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Not Quite How It Works

Many cancer drugs in development don't work quite how their developers thought they did, according to a new analysis. This, Discover's D-brief blog notes, could contribute to the large portion of cancer drugs that never get approval from the US Food and Drug Administration.

Researchers on Long Island used CRISPR-Cas9-based mutagenesis to explore 10 cancer drugs in preclinical or clinical testing and their targets. As they report in Science Translational Medicine this week, they found that many of the proteins these drugs are thought to target aren't essential for cancer cell growth and that the efficacy of the drugs wasn't affected by the loss of their putative targets. Instead, the researchers report these drugs worked through other, off-target means to kill cancer cells.

"Our study showed us that a potential issue with the cancer drug development pipeline is that the ways in which some of these new cancer drugs work is incompletely understood," co-first author Chris Giuliano from Cold Spring Harbor Laboratory and Stony Brook University, said at a press briefing, according to D-brief.

He and his colleagues write in their paper that "stringent genetic validation of the mechanism of action of cancer drugs in the preclinical setting may decrease the number of therapies tested in human patients that fail to provide any clinical benefit."