Researchers at Haukeland University Hospital in Bergen, Norway – where investigations into the use of Rituximab to treat ME/CFS have taken place – are testing another anti-cancer drug called cyclophosphamide to see if it can benefit patients with ME/CFS.
Brief details about the cyclophosphamide trial, written in Norwegian, appeared on the internet at the weekend and a Google-mediated translation appears below.
Dr Charles Shepherd, medical adviser to the ME Association, comments:
This is a short report on an interesting new clinical trial that will be taking place in Norway involving the use of a drug called cyclophosphamide.
The trial is being carried out by the doctors at Haukeland University Hospital, who are involved in the Rituximab clinical trials.
It would be interesting to know if this follows on from any reports of cases where someone with ME/CFS has received this drug and reported a significant degree of improvement.
Cyclophosphamide is drug that is used to treat some types of malignancy – such as leukaemias and lymphomas.
But it also what is known as an immunosuppressive drug, meaning that it can dampen down immune system activity.
So it is occasionally used as a disease-modifying drug in non malignant conditions such as more severe rheumatoid arthritis and lupus where other drugs have failed.
And this is why it might be useful in people with ME/CFS, who have evidence of an overactive immune system response.
Cyclophosphamide can cause serious side-effects involving the bone marrow (causing a drop in vital white blood cells that help to fight off infections), liver, pancreas and lungs, as well as causing a potentially serious bladder complication called interstitial cystitis. So it has to be used with care and carefully monitored.
It can also cause a number of side-effects that form part of the symptom list in ME/CFS.
Going back almost 30 years, when I was involved with Glasgow neurologist Professor Peter Behan in the assessment of a number of experimental (certainly at that time) approaches to drug treatment of ME/CFS, including the use of antiviral drugs (i.e. acyclovir) and various types of immunotherapy, the use of steroids (i.e. prednisolone at 10mg to 60mg per day) produced no benefit and when azathioprine (another immunosuppressive drug) was added no change was seen. In fact, some patients had more complaints at the end of the course of treatment than at the beginning.
Having said that, the numbers involved here were small, and this was not the type of clinical trial that is now being carried out in Norway.
As this is a very speculative form of treatment, and using a drug that can cause serious side-effects, I suspect that other research groups will await the results before any other clinical trials take place.
BRIEF DETAILS OF THE PROJECT
Research project: Cyclophosphamide by myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS)
Cyclophosphamide BY myalgic encephalomyelitis / chronic fatigue syndrome (ME / CFS)
Part A, for up to 30 patients with ME / CFS: An open phase II trial with 6 cyclophosphamide infusions 4 weeks apart
Part B, for up to 20 patients with severe and very severe ME / CFS: An exploratory study with up to 6 cyclophosphamide infusions 4 weeks apart, in collaboration with local health services
The purpose of this study is to investigate whether cyclophosphamide infusions given every four weeks is associated with clinically significant responses and acceptable side-effects in ME/CFS patients, and whether such treatment if necessary. Workable in patients with severe and very severe ME/CFS. The primary endpoint will be based on patients' self-report of symptom development. The application relates Part A: An open-label phase II study for up to 30 patients with ME/CFS (not mild) with six infusions cyclophosphamide every fourth week, 12 months follow-up. If at least 40% response rate in Part A, then part B: A descriptive study with up to 20 patients with severe and very severe ME / CFS, with the same treatment, but where monitoring and intervention carried out in collaboration with healthcare close patient home.
(Project Manager project)
Ref. No.: 2014/1672 EudraCT Number: 2014-004029-41 Project start: 01.01.2015 Project End: 30/06/2017
Treatment Status: Approved
Research Status: Ongoing
Project: Øystein Fluge
Research Responsible (s): Haukeland University Hospital
Initiator: Contributions Research
Study researcher initiated. There is no external sponsor of the clinical trial. Research Group for ME/CFS Oncology Haukeland University Hospital has support from the Kavli Foundation, essential for research purposes to better understand the pathogenetic mechanisms of ME/CFS. Kavli Fund has granted a 50% nurse position for 12 months, to help with the study. This is thought transferred to clinical research Post by HUS which will then be responsible for implementation and interventions (study Part A) in collaboration with the study management at Oncology, HUS.
Drug costs are very low (<200 NOK per infusion), ie 1200 NOK per patient for the entire treatment, and will be covered over the research group for ME / CFS Oncology their budget. It is drawn own drug insurance for study. There is no financial compensation for study participants.
Research Data: Drug Testing, Human biological material Humans
Selection: Patients / clients