C9orf72 Gene Therapy Targeting Trial UK - Phase 1

[Ellie]

This is a big deal for some of our (favourite ) Forum Members.

Phase 1 of its clinical trial started this week, so very early days, but it’ll be very interesting to see the trial results when published.

https://mndresearch.blog/2019/09/19/...ial-in-the-uk/

Love Ellie.

[Onein300]

This is early days but this along with the biogen SOD1 Trial are in my mind the key trials ongoing in the world today.

Rock on!

[Lynne K]

Thanks Ellie. I read all about it previously but forgot. Appreciate the reminder. I read it again. There's other research going on but no other to my knowledge for C9ORF72 in the world. I get the ALS news letters.

I hope that the participants of this trial stay safe and the scientists can learn from it. Obviously I hope that it's the first of 3 associated trials and that it turns out to be a useful treatment but I'm keeping my feet firmly planted. Lynne

[johnburton]

I hope to get on the Phase 1 trial at the National Hospital for Neurology in central London. I was phoned by one of the doctors there yesterday. I plan to visit them in the next few weeks for an informal chat. This is indeed a big deal for those of us with the C9orf72 mutation. They are running 6 cohorts with 8 people in each. Cohort 1 is given a very low dose of drug, Cohort 2 gets a higher dose and so on. The cohorts run in series. The aim, as I understand it, is to assess people's tolerance to the drug.

[Lynne K]

Good luck John. I hope that it doesn't make you feel ill. Take care. Lynne

[Ellie]

Fingers cossed that you get on the trial John and it goes well.

Love Ellie.

[Gillette]

Exciting times! I hope you are, indeed, taken onto the trial, John.

[johnburton]

Thank you, all. I'll post updates.