ME/CFS Advocacy Call - July 19, 2018

Transcript

July 19, 2018

Operator: Welcome and thank you for standing by. All participants will be in a listen only mode until the question and answer session of today's conference. At that time, you may press star then 1 to ask a question from the phone lines.

Also, this conference is being recorded. If you have any objections you may disconnect at this time. I would now like to turn the conference over to your host, Ms. Alissa Gallagher. Thank you, ma'am. You may now begin.

Alissa Gallagher: Thank you and good afternoon. My name is Alissa Gallagher and 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'd like to welcome all of you to this afternoon's teleconference and thank you for your interest in participating in this discussion with us today.

Dr. Koroshetz, the Director of NINDS and the Chair of the Trans-NIH ME/CFS Working Group will introduce our speakers who will provide some updates on the work going on in the field. After these updates, we will open up the phone lines for your questions. We will try to make our remarks brief so that we can answer as many questions as possible in the time that we have available to us this afternoon.

We'd also like to politely request that everyone ask only one question so that we can hear from as many of you as possible. Now, I'd like to hand it over to Dr. Koroshetz.

Dr. Walter Koroshetz: Good afternoon everyone. Thanks for dialing in. We welcome the opportunity to communicate to the ME/CFS community the research areas that we're pursuing either here ar 鶹ý or through NIH-funded research. So I hope that these telebriefings are informative and we can keep the community in very close touch with what is happening here ar 鶹ý or in our universities performing NIH-funded research in ME/CFS around the country.

On today's call, we are fortunate to have Dr. Avi Nath who is the clinical director of the National Institute of Neurological Disorders and Stroke and he is leading a study here in the intramural program of ME/CFS trying to understand in a very well documented, confined group of patients what may be the possible abnormalities and potential causes in a very well defined group of patients. And he'll talk a little bit about that.

Next, Dr. Joe Breen from the National Institute of Allergy and Infectious Disease and Vicky Whittemore from our Institute here at NINDS will describe the latest activities of the Collaborative Research Centers. They'll talk about some of the work that they heard at recent conferences and international collaborations.

We'll then be joined by investigators working at one of the consortia sites, our consortia of ME/CFS research centers, and they'll talk about the work that they're doing in designing an app, cellphone app actually, to track symptoms of patients with ME/CFS in real time and they’ll be on the line to tell us more about that.

Dana March has already joined the line and thank you, Dana, and she's an assistant professor of epidemiology at Columbia University Medical Center and much of her research focuses on health disparities and ways in which social, environmental factors affect health. So very relevant to ME/CFS research. And she's working with the Lipkin Center at Columbia University on their ME/CFS project.

I wanted to say that a new development here at 鶹ý in our attempts to collect information and advice from the wider community to help sculpt the research that we fund here ar 鶹ý, we are forming what's called a Working Group of the NINDS Advisory Council on ME/CFS. The NINDS Advisory Council is a group that is appointed by the Secretary of Health and they meet three times a year and review our funding strategies, funding plans, and in open session, they also discuss with us really important policy questions and research initiatives.

This is an official body that is assigned to advise the NIH and in areas where there's a special need to do special types of planning we form Working Groups of the Council. And we are therefore forming the Working Group of the NINDS Council that will be able to reach out to the community and bring information into NINDS to help inform our decisions. And so invitations have gone out and as soon as the members are confirmed, we will begin planning our first meeting and let, again, the community know about these efforts.

And so we're really looking forward to engaging with this group and hearing their suggestions on how to move the field forward. I want to remind everyone that this group will report through the NINDS Council. Those reports will go out to all the other institutes that are involved in this trans-NIH effort to try to understand ME/CFS and try to work toward a treatment.

So that's the news from my office here, the director's office at NINDS. And with that, I'd like to turn over the call to Dr. Nath who will tell us a little bit about what's going on with the intramural study. Avi?

Dr. Avindra Nath: Thank you, Dr. Koroshetz. So I'll give you a brief update on where we stand on the intramural study. Just to remind you, this study is looking at a subpopulation of patients who have ME/CFS, particularly those individuals whose illness is less than five years of duration and have a very clear infectious disease that precipitated the illness.

So to date, we have had over 200 inquiries for this study and about 74 were screened out at the initial interview. And then Dr. Walitt interviewed 139 individuals. And of them then we screened, then we obtained their medical records and found that 84 of those did not meet our criteria. But for the week’s first visit, which is we initially bring them for what we call the phenotyping visit, the patients stay here for an entire week and we do a lot of extensive work on them. We've had 16 patients and 16 controls, a total of 32 people who have had that evaluation.

After that extensive evaluation we found that actually three of them had some other rare underlying disease that had been missed before. So they had to be excluded. And then we bring them back for a second visit. And so far, we've had four patients and five controls that have gone through the second visit, which is a two-week visit for the patient and one week for the control patients.

And so I think we're making tremendous progress. We've been working very hard and our hope is that by the end of the year, we'll have at least 20 and 20 that will have gone through the first visit and at that point in time, we will start doing the laboratory analysis of the samples to look at the data that we're collecting. But the study will continue and our hope is that maybe in another two years, we'll have finished the recruitment and then after that will be sample analysis and data analysis.

So I'm happy to report that we're doing well. I'll also take this opportunity to maybe ask you to refer patients to us for the next few months. We’re okay now, but my fear is that the number of patients eligible for our study is a small fraction of the people who call in. And usually at the beginning of any study, your recruitment is good and then it tends to taper off. So we would like to continue to keep busy and have patients coming through. So I would urge you to send us individuals that you know and spread the word so that we can continue to have a steady flow of patients into our study. Thank you.

Dr. Walter Koroshetz: Great. Thank you very much, Avi. And now, I would like to turn over the discussion to Dr. Joseph Breen from the National Institute of Allergy and Infectious Disease, and Vicky Whittemore from NINDS. As folks know, they lead the Trans-NIH Working Group on ME/CFS and they have been really great champions for ME/CFS research here ar 鶹ý. So with that, Joe, do you want to start off?

Dr. Joseph Breen: Sure. Thank you, Dr. Koroshetz. So I want to talk to you a little bit briefly about the research centers and the data management coordinating center that were started in the very last part of 2017. The research centers are now fully staffed and working hard. And we actually have a paper published from the Columbia Center recently and I know there are others underway from the other two centers.

In addition, they are starting to interact and coordinate together, and specifically to try and put some resources from all three centers into the data management coordinating center so that they can utilize it across all three centers. And actually, the goal is to then empower the community to see the kind of work that's going on and really to generate some synergy in the overall research community and try and drive the field forward.

I wanted to mention specifically one activity that is underway and that is the collaborative research projects. Each center was designed to hold back some of their support for projects that would interweave between three centers after the original award. And those projects were recently begun and I just wanted to give a brief highlight of those kinds of projects. Because I think, again, that will be one of the strengths of this network where they can really utilize the strength of one center for the other centers.

And they decided to start using some human samples that were already previously collected since all three centers are collecting samples through the lifetime of these awards, but that takes time. Columbia, it turns out, through prior NIH support had collected a number of ME/CFS samples as well as control samples that then they were able to share with the other two centers.

And so this original collaborative project is actually already underway and the Columbia Center is actually going to look at expression of RNA in a specific type of immune cell, called a T-cell, which we think that, we know that there are some immune abnormalities in ME/CFS and we want to look into more detail into a specific type of immune cell called a T-cell. And the Columbia Center is going to do some detailed RNA expression work, again, using samples from Columbia.

And on those same samples that actually the Jackson Laboratory, one of the other centers, is going to be looking at the frequency of immune cell subsets not limited to any specific type of T-cell but across a broader range of T-cells, again, trying to understand what these patients are responding to, try and get a clue as to a little bit more about the nature of this disease. Again, using some of the blood samples that were previously collected.

And then Cornell, which is looking at something a little different, they're going to be looking at extracellular vesicles, which is really a newer area of research but is clearly of interest to the broader scientific community, and in particular there's thought to be a connection to metabolism. And that is a clear connection here to what's been seen and noted in publications for ME/CFS patients.

So they're really looking at cell physiology, looking at these novel vesicles and looking at overall immune cell physiology in the Cornell Center. These are collaborative interwoven projects and the data from these projects will then be shared with the data management coordinating center, which will then, firstly, be allowed analysis across all three centers, which will be an advantage, and eventually will make the data sets available to the public as well.

The second thing I wanted to tell you about was planning for a research conference to be held here ar 鶹ý next April is underway. The conference - Dr. Koroshetz actually mentioned it in his Council remarks earlier this year. The title of the conference is “Accelerating Research on ME/CFS.” We have some co-chairs identified and are currently putting together an organizing committee to help organize some sessions, the scientific sessions.

It's a two-day conference. We certainly can't cover everything in the field, but we're going to do our best to try and address and talk about some of the most exciting work that’s supported all around the world in bringing folks together, which is an opportunity for experienced folks as well as younger folks. And there's a specific opportunity for the younger folks that I'll actually let Dr. Whittemore tell you about. So Vicky, maybe you could pick it up now.

Dr. Vicky Whittemore: Thank you. Sorry, I'm in Denver at a conference and sitting in a hallway. So I apologize for any other stuff that's happening around me. So we are planning the day before the research conference to organize a one-day young investigator workshop, which will model the workshop that was held in conjunction with the Invest in ME Conference in England to bring together graduate students, undergrad, graduate students, medical students, post-docs to come together to talk about their projects and offer advice and suggestions to one another as well as to form a network.

It was a really successful workshop in England and we're hoping to model that again with a one-day workshop in conjunction. And to really bring together some of the folks that are working in the labs, both in the collaborative research centers here in the United States, foreign students and post-docs, as well as people from other supported labs here in the United States.

So I'll just continue on and just tell you a little bit more about the recent workshops and conferences that have taken place. So in early May, there was an ME/CFS conference organized by Alain Moreau and his colleagues in Montreal that brought together people from around the world to discuss ME/CFS collaborations and ongoing research that was a very successful meeting and I think was the first of its kind for Canada. And it also involved individuals from patient advocacy groups and was I think a really well- received conference.

Andrew Breeden, Avi Nath, and I all attended, Brian Walitt sorry, also attended, the Invest in ME Conference in England. And that conference again brought together several investigators from around the world who presented their work. And one of the things that struck me about the conference is - and the presentations -was that there's a lot of really excellent work going on, but it tends to be very fragmented between one lab who is working on looking at B-cells in a certain population of individuals and a different lab that's looking at T-cells, and another lab that's looking at other neuroimaging kinds of things.

And so I think one of the things our hope that we hope is that we can bring the community together to really start to focus on the whole person and looking at each person in a much more comprehensive way to get a better sense of what's happening in the disease and get to the underlying disease mechanisms. So I think that’s going to be one of the goals of the conference we hold next spring and one of the ways in which we hope to really accelerate the research.

And just one last comment about international collaboration. So as you all know, we have a collaboration that we've established with the Canadian Institute of Health Research and they have completed the review of grant applications but have not yet announced the funding of a grant. But as soon as they do, that group will be brought in to collaborate with our research collaborative centers and the data coordinating center. And so we're looking forward to that happening hopefully within the next couple of months.

In addition, we've had ongoing discussions with the Medical Research Council in the U.K. and with individuals representing the funding agencies in Australia in terms of ways in which we can really better collaborate and coordinate research efforts worldwide. And I think this is really terrific in terms of really hoping to move the field forward in a coordinated way where we can all be working together to really move research in a very positive direction.

So I'll stop there and pass it back to you, Dr. Koroshetz.

Dr. Walter Koroshetz: Thanks very much Joe and Vicky. So next, we have really profited by the generosity of our investigators getting on the teleconference calls that we're holding. And today, we're grateful to Dr. Dana March at Columbia University, as I mentioned, working with the center there that was set up by NIH for ME/CFS research. And she's an assistant professor of epidemiology at Columbia. And also joining her as part of this team is Dr. Anthony Komaroff from Harvard Medical School. He's a professor at Harvard and a senior physician at the Brigham and Women's Hospital. So welcome Anthony and Dana.

Dr. Anthony Komaroff: Thank you very much, Dr. Koroshetz. Before we begin, this is Dr. Tony Komaroff, let me just thank you and Dr. Nath, and Dr. Breen and Whittemore for everything you’ve done over the last two years to ramp up the efforts of the NIH. It's really - those of us in the field are so grateful to you. So thank you very much.

Dr. Walter Koroshetz: It’s the least we could do. We'll do a lot more with the kind of scientists that you can bring to the field. So thanks very much. Who would like to start off talking about the collaboration with the Columbia CRC?

Dr. Anthony Komaroff: I'll start off and just summarize. We have, Dr. March and I, have three different projects to summarize. One of them involves an integration of several very large databases that already exist at Columbia, databases of clinical data, bio samples, laboratory test results, et cetera. And it's a huge opportunity to get answers to questions that smaller databases don't allow.

The second is a study of various laboratory test abnormalities that are seen in patients with ME/CFS under conditions of stress, particularly exercise stress and orthostatic stress, which means just being upright for an extended period of time. We'll discuss the rationale for those studies in a minute.

And then the last one is the one you referred to, Dr. Koroshetz, which involves the design of an app for mobile devices that will allow patients to report on their symptoms and the progress of their illness. Dana, you want to start by talking about the integration of the databases?

Dr. Dana March: Absolutely. Thanks so much, Tony. Just to echo Tony's gratitude, thank you so much for having us and it's also a really exciting time for the field. It's exhilarating to be a part of this growing network of centers. So thanks so much for having us today. A little bit about what this set of clinical research projects are, especially with respect to the existing data that we have.

So this is in addition to two other basic science kind of laboratory based projects that are building on a bank of samples that we already have that were collected in the context of an NIH-funded study. So we have an enormous amount of data from previously conducted studies, including one NIH-funded study and several studies that were funded by the Chronic Fatigue Initiative.

So we have a number of different datasets that we have not fully mined. And we could potentially get some insight into subtypes of illness and risk factors for illness if we integrate these datasets in a meaningful way. So just a little bit about what I mean by integration. What I mean by integration is just bringing data together from diverse sources. And it means two things. It means connecting data across various studies that has been collected from the same individuals, so individuals who have participated in multiple studies. And it also means connecting datasets that share common measures but not necessarily the subjects who participated in them.

So two important features that we want to capitalize on in order to shed some light on some critical issues in ME/CFS. There are several goals involved with our dataset integration and analysis project arm of this clinical research project. The first is really to uncover the full scope of the data that we have available on a relatively small but potentially very impactful integration of data across the studies for subjects who have participated in multiple projects.

And one might think that it would be fairly straightforward to integrate various datasets, but the very nature of the data collection itself and efforts to protect privacy of patients and so forth, and the clinical interfaces, the research interfaces that we use make that not such a straightforward task. And this is one potential collaborative opportunity for the DMCC and our research center to get the DMCC's help in unifying these datasets and they're really well poised to do that. So I'm excited to have that resource available for this particular aspect of the project.

The second goal for a data integration and analysis project is really to identify subtypes for illness and we have a variety of different types of data available to us. We have biological data, clinical data, and survey data. And so using these three different types of data across studies that have common subjects that have participated in one or more of those studies and also studies that have collected similar measures will be really helpful for us to identify subtypes.

And the kinds of data that we would use to identify subtypes would be onset type, duration of illness, age of onset, gender, comorbidity profiles, and even certain types of symptom clusters that we might want to explore. Finally, the third goal for this kind of a project would be to explore important risk factors for developing ME/CFS. So past history of particular diseases, history of diseases in first order relatives, or certain prior treatments, or activity patterns would be particularly helpful to uncover certain risk factors that might be really important to dig down into in terms of predisposing people to ME/CFS.

Dr. Anthony Komaroff: Great. Now, let me turn to the exercise and orthostatic challenge studies that I mentioned. As Dr. Breen said earlier, there have been a variety of laboratory or biological abnormalities shown in patients with ME/CFS, including changes in the immune system, in metabolism in general, energy metabolism in particular, and epigenetic studies, epigenetic being shorthand for determining what genes are turned on and off and whether they're turned on and off appropriately.

So all of these different kinds of abnormalities have been demonstrated, but they’ve typically been demonstrated in people at rest. The question is, particularly in an illness like this one, where certain challenges like exercise or being upright for prolonged periods of time make the symptoms worse. It raises the question of whether those same challenges would make the laboratory abnormalities worse. Because all we can say now when we see a particular, let's say immune system abnormality, being abnormal in people with this illness at rest, we can say that it's there. But we can't really tell if it is cause or effect.

But if you show that when people do something like exercise, it makes them feel worse, that those certain metabolic abnormalities get worse along with the symptoms getting worse, it makes it more plausible that these laboratory abnormalities may actually be causal, may be actually contributing to the symptoms of the illness. And if that were true that would be good because they would represent targets for treatment interventions to try to prevent the symptoms from happening.

So that's the rationale for these studies. About 100 patients and healthy controls will be involved in four centers around the country, are involved already, and I think it has the potential to provide some important information.

The third project is one that mentioned earlier, the building of an app for mobile devices. Why do that? Well, first of all, at least those of us who do research on this illness see a pressing need to get much more information from patients about the symptoms they're having, the things that seem to trigger the improvements or relapses in symptoms, any changes in their general activity level that may be related to the symptoms they're having. And the best we've been able to do so far in ME/CFS research is to have patients come back to research practices several times a year and tell us about the symptoms they’ve been having, et cetera. But that's just several times a year. Anyone with the illness knows that it can change day-to-day, week to week.

And now, there is a way of capturing what's happening in real time in many different illnesses, including this one, using smartphones, or tablets that people have, most people have, and software that collects the information you want. That kind of communication can be sent electronically to the research databases. And so the whole process of collecting information and doing research can be made much more extensive and much less expensive by using contemporary available electronic technology like these apps. So that's the rationale.

What would the app do? We're just beginning to define that but it surely would include a capturing of the symptoms, both the presence and absence of them, and how severe they are at the moment they're being reported. A general gestalt estimate by the patient as to whether this is a good day or a bad day. There have been several studies already done of people at days they define as good days versus days they define as bad days. But this will make that kind of study much better and much easier to do. It will capture information on stressors like the amount of physical exertion in the last 24 hours, dietary changes, psychological stressors, cognitive stressors, and any lifestyle changes that might conceivably affect the symptoms of the illness.

When would a person report these symptoms? The app would allow a person to report it any time they wanted. It could be even several times a day. More likely it would be several times a month. And the app would include gentle reminders from people who hadn’t reported anything in some window of time that they're overdue and it would be great to hear from them.

An app like this will create an enormous database, but now the techniques are available to interrogate, to analyze, such large databases and are already being used to learn entirely new things about diseases like diabetes, for example. So we're optimistic that this will be both a way that doctors can learn more about the illness, but that patients can have a way of easily reporting their experience with the illness and have the app also summarize for them what they've reported over the last month, or the last six months, or the last year. See trends in their symptoms, trends in their exercise levels.

So I think it has great potential benefit both for patients and for researchers and physicians. Dana, do you want to talk about the process of building the app?

Dr. Dana March: Sure. So we are concentrating our efforts in years one and two of this project to really conceptualizing this app and what that actually looks like. And we're really approaching that in a very systematic and rigorous way. So I want to tell you a little bit about what that process involves. So firstly, and this work has been underway this summer with the assistance of a student from Columbia who I have brought in, and it's also great because it allows us to get a next generation of researchers interested in this particular illness. And I think we've been successful in that, which we're very happy about.

But we've been performing a rigorous market analysis of already existing apps, mainly apps that are designed to collect information about chronic disease and chronic illness management. There are very few apps available that address ME/CFS directly but there are a number of apps out there that ME/CFS patients use in order to manage symptoms or keep track of what's going on with them, or related illnesses, for example. And we've learned a great deal about what's out there and what ME/CFS folks are, how they're using these apps, what they think about these apps, the kinds of features that they really like, the kinds of features they don't like, what they're really interested in having.

So that's been a really critical piece and it clearly is going to be an ongoing piece because the landscape just changes so rapidly. But that's been a really important step to take. So we're going to continue to build on that work. Now, related to that work is a process that we're beginning to engage in now, which is to form a working group comprising patients, clinicians, folks from other collaborative research centers, community partners. So we partner at Columbia with Solve ME/CFS Initiative, MEAction, and the Microbe Discovery Project.

And the idea is to bring folks together and collect qualitative information, so conducing focus groups with specific questions that we ask and also eliciting information from folks that we didn't think to ask about. And we're going to use qualitative data analysis techniques to really help us understand what the desired user experience and user interface of the app should be.

So what that means is that we're really interested in understanding the features of an app that people want and need. I know anecdotally there is an enormous demand for a way of keeping track of data, a way of connecting with other people who have similar profiles and connecting with the broader ME/CFS community. So we are really interested in using, working with the community to use some information that is provided in order to design features that people want and need, and also inform how those features actually appear and how they perform.

It's important also to understand the needs around ways of visualizing data and summarizing data. There are specific considerations with folks in the ME/CFS community. Some folks have really particular problems with vision. Cognition is always something that has to be considered. So what are the best ways of actually summarizing the data for each person who is actually using the app? And then similarly, summarizing the data so that it can be shared with the patient's clinician and with the research community should people want to be able to participate in research.

And then finally another feature to consider is whether or not to integrate a social network feature. That's certainly come anecdotally from talking to folks in the community that that's been a really helpful feature for other kinds of apps that people have been using. The next step is really to use this information to design the first prototype of the app.

So absent a technological realization of this app in years one and two, to really just get the specs of this app really down pat. And we don't anticipate that there will be any software development in the first year but it's going to be critical I think to connect with app developers and folks who are working in that space technologically from the ground up so that we can do that in a meaningful way. And the idea is that we could either build on an existing platform. We have been in conversations with Linda Avey who is the leader of Precise.ly. A number of folks have used an older version of Precise.ly to use a tag-based system to track their symptoms, and medications, and so forth and have found that to be really helpful.

Chris Armstrong in Australia has also designed a very basic app that's designed to collect survey data. So the question is whether or not we collaborate with these folks or start from scratch. But the idea is that we would like to take this design on paper, this conceptualization of what an app looks like in partnership with the community to work on the development.

Dr. Walter Koroshetz: Dana, that sounds really exciting. I wondered if we could see if there's any of the questions that people have that potentially you could field about the app or about some of the other projects. Because we do have an open line now for questions and so we'll see if there's somebody, if anyone has a question could they indicate that on the website? Dana, that sounds really interesting.

Operator: Thank you. If you would like to ask a question from the phone lines, please press star then 1, unmute your phone, and record your name when prompted. If you need to withdraw your question, please press star then 2. Once again if you'd like to ask a question from the phone lines, please press star then 1 and record your name when prompted. One moment please while questions queue up.

Dr. Walter Koroshetz: So while they're queuing up, Dana, do you think their ability to link in activity monitors as well for the patient reported outcome app you think about?

Dr. Anthony Komaroff: Absolutely. One thing, most smartphones have a rudimentary activity monitor built into them, but there are now so many different Fitbits and even rings that you never detach from yourself that are constantly measuring physiologic variables.

Dr. Dana March: I think one of the challenges with that, there's an enormous promise for that, but one of the challenges is the wide variation in the quality of data that you get from wearables. So I think it's an interesting challenge to overcome.

Dr. Walter Koroshetz:A lot of work in that space. We have a couple of questions queued up, I think. Is that right?

Operator: Our first question today comes from Mark Camenzind. Your line is now open.

Dr. Walter Koroshetz: Mark, are you on mute? Do you want to give us your question?

Mark Camenzind: Sorry. Now I'm not on mute. So my son was a 24-year-old, straight A, Stanford student. Now, he's home semi-comatose with severe eye sensitivities, which are not uncommon. I don't think you are tracking enough the levels of eye sensitivity. It is to the point where it can make him suicidal and goes on for months. And other people have different symptoms like a lady in Sweden had her skin bother her so much that she eventually suicided and well documented that. Completely coherent and my son is coherent.

And so there's a lot of these things that are very specific, need personalized medicine and we need better treatment for the severely ill that have visual issues, especially severe photophobia where it's 100 percent dark room where I can see nothing and any sliver of light under the door will make him scream. And so we need also when manipulating eyes and things, because it's very irritated, eyelashes can get folded under, eyelids can get folded under, things like that. We need vision to see these patients in the dark and there are infrared goggles for distance. Is there anything for up close?

And then similarly retinal scans. We'd love to know why he's screaming. Is it his retina, or optic nerve, or in the brain? It is definitely eye sockets also but we don't know. Could be infection. Could be viral, fungal, who knows. There is no method to get him to an ophthalmologist because he cannot travel without an ambulance. He tolerates no light.

There is, for example, a company, Retina View, that has an app for the cellphone to look at the retina for diabetics. And there's Japanese that have infrared retina scans. So can we combine in some handheld apps these commercial platforms and just extend to other detectors so we could see in the dark, look at the retina in the dark, and do more real-time monitoring like you guys have mentioned.

So I just want to thank you all for doing fantastic research, but you need a lot more funding and SR508 hopefully will drive that Senate resolution and we should be heading toward $200 million. And you guys are doing great, but HHS and NIH have to end the discrimination for decades against ME and increase funding toward $200 million a year. I've taken my time. Thank you.

Dr. Walter Koroshetz: Thank you very much, Mark. Yes, I think the hypersensitivity problems are incredibly disabling and trying to understand the basis of those is, if we could figure out how to solve that problem with the eyes where the light itself is, there are not pain fibers in the retina, but the light actually interacts with the trigeminal nerve cells. And that combination of some irritation to the trigeminal system plus light can cause severe pain.

Certainly, we see it in migraine as one of the core symptoms. Tony, would you want to make a comment about hypersensitivity in ME/CFS and the experience that you had? Any kind of clues that you come across?

Dr. Anthony Komaroff: Well, I wish I could say physiologic clues but there's no doubt that sensory sensitivity, light sensitivity, noise sensitivity, and paresthesia or hypesthesia, odd sensations when people are touched are extremely common in ME/CFS as they are in migraines. I haven't myself seen someone whose light sensitivity is as severe as the caller's son.

But there’s no doubt that it's an important problem and one to pursue. And maybe it's even if there are any patients with severe sensory sensitivity in the intramural study ar 鶹ý, maybe there are some tests of the sort the caller was calling about or suggesting in the call that might pursue what's going on.

Dr. Walter Koroshetz: Thank you. May we have the next question please?

Operator: Our next question comes from William Capell. Your line is now open.

William Capell: Hi. Thank you for this opportunity. Please bear with me because I am suffering a great deal of brain fog at the moment. So if I sound stupid you'll understand why. The mention of the app and research I like a great deal. I've noticed there are a lot of clinics doing biomedical studies, but you have to go to them. And I'd be willing to go to places if I was able.

So I was wondering if there's any way or anything in the line about funding the use of local technicians to collect biological samples when that's all the study requires.

Dr. Walter Koroshetz: Thanks very much, William. I think that is something that we're going to have to broach to investigate folks who cannot travel like that. Dr. Nath is in a special situation where at the Clinical Center here we can bring people in and they can rest up over, Avi, how long are they in the beds here at the hospital when they come?

Dr. Avindra Nath: Yes, they keep them for a week for the first visit and two weeks for the second visit, and we give them a day when they first to come to just recover from the travel itself. But we still have 9 percent or so of our patients have difficulty getting here.

Dr. Walter Koroshetz: Right. So we have used in-home research help for some large projects but I'm not sure that they were at that level where they were going to help us unless we have a clue that we can go out and search for. The one project we used the examiners that worked for insurance companies to go and make the home visits and collect data on patients, large 30,000 patient stroke cognition study. But that's a great idea and we're going to have certainly deal with that at some point getting the research out to the patients who can't travel.

Can we get to the next question? Thank you very much, William.

Operator: Our next question comes from Liz Burlingame. Your line is now open.

Liz Burlingame: Hi, can you hear me?

Dr. Walter Koroshetz: Yes.

Liz Burlingame: On May 25, MEAction sent a letter to Francis Collins. The letter had over 7,000 signatures, photos, and personal comments. The letter went unanswered until just a few days ago and as you note, the reply was not even from Francis Collins himself. As a patient community, the NIH leadership continues to dismiss ME/CFS patients.

So my question is when can MEAction expect a response from our letter from Francis Collins himself? Thank you.

Dr. Walter Koroshetz: Thanks very much for that question and I apologize for the slowness of the system here. But I would say first of all that of course, Francis Collins was really the major driver that pulled all the institutes together and actually funded the first year of the intramural program. So he is a great advocate for ME/CFS.

The process, unfortunately, he gets so many requests that there's a system where the letters come in. They send them out to the ICs to get advice and he doesn't even see the letters until the advice goes back. And, but that recommendation to meet went to his office today and it just goes through the system.

I'm sure he's very interested in the issue but we just have to wait for that system to kind of turn out the answer. And this is not in any way unusual. It's just the way that the system is set up to deal with probably thousands of requests that he gets. But we're hopeful. We have our fingers crossed for sure. So thanks for that.

Next question.

Operator: Our next question comes from Becky Turner. Your line is now open.

Becky Turner: Hi, I was hearing Dr. Nath and I've heard him speak before about the intramural study and I know a few people, at least one person who's been involved in, been a patient there. It does sound to me like things are moving, even with the people who have been through the first week, things are moving pretty slowly.

I realize that there is some concern about getting enough patients but I'm wondering if there are other things holding up the speed of that study. Maybe the number of beds that are available or other sort of equipment or staff challenges that could be met to speed up the study. Because two years to get the data analysis done seems like a long time for those of us who aren't well to find out what is going on.

Dr. Avindra Nath: Thanks for the comment. I hear that quite often from a lot of people and the thing is you can do a small amount of tests on a lot of people and you get it done very fast. But when you design a study where you're going to do an exhaustive amount of stuff on single patients then it just takes a long time to get through.

And so the design of our study is such that really there isn't a lot of room to speed it up. And the team is working day and night. There is not a whole lot more capacity. We will try to double up on patients, try to bring in a control and a patient at the same time. So we will double and increase the speed a little bit. But I think the more important thing really is to do a proper study and not, haste makes waste. So we bring in a lot of patients, you're not able to do it right, something goes wrong. There's just so many moving parts on a single patient that are happening on a single day that the chances for error just go up. And then you'll just get a bunch of garbage data, uninterpretable.

So I know to resist the temptation to do something faster is probably a better approach. I think getting the proper data at the end of the day will be a much greater benefit to the community.

Dr. Walter Koroshetz: I'd just add that the intensity of this study is, there’s nothing that comes close to this type of study. So it is unusual because of the depth of the investigation. So the patients are there for two weeks. The samples, the access, the equipment, all these things are so important to do this correctly. But that makes a lot of sense why people would be asking that question. So thank you for that.

Can we go to the next question?

Operator: Our next question comes from Raquel Sanders. Your line is now open.

Raquel Sanders: Thank you very much. First, I want to thank Dr. Whittemore, Dr. Breen, and Dr. Nath on behalf of all your good work for us. And of course the extramural researchers. Dr. Koroshetz, I have had, last month marked the 40th year, 40, that I've been trying to survive ME. And I've been largely homebound for the past ten years and largely bedbound for the last three years.

On April 20, 鶹ý Director Collins stated, "research done correctly takes time" but he chose not to mention funding. The issue here is I can't go on like this. I want to live. I want to live so badly but I don't want to live like this and you continue to shine us on. You’ve got to fund research on this disease commensurate with the disease burden. Every time I hear Mark Camenzind testify about his son, Tom, it just shatters my heart while you patronize us. You ought to be ashamed of yourself and Dr. Collins most especially. You're moving too slowly. People are dying. You're robbing us of hope because you are moving too slowly and that funding the disease commensurate with the burden.

People stop patronizing us. Please stop with ridiculous excuses. Treat us with civility, humanity, dignity, and respect. And just to finalize I will read very quickly a short piece by Jamison Hill in the New York Times who has ME. He tells us, "I, on the other hand, have had to do everything in bed, brush my teeth, bathe and use the quote unquote bathroom, a plastic bag for bowel movement and, for urinating, a dubious looking plastic container attached to a tube feeding into a bucket on the floor.”

Dr. Koroshetz if you cannot take this seriously and move forward in the way that must be done with adequate funding, not ridiculously low $15 million. 40 years of this is obscene. Thank you.

Dr. Walter Koroshetz: Yes, I think your frustration is embraced by all of us. We feel terrible about the kind of suffering that you’ve had to undergo and millions of people around the world. And we're trying to channel that into answers through research. And we're not going to give up.

And I apologize for how long this takes and we are definitely interested in increasing the funding to investigators who can really make a difference. And as Vicky and Joe have indicated, we're reaching out to try and fill that army that it's going to take to fight this disease and win.

So again, we feel the frustration. We take it to heart. So thank you for that. Okay, next question.

Operator: Our final question today will come from Catherine Suter. Your line is now open.

Dr. Walter Koroshetz: Catherine?

Catherine Suter: Can you hear me?

Dr. Walter Koroshetz: Yes, sounds good.

Catherine Suter: Okay. Great. Thank you for taking my question, well comments and question that I'll squeeze all into three minutes. First off, thank you to everyone for your commitment and your contributions that you make to this, and for presenting it so that lay people, even lay people with cognitive limitations, can understand.

This is my first dance with any sort of engagement in activism, advocacy around this. I've had it for 15 years. It was a slow onset but much slower was my acceptance that something is really wrong. It's so much easier to believe that I'm just a bad person, or a lame person, or guilty because the medical community doesn't or people in general don't accept it. You heard that plenty of times though.

What I wanted really to contribute is to say regarding the app, the symptoms app, it’s a fantastic idea, please, please keep it simple. I have mild ME and I can barely keep up with what's going on. Don't start a new social networking aspect. Maybe link to other social networks that are going on about this. I have a hypersensitivity to sound that makes it extremely difficult for me to work actually and so there's two pieces of information and I had one question.

Maybe I'm not even sure this is the right place for it, but I don't understand why ME, Myalgic Encephalomyelitis, is being chosen as the term for this. I don't see any proof of brain or spine inflammation in me. It seems like the systemic exertional intolerance disorder is a much better term. What's going on with the naming of this? What's that story? Thank you.

Dr. Walter Koroshetz: Boy, that's a good question and I think that our tack here is to try and develop the biological evidence that will inform a name for the subtypes of what we now consider ME/CFS. So as opposed to obsessing about the names, we would like to be able to characterize the patient so that we can give them a biological signature as opposed to a symptomatic signature.

And I would take the opportunity, and I apologize that I want to do it in the most sensitive way I can. But it is not that easy but you did bring up the issue of what is the evidence for Encephalomyelitis or Myalgia. And the point I wanted to make is that for many different diseases, the real clue as to what is going on comes from examining in an autopsy situation for research, the tissues, to see what is really wrong in the tissues.

And we have almost zero evidence. So that's gold standard evidence for diseases and we have almost zero tissue to examine. So the sensitive part of this is that the donation of tissue in folks who die with ME/CFS could be groundbreaking.

And it's a very difficult thing but we have ar 鶹ý a NeuroBioBank program where people can sign up to donate their tissue, should they die, in a variety of different diseases. So ME/CFS is one, but we have hundreds where this is an important issue and in certain conditions it's been very successful and has led to a lot of scientific progress.

So I just wanted, because you mentioned the evidence that's lacking in this area, I just wanted to say that the reason we have no evidence is because there is no tissue to examine. And this is something I was hoping thar 鶹ý working with patients and the advocacy groups can fix. So because there could be, we could get lucky. There could be something sitting there that we and once we see it then things would make sense. But right now, there's nothing to look at.

We work with Tish Hevel in the Brain Donor Project and she has made it a mission, taken on the mission to try to get this message out in the most sensitive fashion possible. So I would urge anyone who is on the phone or any organization, maybe get in touch with Tish and see if we can help kind of answer the question that you started off with.

So I want to thank everyone for listening. We all recognize that we're not giving you the answers that you need but I want to just tell you that all the people working on this project are dedicated to persisting until we can get answers. So thank you very much and thank you very much to Dana, and Tony Komaroff and Dana March for getting on the phone today, and to the staff here. And we'll talk to you soon at the next telebriefing. Thank you.

Alissa Gallagher: Just a few quick reminders that a recording and transcript of the call will be posted to the NIH ME/CFS website next week and in closing today's call, I'd like to also remind you about our listserv for updates from NIH. If you'd like to be added to the listserv, please visit the NIH ME/CFS website at www.nih.gov/mecfs and click on join our listserv at the bottom of the left sidebar.

Thank you again for the thoughtful discussion and we hope all of you have a good afternoon or evening.

Dr. Walter Koroshetz: Thank you very much. Thank you, Tony. Thank you Dana.

Dana March: Thank you.

Operator: That concludes today's conference. Thank you for your participation. You may disconnect at this time.

This page last reviewed on August 2, 2018