OnCo
ideasIdea

Pre-surgery platform trials that test combinations on pathological response in months

Give combinations before surgery and look at how much tumour is left when it is removed. That answer comes in months, so many pairs can be tested quickly.

I-SPY 2 established graduation of arms by pathological complete response in breast cancer. The same model applies to lung, bladder, melanoma, head and neck and oesophago-gastric cancer where neoadjuvant therapy is now standard. Each cancer needs a standing neoadjuvant platform with a shared control arm and pre-agreed rules for graduating an arm to a confirmatory trial.

Hypothesis
Neoadjuvant platforms will screen at least five combination arms per year per cancer, with pathological response predicting event-free survival well enough that graduated arms confirm in phase 3 at least 60% of the time.
Rationale
Pathological complete response and major pathological response correlate with survival in breast, lung and melanoma, and the neoadjuvant window allows paired biopsies that explain why a combination did or did not work.
What would test it
Open a neoadjuvant platform in one cancer without one (oesophago-gastric or head and neck), graduate two arms by pathological response, and track confirmation.
Maturity
being tested at scale
Who has to act
research
Cost to try
Large (over $50M)
Years to first evidence
3
Bottlenecks it attacks

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