CAMBRIDGE, Massachusetts – Biogen thought they had developed a treatment disease Lou Gehrig , a devastating disease that leaves patients trapped in their own bodies until atrophy inevitably die. After the first clinical studies showed encouraging results a few years ago, the biotech company launched a home run hit and launched a huge test of the final stage to test the drug worked.
When the study not , scientists broke into tears at the news, which crushed what appeared to be a godsend for patients was no better than placebo. And then they went to drink.
“We hold all the equipment, like 30 people, one of the best restaurants in Boston,” recalls Steve Holtzman, Biogen executive vice president of development at the time. “We bought champagne, wine fine, and we had an Irish funeral.”
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Such heavy heart celebrations are common in biotechnology, where failures are much more numerous successes. This summer, giants like Bristol-Myers Squibb, AstraZeneca, and Celgene have encountered setbacks with expensive drugs in development. And smaller players, including Seres Therapeutics, Tokai Pharmaceuticals, and Tobira Therapeutics clinical failures have been put into question its long-term survival.
Companies wave through thousands of molecules before hitting on a safe and effective enough to test in humans. And even the chosen few have only a probability of 10 percent, what is always on the ledge of a pharmacist, according to data Biomedtracker , a research service that keeps tabs on the industry.
Pharmaceutical companies use these sad success rates to defend the increasing cost of new treatments. Each therapy approved, they argue, is placed on the shoulders of a thousand minor compounds and should be priced accordingly.
But down among the scientific basis and file, the attrition rate in drug development is only part of the job. Scientists slog through years of research to get a slot in biotechnology, then often spend an entire career working hard in laboratories without touching once a project that matures in an approved drug. It can be emotionally draining, as researchers learned Biogen, leading many companies to remember the many noble assumptions fall short.
And failures are often feted.
“You celebrate the achievement of getting a response,” said Matthew Kennedy, director of neuroscience research at Merck, even if that answer is a resounding “no”.
is the moral of maintenance, in part, but toasting a great idea that fell flat also helps encourage scientists to pursue the next advance, said Seng Cheng, head of research for rare diseases in the division Sanofi Genzyme.
And it’s a good deal. biotechnology companies listed put about a quarter of its revenue in research and development, amounting to an investment of approximately $ 35 million last year, according to a recent report in the journal Nature Biotechnology . Cut the cord in a once-promising program saves companies throwing money away.
“If these faults are found quickly, often handsomely rewarded as successes,” Seng said.
However, the timing of killing a program is usually not easy. If you ask scientists, “there is always a ray of hope,” she said John Butler, CEO of anemia biotechnology focused Akebia Therapeutics. A change in the chemical, a pivot in recruiting patients – rarely do researchers perceive a wall that can not be circumnavigated with a little more time and money
“Two things are always true. drugs that deserve to die refuse to die, and yet more than 90 per cent of successful drugs, failed once or more along the way, but for a champion refuse to let them die, “Holtzman said, now is CEO of Cambridge Decibel Therapeutics .
That makes for the greatest enigma in the direction of a biotechnology company, Holtzman said. Nobody wants to mothball what could be the next breakthrough in cancer, but if it blows its research budget of a drug three times failed, could lose his job.
the trick is to balance passion and objectivity, according to Merck Kennedy. drug hunters at the same time should be able to go full bore in their projects and be clear-eyed about possible faults, he said.
With the ever-present specter of a bad eventual result, companies strive to reward small victories along the path of drug development.
Small trophies, plaques inscribed tablets and jars dot the operating tables in neuroscience at Merck in Boston, distributed in honor of steps along the way. In the Modern Therapeutics in Cambridge, scientists received ceremonial – but empty -. Vials first candidate company drug to commemorate his trial opening, now underway
And when that shot 1-in-10 comes through a drug and win approval, the companies reach a little more moral in their budgets. Two years ago, Genzyme has the blessing of the Food and Drug Administration to market a drug for a rare disease called Gaucher disease, culminating a process of 15 years. And what chemists who discovered that gathered around an oblong table, invited to a slice of cake provided by the company . Then they returned to work.