NNZ-2566 success:  Breaking it down and answering one common question:  “What is this drug supposed to do for girls and women with Rett syndrome?”

November 13, 2014


It’s been an incredible week for the Rett syndrome community with the announcement from Neuren Pharmaceuticals about its successful phase 2 NNZ-2566 Clinical Trial.

We wanted to take the time to answer one common question:

“What is this drug supposed to do for girls and women with Rett syndrome?”

This was reason for the clinical trial. 

Going into the trial we didn’t know the answer to the question. Before we get into the specifics, we need to understand that researchers learned a lot from the preclinical animal studies of NNZ-2566.

It has been extensively tested in traumatic brain injury (TBI) animal models and in MeCP2 mutant mice. In both the TBI animal models and the MeCP2 mouse models the animal showed improvements in motor function (ability to move normally) and in learning (behavioral tests in animals).
NNZ-2566 in the animal studies leads to improvement in the histopathology associated with Rett syndrome.

What does that mean? In Rett syndrome, the nerve cells have fewer connections to other nerve cells. When NNZ-2566 is used with the Rett animal model, more connections are seen.

Watch: Dr. Steven Kaminsky, CSO, breaks down the results


What does this all mean for girls and women with Rett syndrome?

More nerve connections mean more potential brain circuits.

Brain circuits are what control our every movement and every thought. So more circuits are a “good thing” that means more control of movement and thoughts learned through experience.

If the drug can help build more nerve connections the central nervous system can move toward more normal function.

What will that mean for girls and women with Rett syndrome?

A Rett patient will have more circuits to help with learning, moving, and experiencing everyday life. These circuits are what each of us uses to remember, to control actions, to speak, to move, but more about this later.

Let’s go back to the clinical trial goals and results:

The first goal of the study:
Is NNZ-2566 safe and tolerated in Rett patients?

The answer:

With the two doses of NNZ-2566, it was safe and tolerated during the 28 day course of dosing.

The second goal of the study:
Is NNZ-2566 effective in changing any of the Rett symptoms over the course of the trial?

To measure this, the study team looked at various behavior measures and two physiological measures. The behavior measures are done on individual patient basis because everyone’s behaviors are different. So each patient was studied for behavior patterns before the study and then during and after the administration of the drug and placebo.

The answer:

At the high dose, the behaviors changed (improved) from the placebo control.

Behaviors in each patient are different so to say one particular behavior changed would be incorrect because no two persons’ behaviors are exactly the same.

The physiological measures also showed changes and trended toward improvement, but that is still being analyzed.

And now back to the central question, in greater detail, “what is the drug supposed to do for girls and women with Rett syndrome?” 

The most important thing to remember is no one knows how this all works other than the nerve connections are a necessary part of everything we do. If NNZ-2566 increases the number of nerve cell connections, then each patient in the trial may exhibit different progressions in their everyday life.

However, each patient may not progress at the same rate because of individual to individual difference in experiences and how those experiences are stored in the neural connections.

The goal of the study and future NNZ-2566 studies is to correct Rett biology! The nerve connections in Rett are fewer than normal as stated above. This drug in its actions helps to form more connections. By forming more connections we are trying to normalize the Rett biology. If the Rett biology can be normalized, then the nervous system has greater potential for learning, motor function and all the other things the central nervous system does.
It is important to know this drug is not designed to specifically treat one of the symptoms of Rett, i.e. seizures, G.I. tract disturbances, hand movement, etc. But rather it is designed to correct the deficiency in nerve connections globally in Rett.

If the number of connections can be improved, then there is the possibility that those symptoms will be improved by having a greater neural network to stabilize the entire central nervous system.

And finally, we wouldn’t have this wonderful news to share without our heroes, the girls and families participating in the trial, and the doctors and their teams at Baylor College of Medicine, University of Alabama-Birmingham, and Gillette Specialty Healthcare performing the study alongside with Neuren Pharmaceuticals. 

Thanks to all for the outpouring of messages that come into Rettsyndrome.org via different channels, i.e. emails, Facebook, Twitter, phone calls…

We have filled our tanks with your support to advance research and treatments for Rett syndrome.

Your donations are what enabled Rettsyndrome.org to fund these trials, which will deliver meaningful changes to her quality of life. We know that without all your amazing hard work, this would not be happening. We thank you from the bottom of our hearts and we hope that you will continue to be part of our ongoing success.

We have many ways to get involved in with Rettsyndrome.org and we appreciate every single effort.

Alternatively you can donate now to fund the future.

THANK YOU for making the NNZ-2566 clinical trial happen!

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