OnCo
ideasIdea

Test intermittent dosing of targeted drugs to delay resistance, with honest priors

Giving a targeted drug in pulses rather than continuously might slow the emergence of resistant cells and reduce side effects. Early results are mixed, so this needs careful trials with clear rules for when to try it.

Randomised trials of intermittent versus continuous dosing of targeted agents, selected by biology: intermittent schedules are favoured where preclinical data show drug-addicted resistant clones (as in some BRAF-mutant melanoma models) or where toxicity is exposure-driven; disfavoured where continuous suppression is needed. The SWOG S1320 trial found intermittent BRAF/MEK inhibition did not improve PFS in melanoma, so the proposal targets settings with stronger mechanistic support (e.g., some hormonal and ALK-driven settings) and uses ctDNA to define pulse timing.

Hypothesis
In biologically selected settings, intermittent schedules will achieve non-inferior PFS with reduced toxicity and cost, and in a subset will delay resistance; in unselected settings they will not.
Rationale
Resistance is an evolutionary process sensitive to dosing schedule; preclinical models show schedule-dependent outcomes, but the one large clinical test was negative, so selection criteria matter.
What would test it
Two randomised phase 2 trials in settings with preclinical support for drug-addicted resistance, with ctDNA-guided pulse timing and PFS non-inferiority plus toxicity as endpoints.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
4
Bottlenecks it attacks
  • Wrong doses · Most drug doses were chosen as the highest a person can tolerate, which is often more than they need.
  • Acquired resistance to every therapy · Nearly every targeted therapy stops working within months to a few years as the tumour adapts.

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