CLINICAL APPROVAL CONCORDANCE AND TUMOUR CONTEXT IN PRECLINICAL ANTICANCER DRUG RESPONSE
DOI:
https://doi.org/10.65327/bp.v12i1.2538Keywords:
anticancer drugs, mouse xenografts, FDA approval, tumour context, preclinical drug responseAbstract
Both preclinical tumour suppression and approval of an anticancer treatment for a specific cancer are used to prioritize treatments, but the correlation is not clear. An observational analysis was performed to assess the number of xenografts with a US Food and Drug Administration approval matched to the cancer type and the number of xenografts which are not approved by the US Food and Drug Administration and whether there were any differences in drug response. A 2025 data set of 1,173 cases of xenografts was screened, of which 736 single agent records with valid tumour-volume ratios were analysed for breast, lung and colorectal cancers. Response was primarily defined as ≥ 40% reduction in tumour volume compared to the control group. Response occurred in 230 of 441 approval-matched records (52.2%) and 126 of 295 nonmatched records (42.7%). The association was unclear after adjustment for tumour type and clustering by publication (odds ratio 1.42, 95% confidence interval 0.93-2.17; p = .103) and was not supported by any interaction with approval-by-tumour. A stricter 60% reduction threshold yielded an odds ratio of 1.61 (95% confidence interval 1.05–2.49; p = .030). These retrospective correlations don't prove that the xenograft results translate to patient outcomes or eventual approval.
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Copyright (c) 2026 Adams Adah Otakwu, David Acheme Otakwu, Muhammad Auwal Sule, Mathias Okwori Otakwu, Blessing Onyanta Otakwu
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