It's a Phase III, prospective, double-blind, placebo-controlled, randomised trial, in 234 patients, of a drug treatment for severe ME/CFS, replicating and extending previous positive research results with this drug, and finding a difference between the placebo control and treatment groups roughly twice the size considered medically significant by regulatory agencies! That deserves at least three exclamation marks, surely?!!!
The primary endpoint measure of exercise tolerance (presumably based on the Lights' work) seems to me like a more relevant and objective measure than the traditional measures, but they also showed significant improvement on the other, secondary, endpoints, which included drug usage, the Karnofsky Performance Score (KPS), Activities of Daily Living (ADL), and Vitality Score (SF 36). So the more typical SF36 figures are there as well.
Unless I'm missing something, the fact that it's Phase III puts it firmly in the frame to be considered as a scientifically validated treatment option. Surely it should now be on the table for NICE to consider? Do we have any other treatments that have got through a Phase III trial? I don't think so...
I think this is very exciting news. I imagine it was published in PloS ONE at least partly to get the news out there as quickly as possible. It should not require a major traditional journal to gain considerable attention for this study.
This news should really be in all the papers in the next few days...and in the next BMJ...but being UK-based, I'm not holding my breath on that one. It should also be up for consideration by NICE now; even though I doubt it would be approved (it is not cheap), that argument should now begin, if only to be advanced is clear evidence that ME is indeed (and of course) a physical illness, but I don't know how realistic that expectation is.
Of course we should view all research with a sceptical eye, and I'm sure members will dissect the paper in the coming days and weeks, but they will have to come up with something fairly major to dampen my enthusiasm...