Live Science: 'It took the rug right out from under my life': Milestone ME/CFS study begins to explain disease, but will it lead to treatments?’'

[Dakota15]

Live Science: ''It took the rug right out from under my life': Milestone ME/CFS study begins to explain disease, but will it lead to treatments?’'

'An NIH study many years in the making starts to unravel the biology of a misunderstood disease called ME/CFS.'

"We need to validate these findings, but they can be done in the context of a clinical trial," Nath said. There's now enough data to start pinpointing potential therapies, narrowing down which populations of ME/CFS are likely to benefit, and then testing these in trials. Once selected, multiple drugs could even be tested in the same trial, with, for example, four groups receiving different treatments and one receiving a placebo. This could speed up the process, Nath suggested.'

'One possible treatment proposed in the study is an existing cancer therapy called immune checkpoint inhibitors. The idea is that ME/CFS patients might have lingering bits of viruses or bacteria that are constantly setting off their immune systems, exhausting their cells. The checkpoint inhibitors could release the brakes of those exhausted T cells, theoretically enabling them to finish the job and clear away the substances continually triggering the immune system.'

 

[Marylib]

Glad they published what Brian said.."One thing I want people to know is that this illness is a matter of life and death," he told Live Science. "A lot of patients, they don't have any hope that they will ever get better, and they end up ending their lives." Vastag's life partner, ME/CFS advocate Beth Mazur, died in December 2023 after living with the condition for 15 years, and through the years, Vastag has known others with the disease who died by suicide. It's with that in mind that he hopes the NIH will launch treatment trials expeditiously."

But since they did not study the severe or very severe - only those who could actually show up at the study site - no one dies of the disease? We all know that they do. In some interview about the study - can't remember which one - Lucinda Bateman said something about how the patients in the study were not as sick as some of her own patients..

What I say is not meant to diminish or disregard the suffering of the mildly or moderately affected.

 

[BrightCandle]

Almost all studies and trials are biased to moderate or better and often just mild patients. Using especially a double day CPET selects a the mildest of patients only because they can come in even when they have PEM from the prior day. Most studies require transiting to a lab which in itself limits to moderate patients. Then they often have repeated visits over weeks which again requires relatively mild PEM episodes.

To actually get Severe or even Very Severe patients you have to go to them. You either have something you post them and they post back (a carer often necessary for this part) or they are visited in their home for blood to be drawn.

I think inducing PEM is a real problem especially for severe and very severe. While a lot of us die from suicide due to everything about this condition and society one of the main ways we die is from such severe PEM we can't eat and then not even digest, as we are seeing with these 3 women in the NHS right now being starved to death by doctor neglegence. You can't get a diagnostic, study or trial that has a risk of worsening a patient when the consequences of that PEM could lead to their death, its unethical and it certainly should not be allowed as a requirement for disability payments as it often is in the USA.

The other aspect to this mild bias that is being introduced in study design is that the strongest biological symptoms are in all the most severe patients. So while its more expensive to get to them and more care has to be taken they should produce clearer signals and allow trials to be smaller. Right now almost no studies get to these patients at all and that really needs to change. Even DecodeME that tried with its designed biased a bit against severe/v severe as they are less frequently diagnosed due to being too sick to access healthcare for a diagnosis and it looks like its design also biased towards women too.

Its also important to consider when it comes to trials and treatments. Unless a treatment has a very high chance and impact its not worth it for a severe patient, suffering a life threatening PEM episode to get an IV shot of B12 just isn't worth the risk for a tiny increase in overall activity of a few percent points. Large effects are necessary and they don't require complex criteria and tests for efficacy they are really obvious.

We really need this to change and the more people that realise the mechanisms by which studies are harming patients and selecting the most mild hopefully the more we can collectively push for consideration of our most desperately ill people to drive science forward quicker.

 

[Rufous McKinney]

r moderately affected.

I"m moderate and I'd never make it through an hour at NIH.

 

[Rufous McKinney]

We really need this to change

really excellent points: so important

what a mess.

 

[Marylib]

I"m moderate and I'd never make it through an hour at NIH.

Yeah - it must be awful in that place...
Personally I don't have any reason to believe there will be a treatment for me - ever. But I am old...I sure hope that I am wrong but mainly hope that those who are younger or those in the future to suffer this fate - will have something!

 

[Rufous McKinney]

Personally I don't have any reason to believe

same here.

I mean, what would finally cured, at eighty , look like?

I am the type who is convinced if I can just figure something out, I'd be somehow able to do better. So I sort of blame my own inability to figure out this something.

But for others, and the young, and children and those really suffering unable to eat or stand or move I WANT A CURE.

 

[Marylib]

sorry - I messed up the quote/reply feature - glad you got my drift @Rufous McKinney . I guess a cure at 80 would look like the 80 year old people I see out walking, having fun, etc. Or a grandma like yourself being able to enjoy the little ones...

 

[lenora]

I don't think in terms of a "cure" for myself. If I have relief of my symptoms I consider that above and beyond. I'm going to be exhausted anyway, as I can't even sleep for more than 2 hrs., if that. Nothing helps.

Computers now make it possible for more people to join the ranks of NIH via hospitals such as The Cleveland Clinic, Mayo (which is in the process of changing) and other well known facilities. Just like going to M.D. Anderson for a cancer diagnosis, but treatments are done locally. This is a huge plus.

I feel that I've lived my life, have had both joy and sadness, but I had a life, a good husband and two children. Yes, I suffer and can't go anywhere (whereas in the early years I could travel, but not now). However when I hear about bedridden youngsters and young people, I feel they should have their chance.

Help me with pain relief (and that has occurred) and I'll do the rest. I always have some problem or another, but at my age, some of this is related to the older years. I want to laugh, enjoy my family and even watch an entertaining movie. Reading (my favorite thing after my family) had to go when I fractured my pelvis. It hurts too much to sit even almost 2 years later. I've lived through a lot of negativity about ME/FM, insults from the press and I'll keep forging ahead. We have too many really, really ill people and I want a chance for them.

Things will change over the years, I've already seen plenty of it, including different treatments for many of the same problems. We'll have more, but we'll adjust. Things are on the horizon. Yours, Lenora

 

[hapl808]

I feel that I've lived my life, have had both joy and sadness, but I had a life, a good husband and two children. Yes, I suffer and can't go anywhere (whereas in the early years I could travel, but not now). However when I hear about bedridden youngsters and young people, I feel they should have their chance.

I can understand that. I'm in the middle. I struggled greatly with mild-moderate from 25-40 but managed to start building a life nonetheless (good career and friends, but no partner or kids). Then I ended up more severe, and life pretty much ended - along with all my attempts to build a family. I suppose it could've happened younger, but that's my only comfort. No kids, no partner, and if there's no scientific breakthroughs - a grim future.

 

[1gooddog]

I"m moderate and I'd never make it through an hour at NIH.

I would have to be housed at the trials site and wheeled in for testing.