ideasIdea
Intermittent or stop-and-restart nirogacestat in desmoid tumours
Desmoid tumours are not cancers and often stop growing on their own; treating them indefinitely with a drug that causes ovarian failure may be more than needed.
DeFi showed durable responses and ovarian toxicity that reverses on stopping; debate (JCO 2026) questions long-term continuous use.
Hypothesis
Treatment to best response followed by a drug holiday with restart on progression preserves disease control and fertility with fewer adverse events.
Rationale
Slow-growing, non-metastasising tumour; toxicity is dose-duration dependent and reversible.
What would test it
Randomised discontinuation trial after 12 months of response; time to progression and ovarian function as co-primary.
Maturity
speculative
Pages like this
not linked directly; found by shared links- CompanySpringWorks Therapeutics (Merck KGaA)
- PathwayWnt / β-catenin
Shares DeFi, Nirogacestat.