Anavex Life Sciences Corporation recently announced promising results for its new drug, ANAVEX®2-73 (Blarcamesine). This oral medication for mild cognitive impairment (MCI) due to Alzheimer's disease is a readily absorbed small molecule that acts as a sigma-1 receptor (SIGMAR1) activator, believed to stabilize neurons and restore the plasticity of the brain. Researchers believe that activating the SIGMAR1 receptor activates a process called autophagy, in which cells break down and recycle defective proteins.
Blarcamesine achieved significant, visible improvements in patients in the beginning stages of Alzheimer's disease. Participants in the clinical trial who responded to the drug achieved an average improvement of 2.26 points on the Alzheimer's Disease Assessment Scale-Cognitive subscale (ADAS-Cog), the most common measurement scale used in Alzheimer's drug trials.
The medication had its greatest effect in patients with the mildest cognitive impairment, measured by the 30-question MMSE. Volunteers who received the drug for 48 weeks showed increased capability for performing tasks of daily living as well as improved memory and decision-making ability.
Anavex said that the 509 patients in the clinical trial reported few side effects, the most common complaint being dizziness. No other clinically significant side effect reached the 7.5 percent threshold, and there were no reports of clinically significant changes in blood tests, blood pressure, or EEG.
Professor Dr. Timo Grimmer explains, "ANAVEX2-73 is different from other treatment options that currently exist for Alzheimer's because science indicates that ANAVEX2-73 has a better side effect profile. For example, ANAVEX2-73 does not have any GI side effects. Additionally, ANAVEX2-73 has a longer duration of positive therapeutic impact than existing treatment options for Alzheimer's disease."
Anavex researchers believe that the new drug will be effective in about 80 percent of Alzheimer's patients. They have identified a mutation in the SIGMAR1 gene that affects about 20 percent of people that may interfere with the action of the drug. Avenax intends to continue the study until volunteers have received 96 weeks of treatment.
"The potential implications of our research is improved cognitive capabilities for people diagnosed with debilitating neurodegenerative or neurodevelopmental disorders. This is a significant potential worth pursuing, as families across the globe caring for a loved one afflicted with a CNS disease are quite aware," explains Dr. Christopher Missling.
This clinical trial continues a relatively new approach to research that involves associating results with genetic markers. The researchers do not make a priori assumptions about interactions of genes and the effectiveness of the investigational drug, but examine the genetics of those patients for whom the drug works best.
