March 7, 2018

NIH ME/CFS Advocacy Call - March 2018


Moderator: Margo Warren
March 7, 2018
1:00pm ET

Coordinator:  Welcome and thank you for standing by.  At this time, all participant lines are in a listen-only mode. After today's presentation, you will have the opportunity to ask questions and you may do so over the phone by pressing * then 1 at that time.  Today's conference call is being recorded.  If you have any objections, you may disconnect at this time.  Now I will turn the call over to your host for today, Ms. Warren, you may begin.

Margo Warren: Thank you. Good afternoon.  I’m Margo Warren. I'm from the Office of Communications and Public Liaison at the National Institute of Neurological Disorders and Stroke. On behalf of the NIH, I would like to welcome you to this afternoon's teleconference.  And thank you for your interest in participating in this discussion with us today.  Dr. Walter Koroshetz, the director of NINDS and the chair of the Trans-NIH ME/CFS Working Group, will introduce the speakers, each of whom will make some remarks. After which, we'll open the phone call for your questions.  We will try to make our remarks brief so that we can answer as many questions as possible in the time remaining this afternoon.  Now, Dr. Koroshetz.

Dr. Walter Koroshetz: Good afternoon everyone. And thanks to those listening. And we look forward to trying to communicate the activities on the research side that NIH is supporting in our efforts to understand ME/CFS and develop treatments for people who are suffering with this illness around the country.  And so as we had last time, we are overjoyed to have Ian Lipkin join us who is one of the principal investigators on the newly minted ME/CFS consortium to tell you a little bit about what his project is about, and we look forward to bringing in the other PI hopefully on our next call. So you get a sense of what's going on with the consortium as it develops.

Dr. Nath, Avi Nath, who's our Clinical Director here in Bethesda for the National Institute for Neurological Disorders and Stroke, as folks may know, is working on a protocol to investigate potential causes of ME/CFS, which is taking advantage of some of the unique features of the NIH. Those being the fact that there is a research hospital here and so particularly the people suffering with chronic fatigue, where travel and, you know, the stresses of going through a whole bunch of tests on one day are really too much.  We can bring people into the hospital here and do things on a schedule that fits the patient. And so his protocol is moving along quite well. They have an extensive list of studies that are being performed on each individual.

And he reported to me today, unfortunately he got called away to an urgent meeting at the hospital to solve a problem there. But so far, the protocol has had about 220 inquiries. And to determine whether patients are actually eligible for the trial, there are certain eligibility requirements with regard to the diagnosis of ME/CFS and some of the ability to go through some of the testing.  So they've reviewed 70 charts so far.  And they have enrolled for the Week 1 visit, well they enrolled totally 14 yeah, 13 patients with ME/CFS and 16 healthy volunteers to do the comparison studies. And I believe that 10 patients have been have completed the study so far.  So things are working well.

It is, he's still looking for more patients who fit the inclusion criteria and so would be interested, if there are patients out there who would be interested in participating, contact Dr. Nath at the intramural NINDS.  So I think that was the update from the intramural program.

And now I'd like to turn it over to Dr. Breen to talk about the Working Group activities. Dr. Breen's from the NIAID, National Institute of, Immune and Infectious Disease.

Dr. Vicky Whittemore:  Allergy.

Dr. Walter Koroshetz:  Allergy and Infectious Disease. We only deal with acronyms here unfortunately.  And Dr. Vicky Whittemore from NINDS and that stands for ... the National Institute of Neurological Disorders and Stroke. Okay Joe?

Dr. Joseph Breen:  Thank you, Dr. Koroshetz.  So I want to give you a brief update of where the Collaborative Research Centers and the Data Management Coordinating Center are in terms of their activities since the last telephone briefing which was in November, I believe.  Their activities fall into two categories.  One is scientific and then the second is really the coordinating role. Since the last telebriefing, each of the centers, the three research centers, as well as the Data Management Coordinating Center, have had their initial kick-off meeting, where they bring in all the scientists and actually the entire team that involved in each of those.

And that actually turns out to be important because, you know, when the applications are written, if they include scientists and experts from disparate parts of the country actually. And that, getting in one place as the kickoff meeting to make sure that everybody has a clear objective is really important to get a project started like this. It's important for each of the centers as well as for NIH program staff. It's a wonderful chance to interface and see what the status is and what the plans are again for each of the centers.  So that's happened in the last few months. 

Dr. Whittemore and I attended a few of these and I won't go into more detail. Dr. Lipkin had his introductory meeting in January in New York City.  So that's the science side. The teams are starting to really move. Starting to produce, actually, data as they said they would.

The other side which is important in this venture is really the collaborative nature. And in that vein in December, all the PIs and some of the top, key personnel from each of the centers of the Collaborative Research Centers and the Data Management Coordinating Center got together actually here in Bethesda to really have their first face-to-face meeting and decide how they were going to integrate some of their activities. Namely one of the principles was the collaborative projects that they're all, will be undertaking in this year. 

So it was, it's a little bit of getting to know each other. Not all these groups knew each other previously. But also really, how are they going to operate? How are they going to interact and really synergize and build the basic infrastructure around this ME/CFS as intended by the program?  And we were fortunate enough at that meeting we actually had representatives from the Canadian Institutes of Health, CIHR, Canadian Institutes of Health Research were also at the meeting. Because they hope to collaborate with NIH funded centers in the future. And they also announced plans for an upcoming meeting which they'll be holding in Canada in May 2018. So we hope this is a real fruitful collaboration going forward. 

So again, it's an indication of really trying to broaden the base, get more people looking at the science behind ME/CFS and really, better understand the disease from diverse perspectives. Again, from presumably a different patient cohort population that we can then put together and really hopefully execute some collaborative studies to give some real power for the future.  So the collaborative projects, there were some discussed actually on the fly at that meeting, which I think will be a real advantage to the whole network.  And as well in each individual center is really going now. And they are, I know because we see some of the progress that they're formulating manuscripts and working on and trying to move forward and it's an exciting time.

And so it's a mixture right now of beginning to collaborate together as well as undertake this science in each of the centers. And I think I'll stop there because you're going to get a view of Dr. Lipkin's center from Dr. Lipkin himself in a little bit.  And I'll turn it over to Dr. Vicky Whittemore who's going to tell you about some of the other Working Group activities.

Dr. Vicky Whittemore:   So the, as you know, if you've been on the call before, we've talked about the Common Data Elements project which we have initiated together with the CDC. And this was a project to develop common ways in which to report, to analyze, and report data from clinical studies for ME/CFS. And the advantage of this is if someone is, for example, studying sleep or fatigue or pain as it relates to individuals with ME/CFS, the data would all be reported out the same and be able to be compared across studies regardless of where those studies were done.

So the initial draft or the initial version of the Common Data Elements have been completed and posted. It was posted for public comment and we received numerous comments on the Common Data Elements. And those will be, the comments themselves, will soon be posted also on the NIH ME/CFS website. But the Common Data Elements are also posted and available and are being implemented now in studies at the centers and we hope by other researchers as well.

And we also know the researchers that are interested in studying Gulf War Illness are incorporating many of the Common Data Elements and will be modifying them also for their use. So because there's some similarities in symptomatology between the two diseases, we'll be able to compare across those studies and patient populations as well.

And the other thing I wanted to report on is that we've now signed, Dr. Koroshetz signed, a memo of understanding with the Hutchins Family Foundation which was a family foundation that supported CFI, the Chronic Fatigue Initiative, and this Initiative collected biospecimens and clinical data from over 300 individuals with ME/CFS. And those samples now that have been, that are currently stored at Duke, will be transferred to the NINDS-funded biorepository that's at Indiana University that we call BioSEND and the clinical data that's currently at Columbia will be transferred to the Data Management Coordinating Center that's part of the ME/CFS consortium. And then the clinical data and the biospecimens will be linked such that going forward, any investigator who would like to access the biospecimens and the clinical data will go through an application process to gain access to those samples.

So CFI wanted a way in which these samples could be made broadly available and the clinical data broadly available to the investigators across the community. So we're thrilled to be able to partner with them in this way.  And those ae my updates for today. And I'll turn it back over to Walter.

Dr. Walter Koroshetz:    Great, okay, so as mentioned, we're going to ask Dr. Lipkin to talk to us about his project. And after that, we'll have the session will be open for questions to anyone around the table or Dr. Lipkin.  So Ian thanks so much for getting on the line.  As you may know, we have these telebriefings every couple months to update the community about the research that's going on in ME/CFS field and I'm sure everyone will be real interested to hear your thoughts.

Dr. Ian Lipkin: I'm pleased and honored to participate.  So let me just give you a bit of introduction to me personally and to the center and perhaps to the Chronic Fatigue Initiative that Vicky's just mentioned.  So like Walter Koroshetz and Avi Nath, I'm a neurologist.  I spent the majority of my career, however, focusing on understanding the role of infection, infectious disease and the immune system in chronic diseases ranging from autism to chronic fatigue to cancer.  We have a history here of building large interdisciplinary interinstitutional teams where we can bring the best minds together to focus on a problem and I'm thrilled that this has now been turned towards CFS.

My interest in ME/CFS is not recent. It actually goes back to the mid-'90s when I began working with people at the CDC on both chronic fatigue as it was then known as well as Gulf War Syndrome. At that point there was a suggestion that was due to a virus called Borna disease virus that we discovered some years earlier. And I worked at that point with (Birgitta Evengard). We published a paper in '97 that demonstrated that people who had ME/CFS had polyclonal B cell activation, which simply means that the immune system was in overdrive. And this was some of the first evidence to suggest that this was biologically rooted rather than being a psychological condition as many people were discussing at that time.

A few years ago I met Glen Hutchins who was personally engaged with addressing the problem of ME/CFS and I helped him with two of his colleagues, Harry Schroder and Scott Carlson, to develop this program.  That was sponsored by the Hutchins Family Foundation known as the CFI that collected the specimens and the data that you just heard will be deposited in biobanks and at the data coordinating center.

And much of what we do here is based upon that early work.  Both in looking at the role of XMRV which was the first project that we did with NIH in this respect. And then with CFI and more recently with what many of you may know as the Microbe Discovery Project, a crowd-funding initiative that was developed really in collaboration with Vanessa Li who as some of you will know took her life in despair. And it continues to inspire us on a daily basis. And without this crowd-funding initiative, we would not have been able to make the headway that we have.

The people who are participating in this group represent both basic scientists as well as clinicians. The clinicians are probably will be known to many of you: Dan Peterson, Sue Levine, Cindy Bateman, Jose Montoya, who are people who worked in this field for many, many years. And then on the basic science side, we've recruited an individual who runs one of the largest metabolomics laboratories in the world. That's at UC Davis. This is a core for the National Institutes of Health underneath Oliver Fiehn, John Greally who's an expert in gene expression and epigenetics which is the ways in which the genome is modified so that you can see expression of a gene or not.  That John Greally is located at Albert Einstein, Dana March who's the Deputy Director here, and in addition Tony Komaroff who's one of the senior-state people in this field who directs one of the three projects that we support.

We work very closely with ME Action and with Solve ME/CFS. And Avi Nath is a very old and dear friend of mine. He's not an old friend. He's a long-time friend of mine, and we're also working closely together as well.  We're building on the data repository that you've heard, plus the one established with the Microbe Discovery Project and the biobank as well.  And the focus or I should say the foci of our work are on trying to understand the role of the microbiome. And I'm using that term in a larger sense. We look at viruses, bacteria, and fungi in the blood and in the intestines, both the lower intestine and the very low intestine meaning fecal samples as well as in the oropharynx.  And we also look at blood and these other compartments for what we call biomarkers that might be useful in understanding why people feel ill. And in trying to find ways in which we can get markers that would be useful when we have real solutions to offer in terms of drugs, modifiers of the fecal microbiome and so forth.

We are very interested in the impact of exercise.  And we're interested in POTS disease so we have ways in which we look at individuals who have problems standing upright. And having difficulties with cognition and difficulties with exercise that manifests immediately but continue for several days.  And we believe that this is likely to be, when we talk about ME/CFS although it's convenient to talk about it as a single disorder, we believe actually that there will be many, many subtypes. And as a clinician as well as a scientist I can tell you that I've seen many patients with these disorders and there are people who respond to antivirals and there are other people who respond to modifiers of the microbiome. And there are other people who respond to drugs that alter neurotransmitter levels. And I'm not certain that these are all the same. But they manifest with fatigue and cognitive dysfunction.

We also have an effort to try to identify these phenotypes by mining these databases that we have, using state of the art methods that are typically employed by people who want to figure out whether or not the stock market or particular stock is going to go up or down.  Machine learning, ways in which you find patterns that may not be readily apparent using traditional methods for analysis of complex sets of information.  We're also building a phone app, a cell phone app. This work that's led by Dana March and Tony Komaroff that will allow patients and caregivers to track the course of disease and our objective here is to identify triggers that we think might be important in allowing people to feel better or feel worse. And that will, of course, allow us then to say this is the time when it's optimal to get samples when we allow us to follow responses to treatment. And our ultimate goal, of course, is embedded in the name for our center. It's called Centers for Solutions for ME/CFS.

So we're not approaching this purely from the vantage point of what is going to be intellectually interesting. We want to come up with real solutions for real problems so that we can make it possible for people to become active again. And as I say, a guiding principle in all of this is my own interactions with patients over more years than I'd care to count. And in particular, the late Vanessa Li who approached me at a conference in San Francisco four years ago and asked me how can I help? 

So we're trying to return that favor and we are heavily invested in trying to find ways in which we can bring all the different tools that we required over the past few decades in dealing with challenges like biodefense, which would never have been covered with the kinds of research dollars that are allocated here, to bring those same tools, genetic tools, proteomic tools, metabolomic tools, data mining tools, to bear on the problem of ME/CFS, POTS disease, Gulf War Syndrome and anything else that we might discover during the course of this work. 

Thank you very much for your support. We deeply appreciate your efforts and we cannot do this without you as partners.

Dr. Walter Koroshetz: Thank you very much Ian and so I think now we will be able to open up the phone lines for questions to Dr. Lipkin or the folks here at NIH.

Coordinator: Thank you. At this time, if you would like to ask a question, please press * then 1 on your phone's keypad. Please unmute your phone and record your name at the prompt.  If at any time, your question has been answered, you can remove your request by pressing *2. Once again, that is *1 if you would like to ask a question over the phone at this time.  Please stand by for our first questions.  And our first question comes from Eileen Holderman.  Your line is open.

Eileen Holderman: Good afternoon and thank you for the opportunity to ask this question.  I'm Eileen Holderman. I’m an activist. Recently we saw some statements made by NIH that the study and research for ME was housed in NINDS. And we realize that the NIH intramural study for ME/CFS is being undertaken there. But can you please clarify for us whether or not the general research for ME is officially housed in that institute? And if not, can you tell us where it is housed?

Dr. Walter Koroshetz: Sure, so good question. NIH is very complicated and so it does take a little bit of explanation.  So I would say that just to cut to the chase that the type of science and the questions that need to be answered to solve the problems for ME/CFS could potentially come out of multiple different institutes. And truth be told we don't actually know which, where the disease is starting. And we think that the best strategy is for as many institutes to be involved as is appropriate. And so we have a Trans-NIH ME/CFS Working Group and they are the ones, actually all of those institutes, are helping to fund the consortium. So it's a joint effort.

Now like any type of joint effort, there needs to be a leadership taking responsibility and so the leadership of the Trans-NIH committee is myself from NINDS and Dr. Tony Fauci from the NIAID, the National Institute of Allergy and Infectious Disease. And Joe Breen has been the NIAID, kind of, lead along with Vicky Whittemore of that Trans-NIH committee. So that is how it's set up currently at NIH.

Dr. Ian Lipkin: This is Ian Lipkin, I'd just like to add one thing to that.  Through December of 2017, I was on the committee that advises Francis Collins, the Director of NIH. There was broad support within the Director's office and across the institutes for the establishment of this program. I have been funded in the past by NINDS. At present I’m funded by NIAID.  But we are working very closely across the institutes wherever we can find the best expertise to bring solutions in this problem. Thanks Eileen.

Eileen Holderman: Thank you Dr. Lipkin.  I just have a quick follow up about something that you said.  You mentioned that this may be many diseases. And my question because I focus on case definition and using the expert's definition, particularly the International Canadian Consensus, is it because there is no standardization, no acceptance of our expert's definition that we have such different research results?  Because that's what most advocates believe.

Dr. Ian Lipkin: An interesting question that you've raised. And I'm not sure I can get at it directly. Maybe I can give you an analogy.  Because I think we're still in the early days of understanding what, you know, what comprises this set of syndromes.  If you talk to people 50 years ago about cancer, you know, we're going to have a war on cancer. There are many different types of cancer. And they have different causes and they have different solutions. And I think we may be in the same situation with ME/CFS.  I'm not saying that there are going to be as many types as there are forms of cancer, but we have some individuals who have a rapid onset following what seems to be an infectious illness. There are other people who seem to have an allergic prodrome. There are other people from whom this comes out of the blue. I'm not certain that these are all the same thing.

And then you have some individuals who as I said earlier appear to respond to changes in diet that alter their microbiome. There are other people who respond to herpes medications. So until we have a better set of what I would like to refer to as biomarkers, that allow us to distinguish between all these different types of ME/CFS, it's going to be difficult to come up with a coherent plan for diagnosis and treatment. And I think what we're doing right now is chipping away at what we need to learn as rapidly as we possibly can. 

So I'm encouraged by this because I think what it tells us is that as we understand more about this set of illnesses, some people will be treatable with one set of approaches and other people with a different set of approaches. Now the other question that you posed was to the extent this might reflect issues with people who are less sophisticated in making a diagnosis or not. And I think this has been a problem in the past.  But I think since the Institute of Medicine report made available criteria that are a little easier for physicians to understand, I think that's less the case. I do believe that as we begin to characterize these materials that the Hutchins Family Foundation, others have made available, and we made samples available earlier through the XMRV project, we will find markers that are very helpful.

So for example, early on Mady Hornig and others found that there cytokine and chemokine disturbances that were present in both blood and spinal fluid, which were reflective of different types of disease course, different length of disease, duration of disease. And I think we're going to find things that are similar. We have a paper that's now finally getting through review where we find that there are individuals who have irritable bowel syndrome. Others who have a high body mass index. And they seem to differ in the chemicals that we find in their blood. And there may be clues here too. 

Eileen Holderman: Dr. Lipkin I really appreciate that. Do you advocate for the use of one standard case definition for research and one for clinical study?

Dr. Ian Lipkin: Well I think Dr. Whittemore mentioned early on this effort to try to establish Common Data Elements. What we are trying to do is to find ways in which we can, what's the word I'm looking for, formalize definitions even more than they are now.  And I think that will be helpful.

Dr. Walter Koroshetz: Thanks so much. Let's go onto the next question please.

Coordinator: Certainly. The next question is from Tanja Bugas.  Your line is open.

Tanja Bugas: Hi, Tanja Bugas in Littleton, Colorado, patient of ME/CFS for 31 years.  Wondering if anyone is studying in-depth interviewing with patients to kind of get the big picture of what happened to us, what caused the illness?  What were the factors leading up to the illness to make us, kind of, a final common pathway? And I know Dr. Jones always told us chronic fatigue patients here in Denver that, you know, there probably are many predisposing factors that trigger this. There's probably some kind of a stressor. You know, we have stressful events in our lives, a pathogen, a genetic predisposition, and an environmental trigger.  And all of these together then cause us a switch to be flipped or the brain to have exceeded its stress thermostat so to speak.

So I'm wondering is anybody actually like looking at all of these together? It seems like research tends to be focused on just a virus or, you know, something very specific. My analogy would be if you're trying to understand what an elephant is, you know, if one person is studying the tail and another is studying the trunk, you may not - you may be missing the big picture.  You know, what is the elephant? Can anybody tell me if there's anybody out there researching, you know, just really in depth doing interviewing with our patients and trying to figure out why do we stay unwell? Thank you.

Dr. Vicky Whittemore: So part of the multisite CDC study has been taking, through the clinical sites that they're working with, have taken extensive patient histories. So that's part of that multisite study. I know also from, and Ian may want to comment on this, is that part - some of the studies within the collaborative centers will also be taking past exposures and past illnesses into account as they take the histories of patients and individuals who participate in the studies through the various centers. I don’t know Ian if you want to add to that?

Dr. Ian Lipkin: Well I will tell you that Tony Komaroff and Dana March as part of their effort to collect information on this app and look at databases, are trying to get information of this sort.  I'm not aware of any detailed interview plan which is what you're - which is what Tanja has described and this is something that I think we should consider. We do take histories and we try to integrate that information into these databases, but I don't know that it has the detail required to capture what Tanja's referring to. 

And one of the reasons these teleconferences are so important and the engagement with the community is so important is we get ideas about the way we can modify and improve the approach that we're taking to doing research. So thank you for that suggestion.

Tanja Bugas: Thank you.

Dr. Walter Koroshetz: Go onto the next question please.

Coordinator: And the next question comes from Donna Pearson.  Your line is open.

Donna Pearson: Thank you very much.  Whenever we talk about all of the varieties and perhaps there are subsets and there are all these different triggers, I always have a hard time reconciling that back to the epidemics.  When so many people got sick at the same time. And I'm wondering is that always being thought of as you go through your different hypotheses in terms of what could be causing the disease and what treatments could be helping. Because I always wonder if because we're susceptible to opportunistic infections, some of us may have viruses. Some of us may have bacteria. Some of us may have mold toxicity. And that's why different medications help some of us and not others. But the underlying issue may be the same or may be similar.

Dr. Walter Koroshetz: I think everyone's thinking that direction, yeah. I think that I can speak on part of the intramural program here that the big focus is an extensive evaluation with all the possible technologies available to try to see if there's a signal that jumps out in the ME/CFS patients in terms of potential exposures, particularly infectious agents, microbiome. And so that is on the back of everyone's mind. And Ian is, again, you know, as he mentioned, that has been a focus of his research over his entire career. And yeah, so at the intramural program, they're basically specifically focusing on patients who develop ME/CFS after an infectious-like illness with the hope that they're going, that in that population which is a, you know, not representative of everybody, but a significant proportion, that they may be able to see, you know, what was the inciting event.  I don’t know Ian if you want to add to that?

Dr. Ian Lipkin: I think you've addressed it well Walter, but I will be happy to add to it.  There is, there's a challenge with infectious diseases in that, after with most infectious diseases they infectious agent is only present in the blood for example, for a short period of time, a week maybe two. There are exceptions. But you can't really find the agent itself. And that's the easiest way to make a link between an infectious agent and a disease.  Now over the longer course, however, there are antibodies which represent the immune response to a specific agent and you can find differences in the levels of antibodies that are used, that are targeted against specific agents in people who have disease and people who don't. 

So we've published in the past month two papers that illustrate this point I think quite well. One focused on tick-borne illnesses because you frequently cannot find direct evidence of Borrelia in an individual who's been bitten by a tick and where you find infection unless you're lucky enough to get the rash and to biopsy the rash at the time that the patient first gets exposed.  More likely that you make a diagnosis by finding antibodies to the bacterium that was carried by that particular tick. And then yesterday we published a paper in which we talked about Zika. As you know, the risk of, you know, of mental retardation and microcephaly with Zika persists for a long period of time after the blood will have left - the virus will have left the blood of the mother. So you have to find ways in which you can detect the antibodies as markers for the presence of something - a fossil if you will, to indicate what happened. And then you can reconstruct what someone might have seen.

Part of what our center is doing is taking this sort of technology that allows you to look at three million peptides. These are small protein fragments so that you can describe a wide range of illnesses that might be associated with triggering disease. And they can be fungi, bacteria, viruses to address the problem that you've described.  Now there are a few examples where there appear to have been an outbreak that affected many people simultaneously.  Unfortunately, those outbreaks such as the Incline Village outbreak and there was another one in Sweden. There are no samples left that we have been able to find that we can access to study using these kinds of methods. So we're increasingly focusing on the use of these antibody-based technologies that are less well developed to try to figure out what someone might have seen in the past.

So that I think will be fruitful and you've raised an excellent point about some of the difficulties that we encounter in trying to understand the original events that triggered the onset of disease. 

Dr. Walter Koroshetz:    Thank you. Can we go to the next question please?

Coordinator: Certainly. The next question is from Terry Gilmete. Your line is open.  Please check the mute button on your phone.

Terry Gilmete.  Hi, this is Terry Gilmete and I've had ME for 32 years now.  And I have a couple of questions. The first question is for Dr. Lipkin. I’m interested a little more about what's happening at UC Davis. What is the name of the project and who is in charge of it? And could you tell us a little bit more about what's happening at UC Davis?

Dr. Ian Lipkin: The person, so the person who's doing the metabolomic research there is Oliver Fiehn, F-I-E-H-N.  He's the director of the metabolomics center there. He's a member of the Metabolomic Society, an officer of the Metabolomic Society, a graduate of the Free University and from the Max Planck and is absolutely top draw. And when we decided to integrate metabolomics in our center, I interviewed directors of metabolomic centers across the United States, actually across the world and flew out and met with him and decided that his team was the one with which we wanted to work.

We have now done two projects with the Fiehn group: one on plasma using materials from the Chronic Fatigue Initiative supported by the Hutchins Family Foundation and the other is a set of spinal fluids that we analyzed in collaboration with Dan Peterson in Incline Village.  And I will tell you that this is tantalizing and unfortunately not very satisfactory for you. But and I wish it were different, but I can't present the data because if I do that then it will never get published.  That's just the way these things go. But I'm hopeful that they'll be out there soon.

If there were something that would immediately impact the community in a profound way, in such a way that it would change treatment and so forth, I would break that embargo. But given that it doesn't meet that bar, I can't.  But I think you will find that we are able to confirm some of the findings that came out of UC San Diego, but we have differences too.  And things that I think extend that work, change it a bit and suggest certain treatment modalities that might be helpful.

Terry Gilmete: Thank you so much.  And I have a question for NIH, please.

Dr. Walter Koroshetz: Sure thing, go ahead.

Terry Gilmete: Yes, I'm wondering about, you know, patients continue to suffer without accessibility to care. There doesn't seem to be medical tracking of folks that come down with this.  What can be done to alleviate some of the suffering that so many patients go through that don't have accessibility to the ME specialist.  Really concerned about that.  I am fortunate in that I'm able to access an ME specialist but many of the folks that I know online are not.  And they do become desperate and suicide does come to the forefront and for them, after years and years of living with this disease and not having anybody to refer to or anybody that will believe in them and help them. 

Dr. Walter Koroshetz:    Yeah, so we agree. And unfortunately, that's true for a number of our diseases particularly the rare neurological diseases, that expertise is in short supply.  And NIH is the country's research agency. So we are not the part of the government that is involved in healthcare systems. But we are confined to funding the scientific research. Now that being said, we have certainly seen is that as the science progresses in an area, that often draws clinicians into the condition. And so we are hopeful that as the research effort builds, this will bring more people who will want to become clinical experts in ME/CFS and that we can spur a greater access. But we are certainly limited because the practice of medicine is not, certainly out of our bailiwick. 

Dr. Vicky Whittemore: If I could also add, I know that the Chronic Fatigue Syndrome Advisory Committee also has a working group focused now on, excuse me, on medical education. So it's really focusing on how to get the information out to a broader network of physicians.  I think one of the things we've also talked about with some of the federal agencies is ways in which to promote telemedicine, such that a physician in a remote area or an area where there isn't an ME expert, could utilize telemedicine to work with some of the medical experts. So that's something that I think we hope to explore in the future as well.

Dr. Walter Koroshetz:    Can we go to the next question please.

Coordinator: Yes, our next question comes from Carol Barash. Your line is open.

Carol Barash: Hi, this is a question that was just previously in part answered.  It's the question for you Ian. I was interested in learning a little bit more about how you're using metabolomics and what you anticipate finding, if possible. How you're going to use proteomics, if you're working with anyone, because I didn't hear you mention a group that you had chosen. And lastly, if you're focusing on specific genes that you're looking at the gene expression of. I know at least one gene, I believe it's NCSKN1 that is associated with the inability to fight viruses, bacteria and fungal infections. And so if you could comment any more deeply on these genomic areas, and what you're hoping to get out of the research that you're doing.

Dr. Ian Lipkin: Carol thank you very much for calling in. It's a pleasure to hear from you.

Carol Barash:  Nice to hear from you too.

Dr. Ian Lipkin:  Carol and I have been pen pals for about a year now.  So we're working with Penn in proteomics. This is the reason we've done so, is that we've been doing, we have a project that focuses on emerging infections with the team there that's supported by DARPA. And I've been very pleased with the quality of the data that we've received from this particular group that's run by Ben Garcia. I don't know if you know of Ben Garcia, but he's excellent. And he's on Fiehn's level, I think, in his own field.

Now in terms of genes, one of the things that I think we need to do because there has been some discussion of looking at GWA studies. I think there's one GWA study that came out of Nevada that was done by Lombardi what was quite interesting.  But and they looked at something like 650, I'm trying to remember.  I don't want to misspeak. But in any event, they had a fairly comprehensive microarray approach for looking at exomes and as well as introns. We are hoping to move into whole genome sequencing because I think the price has now come down to the level where it's tractable.  And we need a large enough sample size so that we can find things that will be useful.

In addition, we will join this with epigenetic studies so that we can not only get the baseline but look at genes that are modified. And as you know, Carol, the important thing with epigenetics is to analyze the appropriate DNA sample.  And you can't do everything. My thought is we will probably focus initially on peripheral blood mononuclear cells because they're accessible. And because there are these immunological abnormalities that we've all defined.  My hope is that through the proteomic and metabolomic analyses we will get clues that will allow us to swim upstream to the genes that are potentially implicated. And we'll study them both in an unmodified fashion as well as in a modified fashion.

Carol Barash: Okay.

Dr. Ian Lipkin: I think that's really all I can say without getting into science fiction.

Carol Barash: And we won't go there today.

Dr. Ian Lipkin: Okay.

Carol Barash: Thank you very much. It's helpful.

Dr. Walter Koroshetz: Thank you very much. The next question please?

Coordinator: The next question comes from Mary Lemkemeier.  Your line is open.

Mary Lemkemeier: Thanks for taking my call. This is Mary Lemkemeier from St. Louis. I'm the parent of an ME/CFS patient. And I, she's been studied at NIH and I am wondering. You mentioned three different potential, sort of, trial treatments. One is changing the diet to alter the microbiome.  One is taking antiviral drugs. And I missed what the third one was. 

Dr. Ian Lipkin: So first of all, I want to be clear in saying that I'm not recommending anything. At present what I'm saying is that we have anecdotal data from individuals who have tried these different approaches and have reported that they have felt some subjective success.  It's important as with any of these sorts of things that we have controlled clinical trials because we can frequently be misled by what we call anecdotal evidence. But that's frequently where things start, with anecdotal evidence that then leads to some sort of clinical trial.

There are individuals who reported improvement with prebiotics, which is ways in which you change the nutrients that support certain bacteria within the microbiome.  There are other individuals who have reported responses to anti-herpes drugs. Many of those individuals have been followed by Jose Montoya. There are people who have been reported to respond to Ampligen, which is basically a genetic treatment.  It's a polynucleotide. Something called inosine cytosine that triggers the innate immune response. I'm not advocating for anything of these things. And then there are a group of people who have been found to have serotonin abnormalities who respond to SSRIs, which are drugs that are used to treat certain other sorts of illnesses.   The fact that those are psychiatric illnesses doesn't mean that we consider this to be a psychiatric disease.  It simply tells you that those individuals have responded to that sort of an approach.

Mary Lemkemeier: Correct.  Thank you for clarifying that. 

Dr. Walter Koroshetz: Can we go to the next question please?

Coordinator: Yes, the next question is from Susi Varvayanis. Your line is open.

Susi Varvayanis: Hi, so I'm not a patient or a parent. But I am the Executive Director of the Cornell Collaborative Research Center and I just wanted to address the question of the person who was concerned with access to specialists. And that's something I think that all of our collaborative research centers are tasked to do. We are all partnering with clinicians and patient advocates. So that we can get the news out once we do have more information about more clear criteria for inclusion to the disease. And in fact, Maureen Hanson who's the point of contact for our Cornell collaborative research center just gave a Grand Rounds a few weeks ago to talk to clinicians and say, you know, here is some of the things that we found and here are things that you shouldn't be prescribing for instance.

So I think we're all going to be working towards that. And hope to have better information for physicians in the future.

Dr. Ian Lipkin: So this is Ian Lipkin. I think Maureen has done an excellent job of highlighting some of the things we now know.  It is true that we have a shortage of people with expertise in ME/CFS because it's a challenging disorder or set of disorders to study.  We tried to help Dan Peterson put something together so that he could recruit people who could help take over his practice. I know that everyone who's, I've spoken to who's in practice who has a focus on ME/CFS has expressed concerns that there aren't enough hours in the day for them to see the patients who want to see them.  And frequently the waiting lists are months long.

So we do need to do a better job of persuading people to go into this field. And as Walter Koroshetz has said, as we develop real treatments, better diagnostics, we hope to be able to attract more clinicians to the field and we hope to be able to do as you just heard find ways in which general practitioners, internists, family medicine people can also be brought on board to help share the responsibility for taking care of these people who've been neglected for so long.

Margo Warren: We have time for one more question. I know there are many of you waiting to ask questions, but please send any of your unanswered questions to our email Or go to the NIH ME/CFS website and click on “contact us” to submit those questions.  So let's hear one more question please.

Coordinator: Certainly. Your final question comes from Sandy Carl.  Your line is open.

Sandy Carl: Yes, thank you. This is a question for Vicky Whittemore and in general NIH. What is the status of the proposed interagency collaborative to advance research on ME/CFS that you, Dr. Whittemore mentioned at CFSAC in December.

Dr. Vicky Whittemore; So we're still in the planning processes and hope to be able to finalize that within the coming weeks.  It's something that we think is very important, to bring together the representatives from the federal agencies and patient advocates. So we're moving forward with trying to figure out the infrastructure to how to make that happen.

Sandy Carl: And when you say finalize that in the next, I'm sorry, couple of weeks.  What does that mean?  In other words, when will, I mean I'm guessing that means, kind of, figuring out a plan. What do you, can you just talk about a timeline for both planning and possible implementation?

Dr. Vicky Whittemore:   So we need to finalize, well so let me back up. So we have a Trans-NIH Working Group meeting coming up in March where we will discuss this. And then we will have to see what the steps are to actually implement it. So I'm, I think we're hoping to have something in place within the next several months, but I can't give you a more specific timeline that that.

Margo Warren: I want to thank everybody for joining us today. A recording and transcript of the call will be posted to the NIH ME/CFS website next week.  In closing the call, I'd like to remind you about our list serv for updates from NIH.  If you received a message about today's call from the NIH ME/CFS Working Group, then your email address has already been added to the list serv. If you received a notice from a friend but would like to be added to the list serv, please visit the NIH ME/CFS website, which is  And click on join our list serv at the bottom of the left-side bar.  Thank you all for an informative and thoughtful discussion and I hope you have a great afternoon.

Coordinator: Thank you for your participation on today's conference call. At this time, all parties may disconnect.

This page last reviewed on March 26, 2018