What are the most important outstanding research questions for people with MS?
Depending on how long you have been following this blog you may or may not be aware of the COMBAT-MS study. This is a study that is funded by the US government as part of the Patient-Centered Outcomes Research Institute (PCORI).
COMBAT-MS is a US-Swedish initiative for a hybrid study between a traditional retrospective cohort and a structured prospective cohort study. COMBAT-MS (clinicaltrials.gov NCT03193866) aims to recruit up to 3,700 MS patients at all of Sweden´s seven university clinics (serving about 50% of the total population) who are starting their first MS therapy or switching therapies. By doing the same structured follow-up with annual disability scorings, MRIs and patient-reported QoL outcomes the COMBAT-MS study will generate high quality, real-world long-term efficacy outcomes. This will help the MS community sort out which drugs work or don’t work in a real-world setting. To address safety concerns, COMBAT-MS will obtain data from Swedish national healthcare registries, as well as from Kaiser Permanente Southern California. By combining these resources, COMBAT-MS will be able to address major risks, such as cancer, as well as transient treatable concerns like recurring bladder infections and bothersome skin rashes. So, which of the approved MS drugs will be the winner?
I will be attending the annual COMBAT-MS stakeholder meeting for an update this week and have been asked to give a talk on ‘Identifying and addressing research questions of high importance to patients’. I could simply use the MS Society’s James Lind Alliance Top 10 list (below) or ask you the Barts-MS Blog readers to add to, or critique, this list for my talk this Thursday. Please note that this list below was published in 2013 and what was a priority then may not be a priority today.
- Which treatments are effective to slow, stop or reverse the accumulation of disability associated with MS?
- How can MS be prevented?
- Which treatments are effective for fatigue in people with MS?
- How can people with MS be best supported to self-manage their condition?
- Does early treatment with aggressive disease modifying drugs improve the prognosis for people with MS?
- Is Vitamin D supplementation an effective disease modifying treatment for MS?
- Which treatments are effective to improve mobility for people with MS?
- Which treatments are effective to improve cognition in people with MS?
- Which treatments are effective for pain in people with MS?
- Is physiotherapy effective in reducing disability in people with MS?
The following is this week’s programme; any comments to help me prepare my talk would be much appreciated. I was invited onto this trial as a stakeholder because I am considered a patient advocate and for my role in formulating and promoting an ‘Essential Off-label DMT‘ list. To fulfil my role as a patient advocate I need your help. Thank you.
Physiotherapy is effective in reducing disability in patients with, MS along with a healthy lifestyle. The best drugs in the world alone will not improve EDSS without exercise, but exercise and no treatment can and is beneficial. It needs to be individually targeted.
This will need to be bungled with restorative trials. I will be it on the agenda.
Yes Anonymous , it is so so so difficult to get any physio. I am still battling with bad arm. I was seen in August at the spasciticy clinic and I am still waiting to be seen again, in the waiting list . Waiting and waiting in pain, by the time they see me hopefully it’s not too late otherwise the arm will be inmobilised . And I can see benefits when doing physio. Physio is very important and almost impossible to get.
How could we be made more accessible ? That would be a question for you to take.
Yes Maria, I waited a year after being referred by my Neurologist. I’ve had MS for many years and now SPMS. I know that physio works for me in my legs. Upper limb physio is hardly ever offered. I have weak arms and hands and I completely understand your frustration. When i’ve chased where I am on the waiting list, i’ve been told how many patients are on the books. Not helpful.
Keep chasing. Anonymous
Possible question: Given that Tysabri is apparently very effective and well tolerated by many what is being done to make it suitable for more people for longer? (Eg. What is the news on JCV vaccine?)
This is in Biogen’s court. They are doing a lot to derisk and keep natalizumab alive and kicking. We need it for DrK’s #AttackMS trial.
Two suggestions:
5) should include something on HSCT, in addition to medicines – maybe already thought of….
Add a point concerning affective disorders (mood disorders): depression, bipolar disorder (type I and II) and suicide (which all have a higher incidence rate in pwMS).
What is causing MS?
More to get Scientists together to analise what is going on. Yes, new drugs are important, but finding the cause is even more important.
Best drug for people on SPMS?
We spms patients have no time to spare. I sometimes think I won’t see a major break through in my lifetime! And i am only 46!
These topic are already on the James Lind list.
A few suggestions:
– how does aging contribute to increasing disability and how can the impact of aging on disability be mitigated?
– why has research to identify therapies to repair MS damage still not delivered any effective treatments?
– if MS is one disease, why are the outcomes so varied (I knew an MSer who died at 42 from complications of MS and I know an MSer who is 82 and who is still walking with a cane)?
– what is research on paediatric MS telling us about the likely causes or triggers of the disease?
– why do some neuros still wear bow ties and why do some MS researchers wear silly ponytails?
1. The role of viruses and the potential of anvirals as treatments in MS to be exhaustively researched.
2. Research into microglia, the innate immune system, such as Katerina Akassoglou’s work.
3. Neuroprotectives for progressive – including inactive – disease.
Agree 🙂
Fasting
Good one!
This!
How may health care personnel make sure to diagnose and support pwMS when they develop mood disorders?
Mood disorders due to structural changes in the brain, may be rather difficult to treat employing standard psychiatric approaches. It was rather annoying when I became bipolar after some years with MS. In the beginning I certainly did not understand what was going on…and it took too long before others found out and helped me….
I would like to see research carried out for future generations that would allow for earlier identifiers of ms in childhood/teenage years, including studies involving children of pwms.
This could be badged under prevention.
It has been discussed on this blog before, but how about a question around the future of patient specific dosing of high efficacy DMT’s, or even personalized cocktails of various DMT’s? I think back to Mouse Doctors post of “Are we overdosing Ocrelizumab” and can’t help but feel a one size fits treatment regimen could be overdosing some patients and underdosing others. Could the Barts Cladribine protocol (weight and lymphocyte count) be utilized to some degree for other DMT’s? I would think personalized dosing we help de-risk some of these potent drugs. We need to get more people thinking about these concepts and creating methods to administering them.
It has been discussed on this blog before, but how about a question around the future of patient specific dosing of high efficacy DMT’s, or even personalized cocktails of various DMT’s? I think back to Mouse Doctors post of “Are we overdosing Ocrelizumab” and can’t help but feel a one size fits treatment regimen could be overdosing some patients and underdosing others. Could the Barts Cladribine protocol (weight and lymphocyte count) be utilized to some degree for other DMT’s? I would think personalized dosing we help de-risk some of these potent drugs. We need to get more people thinking about these concepts and creating methods to administering them.
I would classify this as making treatments safer.
What about suggestions there is a link between the gut and MS? What’s the evidence?
There has been tremendous progress on DMTs, but it seems that the focus is on preventing relapses.
How can we go beyond this point and start treating MS avoiding smouldering MS?
A database including the genome and the microbiome.
Supplements:
Lipoic acid.
Boswellia Serrata.
Nano Curcumin.
Biotin.
CDP Choline.
D-Mannose with cranberry extract.
Etc. Etc. Etc.