Precision oncology using a clinician-directed, tailored approach to molecular profiling

Area of Science:

• Oncology
• Genetics
• Clinical Trials

Background:

• Precision oncology relies on molecular profiling (MP) to match patients with targeted therapies, particularly in early drug development (EDD).
• Comprehensive MP platforms can be costly, necessitating practical alternatives in resource-limited settings.
• A clinician-directed, tailored MP approach was developed to support EDD and precision oncology.

Purpose of the Study:

• To evaluate the feasibility of a clinician-directed, tailored molecular profiling approach for patient enrollment in molecularly targeted trials.
• To assess the impact of this approach on patient enrollment and outcomes in an early drug development program.

Main Methods:

• Patients referred to the Royal Melbourne Hospital EDD program (Sept 2013-Sept 2015) were analyzed.
• Clinician decisions for ordering MP were based on patient consultation, tumor type, and trial availability.
• Data collected included clinicopathological details, MP results, identified targets, and clinical trial enrollment.

Main Results:

• Of 377 referred patients, 216 (57%) underwent MP.
• MP identified 98 actionable targets in 86 patients.
• 98 patients (25%) enrolled in clinical trials, with 40 (11%) receiving molecularly matched treatments.
• Median progression-free survival was significantly improved in molecularly matched patients (3.6 months) vs. unmatched (1.9 months).

Conclusions:

• A clinician-directed, tailored molecular profiling strategy is feasible for early drug development.
• This approach successfully identified actionable targets and facilitated enrollment in molecularly matched clinical trials.
• The study demonstrates the potential of a practical MP approach to advance precision oncology in resource-constrained environments.