Rett Syndrome is Such a Trial… #1 Anavex 2-73

seven…and 2019 looks to change that up a bit- Rett Syndrome, so many trials! According to the most recent GP2C newsletter (GirlPower2Cure)there will be at least 7 trials this year, currently in various stages. This is SO exciting! What does this mean for parents? Well, of the trials already listed on it means that there are trial sites in 15 different states and two in the UK; this translates to, “there is a trial site near YOU!” and the Rettland Foundation is there to help you make it all possible.

Starting off the pack of currently enrolling trials is Anavex with their compound Anavex 2-73. Anavex 2-73 appears to be a versatile compound and may be effective in a variety of conditions. It also appears to have a good safety profile since it was first trialed back in 2014 and Anavex has multiple trials with it in various stages; the three  disorders currently being addressed are Rett syndrome, Alzheimer’s and Parkinson’s Disease Dementia.

Anavex has a great video that gives an overview of how Anavex 2-73 works and the end goals they hope to achieve with their compound in Rett syndrome. One of the main “targets” is neuronal growth, which is critical for our children because the nerve cells in the Rett brain have dendrites (fingerlike projections) that are a combination of too few and too small. If you think of the brain as a network of telephone wires, basically the wires have been cut and the brain can’t talk to the body like it needs to; it’s firing off all the right messages but they are getting lost in the gigantic space between telephone poles.

How are they to do this? They hope to provide the brain with homeostasis- “the tendency toward a relatively stable equilibrium between interdependent elements, especially as maintained by physiological processes.” I heard Dr. Kaminsky speak about it once. He said, and I paraphrase here, “Rett syndrome stresses out the brain and so it doesn’t function properly; Anavex 2-73 makes the brain happy and in that environment things tend to work better.” While certainly an over simplification of the process at hand, it does provide insight into the ultimate goal- a happy brain functions better.

I had to dig around a bit to get information about the potential in Rett syndrome based on findings in other trials. One very promising thing I found is it’s potential in controlling seizures. You can also read about it from their presentation at the 2016 Rett Syndrome Symposium. Just a couple of weeks ago, Anavex presented at the American Society for Experimental Neurotherapeutics (who knew that was a thing??) Dr. Kaufmann presented:

Longitudinal 148-Week Extension Study Of Anavex®2-73 For The Treatment Of Alzheimer’s Disease Demonstrates Maintained Activities Of Daily Living Score (ADCS-ADL) And Reduced Cognitive Decline (MMSE) For Patient Cohort On Higher Drug Concentration And Confirms Role Of Precision Medicine Patient Selection Biomarkers 

(For those of you who have not heard of Dr. Kaufmann, he is a juggernaut in the field of Rett syndrome research and one of the best doctors Katelin has ever had. He understands the need for wrap around services and a team approach to the challenges our children face. In his extensive career he has participated, in one facet or another, in almost every single Rett syndrome trial. Knowing that he is on Anavex’s team instills great confidence in me that we can place our children in their hands.)

The thing that jumps out at me is “reduced cognitive decline.” Whether this was achieved via a mechanism that would be helpful in Rett syndrome, I don’t know, however, I find it to be an optimistic observation. What else does Anavex 2-73 have going for it? It’s been given Orphan Drug Designation by the FDA; it is an oral medication; has demonstrated “favorable  safety  and  bioavailability” in the Alzheimer’s trial.

By this time, you’re probably thinking…Cut to the chase, already!

This trial is for adults, 18 and over. You can review all the criteria, goals etc. on The sites are:

United States, Alabama (recruiting)
UAB | The University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Principal Investigator: Alan Percy, MD

United States, Illinois (not recruiting yet)
Rush University Medical Center
Not yet recruiting
Chicago, Illinois, United States, 60612
Principal Investigator: Peter Heydemann, MD

United States, Ohio (recruiting)
Cincinnati Children’s Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Principal Investigator: Shannon Standridge, DO, MPH

For more information and to register interest in the trial you can go to:

For possible assistance with trial expenses contact: Colleen English, Rettland Foundation

They only need a total of 15 participants for this trial, which is 7 weeks long. They’re giving themselves until DECEMBER! We can do better than that. Let’s get this done!

This entry was posted in Anavex, Anavex 2-73, cure, Dr. Walter Kaufmann, FDA, gp2c, hope, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome conference 2016, Rettland Foundation,,, Trail to a Texas Trial, Uncategorized and tagged , , , , , , , , , , . Bookmark the permalink.

3 Responses to Rett Syndrome is Such a Trial… #1 Anavex 2-73

  1. Georgejjl says:

    The latest information from and about Anavex.

    Good luck and GOD bless,


  2. Georgejjl says:

    Last year’s 2018 presentation regarding preclinical (animal) eperiments

    ANAVEX®2‐73 as a Potential Treatment for Rett Syndrome and other Pediatric or Infantile Disorders with Seizure Pathology

    Good luck and GOD bless,


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