Steve Perrin, an amyotrophic lateral sclerosis (ALS) researcher, recently reexamined more than 100 pharmaceutical compounds that had once been flagged as candidate drugs for ALS symptoms. But in retesting, Perrin found that the vast majority failed to slow the fatal degenerative disease, also commonly known as Lou Gehrig's disease.
Perrin, chief scientific officer at the ALS Therapy Development Institute in Cambridge, Massachusetts, found eight studies that originally showed promise in lab mice -- but ultimately failed in humans -- shouldn't have made it past the early stages of research.
Perrin says his research is proof the better quality controls are needed for early drug trials involving mice. Too often, Perrin argues in a recent paper published in Nature, problems that arise in human drug trials could have been predicted had researchers given more thought to the differences between mouse and human anatomy.
Perrin says the problem extends beyond ALS drug trials.
“I hear too many stories about patients who have used their one shot at getting into a trial on a drug that didn’t have enough legs to begin with, and that’s a tragedy,” Perrin told Nature.
Other researchers agree. Caterina Bendotti, a neurobiologist at the Mario Negri Institute for Pharmacological Research in Milan, Italy, told Nature: “The poor reproducibility of preclinical results, particularly in animal models, goes beyond ALS."
Perrin says more work needs to be done in early trials to better understand how drugs will translate from mice subjects to humans. Others say that kind of extra effort is often cost prohibitive.
Still, Perrin says extra precaution at the beginning would save money in the long run. “Somebody has to do this, or else we're wasting precious resources,” he said.