Phase III: Dexpramipexole

[StillSteve]

Good to hear that you have good things to say about Biogen, Alyoop. Seems like they are pretty confident about this drug, and hope to get it to market soon.

The answer to the question I got privately, "Why not 'til August?" is contained in the overlong update I tried to post on another thread here (it is stuck in moderation limbo; I dunno why).

The short version is that I chose to start on riluzole Friday and have been told that it is almost certain that a spot will still be available to me after the 60-day wait that will be required because of this. The long version (virtually identical to what I posted here) is available on the Patients Like Me site.

 

[SueG]

Randy, both expert researchers (one of whom is the PI for the whole trial) I've talked with say this is by far the most promising drug yet-- not even in the same league as riluzole-- and so if you can swing the long days to Minneapolis, I'd encourage you. Mass General has arranged a home nurse visit program for alternate months so we only have to go to Boston (3 hrs @ way) every other month. I wonder of they're doing that where you are? I have the nurse coming on Tuesday for the one-month visit (blood draw and vitals) and then will do the rest by phone/email with the study coordinator.
Good luck!
Sue

 

[Happyphysicist]

By "not even in the same league as riluzole" do you mean that Dex is in a better league than riluzole or the other way around?

 

[HelenL]

From what I recall at Mass General while going through the preliminary workup, this is a mirror image of a drug already approved for MS patients (sorry, don't know the name), and yes, I would say that its in a "better league" than Riluzole. I've been waiting for this drug trial to start since last fall when my neuro at Beth Israel in Boston told me about it and that he considered it to be one of the more exciting breakthroughs. (And he was the neuro who won a million dollar prize for an invention to hopefully replace emgs in testing)

I'm going in on Thursday this week for my first "official" workup and then start the drug... hopefully! I guess it will be hard to tell if you have the placebo or not, as there are very few side effects of the drug. Hopefully I'll be able to tell I'm getting it when I see slower progression! But I would highly recommend that you get in the study if you can, as even if you don't get the drug during the double blind stage, at the end of the 18 months total (depending on when you start your time will be less) ALL the participants still in the study will then be eligible to get the drug during an "open phase". I understand that many of the people in phase 2 are still on the drug now for that reason.

Good luck everyone, Sue, I'll be pm'ing you to compare notes on this!

 

[frankb]

For rmsooo: Emory ALS Center is gearing up for the Dexpramipexole study. The staff is universally professionsal, efficient, and they answer e-mails promptly. I can't praise them too highly./Frankb

 

[Chris_b]

Got appointment for next week for day 1 of the study as my screening visit tests came back satisfactory

 

[HelenL]

Chris, that's awesome news! I started the trial today... hopefully got the real stuff and not the placebo, but I guess only time will tell! Let's compare notes when you get started on in the trial.
Helen

 

[rms000]

Thank you, Frankb. They have put my mom on the list to be considered. I hope to hear from them soon

 

[Chris_b]

Started the trial today so glad to get the chance. Lets hope it turns out to be something special for the future

 

[SueG]

Good luck, Chris & Helen!
In my first month of the trial I lost 5 lbs (a fair amount for little me), but that could be due to lots of things including that chewing is getting harder. Been working to turn it around-- butter on everything!

Sue

 

[twinmommie72]

I started yesterday. Praying it's the real stuff!

 

[Alyoop]

Good luck to you all, hope everything goes ok and always remember, you are all pioneers and heroes. If not for people like you we would have no medication.

Bless you all
Aly

 

[HelenL]

I don't feel like a hero, those are really the folks who take this in the first 2 phases, where much more is unknown about the meds. Not to mention the people who dedicate their lives to finding cures for all diseases, not just the ones we're lucky enough to have.

 

[Alyoop]

Hey Helen phase 1 people are usually paid big bucks to take the risk. Researchers get kudos and research grants ( they care as well), you guys do a lot of hard work. If I think you are heroes then you are. after all I am never wrong lol lol
Aly

 

[tdamess]

i have a question once trial 3 is done how much longer till fda approves it a then pals all pals can get it