Jonathan Cohen, Director of Clinical Research at Sharett Institute of Oncology of Hadassah Medical Center, shared on LinkedIn:
“All Genetic Alterations are Equal, But Some are More Equal Than Other
DRUP trial (published in Nature), 1610 advanced cancer patients treated off-label based on presumed actionable genetic alterations. Data for often-utilized precision medicine.
Some take-home messages:
- Not all predictive biomarkers performed equally: MET amplification, BRAF V600E mutations, MSI-H, TMB-H, and BRCA1/2 inactivation were strong biomarkers.
- Tissue context matters – We knew this from BRAFi in CRC vs. melanoma; validated here in other targets and tumor types.
- Whoever saves a single soul,…: 7.0% exceptional responders with durable response.
- However, for each exceptional responder, there were ~4 with severe toxicity (28.4%).
Structured trials like DRUP are the bridge from “empirical” to “standard of care”. These will eventually allow us to prioritize the most-effective off-label prescriptions and generate a sufficient mass of evidence for drug registration and reimbursement.”
Title: Prospective evaluation of genomics-guided off-label treatment
Authors: K. Verkerk, A. C. Spiekman, S. F. Haj Mohammad, F. A. J. Verbeek, H. Timmer, M. A. van Maren, L. J. Zeverijn, B. S. Geurts, V. van der Noort, P. Roepman, A. M. L. Jansen, W. W. J. de Leng, H. Gelderblom, H. M. W. Verheul, E. E. Voest and The DRUP Trial Investigators
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