Wednesday, May 16, 2012


I got goosebumps a couple times last night.  And at least twice today.
Because of this news:
Pursuing the first Investigational New Drug (IND) for Rett syndrome:
Neuren Pharmaceuticals announces a program to the FDA
about a proposed Rett syndrome clinical trial.

Umm, yeah. Another possible clinical trial. It is kind of huge.  Gigantic.

Last night I attended the Samantha Corpus Golf Tournament dinner at a local country club thanks to a great friend.   A wonderful family in the area has been hosting this tournament for the past 7 years and have raised over a million dollars for IRSF. That is huge in and of itself. Huge.

Among some other very important people in attendance was non other than the new CSO for IRSF, Dr Steven Kaminsky.  He was there, in person to speak to the crowd-all the way from Maryland.  Whoa.  Also there, Barry Rinehart-he is currently on the Board of Trustees at IRSF and just happens to be the writer and director of the Silent Angels Documentary from 1999.  Very cool. If you havent seen it, you must. 

I was lucky enough to be introduced to these gentlemen and get to talk with them prior to the dinner.  Hearing Dr Kaminsky explain his thoughts on tackling Rett was thrilling-it gave me goosebumps. 

It is always thrilling to meet smart people-but it is a whole other kind of thrill when those people have no familial connection to Rett and are excited about figuring out a way to get this thing cured.  It blows my mind.  Really.

So, we got to sit with Dr Kaminsky and Mr Rinehart.  Dr Kaminsky spoke to the crowd.  He mentioned the above announcement-and we knew we were the first public crowd to hear anything about it.  goosebumps. He has a way of connecting to the crowd and making it interesting and understandable.  Basically, he told us this new drug has been used to treat mild traumatic brain injury and when used on rett mice-it reversed some of their symptoms.  You can read more about it HERE.  goosebumps.

The thought that there is currently a clinical HUMAN trial going on now and one more in the works for Rett syndrome is just mind blowing.  It has me laughing and crying at the same time. 

Some might ask: will it work for my daughter?  will it happen in time to help my daughter?  Sure, I am curious about both-but you know what?  It doesnt matter to me at this point. Really. Sure, I would love for this to be the "magic bullet" or part of a cocktail of medicines I could give to my daughter to make her able to lead a more typical life ( and it may very well be!)-but for now Im just so thrilled that the possibilities keep coming.  so do the goosebumps.


Zenaida said...

I am so with you Erica! I came home and I couldn't sleep thinking of the possibilities! This is happening. I can see it! I can feel it. I loved, loved his approach, his thinking behind how to tackle Rett syndrome.

Janie Beaumont said...

Thankyou so much for sharing this........It so good to hear such positive news... love to you & your family xx

Catriona said...

It's exciting! And look at you, getting to be right there in the middle of it! xx

Lindsay said...

I got goosebumps as I was reading this. I will continue to think good thoughts & pray for those smarty-pants who can find a cure for Rett!!!

Love you friend!

The MacDonald Family said...

Goosebumps here too!!!