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When the Target Isn’t Really the Target: One Way Cancer Drugs Fall Out of Clinical Trials

October 24, 2019 / RICKI LEWIS

Ninety-seven percent of potential new cancer drugs never make it to market, dropping out of clinical trials when they don’t meet measures of safety or efficacy. “Why that is, we don’t really know. But I think that this extremely high failure rate suggests that there are some fundamental issues in how new drug targets are studied and how new drugs are characterized,” said molecular biologist Jason Sheltzer, PhD, an Independent Fellow at the Cold Spring Harbor Laboratory on Long Island, NY. He decided to investigate, and uncovered the potential power of publishing negative evidence. The work fits in with Open Access week here at Public Library of Science. CRISPR Improves Precision. The team reports in Science Translational Medicine on using the gene editing tool CRISPR-Cas9 to test whether 10 experimental cancer drugs work exactly how their developers predicted they would. And they found a tunnel vision in the way that drugs are targeted that might explain why certain patients do not respond as hoped.