OnCo
ideasIdea

Test drugs on the patient's own cancer cells when there is no trial to join

For very rare cancers there is often no genetic clue and no trial. Growing the patient's cells and testing drugs on them directly can suggest what to try.

Functional precision medicineex vivo drug sensitivity testing on fresh patient material — guided treatment with reported benefit in a randomised haematology study and in paediatric and rare solid tumour case series. Turnaround, tissue quality and the absence of standardised reporting are the barriers. For rare cancers where genomics is uninformative, function is the only remaining evidence source.

Hypothesis
Ex vivo sensitivity-guided therapy improves progression-free survival ratio compared with the patient's own prior line in rare cancers without actionable mutations, in at least a third of tested patients.
Rationale
Functional testing bypasses the need for a biomarker hypothesis, which is what makes it suited to rare and unclassifiable tumours. Haematology results show the approach can outperform physician choice when turnaround is fast enough.
What would test it
A multi-centre study in rare solid tumours reporting assay success rate, turnaround, proportion of patients whose treatment changes, and progression-free survival ratio against their own prior line.
Maturity
early clinical
Who has to act
clinic
Cost to try
Medium ($1M to $50M)
Years to first evidence
5
Bottlenecks it attacks

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