Rituximab - A Promising Treatment for ME/CFS

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I have been watching and reading the ground-breaking research on using a cancer drug, rituximab, for ME/CFS with great interest and wanted to provide a brief update here for those who haven't heard about it yet or who want more information (or, as always here, a simple explanation).

Rituximab is a drug that affects the immune system, used for patients with lymphoma, a form of blood cancer, and also for autoimmune conditions like Rheumatoid Arthritis (RA). There is now growing evidence that it might work for a majority of ME/CFS patients, either greatly improving their symptoms or even providing complete remission.

A simplified timeline of what's gone on so far:

1. In 2004, two Norwegian doctors using rituximab for a group of lymphoma patients noticed that one patient - who also happened to have ME/CFS - was not only cured of his/her lymphoma but that all of his/her ME/CFS symptoms had disappeared as well.
2. Excited by this unexpected occurrence, those same two Norwegian doctors conducted a small, 1-year study of 15 people with ME/CFS in 2011. A full two-thirds of the patients (10), experienced significant improvement on rituximab. Two of those 10 seemed to be completely recovered, even 3 years after their initial doses, and were back at work.
3. In July 2015, results were published from a larger Norwegian study of rituximab in 29 ME/CFS patients, showing that once again, almost two-thirds of the patients (18) responded well and improved significantly, some achieving full remission. Eleven of the 18 who responded were still in remission three years after beginning the treatment, and some have not had symptoms for five years. This study included a variety of ME/CFS patients, ranging from moderate to severe (bedbound).
4. A much larger study, of 150 ME/CFS patients, is now underway in Norway and includes a control group. Results are due to be published around 2017-18.

Can you believe those facts?? Two-thirds significantly better? Complete remission? These are words that many ME/CFS patients never thought they'd hear.

So, how does this miracle drug work?
Rituximab, a cancer drug, wipes out most of the body's B-cells, a kind of white blood cell that makes antibodies. In the ME/CFS trials, the doctors have generally seen a 4-6 month lag in response time after the first dose is administered, which is about the length of time it would take for existing antibodies to be cleared from the body. Patients showed signs of relapse at about 1 year - approximately the amount of time it would take for the body to make more B-cells and re-grow antibodies. The researchers have found in the series of studies that giving multiple doses of rituximab over a long period of time helps to increase the number of people who respond and decrease relapse.

Is there a down side? Definitely. Killing off all your B-cells is not without risks, and there can be serious side effects, just as there are with immunotherapy in cancer treatments. Some people feel sicker during that initial period. I don't know about you, but I would be very willing to put up with worse symptoms for a few months if it meant a 67% chance of remission. Rituximab is currently a very expensive drug.

Also, although two-thirds of patients responding is an amazing number in any treatment study, that still leaves one-third that does not. You could potentially pay a lot of money and suffer through that initial worsening, with no benefit...though the odds are in our favor so far. Future studies will try to figure out what separates the responders from the non-responders and what factors can help to predict who will benefit from rituximab.

I'm also not sure whether rituximab could be used in ME/CFS patients who have active infections, like my son who has Lyme disease plus two other tick infections. I would think that killing off all B-cells and clearing the body of antibodies wouldn't be a great idea with active infections present, but I'm no expert.

When will rituximab be widely available? So far, Norway has conducted all of the ME/CFS rituximab studies to date. UK advocates at Invest in ME have raised $600,000 for their own rituximab study of 30-40 people.

Rituximab is already FDA approved in the U.S., for lymphoma patients and also RA. It is sometimes used in the U.S. off-label (i.e. for purposes other than its official approval) for other autoimmune conditions, like MS, lupus, and others. I have no idea whether any ME/CFS specialist are yet prescribing it for patients here or whether they are waiting for the results from the largest Norwegian study. As for the cost, if it is eventually FDA-approved specifically for ME/CFS, then it should be covered by most health insurance; used off-label, as it could be now, insurance coverage will vary based on the insurer and the specific policy.

So, those are the basic facts - and exciting facts they are! Besides the links above to the two early Norwegian studies, you can read:

• a brief summary from New Scientist
• a brief summary from the UK's ME Association
• a much more detailed, in-depth explanation written by the talented Cort Johnson at the Simarron Research site.

I plan to ask my ME/CFS doctor about rituximab on my next visit. I am interested to know whether she or any of her colleagues are using it yet here in the U.S. or whether there is any information yet to predict who will respond to it.

Do you know of any patients on rituximab or any doctors using it for ME/CFS yet?

What do you think of this exciting new possibility?