What research would you do if you had £1M?

[digital dog]

Definitely get the media on our side.
I would look at the immune system and autoimmunity.
Put money into Rituximab.
Then, when it was proven that we are not lazy malingerers or mentally ill, I would name and shame all those doctors, psychiatrists and specialists that thought we were.
It will happen. We will find a cure or treatment for this condition. People will look back at the way we have been treated with horror.

 

[alex3619]

Well, I would use that money to launch a major PR campaign on all media about the realities of ME/CFS, and start a major fundraising effort targeting potential donors with deep pockets, so we can turn that million into 1 billion. The PR and goodvertising effort should also shame the governments of major industrialized nations (and shame them hard!) for neglecting research funding and treatments for the disease. The goal would be to change the public perception of the disease and make the public demand that governments properly fund research for this devastating illness.
That would be my take.

Perhaps a bit ambitious? Yet if we don't think big we wont make big gains. So it might be worth trying. If we aim for a billion, but only get a hundred million, or even ten million, its still a win. If we get two billion then, hey, its a miracle.

This has been tried to some extent though, with not much gain. Its hard to get traction. There is already a public relations campaign, or was. I have not read an update on it in quite a while.

 

[unto]

I would give priority to research on the cause of ME, because I think that might be the best starting point to understand and defeat the disease;
I have always been convinced (by personal experience ....and the history of ME...) that a virus has made me sick
then I would give the ML. in search of new pathogens (mainly viruses) in our bodies,
doctors like Dr. W.John Martin and dr.Judy Mikovits' that made me hope.

 

[snowathlete]

I'd give the money to the OMF because they have the best chance I've seen of coming up with a diagnostic biomarker which would improve legitimacy, diagnostics, future research and may provide clues to disease processess. All of great benefit to us.

 

[picante]

I suspect we only need gas analysis in severe patients. They are probably living at or over their anaerobic threshold.

Enlighten me, Alex. Which gases are you thinking of?

 

[alex3619]

All you need to do is look at the O2 to CO2 ratio and volume from exhaled breath. Its what they do anyway in a full CPET but without the exercise part. I have also had it done as part of metabolic testing, and I always come out poorly.

 

[Antares in NYC]

Perhaps a bit ambitious?

Indeed. But a guy can dream...

 

[picante]

From the "Sick but Never Sick" thread:

I'm just confused at the apparent dichotomy. Apparently, people are helped by things that activate macrophages and by things that calm them down. How could this both be true?

It may be that it's two separate groups who are helped, but perhaps there's a model where both make sense.

If you have a chronic infection, killing it off would make you feel worse at first, but eventually better. If you have a chronic infection, calming your immune system for a second might make you feel better in general, or get you to stop attacking your own tissues. Perhaps it's a good thing in either direction... for everyone.

It would make an interesting study.

* Start with a large patient cohort, MS or ME or both.
* Assign randomly to LDN, GcMAF, or control group.
* At four months, if a patient in the LDN group hasn't been helped by LDN, switch them to GcMAF and vice-versa. (Consider publishing as a second study rather than all together?)
* At one year, publish findings.

Now, where's the several million I'd need to make this happen?

 

[Scarecrow]

I want to see a whacking great trial on rituximab or whatever needs doing so that we're not all hanging about still waiting when the Norwegian results are published in 2018.

I'd go for a phase 3 trial of cyclo in mild to severe ME (ICC) and start recruiting participating centers. As soon as the results from the norwegian phase 2 trial are ready next spring I would try to treat as many as possible. Never mind the exact disease mechanism if we can get a treatment faster. Rituximab as plan B

If we only have 1 million I agree - we need to focus on treatments and not diagnosis. More phase 3 trials of anything and everything !

Treatment would be my priority too ...........and with the focus on rituximab. A successful treatment provides legitimacy - it would be impossible to argue that ME is not a biological disease. And with successful treatment would come some very motivated and able advocates. All sorts of floodgates will open.

Even if the Norwegian Phase III is less successful than hoped and say only 1 in 4 responded rather than 1 in 2, that's still a massive result. I can't conceive that it will get any worse than that and that's based on not being able to identify likely responders before giving treatment.

In my rituximab trial, I would include a battery of neuropsych tests and would hook up with Stanford to see if there were any MRI changes in responders..............and I've just blown my budget!

 

[Scarecrow]

I would fund immune and mitochondrial research on moderate-severe patients only. I'd insist on CPET testing to confirm that patients in the study actually had PEM, given that's the closest test we have for it so far.

I think this may well be true. The report of my last (submaximal) CPET test said I was hypermetabolic at rest before the test even began, so my guess is that severe patients will show sufficient abnormalities without the 2-day CPET. The 2-day CPET may be necessary to identify mild patients, but possibly not moderate-severe patients. If we can find a way to avoid maximal exertion testing, we could get more severe patients in these studies, which is crucial, imo.

I think we need to get at the core of PEM. That is the identifying feature of ME. It's not fatigue, so simple fatigue research is not the way to go. The most likely sources of PEM are in the immune system and/or the mitochondria, so that's where the money should go. Once we can identify what makes PWME different from patients with other illnesses, then we can start looking into what might be causing that difference -- pathogens, autoimmunity, cardiac, or neurological abnormality, genetics. Ninety percent of the problem with current (and past) ME research is that it is highly likely that the cohorts of mild patients most researchers study probably contain a very high percentage of non-ME patients, so the results are inconclusive.

Something along these lines would be my next move but I'd include an autonomic component to the study. I'm always very mindful of the Nancy Klimas PCOCCA where she talked about exercise challenging the autonomic system and the immune system following. (When are they going to publish on that?). I'm always very aware of the period when, having been physically active, I just want to drink several glasses of water, stop and lie down. It's such an extreme change.

I tend to share your reservations about including mild patients but there may be an argument for it. When I was moderate I was permanently fighting against heavy brain fog and I never felt like I hadn't just run a marathon. But as a mild patient, I find that there is much more in the way of light and shade. I suspect that there might be quite a lot to glean from mild patients from good day/bad day studies and from exercise challenges but probably less so from patients v. controls.

I think that the lab work for the profiles you describe are very quickly going to eat into my budget, so definitely it's rituximab first and then going after the funds for your study.

 

[Sasha]

I think that the lab work for the profiles you describe are very quickly going to eat into my budget, so definitely it's rituximab first and then going after the funds for your study.

I like this prudent and strategic management of your imaginary money.

 

[BurnA]

I tend to share your reservations about including mild patients but there may be an argument for it. When I was moderate I was permanently fighting against heavy brain fog and I never felt like I hadn't just run a marathon. But as a mild patient, I find that there is much more in the way of light and shade. I suspect that there might be quite a lot to glean from mild patients from good day/bad day studies and from exercise challenges but probably less so from patients v. controls.

I think there is an advantage to studiing mild patients once the diagnosis is clear. The results are less likely to be clouded by comorbidies or indeed non cfs/me signals that may be as a result of severe me/cfs. In other words the specificity might be higher in mild patients even if the results are more pronounced in severe patients. So compare both sets might be the answer which is what the OMF plan to do

 

[BurnA]

Treatment would be my priority too ...........and with the focus on rituximab. A successful treatment provides legitimacy - it would be impossible to argue that ME is not a biological disease. And with successful treatment would come some very motivated and able advocates. All sorts of floodgates will open.!

Agree and hopefully this will happen even if we don't get to spend our imaginary 1m !
A successful trial will demonstrate biological cause, generate PR and hopefully stimulate more research and more trials from big pharma who generally only move in on something when the little guys have done all the homework. Pity we have to wait but lets hope it's worth waiting for.

 

[SOC]

I think there is an advantage to studiing mild patients once the diagnosis is clear.

Aye, there's the rub. We need to know that the mild patients are definitely ME patients before we use them in research. Finding biomarker(s) usually requires studying the most severely affected. Once you have the biomarker, all kinds of interesting work can be done on the whole group as well as subgroups. What makes mild patients different from moderate or severe patients? Which features/symptoms are distinctly ME and which are common cormorbidities? But we can't do that until we know we're actually studying a single entity.

Finding biomarkers is critical both from a research and a political point of view. Once we have biomarkers, we are suddenly no longer medically unexplained. That kicks the butt of the BPS school and gets a lot more researchers (and clinicians) interested in us. We desperately need both.

 

[bsw]

Flesh out a proper treatment protocol that centers around lowering oxidative stress, reducing autoimmune issues and increasing mitochondrial function, along with normalizing the gut.
I think that would be a fantastic step in creating a standardized protocol that could be a first line of defense against CFS.

 

[gregh286]

Immune system...b cell and mast cell interaction and activity during latter half of sleep cycle.