1. How do I know that my money reaches the right project?
Our finances will be available to be checked in the Brønnøysund Register and anyone wishing will be able to gain full access to check all revenues and expenditure. The funds coming into the Haukeland Hospital for the project will be separate from other project funds in Haukeland’s accounts. The Cancer Department’s Controller will have a continuous insight into what is coming into the account. MEandYou will report on revenues as frequently as possible on their website.
2. What is the difference between giving to us and giving to the ME Association’s Krone Rolling Action?
To put it simply, MEandYou is time limited and focused on one study while the ME Association’s initiative has a longer-term outlook. You are welcome to support both! Read more about it here: MEandYou as an independent and action oriented charity.
3. Why is it called a Phase 3 study?
The study we are raising funds for is the last of three stages in a study where researchers at the Haukeland Hospital examine the effect of the drug Rituximab on ME patients. Three patients participated in phase 1, the second phase consisted of 30 patients and this last phase will consist of 140 patients. In phase two, it was shown that 2/3 of the patients had good or moderate response to Rituximab. Most of them got sick again after a while. This is common when using this drug in other diseases. Other disease groups need to have regular new medicine to maintain the benefits. The researchers will also examine if amended and more frequent doses make the effect of the treatment last longer.
4. How will the Phase 3 study be implemented?
The third phase of the Rituximab study at the Haukeland Hospital will involve 140 patients and it will be a multi-center, double-blind, placebo-controlled clinical trial. This study is necessary to find out more reliable answers about Rituximab, if it can be used as a treatment for ME sufferers.
5. What does “multi-center, double-blind, randomized, placebo-controlled clinical trial” mean? When researching a medication or a treatment, we want to explore the effect of the treatment itself. The kind of study that has the greatest potential to tell us this is called a randomized controlled trial, abbreviated to RCT. In an RCT study, we attempt to the fullest extent possible to separate the effect of the treatment we want to examine from the control group.
By a control group, we mean those who do not receive the therapy we want to test. They may receive a placebo or another type of therapy we know the effect of, and then we would examine whether it has better or worse results than the new treatment we want to test.
A randomized study means that participants are randomly allocated into the group receiving the treatment and the group of those who do not. To increase the quality of the study further, what we call a double-blind study is carried out. This means that neither the person who gives the medication nor the person receiving it knows if the medication is real or not. It helps to prevent a person from showing a response only from the expectations that they are receiving the treatment.
Multi-center: When a research study is conducted in several different places. It is important so that scientists can verify the results separately.
Placebo: An inert substance which cannot produce the effect we want to investigate.
Randomized: Patients are randomly placed in either the placebo group or the group receiving the treatment.
Double-Blind: When neither the researcher nor the patient knows if the patient is receiving the treatment or not.
6. How long will this study be?
This multi-center study will last approximately 2 years from the date when the first patient starts until the last of the 140 participants receives their last dose of Rituximab.
7. Who can participate?
Rituximab will be tested on 140 patients. The group will be divided into two and 70 participants will receive the active treatment and 70 participants will receive saline. No one will know which group they will be in. It is the researchers who select the patients. First, the researchers will consider whether the patients meet the set criteria. After this, those who have been accepted will be randomized. The research will take place in several locations in the Norway. We do not know where yet.
The official healthcare system only treat Norwegian citizens, so you have to live in Norway to be included.
8. Why would Rituximab work for ME? What are the researchers’ hypothesis(es)?
The researchers’ hypothesis when the study was published was that ME is an autoimmune disease. The reason that they thought this was because ME patients had a good response to the same medication as in other autoimmune diseases. All good researchers can modify their hypotheses as they find new answers during their investigations. We must wait for Fluge and Mella’s final publication of the results.
9. Do we have the answer now, as Rituximab has been shown to have an effect on ME sufferers? We do not have the answer and do not know what mechanisms are at work. We also do not have the answer as to whether Rituximab only works on a few ME patients or the majority. We therefore need Haukeland Hospital’s Phase 3 trial with many patients to confirm the promising results from Phase-1. The significance of this result is that it points towards the area to continue to research: the biomedical.
Results from the Phase 3 study may arouse great interest among many more researchers. Our concern is knowledge and research on ME, it is therefore important that as many researchers as possible are interested.
We also need to understand the disease and its mechanisms better. It is necessary to identify different types of drugs that can have an effect, not only Rituximab. Researchers are also looking for a test that can differentiate ME from other diseases. The beauty of this is that we can get the answer if we keep on researching and do not stop here.
10. How long will it take before I can possibly get Rituximab?
The short answer is: In the best case scenario, it will take 4-5 years before we could see Rituximab provided for the sick. The important thing now is that a major study on Rituximab is carried out in order to make progress. Then, several research places will have to do the same study and confirm the results. The Haukeland Hospital’s Phase 3 trial will take approximately two years to complete.
When the study is completed and it may be shown that there is a good response to Rituximab, the researchers will write their report and submit it to a medical journal. This can take between 6 to 12 months. Then the medication may be approved for use for ME sufferers with specific criteria. Most likely various type of medication will be needed for ME patients, just as different types of medication are used by diabetics and people with high blood pressure.
11. Who are these 140 that you speak of?
In the next phase of the study, 140 ME patients will be considered for a trial of the drug Rituximab. Half will receive saline, half Rituximab. These 140 people will be determined by the scientists. They will first select patients who meet the strictest criteria. Then they will randomly select persons from this group again. The study will be carried out in multiple locations in the country but it is not known yet where.
12. How can I secure myself a place in the study?
No one can buy places in the study nor secure places by any other manner. In order to participate, an application to Haukeland Hospital must be made in the usual way: via your doctor who can document your illness, diagnosis and symptoms etc.
13. Why should I support MEandYou if I do not get to participate in the study?
If you do not get to participate in the study, the results will still be beneficial to you, knowing that scientists are learning more about the disease mechanisms and treatment options. If this study does not receive funds for starting, then we will not know if Rituximab is something that will help this group of patients and this treatment will not be used on patients.
14. Will this study be enough or will further research be needed?
The results from this study will provide important new knowledge about ME and the disease mechanisms. This is a major and essential step towards a treatment for ME. It will probably still be necessary to do more research to find out exactly what ME is, how people get ME, how to detect and how to treat ME.
15. What can “go wrong” so the that study doesn’t get started?
Haukeland Hospital can be at risk of not getting enough funds for further research. With no funds, there will be no answers.
In the first study, it was found that 2/3 of patients had a good or moderate response. This may change in a larger study. When research is done on small groups, it may be that we just selected the patients who had a good response. In a larger study, we could see different numbers.
16. What does it take for a new medication to start being used?
To use drugs for ME, the medication has to be approved for use first. Before medications are used, there is an assessment of the benefit, severity and cost. Rituximab is an expensive medication and to receive it through the reimbursement scheme, it must be approved.
17. Can my doctor prescribe the treatment on the same day as the research project ends, and if not: what are they waiting for?
No, the doctor cannot prescribe treatment on the same day as the research ends. The drug must first be approved for routine use. Rituximab is a medication that must be given by a specialist, and under the supervision of medical personnel.
18. Why can we be more optimistic about Rituximab then we were about XMRV? Will this be yet another disappointment?
In this study, a drug has been tried out. There has been a small study with a solid study design and this means that we can more likely trust the results. There are dozens of pilot patients who have already had good results in the first two Rituximab trials.
The theory of XMRV arose when scientists thought they had found a new virus in the blood of ME patients. Many sick people hoped to get treatment when this came out. After testing by other scientists, it turned out that this was only a contamination of blood samples in the laboratory. Rituximab is a well-tested drug which is usually used either in the treatment of cancer or autoimmune disorders and as we know, it works with these diseases. The researchers at Haukeland Hospital have experience that this medication can also be effective in ME patients.
19. What is Rituximab and how does it work?
Rituximab is a type of protein that must be taken as injections regularly. Rituximab binds to the surface of a type of white blood cells called B-lymphocytes. B-cells are important cells in our immune system. B-cells make what we call antibodies against the body, the body recognizes these as “foreign” and not like itself. When Rituximab binds to the surface of these cells, it causes the B-cell to become inactive.
20. Is Rituximab dangerous?
Rituximab is given as in an injection, as an infusion, directly into the vein. Most adverse events occur during the infusion. It may be that you become more vulnerable to infections during treatment with Rituximab. In general, Rituximab is tolerated very well but when many people get treated, one cannot rule out that some of these patients can have serious side effects.
You can read more about the side effects here: http://www.felleskatalogen.no/medisin/pasient/pil-mabthera-roche-561181
There has been a study on the long-term effects of Rituximab: http://www.ncbi.nlm.nih.gov/pubmed/20110520
21. Is Rituximab used to treat other conditions?
Rituximab is used to treat cancer (non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia) and autoimmune diseases such as rheumatoid arthritis (arthritis) and lupus.
22. What is an autoimmune disease?
Occasionally the body’s immune system attacks its own cells. This is called an autoimmune response and is present in many different diseases. The researchers believe that the B-cells produce antibodies that attack the ME patients’ own cells. Rituximab works by knocking out B-cells so that fewer antibodies are produced and the body attacks itself less. Scientists do not yet know what causes an autoimmune reaction but many have theories that this is connected to infections.