A detected mutation is not automatically a treatment target
Targeted therapies interfere with proteins or pathways that help some cancers grow. Small-molecule drugs and therapeutic antibodies work differently, and their evidence is tied to a specific tumour type, biomarker, disease stage and treatment setting.
A genomic report may list many alterations, but only some are clinically actionable. The care team distinguishes validated treatment-linked findings from uncertain variants and verifies whether the exact assay and specimen support the proposed use.
The same alteration can have different treatment implications in different cancers. Approval, guideline support, resistance markers and access must all be checked.
Who may be considered?
This pathway may be discussed when the confirmed diagnosis, disease extent and treatment history make targeted therapy a reasonable question—not simply because the technology is available.
- A cancer with an established predictive biomarker and an appropriate approved targeted treatment.
- Advanced or recurrent disease where profiling may identify another evidence-based option.
- Progression on prior targeted therapy where resistance testing could affect the next choice.
- A clinical trial requiring a defined molecular alteration.
- A review of an outside report that labels a finding actionable without clear disease-specific evidence.
What the specialist team must confirm
Specialists review pathology, stage, specimen source and date, assay method, variant details, previous targeted agents, response duration, resistance findings, current disease burden, organ function and interacting medicines. When the sample no longer represents current disease, repeat tissue or liquid testing may be discussed.
Key points for this treatment
From biomarker report to an evidence-matched plan
The molecular finding is confirmed, graded by evidence, checked against the clinical setting and translated into a monitoring plan rather than treated as a stand-alone answer.
Response, resistance and the next molecular question
Targeted therapy may be continued while it controls disease and toxicity remains acceptable. Imaging, symptoms and selected laboratory or molecular measures are interpreted together.
At progression, the team asks whether resistance is focal or widespread and whether a new biopsy or liquid biopsy could change treatment. Continuing, combining or switching therapy requires disease-specific evidence.
Limits, burdens and realistic expectations
Tumours are heterogeneous and evolve. Testing can miss alterations, a matched drug may not work, and resistance can emerge. Off-label access may be limited, interactions and organ toxicity matter, and a variant of uncertain significance is not a proven reason to treat.
Severe rash with blistering, breathing difficulty, chest pain, fainting, sudden neurologic symptoms, uncontrolled diarrhoea or other rapidly worsening symptoms need urgent local assessment.