Potential First-in-Class Therapy for Spinocerebellar Ataxia


Thanks Alan🙂 So, this is a potential treatment specifically for
Spinocerebellar Ataxia itself? Something we’ve all been hoping for.

I’ve read the link but am unable to find out what it actually does🤔xB

Thanks for the info Alan. Like Beryl says I wonder if this news could be the one.
I certainly don’t want to get ahead of myself but you can only live and hope

Hi. My daughter starts the clinical trial on Friday at UCLA. Sounds very hopeful. Her diagnosis is SCA8.


Hope all goes well Cynthia

What this trial is about? Can you share more info? Thanks!

Hi. This is a trial for a drug by Biohaven called BHV 4157. Just google it. It is to replace the glutamine in the brain. It has been put on the fast track by the FDA. The doctor at ucla is very excited about this. Look up clinical trials they might still be looking for candidates for phase 2. it might help people with certain heriditary ataxias. We go Friday for placebo or real drug. Then in the 3rd phase we get the real drug. I hope this is the answer

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Hi Cynthiafinnell, I am going through phase 2 now and will get the real thing June 2. I am almost positive I am on a Placebo pill now and can not wait for the real thing. My condition is getting worse! Not to mention with all the falls I am doing, my back is in also bad shape. Will keep everyone posted.

Hello, do you Cynthia, or anyone happen to know, if the results from this trial are good, when will the drug be available to patients? I mean so that the doctor could prescribe it to a patient?

I know they have to do this trial first before the FDA will approve this. This is on a fast track and if all goes well it will be available 2018. i am unsure what ataxias it will work on if it works. Just came from ucla today with the pills. Fingers crossed.

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Hi Henry. I know it’s only been a few days but how are you doing on the mess?

Hi Henry. From the previous post I mean medicine not mess. Lol

Hi Cynthia, sorry for the delay. You had it correct the first time - what a mess! Can you believe they ran out of the real stuff (BHV-1547)? I have to wa

Damn, fat fingers! I was saying I have to wait for pills to be delivered to my house! I hope to get them soon because I can hardly get around. Will update you when at least I am on it for 1 month.

Wow. What a bummer. What state are you in?

I am located in Madison NJ (look at profile) but the trial is at Columbia University NY. Just found out that I will finally get pills delivered to my house next week (6/14/17). CAN NOT WAIT!

That’s awesome. So I assume you were on a placebo. My daughter seems to be improving but maybe it’s my imagination. My daughter said I was on a placebo. Lol. She seems to be more active and happier. We will find out soon enough.

How is it going Henry. I hope you are feeling better.

Hi Cynthiafinnell,

Just completed 7 days on BHV-4157 and have not noticed anything new. I’d love to hear how your daughter is coping!

Hi Henry. Her spirits seem way more positive. I don’t know if this is because there may be a treatment for her sca8 or if it really is working. She just finished 4 weeks and saw the doctor last Thursday. Feeling hopeful. :blush: