Related Experiment Video
Updated: Jan 7, 2026
05:10
Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
10.1K
Drug Development
Marwan N Sabbagh1, Audrey Gabelle2, Timo Grimmer3
1Barrow Neurological Institute, Phoenix, AZ, USA.
Alzheimer'S & Dementia : the Journal of the Alzheimer'S Association
|December 26, 2025
Summary
Precision medicine in Alzheimer's disease (AD) shows promise. Blarcamesine, targeting the Sigma-1 receptor (SIGMAR1), demonstrated enhanced efficacy in patients with the wild-type (WT) SIGMAR1 genotype, supporting personalized treatment strategies.
Area of Science:
- Neuroscience
- Pharmacology
- Genetics
Background:
- Precision medicine tailors Alzheimer's disease (AD) treatments based on individual profiles.
- This approach optimizes outcomes by identifying patient subgroups likely to benefit from specific therapies.
Purpose of the Study:
- To evaluate the efficacy of blarcamesine, a Sigma-1 receptor (SIGMAR1) modulator, in Alzheimer's disease patients.
- To investigate the role of the SIGMAR1 genotype in predicting response to blarcamesine treatment.
Main Methods:
- Conducted a Phase IIb/III clinical trial (ANAVEX2-73-AD-004) using oral blarcamesine.
- Performed pre-specified efficacy analyses based on the SIGMAR1 genotype (wild-type vs. others).
Main Results:
- Blarcamesine significantly improved cognitive and functional measures (ADAS-Cog13, CDR-SB) compared to placebo in all participants.
- Patients with the homozygous wild-type (WT) SIGMAR1 genotype showed substantially greater improvement versus placebo (49.8% for ADAS-Cog13, 33.7% for CDR-SB).
Conclusions:
- The SIGMAR1 genotype is a key determinant of blarcamesine efficacy in AD, highlighting its potential in precision medicine.
- Personalized therapies targeting specific molecular profiles offer more substantial and sustained benefits for AD patients.
- Realizing the full potential of precision medicine in AD requires addressing challenges in cost, data privacy, ethics, and access.
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