Efficacy of rintatolimod (Ampligen) in the treatment of cfs/me

[Remy]

Expert Rev Clin Pharmacol. 2016 Apr 5. [Epub ahead of print]
Efficacy of rintatolimod in the treatment of chronic fatigue syndrome/ myalgic encephalomyelitis (cfs/me).

Mitchell WM1.
Author information


Abstract

Chronic fatigue syndrome/ Myalgic encephalomyelitis (CFS/ME) is a poorly understood seriously debilitating disorder in which disabling fatigue is an universal symptom in combination with a variety of variable symptoms. The only drug in advanced clinical development is rintatolimod, a mismatched double stranded polymer of RNA (dsRNA). Rintatolimod is a restricted Toll-Like Receptor 3 (TLR3) agonist lacking activation of other primary cellular inducers of innate immunity (e.g.- cytosolic helicases). Rintatolimod also activates interferon induced proteins that require dsRNA for activity (e.g.- 2'-5' adenylate synthetase, protein kinase R).

Rintatolimod has achieved statistically significant improvements in primary endpoints in Phase II and Phase III double-blind, randomized, placebo-controlled clinical trials with a generally well tolerated safety profile and supported by open-label trials in the United States and Europe. The chemistry, mechanism of action, clinical trial data, and current regulatory status of rintatolimod for CFS/ME including current evidence for etiology of the syndrome are reviewed.
KEYWORDS:

Ampligen; Chronic Fatigue/Myalgic Encephalomyelitis; TLR3 agonist; clinical efficacy; clinical safety; clinical trials; dsRNA; primate/non-primate disassociation of toxicity; rintatolimod

 

[Who Me?]

Sign me up! And find me a sugar daddy to pay for it.

 

[IrisRV]

Ampligen has always been at the top of my wishlist. I even went to Hunter-Hopkins soon after my initial diagnosis to try to get Ampligen. What I didn't know then is that I had to live near the clinic to get Ampligen.

The problem I see currently is that the FDA isn't going to approve Ampligen the way Hemispherix is (not) jumping through the right hoops. Hemispherix is not going to let go of the patent. So we're all screwed.

Ampligen has been in existence a long time. It appears to be safer than other things used in ME/CFS. It gives "statistically significant improvements in primary endpoints." And we can't get it for political/administrative/business reasons.

I keep hoping, but it's no longer anything more than a vague hope. I hope to be rich someday too, but that's not going to happen either.

Does anyone have an idea if this publication is likely to have any impact whatsoever on the accessibility of Ampligen?

 

[Cort]

Good timing - I have an interview with the new Prez coming up. He said Hemispherx is totally focused on bringing Ampligen to market.

 

[IrisRV]

Good timing - I have an interview with the new Prez coming up. He said Hemispherx is totally focused on bringing Ampligen to market.

That's great news! I hope you will ask him how long he thinks that will take. I'm assuming they'll have to do the testing the FDA wanted, which will take a few years. Then it has to get in line to be approved (hopefully) by the FDA. Lord knows how long that will take. Am I likely to be dead before Ampligen is available to a Joe Shmoe in the middle of nowhere?

 

[Strike me lucky]

I hope they find an easier way to administer it so it can be done at home.

 

[IrisRV]

I hope they find an easier way to administer it so it can be done at home.

Hey, I'd be happy if I had to go to the local hospital for an infusion at this point. Anything to get it to those of us not within an hour of the few places it's currently available. It would be nice if there was a home version, though.

 

[Who Me?]

I hope they find an easier way to administer it so it can be done at home.

Like Hizentra.

 

[Cort]

That's great news! I hope you will ask him how long he thinks that will take. I'm assuming they'll have to do the testing the FDA wanted, which will take a few years. Then it has to get in line to be approved (hopefully) by the FDA. Lord knows how long that will take. Am I likely to be dead before Ampligen is available to a Joe Shmoe in the middle of nowhere?

It really is a new day for them...They recognize they have a real opportunity with the NIH opening up and they don't want to miss it. On the other hand their finances are in the pits. The new guy is coming in it sounds like to show investors that Hemispherx has got its act together and can be trusted. It seemed pretty clear that they need investors...

Actually if they get a trial started it might not take that long at all....They have the doctors lined up...They have some results from past trials. I imagine the FDA would fast-track their end of it.I think it could go pretty fast - I know that's a relative term in the medical field - if things worked out.

 

[Who Me?]

It still requires someone to rx it. There in lies the problem for lots of us.

 

[IrisRV]

It still requires someone to rx it. There in lies the problem for lots of us.

I'm hoping, at least as an intermediate step, that ME/CFS specialists could prescribe it, but it could be administered locally. I realize that's still a problem for patients who can't get to a specialist at least once a year.