profit CureDuchene earned was reinvested in three new research projects.
BioMarin, meanwhile, said last week that the U.S. Food and Drug Administration would review a new-drug application for Drisapersen, a Duchenne treatment, according to CureDuchenne.
Another investment is in Cambridge, Mass.-based Sarepta Therapeutics, which has announced the completion of a new-drug application for its Eteplirsen.
CureDuchenne is now raising money for two undisclosed projects and has a $10 million budget for the next three years that it hopes will be met through donations.
“We just need the next heroes to help us fund this research,” Miller said.
Miller is optimistic about the future of Duchenne therapy and treatment. Her son is 18 and plans to start classes at the University of Southern California in the fall. He’s also done some public speaking to raise awareness of the disease.
“He knows he has a purpose on this earth,” Miller said. “We are all working to find a cure.”
