This Alzheimer’s disease investigational drug is actually promising against Rett syndrome (a rare disease)


ANAVEX-LOGO_JPGAnnavex Life Sciences Corp-investigational drug, Anavex 2-73 has shown positive results during its exploratory study on  Rett  syndrome.


Interestingly, Annavex 2-73 was earlier developed to potentially treat the Alzheimer’s disease and successfully cleared the Phase I clinical trial. Later when the drug was explored for its effect on the symptoms of Rett syndrome it showed encouraging results.

In the MeCP2 Rett syndrome disease mouse model, chronic  oral daily dosing of ANAVEX 2-73 started at ~5.5 weeks of age  and continued for a 12‐week behavioral testing time point 60 minutes pre‐treatment during the behavioral testing. Various levels of muscular coordination, balance, motor learning and muscular strengths and  some  of the characteristic  problems observed in Rett syndrome were tested under  their  behavioural criteria. PsychoGenics, Inc., NY performed the experiment and was sponsored by Scout Program. 

Steven Kaminsky, science officer of said The data demonstrate dose-related and significant improvements in an array of behavioural and gait paradigms in a mouse model with a MeCP2-null mutation that causes neurological symptoms that mimic Rett syndrome. There is a tremendous need for therapeutic solutions for the individuals living with Rett syndrome and their families, and we are very encouraged by the data we have seen with ANAVEX 2-73” .

Rett syndrome is a rare genetic postnatal neurological disorder of the grey matter of the brain whose symptoms are shown between 6 to 18 months of age. It’s characteristic symptoms shows repetitive stereotyped hand movements, decreased rate of head growth, small hands and feet. Rett syndrome is caused by mutations in the gene MeCP2 located on the X chromosome (see below).


Figure 1. Causative mechanism of Rett Syndrome

Only symptomatic treatment is available for the disease and is currently incurable. In the present situation the efficacy shown by Anavex 2-73 for Rett syndrome  and its safety data sheet  promises  a bright future for Rett syndrome treatment.  

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