Paul Miller, co-founder of the CureDuchenne nonprofit group and chief operating officer of Irvine-based Patman Meat Group, recalls the first time he met the woman who would become his wife and the mother of their son, Hawken.
When he sat next to Debra on a plane in 1992, he was instantly captivated by her warm smile and bubbly personality.
Fast forward to 2002. The couple and Hawkin seemed to have it all. She was thriving in her career in advertising sales, working with top publications such as PC Magazine. Paul was a senior executive with a company that makes food-to-go products for convenience stores.
But something was wrong with Hawken. Multiple visits to doctors, including specialists, confirmed Debra’s worst fear: the 4 year old had a rare, incurable disease. Duchenne is a form of muscular dystrophy that affects only boys, caused by the absence of the protein dystrophin, which helps keep muscle cells intact. The muscle-wasting disease is nearly always fatal by the patient’s mid-20s, usually due to heart failure.
Duchenne is what’s often referred to as an “orphan disease,” a condition that affects fewer than 200,000 people nationwide. About 15,000 boys are living with Duchenne in the U.S. and 300,000 worldwide. And since it’s an “orphan,” the market isn’t big enough to gain support and resources to discover treatments.
After the Millers absorbed the major life change the disease introduced into their lives, they did what every parent would—started searching for possibilities of finding a cure. To their dismay, resources were limited. There were a handful of Duchenne nonprofit groups, but the Millers said those focused on providing support and services, not medical research.
“We said to each other, there has to be a solution,” Paul said.
Unique Funding Model
The Millers founded the nonprofit CureDuchenne in 2003, selling all of their stocks, emptying their 401(k)s, and borrowing about $400,000 from friends and family.
They approached it less as a nonprofit than as a business, creating a detailed business plan, financial projections and benchmarks to meet. Debra devoted herself full time to the cause as Paul continued his career in the food services industry while working closely with her on CureDuchenne.
“We wanted to do whatever we could to accelerate drug development for the treatment of Duchenne,” Debra said.
From the outset, CureDuchenne was different from the other Duchenne organizations. While the organization does provide education and support resources for patients and their families, it’s dedicated to identifying, funding and fostering early-stage science that could eventually lead to a cure. One example is exon skipping, a treatment in which a faulty section of genetic code is “skipped,” allowing it to correct and restore dystophin production.
Those first few years, Debra worked from home. She had few contacts in the pharmaceutical or science industries, so she started introducing herself and studied every aspect of her son’s disease and the scientific developments that might give him and others like him around the world a higher quality of life.
Today, CureDuchenne uses a venture fund model, investing philanthropic dollars into scientific research entities through grants, loans and equity financing. Then it exits the company at the appropriate time and reinvests the returns in other scientific opportunities that could hold promise.
It pursued its first such venture in 2004, investing $1.3 million in Dutch biotech firm Prosensa financed by two early CureDuchenne board members, Vinny Smith, founder of Newport Beach-based firm Toba Capital, and Kelly Thornton Hallman, founder of the Living Peace Foundation.
Over the following 11 years, CureDuchenne stayed invested in Prosensa as it continued its exon-skipping research and developed experimental medicines. In 2014, Biomarin Pharmaceutical Inc. acquired Prosensa for $840 million and CureDuchenne used the proceeds to invest in four other companies.
Also that year, it created investment arm CureDuchenne Ventures LLC, which funds opportunities, investments ranging from $50,000 to $5 million. Its current portfolio includes Bamboo Therapeutics, Capricor Therapeutics and PTC Therapeutics, all of which are working on a variety of approaches to treat Duchenne’s.
Overall, CureDuchenne’s portfolio includes 12 wide-ranging projects with three successful exits. All 12 have made it to human clinical trials, one of them getting Food and Drug Administration approval.
“Our early investment in these organizations has helped draw the attention of VC firms,” Debra said. “They watch what we’re investing in. They know we do all our due diligence and have very specific goals. They trust us and have often invested in the same companies we have.”
CureDuchenne has also worked with leading-edge scientists in the field and founded two companies: Exonics Therapeutics, which develops gene-editing therapies for Duchenne, and Myotherix, which is developing therapies to improve muscle function and enhance steroid tolerance.
Key to Success
To raise both money and awareness, CureDuchenne hosts events, such as the 8th Annual Getzlaf Golf Shootout, hosted by Anaheim Ducks Captain Ryan Getzlaf, and the Napa in Newport wine auction.
The organization is also sponsored by well-known companies, such as Bristol-Myers Squib, Lilly and Pfizer. Its annual budget is $2.5 million, according to the Guidestar nonprofit report.
Paul is quick to point out that while Duchenne Ventures has had successes, every penny made is reinvested in more research.
End Goal
Hawken is now 21, and despite the challenges, is doing well. He’s a communications and journalism major at the University of Southern California and is doing many of the same things as his peers. He interned at a sports marketing company this summer, negotiating product placements. But unlike his peers, he takes more than 30 pills a day to maintain his current level of health.
Taking the even longer view, the Millers forge ahead with their vision.
“We need 10 times as many projects in the pipeline,” Paul said. “We would be so much farther ahead if we had more funding. Our goal is to fund the right science that will lead to a cure. Financial returns are secondary. And we are so, so close to a cure.”
