By Thomas C. Südhof in The Washington Post.
When the news emerged in November that a major clinical trial for a drug to treat Alzheimer’s disease had failed, the questions naturally focused on the trial itself: Was the drug poorly designed? Was the science bad?
Yet these questions fall short in explaining why the $1 billion trial fizzled. The real problem is that there simply is not enough underlying science to begin with. The trial, led by Eli Lilly, was based on a vague hypothesis without a fundamental understanding of Alzheimer’s disease itself. The trial — like too many recently — was essentially a moonshot in the dark.
The episode underscores that it is time to rethink how we pursue biomedical research and drug development. This shift is especially pressing for diseases of the brain, such as autism and Alzheimer’s disease, which are becoming increasingly important for our society but lack effective treatments. One reason for this lag in treatments is that we have failed to invest enough in obtaining a fundamental understanding of diseases. Instead, we have spent too much on costly clinical trials and studies on human patients that, for ethical reasons, have to be guided by the interests of the patients rather than research goals.
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