Norway funds Major Rituximab Trial
After a torrent of protest six months ago at the Norwegian Research Council’s (NRC) failure to fund a Rituximab trial, the NRC said they would revisit the issue. This time they got it right, agreeing to provide funding for a big Phase 3 multi-center Rituximab trial in chronic fatigue syndrome (ME/CFS). It’s not clear how much of the approximately $7,000,000 of their funding announcement, is going to go to the Rituximab trial, but with Fluge/Mella requesting between $1.1 – 1.7 million dollars (US), and the Council agreeing to fund it, it’s hard to imagine the council not providing enough to support it.
(Phase 2 trials assess safety and efficacy in a small group. Phase III trials do so in a group large enough to fully assess safety and efficacy. )
The NRC’s commitment to producing a Phase III trial could produce a breakthrough in our understanding of this disorder
Norway’s Health and Human Services Minister, Jonas Gahr Støre, asked the research council to give ME/CFS applications priority, and they did, also funding a study on the risk factors and long term consequences of a Giardia outbreak that produced many ME/CFS/IBS patients . With two out of the ten funded studies focused on ME/CFS, the NRC was clearly in a supportive this time around – a real testament to the hard work of Norway’s ME/CFS advocates.
The remarkable response to Dr. Maria Gerpe’s ninety-day crowd-sourcing effort that raised a $430,000 no doubt helped the NRC to think again about their miserly approach. With the NRC’s commitment and Maria’s funding in the bag, Fluge/Mella will be able to start the trial soon, long before the NRC’s funding becomes available in January of next year. That’s a good thing given the trial may take up to two years.
That’s a lot of money and this trial, in particular, with an expensive drug is going to be costly Maria reported that almost all the money for the trial will go to pay for the drug and the Fluge/Mella will be contributing their time free. That’s an enormous amount of pro-bono time in a trial this size.
According to accounts on the internet, the trial will include about 140 patients
Cautionary Notes
Some notes of caution are in order. Several doctors I’ve talked to are taking a wait and see approach. While some of Dr. Kogelnik’s patients are, for instance, are getting the same very positive results seen in the Norway trial, he didn’t seem anywhere near suggesting this was the answer for ME/CFS.
There is concern about the ‘first study’ effect which results in really positive results in carefully selected patients in a first trial and then much less positive results in bigger, later trials. We saw this happen when the extraordinary results of Dr. Montoya’s preliminary Valcyte trial turned to quite underwhelming results in a larger, more rigorous trial. Just as in the Rituximab trial, several participants returned to full health in the first trial.
Fluge/Mella are ahead of Montoya in that their Phase II trial was successful (Montoya’s was not but Phase III trials, with their large numbers of patients spread out across several centers, are the testing point. How important duration of illness is in determining how responsive patients are to the drug will be very interesting given reports from several physicians that longer duration patients are more difficult to treat and some research evidence suggesting that the disorder does change over time.
Importantly, both Kogelnik and Olav/Mella are engaged in trying to figure out why one person benefits while another does not.
David and Goliath II: David Wins Again
The leader in drug development for ME/CFS? Not the US – Norway….
Big things can happen in small places. Norway is about the size of New Mexico. By the time this trial is though the NRC may very well contribute several million dollars to ME/CFS research. Contrast that with the four or so million dollars the 28 billion dollar National Institutes of Health in the US can bring itself to spend on ME/CFS.
Let’s put this in the context of Ampligen – a drug that’s been much better studied in ME/CFS, and shows benefits, and, of course, would treat a disorder which has no drugs approved for it….and yet is left to sink or swim in the marketplace. There’s something gravely wrong with the US approach that says a drug is only of interest if a drug company will provide X millions of dollars to study it. One wonders if the NIH/FDA has any second thoughts when a country the size of Norway does what the NIH/FDA cannot do; funds a clinical trial on a promising drug into a disorder the FDA says is serious, has huge unmet needs, and is a priority for them. Who’s doing for ME/CFS?
Other Trials
Work on at least three Rituximab trials for ME/CFS appears to be underway; the Fluge/Mella Norway trial, the OMI-Merit trial in the US and an Invest in ME trial in the UK.
Conclusions
This is a big, big deal. One of the most promising drugs ever for ME/CFS is going to get a full-scale trial and that pretty much means irrefutable evidence either for or plus Rituximab. A Rituximab trial was priority #1 on OMI-MERIT’s list of groundbreaking projects and to see it happen, in little Norway, of all places, is a head-shaker.
Significant numbers of responders would open up a much needed new treatment option, send immune researchers scrambling to figure out what’s going on and redefine how this disorder is researched, treated and funded.
Cracking article, thank you. Interesting too that others might not be getting the same results as the original Rituximab study, though Fluge & Mella are about to publish a slightly larger, open-label trial, n=28
You’ve always got the inside scoop Simon :). Looking forward to that study…Fluge/Mella’s preliminary studies are much more rigorous than Montoya’s – that’s good news..
Hi, Simon, Can you add anything else? Are you saying that their study is about to be published, or to be submitted for publishing? Also, do you know anything about their results? Any interviews they’ve done or hints they’ve given?
>Importantly, both Kogelnik and Olav/Mella are engaged in trying to figure out why one person benefits while another does not.
I wonder if they have considered the extent to which people are living in a toxic environment as possibly relevant here.
Great article! Made it clear to me the scope of what Norway and its researchers have accomplished by persistance. Letting them do a Phase III trial! Amazing.
I am always glad of the people who are treated and improve or even get cured in Phase II trials – every person helped counts for real. One more life improved, and that is the right direction.
One small question: Did Dr Gerpe raise $43,000 or $430,000?
Sarah
It’s amazing but that’s what Maria’s blog said –
What an amazingly successful effort.
Great article, Cort! I couldn’t stop smiling yesterday after I heard the news.
It’s worth noting that, even with this additional Norwegian government grant, the trial isn’t yet fully funded. The researchers didn’t want to apply for the full shortfall because they didn’t think that an application for such a large amount would be successful, so even though we don’t know the amount of the grant yet, we know it won’t be enough to fully fund the trial. But it is enough to give Drs Fluge & Mella enough confidence about funding to begin the trial immediately.
To make further donations, go here and scroll down for instructions for overseas donations:
http://www.meyou.no/donate-doner/
Even though Maria will shortly be standing down after her 90-day campaign, the accounts will still stay open for donations.
Please also sign this e-card thanking Maria for what she has done for us all:
http://www.groupcard.com/c/zMGag9wWEjE
As a severe ME sufferer in Scotland, UK for over two decades(and we know the “treatment” advocated here….)I am so proud that my son having chosen to live in Norway(after twice going on a student exchange there,and seeing the Norwegian attitude to life in general),that this small country has taken such a big step.
As I say, hearing so much from my son re. the “modus operandi” of Norway`s people and government,I feel this is somewhere we sufferers can have faith in the integrity of the scenario which will unfold.
Yes,I suppose it gives me(and hopefully others)HOPE!.After 22years of an “existence”,hope can literally keep you going.
God bless Norway and Drs Fluge and Mella!
This drug is very dangerous and toxic. It is very important to find which patiënts will benefit with objective markers. I think ”only” 30% will benefit from this medicine. I am a little sceptical because ME is primary not an auto-immmune disease. If you read the papers it does not fit. There is more. But time will tell us. We wait and wait and wait and wait 🙂 I think that primary the ‘stressmechanisme’ is defect or changed which lead to all problems we see.
As I noted some US doctors are being quite cautious. Let’s be conservative, cut the 65% in half and say it’s a lifechanger in 10-20% of patients overalll. That’s still a huge chunk people getting their life back if they can get access to this drug.
It’s also the opportunity to weed a significant portion of patients who respond out of research studies (eventually)…I think 30% response rate would be really good, actually. That’s alot of people right there.
Cort,
I agree with you that’s it’s great if even a small proportion of us get their lives back. But that’s not all, because that result can help point us to underlying mechanisms that could help others as well.
Thanks for this thread, I haven’t heard from you since before the London conference, which Mark from Phoenix Rising covered so well, but I was wondering if you’ve written anything on that also?
Regards,
Nothing on the London Conference from me unfortunately…Too far for me to travel. Mark did a good job with that.
This is excellent news and very exciting. Just to correct you on the subject of “little Norway”. It may be small but you should appreciate it is hugely wealthy due to oil and gas exports. Norway is the fifth largest oil exporter and third largest gas exporter in the world. For the last 10 years Norway has generated a large balance of payments surplus each year. So it is no surprise at all that the government could afford to fund this study. We are fortunate that Fluge and Mella live in Norway rather than somewhere else.
Tony thanks for the info. I had no idea Norway has a surplus balance of payments each year; that’s amazing given what’s happened economically over the last five years.
These trials are kind of scary to me. They’re necessary to advance treatment but I can’t help but think that there are going to be some casualties based on the anecdotal reports on the Phoenix Rising forums. Apparently if a person is a responder to Rituximab then it works good but it can also apparently jack you up pretty good if you’re not a responder.
I’m a patient of Dr Kogelnik’s in California and received my fifth infusion of Rituxan last month. I have been deemed a responder because I have experienced *some* improvement. However, I was not one of the fabled few who miraculously recovered by week 30. I still have hope that I will experience further improvements on Rituxan but it’s still disappointing. I took a gap year after graduating high school to take Rituxan and am now well enough to study part-time at my university this fall, so that’s somethung. Through Kogelnik, I have also given blood and bone marrow samples and am part of a CDC study. So that’s my story. Exciting developments, and thank you for the article.
Thanks for letting us know Hannah 🙂 and good luck with it. I imagine there are many different ways to respond and who knows – maybe things will kick in. Good luck with the part-time study – that’s good news…
Please keep us informed..
Hannah: I, too, wish you well with the Rituxan. That’s great that you can start your college. Take care. Joy