[medicalxpress.com]

Reading the article now! This is a big deal, folks!!!! It's a continuation of the phosphorylation research that I covered two years ago, the research by Dr. William Yang and Dr. Joan Steffan that Nancy Wexler was so excited about.

I'll provide a link when I get the update finished.

Thanks for posting this. It's very exciting and I'm looking forward to reading Marsha's update.

Patty

I google hd pretty much everyday and saw this. Nice Valentine's present. Another path of possibilities!!

Marsha-

Because this has an already good safety profile, any sense of how quickly Phase 1 trials would begin?

Also, do you have any insight on whether this group plans to test in primates before moving to humal trials?

thanks!!

Orzoro, thanks for posting this! Also thanks to Marsha for commenting. I LOVE to hear promising treatment news! This is the best Valentine's Day present I could ask for!!!

Still working on reading the articles and other supporting articles so I can relate all the important stuff. I expect to have the update posted tomorrow morning.

According to Dr. Sipione, the lead investigator, clinical trials could begin in one to two years. There is no need for primate studies since GM1 has already been used in a pilot study back in 2002 with AD patients. There's a five year study done in PD which resulted in a good safety profile as well but I'm not sure if that was oral administration or infusion.

Two caveats:

1) GM1 doesn't efficiently cross the blood brain barrier so it would have to be administered into the ventricles of the brain through a Medtronics pump.

2) Two years ago, Dr. Joan Steffan raised the possibility that a treatment to phosphorylate serines 13 and 16 might need to be an early treatment because after the protein clearing mechanisms of brain cells are no longer functioning at top efficiency, it could potentially make things worse by piling up even more HD protein in the nucleus of the cell. I've written to Dr. Yang who was going to look at this in older HD mice to see if he has any data (I haven't seen any published).

The mice in the current study are five month old YAC128 mice. They have fairly severe motor symptoms but no neurodegeneration so it wasn't a really early treatment but not a later one either. As I said, I'm trying to find out about any data supporting or not supporting Dr. Steffan's hypothesis.

The good news here is that this is the FIRST HD treatment in which the mice got better. We have treatments which postpone onset and delay progression in the mice but this is the FIRST to improve them, reversing their symptoms.

Wow, this is DEFINITELY interesting...look forward to your article Marsha!

Marsha,
Thank you for your information.
Paula

This is great news! HDbuzz also covered it the other day: [hdbuzz.net]

Look forward to hearing more Marsha!

Matt

[www.hdyo.org]

Still working. Have heard back from Dr. Yang and Dr. Truant re questions that I had. Our researchers are wonderful!.

Okay, my update has been posted on the HDSA website. I wanted to get as much information as I could to share with you about what is going on with this research and it took a while.

[www.hdsa.org]

Thank you Melissa for your follow-up. This is indeed so exciting. I guess though I couldn't get if indeed there is still concern on the latter stages, or this is still yet to be worked out.

It feels good to see such great commitment out there from the researchers and loved that they said "jumped for joy". Please let them know who much that means. Please keep us posted.

It's still to be worked out whether this would work in the later stages of the disease. The concern raised was based on cell model and fruitfly research and there might be no problem in mammals. I am encouraged that the YAC128 mice were five months old and the BAC mice were one year old but I think we have to look at older mice. I am going to advocate for research funding for this.

I'm sorry I meant Marsha. I am new to still sight. Thanks again

So....Marsha, can you honestly say if this worked what sort of time frame we would be looking at before it is fully approved? We are getting to the point for my hubby that he will probably start showing signs REALLY soon. Even a couple of years will make a difference for us. Do you think it will be in time for him?