Related Experiment Video
Updated: Aug 14, 2026
Development of a Virtual Reality Assessment of Everyday Living Skills
Published on: April 23, 2014
Efficacy of flupirtine on cognitive function in patients with CJD: A double-blind study
1Departments of Neurology, University of Goettingen, Germany. motto@gwdg.de
Background:
In cell culture experiments, flupirtine maleate (FLU), a triaminopyridine compound, was able to protect neuronal cells from apoptotic cell death induced by prion protein fragments and beta-amyloid peptides. As FLU is a clinically safe drug, the authors started a double-blind placebo-controlled study in patients with Creutzfeldt-Jakob disease (CJD).
Methods:
Twenty-eight patients with CJD were randomized to an oral treatment with either FLU (n = 13) or matching placebo (PLA; n = 15). For inclusion and continuing the study, the patients had to achieve at least 50% in two of the subscales of the dementia tests employed. A battery of standardized questionnaires was employed to monitor the progression of the disease. The main outcome variable was the cognitive part of the Alzheimer's Disease Assessment Scale (ADAS-Cog); the difference between baseline and the best score under treatment was defined as the primary efficacy variable for hypothesis testing.
Results:
CJD types were homogeneously distributed among the treatment groups. Patients treated with FLU showed significantly less deterioration in the dementia tests than patients treated with PLA. The mean change in ADAS-Cog (baseline to best) was +8.4 (+/-15.3) in the FLU group and +20.6 (+/-15.1) in the PLA group (p = 0.02, one-sided t-test).
Conclusions:
FLU has beneficial effects on cognitive function in patients with CJD. These positive results also may suggest a treatment potential of FLU in other neurodegenerative disorders. However, further studies are necessary.
Insights
Flupirtine maleate (FLU) demonstrated beneficial effects on cognitive function in Creutzfeldt-Jakob disease (CJD) patients. This clinical trial suggests FLU may slow cognitive decline in neurodegenerative disorders.
Area of Science:
- Neuroscience
- Pharmacology
Background:
- Flupirtine maleate (FLU), a triaminopyridine, showed neuroprotective effects against prion protein and beta-amyloid-induced apoptosis in cell cultures.
- Given its clinical safety, FLU was investigated in a study for Creutzfeldt-Jakob disease (CJD) patients.
Purpose of the Study:
- To evaluate the efficacy of flupirtine maleate (FLU) in treating patients with Creutzfeldt-Jakob disease (CJD).
- To assess the impact of FLU on cognitive function decline in CJD patients.
Main Methods:
- A double-blind, placebo-controlled study involving 28 CJD patients randomized to FLU or placebo (PLA).
- Cognitive function was monitored using standardized questionnaires, with the Alzheimer's Disease Assessment Scale-Cognitive (ADAS-Cog) as the primary outcome measure.
Main Results:
- Patients receiving FLU exhibited significantly less cognitive deterioration compared to the placebo group.
- The mean change in ADAS-Cog from baseline to best score was significantly better in the FLU group (+8.4) than in the PLA group (+20.6) (p=0.02).
Conclusions:
- Flupirtine maleate (FLU) shows beneficial effects on cognitive function in Creutzfeldt-Jakob disease (CJD) patients.
- These findings suggest potential therapeutic applications for FLU in other neurodegenerative diseases, warranting further investigation.
