- The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy — described today in the WSJ— is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development.
Before the Seckler and Wicka families bought halofuginone, a drug that showed promise in experiments done with Duchenne mice, they set up Dart Therapeutics. Dart is funded by foundations set up by the two families, and run by Eugene Williams, a drug industry veteran with 25 years’ experience, including seven years at Genzyme.
Mutual acquaintances led the Secklers and Wickas to Williams in 2010, when the families were discussing how to advance drug development in Duchenne muscular dystrophy. The disease is rare, with only around 20,000 new cases a year, and both families were increasingly frustrated that despite raising many millions to help fund research and experimental drug development programs, promising compounds frequently went nowhere.
They wondered if, with expert advice, patients and advocates in some cases might be able to develop drugs themselves.
That idea led to the formation of Dart in June, 2010. Williams, who is the chairman, put together a “virtual company” — with no big in-house staff — comprised of former senior executives and consultants expert in developing drugs and getting them through the FDA approval process. The idea, says Tracy Seckler, whose son Charley has Duchenne and whose Charley’s Fund foundation is co-owner of Dart, was to create a vehicle to identify potential compounds, make deals, and develop the drugs.
Williams says that in developing drugs for rare diseases, patients and advocates have to take a variety of approaches. Dart’s job is to assess how to best move a drug forward. In some cases, that may mean putting up some money to get other companies to focus on a compound or to pay for testing or other studies to move the program forward. In other cases, Dart will provide advisers and consultants to oversee pre-clinical work that a traditional pharma or biotech company might not want or be able to take on. “We want to help reduce the risk so drugs don’t get stalled,’’ says Williams.
By Amy Dockser-Marcus
In cases where Dart decides it’s worth it to buy the drug — which is what happened when it took a look at halofuginone — a separate company will be formed. (In the case of halofuginone, the company is called Halo Therapeutics.)
Williams presented the model at a FasterCures conference last year in an effort to spread the word to other patient-driven foundations.
Ultimately, some of those foundations may become customers of Dart. Seckler tells the Health Blog that the goal is not only to help do-it-yourself drug development for Duchenne but also other orphan diseases that can use outside experts to negotiate deals, oversee experiments, and work with the FDA.
“We hope to generate revenue to continue what we’re doing in Duchenne muscular dystrophy by providing these services,” she says.