OnCo
ideasIdea

A funded organoid and PDX panel as the go/no-go gate before IND-enabling money

Before any academic compound gets money for pre-trial studies, it would have to show activity in a standard panel of patient-derived tumour models run by an independent centre, so weak candidates are stopped early.

A central facility maintains a standardised, molecularly characterised panel of patient-derived organoids and xenografts across cancer types (drawing on EurOPDX, the NCI Patient-Derived Models Repository and Human Cancer Models Initiative collections) and tests every candidate nominated for IND-enabling funding in a blinded, pre-registered protocol with clinically relevant exposure. Results are published regardless of outcome. Funding for toxicology and CMC is contingent on passing pre-agreed activity criteria. This replaces the variable, investigator-run efficacy evidence that currently supports translational decisions.

Hypothesis
Candidates that pass an independent, blinded PDX/organoid gate have a phase 2 objective response or progression-free survival signal at least twice as often as historical academic candidates that were advanced on investigator-generated data alone.
Rationale
Retrospective studies show PDX and organoid response tracks patient response for many drug classes; NCI's PDMR and EurOPDX exist but are not used as a formal gate. Independent, blinded testing addresses the reproducibility and selection biases that inflate academic efficacy claims.
What would test it
Gate one translational fund's candidates for three years, publishing pass and fail results, and compare downstream clinical signals with the fund's prior ungated cohort.
Maturity
preclinical evidence
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks

Connected

9top

technologies

3
Functional (ex vivo) drug testingPatient-derived organoidsPatient-derived xenografts

ideas

3
A fast IND-enabling fund that pays for toxicology and manufacturing in eight weeksPatient-derived organoids to pick ADC payloadsSet aside 3% of grant budgets to replicate findings before translation

collections

1
Cancer Models (PDCM Finder) & HCMI

bottlenecks

2
Preclinical models that do not predict peopleThe valley of death between lab and product