There are lots of really good things about this, starting with over $0.5 million of donations. Then they focus on changes in genes, metabolites and more with improvement and response to treatment, which increases the odds of real rather than chance differences between groups.
Then they collective objective data on activity as well as heart rate etc, all collected by patients using new technology. That really is leveraging technology to make the most of patient input. Putting the congnitive test online is another example (all cognitive tests are done on a computer in any case). So they are creating tremedously rich data incorporating biochemical and physiological and congnitive and activity measurements
I do have a concern is that they seem to have a lot of studies (at least 10) here and I wonder if they will be big enough to produce more than very tentative findings. Certainly 20 patients for gene expression studies is very small - even though it helps that they are trying to correlate the gene expression changes with changes in health status. If ME/CFS is indeed
many different illnesses (even CCC definition) then a study of 20 patients won't be able to find much.
I don't want to sound negative because they are doing so many good things here, but I can't help wondering if it would be better to focus the money on a couple of much larger studies.
I've reformatted their post to make it easier to read, please shout if you think I've split it up wrong:
... Compelling areas include:
an in-depth study of 20 ME/CFS patients:
- 1. Whole genome sequencing
- 2. A virome survey of oral viral flora
- 3. A gene expression study to examine 30,000 human genes for changes in gene expression patterns correlated with improvement/response to treatment
Two proteomic/metabolomic pilot studies: [how big?]
- 4. One looking at over 250,000 protein features per sample. Resulting in a protein profile to compare a baseline ME/CFS patient with responders and non-responders.
- 5. The second analysis will examine 30,000 smaller molecule profiles representing toxins and metabolites in the same set of samples. This will further our understanding of what a patient’s profile looks like on a molecular level pre and post-treatment.
Immune studies
6. Natural killer cells, B-cells, and T-cells will be looked at for functional performance, activation markers when
compared between ill and recovered patients
7 (+)Other Immunology studies will include single cell measurements, acetylation and other cellular parameters
Breath testing for GI flora gas production with dietary changes to start looking at the effect of the gut microbiota on the
disease, recovery and treatment
Other work
8 Measurement of mitochondrial and related biochemical pathways that play a role in oxidative stress and energy production, adrenal and thyroid pathways that regulate metabolic function along with full panels of pituitary, gonadal, adrenal and other hormones.
9 Brain/Cognitive Data will be collected using unique on-line programs including reaction time, short-term memory, latency, and additional cognitive assessments. These data will be
correlated with clinical and molecular endpoints
10 Quantified-Self (self measurement) projects will rely on wearable
devices logging data of activity, heart rate, sleep patterns, blood pressure, heart rate variability (a measure of stress) and others
