Timelines: 2012

Two families looking for a cure take business into their own hands

 

When Israeli biotech company Collgard Biopharmaceuticals recently halted development of a drug for muscular dystrophy, two American families affected by the disease decided to become actively involved. In an unprecedented transaction, the Seckler family of Stockbridge, Massachusetts, and the Wicka family of Minneapolis, Minnesota, negotiated with Collgard to buy its promising experimental compound halofuginone (HT-100) and oversee its development. Both families have sons, Charley Seckler, age eleven, and Nash Wicka, age twelve, who suffer with Duchenne muscular dystrophy (DMD), a progressive muscle wasting disease that confines most patients to a wheelchair by adolescence. DMD has no cure and affects an estimated 20,000 American males.

Collgard had experimented with halofuginon for other diseases, but concentrated its efforts on DMD after studies showed that halofuginone alleviated symptoms of muscular dystrophy in mice. The company conducted preliminary human trials and collected safety data, but decided not to pursue the project further. The stall was unacceptable to Charley’s mother, Tracy Seckler, who told the Wall Street Journal: “As a parent, even worse than having no treatment prospects is having a treatment prospect going nowhere.” With funds from their separate DMD research foundations, the families paid Collgard $1,000,000 for the compound and hired Marc Blaustein, a former biotech executive at Dyvax Corp., to lead the renewed research effort. As head of the newly established Halo Therapeutics, Mr. Blaustein plans to begin clinical trials of the drug within a year.

While this model of do-it-yourself drug development is innovative, industry experts predict challenging roadblocks ahead. Moving the drug through Phase II clinical trials will require an additional $8 million, and there is no guarantee that early clinical results will be reproduced. Pat Furlong, President of the Parent Project Muscular Dystrophy, points out that while patient groups often become emotionally attached to a promising compound, “the odds are against picking a winner.”

Despite the odds, the Secklers and Wickas are making progress. The two families were commended for a recent European clinical trial proposal. They convinced a panel of drug development advisors at Treat-MND that Halo Therapuetics is “ready for the clinic and extraordinarily well-prepared.” On January 4, HT-100 was granted orphan drug status by the U.S. Food and Drug Administation. For both families, active progress is the goal. Says Mr. Wicka “We are ready to be the ones who get to make the decision of go or no go on this one."