Here is a great article on Phase 1 of the IGF-1 study that has been going on at Boston Children’s Hospital. IGF-1 Phase 1 see also http://medicalxpress.com/news/2014-03-mecasermin-rh-igf-treatment-rett-syndrome.html
But for those not familiar with it-
IGF-1 stands for Insulin Like Growth Factor 1
It shows promise in helping to address some of the symptoms of Rett Syndrome and alleviate them.
It cannot be given to post-puberty children and women, as explained to me, since it is a growth factor it could cause some bones to grow (particularly in the face/jaw) and cause deformities and other issues.
It is given as an injection.
Some difference between IGF-1 and NNZ-2566 are
NNZ-2566 is a modified portion of IGF-1
NNZ-2566 can be given to post-puberty children and women
NNZ-2566 is an oral solution vs an injection
Here is a brief summary about NNZ-2566 compared to IGF-1 taken from Neuren Pharmaceuticals-
NNZ-2566 is a synthetic analogue of a naturally occurring neuropeptide derived from IGF-1, a growth factor produced by brain cells. In animal models, NNZ-2566 inhibits neuroinflammation and normalises the function of microglia with consequent improvements in molecular, cellular, anatomic and behavioural outcomes. Click here for the full article– http://www.neurenpharma.com/ssl/cms/files_cms/486_131001FRAXA%20meeting.pdf
One thing these trials need is Participants! Clinical trials are very costly….for PARENTS. Often, they have to stay away from home for long periods of time or make frequent flights to the designated study locations. Please help as you are able and donate to Rettland.org, helping parents help their children and others by assisting them in the costs of hotel fees and airfare.
I think of all of these studies and it’s like- at first hope for a cure could only be seen with a telescope, and then binoculars and now I just need to shield my eyes from the sun and I can see it on the horizon. Thank you to all the researchers and families who participate and make the sacrifice to bring hope to our girls and boys and a possibly normal life to those who have yet come to be.