Implementing precision medicine initiatives in the clinic: a new paradigm in drug development.
OBJECTIVE
With the recent advances in genome sequencing technology, greater knowledge of cancer biology allows the identification of different molecular alterations in tumor samples from patients with advanced cancer, some of which are promising potential therapeutic targets. This review highlights the different methods for molecular screening, examines the recent success and preliminary precision medicine programs' results. Challenges and perspectives of this approach are discussed.


RESULTS
The emergence of high-throughput technologies has allowed the stratification of most common diseases into rare molecular segments. The recent success of predictive biomarkers showed that molecular screening has become essential in the development of drugs in early clinical trials. Several prospective trials are ongoing worldwide with very promising results.


CONCLUSIONS
Characterization of the genomic alterations in cancer patients is now critical to better select molecular-targeted therapies and to accelerate the drug development process in phase I/II. High-throughput analysis is feasible in daily practice, and allows enrichment of phase I trials in molecularly selected patients.
aDrug Development Department (DITEP: Département d'Innovations Thérapeutiques et Essais Précoces) bInserm Unit U981, Gustave Roussy Cancer Campus, Villejuif cFaculté du Kremlin Bicêtre, Université Paris Sud, France.
Antoine
A
Hollebecque
Christophe
C
Massard
Jean-Charles
JC
Soria
D000970:Antineoplastic Agents / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000235:genetics; D002986:Clinical Trials as Topic; D004252:DNA Mutational Analysis / Q000379:methods; D055808:Drug Discovery / Q000379:methods*; D006801:Humans; D058990:Molecular Targeted Therapy / Q000379:methods*; D009369:Neoplasms / Q000188:drug therapy* / Q000235:genetics
D000970:Antineoplastic Agents; D014408:Biomarkers, Tumor