Disappointing Results in Sporadic CJD Treatment Trial

Brandy R. Matthews, MD

Despite efficacy in vitro, quinacrine did not prolong survival in humans with sporadic Creutzfeldt-Jakob disease in a randomized trial.

Sporadic Creutzfeldt-Jakob disease (sCJD) is a human prion disease that results in rapidly progressive dementia and causes death within 1 year in 90% of cases. Quinacrine is an antimalarial drug known to have central nervous system penetration and eliminates prions in vitro. Open-label administration suggested that the medication was well-tolerated by sCJD patients. Therefore, researchers undertook this randomized, controlled clinical trial of oral quinacrine (300 mg daily) to assess survival benefit. Of 425 patients, 69 patients were evaluated, of whom 54 were randomized to quinacrine therapy or placebo. An additional 3 subjects were excluded from analysis following the discovery of a known genetically determined variant of CJD. Open-label quinacrine was offered to patients who attended a 2-month follow-up visit.

Survival at 2 months (the primary outcome) did not differ significantly between the two groups. Secondary endpoints of functional decline, also assessed at month 2, suggested potential benefits of quinacrine on two out of three measures. A planned, open-label extension of the trial to 12 months did not reveal any survival benefit in those treated with quinacrine.

Citation(s):

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