ideasIdea
An open organoid bank for cancers too rare to have models
For many rare cancers there is not a single laboratory model in the world, so no one can test drugs. A shared bank with free distribution would change that.
Rare and paediatric cancers lack cell lines and xenografts, which blocks even basic drug testing. A distributed programme would fund collection at referral centres, derive organoids and xenografts under a common protocol, characterise them genomically, and distribute them at cost with no reach-through rights. Precedents include the Human Cancer Models Initiative and paediatric preclinical testing programmes.
Hypothesis
Providing at least three characterised models for each of 50 rare cancer types measurably increases the number of published drug-testing studies and industry programmes in those diseases within five years.
Rationale
Model scarcity, not biological intractability, is the first barrier for rare disease drug development; a model is a prerequisite for any preclinical package.
What would test it
Fund derivation for ten rare types, distribute openly, and count downstream requests, publications and programme starts against matched types without models.
Maturity
preclinical evidence
Who has to act
philanthropy
Cost to try
Medium ($1M to $50M)
Years to first evidence
5
Bottlenecks it attacks
- Preclinical models that do not predict people · Nine in ten cancer drugs that work in mice fail in humans. Our models are the reason.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.