After an Israeli biotech company stopped working on a promising medication, two U.S. families took an unusual step: They bought the drug and decided to develop it themselves as a possible treatment for their children’s muscular dystrophy. are hoping the experimental compound, called halofuginone, can lessen the impact on their sons and other people who suffer from Duchenne muscular dystrophy.
Halofuginone isn’t able to correct the underlying genetic defect that causes Duchenne. But published research studies using the drug have shown good results at easing symptoms in mice with Duchenne. And Collgard, which was exploring halofuginone for other diseases, had run trials in people that provided safety data on the drug.The families hired a biotech industry veteran to arrange a deal with the Israeli firm, Collgard Biopharmaceuticals.The families, through research foundations they had set up, agreed to pay $500,000 in March to buy the drug and $500,000 more 15 months later.Collgard also will get a cut if the drug proves valuable down the road.
Although the costs of developing a drug might seem out of reach for most consumers, but this model is very workable,.
grew frustrated after years of putting money into research and drug-development projects that stalled, went too slowly, or were switched to focus on another disease.we could not control the pace until we had operational control” of a drug’s development
Companies also drop drugs for commercial reasons—sometimes the market is deemed too small, the company ran out of money, management changed focus to a new disease, or the company got bought and lost control of the project.