Metastatic cancer is a medical challenge that has been historically resistant to treatments. One area of leverage in cancer care is the development of molecularly-driven combination therapies, offering the possibility to overcome resistance. The selection of optimized treatments based on the complex molecular features of a patient's tumor may be rendered easier by using a computer-assisted program. We used the PreciGENE® platform that uses multi-pathway molecular analysis to identify personalized therapeutic options. These options are ranked using a predictive score reflecting the degree to which a therapy or combination of therapies matches the patient's biomarker profile. We searched PubMed from February 2010 to June 2017 for all patients described as exceptional responders who also had molecular data available. Altogether, 70 patients with cancer who had received 202 different treatment lines and who had responded (stable disease ≥12 months/partial or complete remission) to ≥1 regimen were curated. We demonstrate that an algorithm reflecting the degree to which patients were matched to the drugs administered correctly ranked the response to the regimens with a sensitivity of 84% and a specificity of 77%. The difference in matching score between successful and unsuccessful treatment lines was significant (median, 65% versus 0%, p-value <0.0001).
Keywords: exceptional responders; molecular pathology; neoplasms; precision medicine; therapeutic decision.