From Trail to a Texas Trial Trofinetide #1 So, we are narrowing...

From Trail to a Texas Trial

Trofinetide #1

So, we are narrowing in on the start of Phase III of Trofinetide. It’s a word that causes caution, skepticism, hope, giddiness and a myriad of other emotions. Even though I am completely convinced of it’s effectiveness, how it affects each person is still unknown. That’s the scary, yet tremendously exciting unknown. But what IS it.

This is Trofinetide. (read Neuren’s indepth description)

all following excerpts are taken from the above page.

“Trofinetide is the name proposed by the World Health Organisation for our lead clinical-stage drug candidate (previously designated by Neuren as NNZ-2566). It is an analog of a molecule which is derived from IGF-1 and occurs naturally in the brain. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease.”

So, here we have the IGF-1 protein showing the portion of it that was modified and comprises Trofinetide.

Trofinetide (initially known as NNZ-2566 was discovered by Margaret Brimble’s lab. Margaret Brimble is a world renown researcher with multiple awards under her belt, lest you think this is her only achievement. Not by a long shot!

But, let’s cut to the chase, what does it actually do?

When speaking to someone about Trofinetide, I usually use this visual…take your hands, open them and hold them close so the finger tips are almost touching-that’s the normal brain, before Rett syndrome. The space between is called the “synaptic space”, the distance a signal has to jump from one nerve cell to another. Now, make a fist. That space is now HUGE and not only that, the “fingers” are gone. This is the Rett brain. Now, reopen your hands, that’s what Trofinetide does. But here you go, from Neuren’s site.

This is surely simplified, even I know that. There were so MANY hurdles to overcome. One of the main ones-breaking the blood/brain barrier, I’m not a chemist so I doubt if I would comprehend the fundamental basics of how it was done, but since I’m not a chemist, I don’t care. (Sorry, Margaret) But, if a picture is worth a thousand words, this picture will one day be worth a million words and more.

Next week… why were Rett syndrome and Fragile X chosen?

http://www.neurenpharma.com/irm/content/the-science-behind-neuren-s-products1.aspx?RID=304