A quick transcript of Dr. Hagerman's presentation. Sorry for any errors or omissions.
"Hi I’m Dr. Randi Hagerman,
I’m a Clinician, I’m a developmental and behavioral Pediatrician and I have a great interest in the use of targeted treatments for a variety of neurodevelopmental disorders. And again by targeted treatments, I mean medications that can modify the neurobiological changes that are made in the brain and individuals with neurodevelopmental disorders. I participated in the Adult Rett Syndrome Study using Anavex2-73.
The results are being released now and these are very exciting results.
I was blinded during the control trial where individuals were randomized either to active drug, about 5mg a day vs. placebos so I didn’t know who was on what.
I saw a number of individuals who did very well on this medication and others who didn’t but also I have been involved now in the follow up where they go on the active medication so I can see actually the results now in individuals that I know are on medication.
I saw a variety of improvements and again as you heard in the last talk from Jeff, these individuals are very severely impaired.
They can’t talk or talk very little, often they’re in wheel chairs. They can’t use their hands well . They often times can have agitation, other types of problems like constipation and what we saw is that individuals were calmer, less agitated, they seemed to be happier. They were able to concentrate better. They made better eye contact and they seemed to understand questions or directions given to them and were able to follow through in many cases on some of the commands that their parents made.
There was less anxiety, better understanding and in general the parents felt they were calmer and less agitated and often we saw improvements in the gastrointestinal problems.
One patient that stands out that I just recently saw, was able to swallow her food better. She didn’t have any problems choking now that she’s on the active medication.
She was able then to eat better, happier. She had less bloating, less constipation and having better function GI wise I think can lead to less agitation.
The families were able to mark on the main outcome measures that were improved. The Rett Syndrome Behavior Questionaire that the parents answered and also the Clinical Global Improvements Scale. The CGII that the Clinicians do. There were significant with the Anavex2-73.
So this is very exciting.. So far the patients want to continue on the Open Label Extension on this medication.
This is a relatively unique medication that can actually improve the calcium disregulation, the mitochondrial function, the oxidative stress and the neural connections. These problems can be seen in variety of other neural developmental disorders and also neurodegenerative disorders.
They’ve carried out studies in Alzheimer’s and in Parkinson’s Disease looking at cognition in Parkinson’s disease. It improved cognition in both Alzheimer’s and Parkinson’s disease.
I do a lot of research also in Fragile X syndrome and this medication can improve the Fragile X symptoms in the mouse model of Fragile X so I’m very excited about this medication causing improvements not just in Rett Syndrome but in other neurodevelopmental disorders and neurodegenerative disorders.
This medication can be a boon. I think in general for aging and certainly for aging syndromes like Parkinson’s Disease and Alzheimer’s and I can’t wait to start it on other neurodevelopmental disorders like Fragile X Syndrome.
I am very excited about this medication and also as I age I would definitely like to take Anavex myself.
I’m excited that this is a break through medication and for not just Rett Syndrome but other disorders of aging and also neuro-degeneration.
This is an exciting new treatment."
