Breakthough! Norway Wakes Up – Funds Big Multicenter Chronic Fatigue Syndrome Rituximab Trial

by Cort Johnson | Jun 7, 2013 | Rituximab | 18 comments

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.

18 Comments