The Generative Brain

Quinacrine in CJD (UK PRION-1 and UCSF placebo-controlled trial)

Quinacrine studies in prion disease: the UK PRION-1 patient-preference study (107 volunteers, 300 mg a day, reported March 2009), a French compassionate-use series and a US randomised trial in 54 patients (published December 2013). None showed a survival benefit.

Status as of 2026-10-16. Please check the study registry for the latest position.

Key facts

  • Status: Completed; no survival benefit.
  • Who can join: Sporadic CJD
  • Where: UK and US
  • Sponsor: MRC and UCSF
  • Registry NCT00104663: National Prion Clinic, London; PI John Collinge; MRC Clinical Trials Unit; inclusion age 12 or more with any human prion disease.
  • PRION-1 enrolled 107 volunteers taking quinacrine 300 mg a day; after adjusting for large differences between patients who chose the drug and those who did not, the authors found no evidence of longer survival; published in Lancet Neurology on 10 March 2009.
  • A French compassionate-use series of 32 patients (published December 2004) reported no significant effect.
  • The US randomised double-blind placebo-controlled trial, led at UCSF, enrolled 54 patients on 300 mg a day, saw no benefit and was published in Neurology on 3 December 2013; its randomised phase was only 2 months, which the lead author calls a design weakness.
  • Quinacrine did not prolong survival in a mouse model, so the negative human results could largely have been predicted.

Timeline

  1. 2004French compassionate-use series published.
  2. 2005US randomised trial recruitment begins.
  3. 2009PRION-1 results published in Lancet Neurology.
  4. 2013US quinacrine trial published in Neurology.
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