ME CFS conference in New York 20th November 2013 relating to treatment .

[Andrew]

I've long wondered if we would be better off putting more attention on soliciting aid from private foundations, then trying to change the mind of the NIH.

 

[john66]

I was at the conference and just got home. My head and body are fried, crashing in a major way, but it was worth it. All of the speakers were outstanding and it give me hope that progress is being made on a shoestring of a budget. All of the doctors there are making great use of new technological capabilities that leverage data analysis. I have notes which I will try to distill over the next couple of days and report more. Thanks to Dr. Enlander, Mt Sinai and Hemispherix for sponsoring it. Most of all thanks to the super generous man who started the whole thing at Mt Sinai with the heart of gold.

 

[justinreilly]

@justinreilly I was the person who met your parents. I can't go this time .

Yes, now I remember.

 

[justinreilly]

A tweet of Jeannette's that I think captured something interesting-
#MECFS Dr. K: it's all about studies. If there is only $3 mill per year in research money, who will do validation studies? W/o that, study won't catch on. Grant money not given for validation studies, typically. Also need business partners who will market biomarkers.

http://tl.gd/mvbvl9

My Bolding

 

[justinreilly]

I saw Simon Wessley's name tag on the table at Mt Sinai. I asked if he really was coming and the woman said yes. But didn't see him or anyone else with Bozo the clown hair.

 

[leela]

Bozo the clown hair

Justin! you're wicked!

 

[Wally]

Justin! you're wicked!

All Points Bulletin - New York City Please Be On The Lookout For This Suspect (a.k.a. "Bozo The Clown")

 

[Wally]

Another tweet of Jeannette's that I think captured something interesting-
#MECFS Dr. K: it's all about studies. If there is only $3 mill per year in research money, who will do validation studies? W/o that, study won't catch on. Grant money not given for validation studies, typically. Also need business partners who will market biomarkers.

http://tl.gd/mvbvl9

My Bolding

If I was going to give money to a private institution/foundation. I would suggest that this one be at the top of the list.

Open Medicine Foundation (Founder Dr. Andreas Kogelnik) http://openmedicineinstitute.org/foundation/mission/

Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS)
ME/CFS is a debilitating disease affecting neurologic function, metabolism, hormonal regulation, circulation/blood pressure, and overall well being. ME/CFS affects over 8 million patients worldwide. It has few options for accurate diagnosis and treatment.

Patients with ME/CFS desperately need answers. We are convinced these answers are readily available if we apply the best resources in a large-scale, concerted effort. Bringing together the right experts and the most advanced technologies to deliver actionable results is a necessary condition for success that has been a long time coming to this field.

The OMI-MERIT Initiative—Changing the Game for ME/CFS
OMI-MERIT (ME Roundtable on Immunology and Treatment) is a strategic initiative of OMI and its collaborators to bring together leading clinicians and researchers to tackle this debilitating but underserved disease. The MERIT group is focused on developing and applying a multi-factorial approach to the discovery of new diagnostic and treatment solutions for ME/CFS.

In June 2012, OMI convened an initial group of game changers to advance and prioritize the conversation around urgently needed treatments for ME/CFS. We invited engaged, established scientists and physicians to a two-day session with the goal to address practical issues and identify achievable targets in ME/CFS with a focus on research projects that deliver results for patients in the shortest amount time. We continue to expand the list of OMI-MERIT collaborators and hope to add additional projects as funds allow.

OMI-MERIT Priority Projects

1. Treatment: Phase 1: A Large-scale, Randomized, Placebo-controlled Trial of Rituximab and Valgancyclovir
   • Goal: This rigorous, four-armed study will examine and further validate two of the most promising therapies in the field by comparing placebo, rituximab alone, valgancyclovir alone, and combination therapy of valgancyclovir plus rituximab. Measurements of physiologic, genomic, virologic, and immunologic markers will be made throughout the course of the trial.
   • Importance: A large-scale, rigorous trial is needed to confirm the initial findings of earlier smaller studies and move ME/CFS to a molecularly trackable disease. Success of such a trial could move ME/CFS to a mainstream process for additional diagnostic and treatment trials.
2. An International Neuro Registry and Biobank
   • Goal: Supporting and expanding the largest and most comprehensive longitudinal ME/CFS information source for research and collaboration will be the result of this project. We will collect longitudinal data and biological specimens from ME/CFS patients and controls, and characterize the ME/CFS population by patient symptoms and laboratory and molecular profiles through crowd-sourced informatics and cutting-edge tools in immunology, genomics, and molecular biology. Comprehensive, standardized sampling will include blood, cerebrospinal fluid (CSF), urine, stool, brain/CNS, and other tissues. Samples will be available for additional studies in the MERIT list and beyond.
   • Importance: There has been no large-scale, chronologic characterization effort across the ME/CFS population. The Registry and Biobank will help establish clinical and biologic clusters in the population, paving the way for diagnostic biomarkers and cluster-specific treatments. In addition, this will provide a community resource for patients and is central to additional collaborative projects.
3. Protein Panel in Treated and Untreated Patients
   • Goal: Performing in-depth, cutting-edge protein analysis of selected specimens from the Biobank to identify bacteria, viral, hormonal, antibody, cytokine, and other protein-based substances that might be present in patient specimens. Specimens will be selected based on expected yield from clinical data and then discoveries confirmed in the larger patient population.
   • Importance: This project aims to apply cutting-edge protein detection systems with specific, ultra-sensitive ME/CFS-related targets identified. Protein markers are key in identifying potential biomarkers, and many new advanced technologies have never been applied to ME/CFS before.
4. Treatment: Phase 2: Other Therapy Mono and Combination Pilots
   • Goal: To assess the effect of other touted treatments that are currently available in the field and establish immunologic and molecular parameters for measuring the efficacy of such treatments. Treatments assayed will include Ampligen, Etanercept, rifaxamin, Issentris, famcyclovir, and possibly others.
   • Importance: To determine a direction and baseline for other potential drug therapies in the field and assess which should receive additional allocation of funds for research.
5. Immunologic Biomarker Exploration Studies
   • Goal: These exploratory studies will examine B-cell, T-cell, and Natural Killer cell responses to disease and treatment groups using comprehensive, rigorous methods, many of which have never before been applied to ME/CFS. It will seek to establish immunologic baselines and variants from that across the patient population.
   • Importance: For a disease that appears to have a solid immunologic component to it, this study will provide the most advanced, longitudinal characterization of immune changes in critically implicated cells over selected treatment and control patients.
6. DNA Genetics
   • Goal: Use the most advanced methods to sequence key areas of the human genome in a set of patients, controls, affected families, and unrelated individuals. Utilizing advanced Human Genome Project technologies, this project will undertake HLA and other regional sequencing of areas of interest for selected patients and families.
   • Importance: Establishing or refuting a role for genetics and potential heritable risk in ME/CFS.
7. Mass Spectroscopy/Environmental Measurements
   • Goal: This exploratory study will search patient samples for unknown compounds, toxins, proteins, and other substances that may be implicated in the genesis of the disease or otherwise contribute to immune dysfunction.
   • Importance: This would be the first systematic examination of samples by the most reliable substance identification techniques to begin to establish an understanding of the contribution of nutritional and environmental factors to ME/CFS.
8. Comprehensive Viral Testing
   • Goal: Establish a core of viral testing methodologies that are useful and could be useful clinically. Testing will include blood, urine, saliva, and other tissues where available for specific viruses such as EBV, HHV6, CMV, Parvovirus, HSV1, and HSV2, and additional panel-type testing for novel viral identification and high throughput methods.
   • Importance: This project will set the standard for clinical viral testing in ME/CFS and establish a guideline for evaluation and treatment directions for patients. Priority will be given to assays that have already yielded promising clinical results in partner labs.
9. Advanced Immunologic Biomarker Study 2
   • Goal: This secondary immune study will look at additional cell types that complement project #5 above, such as monocytes, macrophages, and dendritic cells.
   • Importance: This study extends our immunologic understanding of the disease and its extent.
10. Treatment: Phase 3: Natural and Over-the-Counter Substances
    • Goal: Examine the potential benefit of several over-the-counter/natural therapies in a vetted scientific setting. Substances examined will include Moringa oliefera, GcMAF, Vit B12, and artemesin.
    • Importance: This project will be a first application of vetted scientific method and molecular science to non-pharmacologic substances that have had anecdotal benefits reported, thereby setting a standard for mainstream measurement of ME/CFS.

OMI-MERIT Initiative Signators (in Alphabetical Order)
Lucinda Bateman, MD (Fatigue Consultation Clinic & Univ of Utah, UT, US)
Alison Bested, MD (Complex Chronic Disease Clinic, Canada)
Yenan Bryceson, PhD (Karolinska Institute, Sweden)
Ron Davis, PhD (Stanford Genome Technology Center, CA, US)
David Dreyfus, MD, PhD (Yale/Private practice, US/Israel)
Oystein Fluge, MD (Haukeland University Hospital, Norway)
Mady Hornig, MD, PhD (Columbia Univ, NY, US)
Nancy Klimas, MD (NOVA Univ, FL, US)
Andy Kogelnik, MD, PhD – Chair (Open Medicine Institute, CA, US)
Charles Lapp, MD (Hunter Hopkins Center, NC, US)
Jay Levy, MD (UCSF, CA, US)
Alan Light, PhD (University of Utah, UT, US)
Kathleen Light, PhD (University of Utah, UT, USA)
Sonya Marshall-Gradisnik, PhD (Griffith University, Australia)
Mauro Malnati, MD, PhD (San Raffaele Scientific Institute, Italy)
Olav Mella, MD (Haukeland University Hospital, Norway)
Jose Montoya, MD (Stanford University, CA, US)
David Patrick, MD, PhD (Complex Chronic Disease Clinic, Canada)
Dan Peterson, MD (OMI and Sierra Internal Medicine, NV, US)
Simone Pensieroso, PhD (San Raffaele Scientific Institute, Italy)
Charles Shepherd, MD (Private practice, UK)
Ila Singh, MD, PhD (Mount Sinai School of Medicine, NY, US)
Carmen Scheibenbogen, MD (Charité Berlin, Germany)
Chris Snell, PhD (University of the Pacific, US)
Eleanor Stein, MD (Private practice, Canada)
Staci Stevens (Pacific Fatigue Lab, US) and
Rosamund Vallings, MD (Private practice, New Zealand).

We are adding more all the time. If you are a physician or researcher and want to join us, please contact the OMI.

Of course there are many other private institutions/foundations that also would be worthy of a donation - here are a few others that I would feel comfortable making a donation to fund their research initiatives.

Simmaron Research - Dr. Dan Peterson - http://simmaronresearch.com/

Stanford Medical School's ME/CFS Initiative - Dr. Jose Montoya - http://chronicfatigue.stanford.edu/

Perhaps others will have suggestions of other private institutions/foundations that they feel confident enough about to support. If you decide to donate to any research initiative be very careful that they have enough transparency/accountability that you can see where your dollars are being spent.

I do have concerns that there may well be a man (men) behind the curtain who does not have the best interests of ME patients on his agenda.

 

[*GG*]

This is very hard to swallow.

At the same time, based on how government government agencies have acted for as long as I can remember, it sounds like the hard truth.

What? That we shouldn't count on the gov't and Private Foundations is where it's at? I don't see why we should keep banging our heads against the wall, expecting things to change. Why would they change now? Money's tight!

GG

 

[justinreilly]

Ruscetti noted in the old days before there was an NIH, all research was funded by private foundations.

By private foundations for funding, I think he was talking more about apply for grants from foundations that have nothing to do with ME, like the Rockefeller foundation, etc. But he didn't specify, so I don't know.

 

[justinreilly]

Dr. Enlander said: This is NOT a psychiatric disease!! It is a physical disease.
Applause. Right then his phone rang loudly. I said "Simon Wessely calling." Thanks for those of you who politely chuckled! : )

 

[justinreilly]

This email from my Mom (72yo) sums things up nicely:

"Good morning Justin!
Dad and I are very glad we attended the symposium yesterday. Much eluded us but we had a sense that things are moving forward on the ME/CFS front compared to the symposium two years ago. We see that Eric is enthusiastically charging ahead & Mikovitz is not in jail"

 

[justinreilly]

It was a little funny. Dr. Enlander in his dark suit and french tie standing next to Eric at the podium in his shorts and bookbag. I think that Eric had a short sleeve polo shirt, instead of a tshirt i think he wore two years ago. I guess he's getting uptight being on the east coast so long.

At first I thought Dr. Gupta was a sharply dressed intern or resident. I was surprised he was so young looking. (Im not sure why I am doing the runway report, not my usual, but it just kind of struck me)

Eric knows how to give a good powerpoint! The graphics were tight.

Sorry to sound chipper like Cort on a conference high, but all this stuff he and Klimas are doing with networking is so exciting. He hasn't done much with ME yet, so there wasn't much to show specifically except there was at least one slide where he showed a representation of a network of genes associated with neuroimmune disease and all the many aberrant genes were clustered together (rather than randomized as would be expected if there were no connection between this network and ME).

He and Klimas emphasized that its not just which biomarkers such as cytokines and genes were aberrant, but how they interacted with other cytokines and genes. For example, Dr. Klimas showed that in normal controls, IL1 produced in response to exercise did not trigger any other cytokines, hormones, genes, neurotransmitters, etc. Whereas in GWI, the exercise induced IL1 caused a huge cascade of pathology including up regulation of many specific messenger molecules. This is vital to know because we can focus treatment then on the 'heavy-hitters' like IL1 that cause all the other problems instead of trying to down regulate all several dozen molecules up-regulated downstream by IL1.

Eric also is taking a network model approach to the pathology. He seems to take it a big step farther, although he hasn't done much yet on ME. He collects tons of data from many patients and then somehow sets up a model of the networks 'in silico' as he called it, so he could do virtual experiments on the computer very quickly to see what tweaking this or that molecule or group of molecules would do to the network.

In this way he can then correlate the computer model predicted positive changes on the one hand to already existing interventions such as specific drugs, nutrition or exercise on the other. He gave two good examples of drugs his lab had repurposed in this fashion. One was an unexpected existing drug that turned out to have efficacy on some type of severe GI inflammation/ulceration, iirc. The other one just floored me. He said his models predicted that an old tricyclic antidepressant would work on lung cancer! They did a trial and it worked better than the most recently approved lung cancer drug!! Wow! I can't wait for him to get further into ME.

Imo, If you are looking to donate money to ME research, I would start with the Mt. Sinai project.

CAA has a grant out for a company doing some kind of computational prediction of drugs that could be repurposed. I'm sure they are not as good as Eric, looking at networks etc, but hopefully they are on the right type of track.

Klimas looked like she was doing really great stuff. But the constant 'chronic fatigue', 'chronic fatigue' out of her mouth was like nails on the chalkboard as usual. Every time she said 'chronic fatigue' I wanted to yell out 'syndrome slash M E!"

I heard the next one will be in a couple of years again. Can't wait.

 

[Nielk]

Thank you @justinreilly for this. I almost feel like I was there, with the runway report...

 

[justinreilly]

Eric also said something interesting about how he is able to do virtual randomized trials on the computer. I didn't understand it, but it was something like he figured out that during cellular reproduction, when the alleles cleave in two this creates sufficient randomization for a trial. Sorry I don't have the head for science. He said this insight was named one of the top 10 science ideas for whatever year a few years ago.

If you like Judy you will love her talk, if you haven't heard it before from another conference (don't follow her so I don't know how much of her presentation was new). I did not understand it, but it actually seemed convincing even though I didn't know what she was saying (as i think i have said before)! Just her approach of laying out and explaining the right primary evidence, result after result, study after study, boom, boom, boom impressed me. I had seen a couple of her talks on the internet, but it kinda wasn't the same, I guess being in the room; she has a 'convincing presence' for lack of a better term.

This is probably old news to some of you, but I think she was saying that her samples were not contaminated in the science paper, which she proved by getting the samples out again and western blotting, but the fact she couldn't reproduce the results in the Lipkin study meant the finding wasn't yet valid (Im not sure of the wording she used here, but she seemed to be saying that now they were 'officially' not valid results, but seemed to be allowing for the possibility that they could in the future be replicated).

She also talked about XMRV-2 which was discovered by another scientist and was very homologous to XMRV. But which got quickly renamed to something else 'under political pressure', but she still likes to call it XMRV-2. Noted that her skeptics had been saying that the origin of XMRV was such an unlikely event , that it wouldn't happen again, But she points out that it did happen again in XMRV-2. She is worried that these and other hardy gammaviruses which can spread by air and replicate fairly quickly will at some point be able to adapt to infect humans.

Which is, fwiw, a worry of mine too. It seems like a fine line between a 'lab contaminant' and a plague.


She said other things my brain wasn't able to absorb.

 

[leela]

Erm, Eric Who? I haven't sufficiently dosed on the green tea yet today, sorry.

Runway report=hilarious

 

[Purple]

Eric Schadt - http://en.wikipedia.org/wiki/Eric_Schadt

 

[Wally]

Justin,

Thanks so much for attending this talk and reporting back on what you learned. I thoroughly enjoyed the "runway" critique and your Mom's e-mail. It is nice when a little levity can be thrown into the mix.

I had posted earlier, in response to the comment from Frank Ruscetti about private funding of ME research, about three (3) private research initiatives that I would feel comfortable donating to. I was hoping that someone would make a comment about others.

Imo, If you are looking to donate money to ME research, I would start with the Mt. Sinai project.

Can you provide a link to where people can find more information about the Mt. Sinai project?

Wally

 

[justinreilly]

Wally,

Thanks for your interest. Actually, I couldn't find any official webpage on the center, which needs to change. I sent an email to his office and patient Jay Spero who helps Dr. Enlander with his web presence re registering that opinion. That may have to wait until the IoM thing is over.

In the mean-time, the reason I would give to the Mt Sinai ME CFS Center is
(1) the quality of the research as I spoke about (as best I could)
(2) it is the only ME research or clinical center at a leading teaching hospital, which counts hugely for credibility in the medical world
(3) the marquee name of Eric Schadt

These last two may seem superficial, but we all have experienced how doctors are unduly enamored of authority. These last two reasons mean that ME and bona fide ME research will be accorded more respect in general and the related benefit that their groundbreaking research will get into top journals much easier.

Mt. Sinai has been rising in the rankings lately as they get huge donations from NYC billionaires and have sold several fifth avenue residential buildings to raise capital which they have used to massively increase floorspace and cutting edge equipment and also lure top docs from other hospitals. Mt. Sinai is on a roll that we can benefit from.

It's also my understanding that the hospital throws in its own money for the center in addition to donations specified for it, but I will check on that.

(The best I could do is this donation form, but I like you would want to know more before I donated; the best info will be the conference DVD when it comes out)

https://philanthropy.mountsinai.org/donate

 

[justinreilly]

Another thing, not ME related really, that Eric S. said that i found interesting:

He's collaborating with Mars, which owns pet-clinics as well as mars bars, etc. It turns out homes with pets are more likely to share similar micro biomes among family members. They would test the dogs micro biome, maybe because its easier than testing the humans, not sure. Then they were thinking of trying to change the whole family's micro biome by changing the dogs diet and i guess adding probiotics.

Out of the box, this guy.