October 17, 2019

NIH ME/CFS Advocacy Call October 17, 2019

Updates on NIH’s Myalgic Encephalomyeltis /Chronic Fatigue Syndrome ME/CFS-related activities.

Dr. Walter Koroshetz: [National Institute of Neurological Disorders and] Stroke, with the team from NIH, and we're excited to once again let folks know about what's been going on our end in ME/CFS research and this is our third telebriefing of 2019. In today's teleconference, which we're going to do on WebEx, so folks should be able to see the slides, Dr. Andrew Breeden will briefly introduce the council Working Group that presented their findings to the NINDS Council just a month or so ago on ME/CFS research. And in that vein we're delighted to be joined by Steve Roberds. Dr. Steve Roberds is the chief scientific officer at the Tuberous Sclerosis Alliance. He's a member of our Council and he worked very hard with a great team of folks to chair the council Working Group on ME/CFS research.  We've invited Dr. Roberds to give us an overview of the Working Group and discuss the Working Group's report, which is really the focus of today's teleconference. Afterwards we'll hear from Dr. Vicky Whittemore who works very closely with Dr. Joe Breen from the NIAID Institute here at NIH and they'll discuss how Trans-NIH ME/CFS Working Group will work to implement the report. As always we're very eager to hear from folks and take questions, so we'll keep our remarks as brief as possible before opening the phone lines for your questions. With that I'd like to turn the call over to Dr. Breeden.

Dr. Andrew Breeden: Thank you Dr. Koroshetz. So as Dr. Koroshetz mentioned, today we'll hear from two speakers about the recent efforts of a Working Group of the NINDS Advisory Council that was formed to identify gaps and opportunities in ME/CFS research, as well as strategies to move the field forward. First Dr. Steve Roberds, who chaired the Working Group, will describe how the Working Group produced its final report and summarize the report's findings. Next Dr. Vicky Whittemore will describe plans for implementing the strategies outlined in the report. Before we hear from Drs. Roberds and Whittemore with more details, I first want to briefly give some background about the Working Group. Each NIH Institute or Center has a National Advisory Council that's made up of scientific and medical experts who can advise the Institute director, in this case Dr. Koroshetz, on important policies and procedures. In the summer of 2018 NINDS formed the Working Group that we've been discussing of our Advisory Council that was focused on how to best advance ME/CFS research. The Working Group was composed of scientists, clinicians, representatives from advocacy organizations and individuals with ME/CFS. The Working Group was charged with three main things. The first was to identify gaps in current knowledge, opportunities for future action, and a series of potential strategies to move forward in each gap and opportunity. The second was to examine how to attract and train a pipeline of new and early career ME/CFS investigators. And finally moving forward to examine how to enhance ongoing research collaboration and communication. Dr. Roberds will soon describe the many productive meetings held by the Working Group that culminated in a report that was presented to the NINDS Advisory Council in September. The report is publicly available online and we will soon include a link on the slides that you'll be able to see where to access the report. And the report was approved, was accepted, by the NINDS Council and so the 23 NIH Institutes, Offices, and Centers that compose the Trans-NIH ME/CFS Working group have been holding discussions on how to prioritize and implement the strategies outlined in the report. Dr. Whittemore will describe these efforts. With that I will turn it over to Dr. Steve Roberds who chaired the Working Group. Dr. Roberds is the chief scientific officer of the Tuberous Sclerosis Alliance and has played a key role in leading the development and execution of research strategies in a variety of contexts. We are very grateful to Dr. Roberds for leading the ME/CFS Working Group. Dr. Roberds.

Dr. Steve Roberds: Thanks Dr. Breeden, I really appreciate the kind introduction and setting the stage and thanks to Dr. Koroshetz for asking me to take on this responsibility. I was really truly honored to be a part of this. It was a great opportunity to come from outside the ME/CFS field, to learn a lot about it and what's going on, but you know and really bring perspectives together with you know what I think was an open mind, right, unbiased view because I wasn't in the field. So you know I really appreciate the trust that was placed in me and the other members of the Working Group. So if you could go to the next slide please, I wanted to just start by stating what might be obvious, that the burden of the illness is not only substantial but severe and high and because the burden of the illness is so impactful and we don't know anything about the etiology and pathogenesis of the disease, there's no diagnostic tests, there are no approved therapies, all of this as all of you on this on the call know really set the stage for an urgent need to expand research in ME/CFS and, next slide please. So as Dr. Breeden outlined the Working Group of Council had three charges and I reiterate those because I'm going to address each of those as, in the next few minutes. So the first was to identify gaps and opportunities in ME/CFS research and then the second was to consider strategies for NIH to support ME/CFS research to attract and train a pipeline of new and young investigators and then the third was to identify potential ways to really enhance the ongoing collaboration and communication and research in ME/CFS. As I'm sure you're all aware, we can do a lot more together than we can give you separately and individually. And speaking of working together, on the next slide, is the Working Group. So I'd like to acknowledge all of these individuals for their time and effort. I like to think of the way this slide is organized that the people on the lower right are actually the ones who put in huge amounts of time and kind of as you work backwards to the upper left you know we were privileged to be a part of it  and to provide that you know that insight and guidance with the NIH staff. Really I want to express my appreciation for all the work that they did in the background to you know get data together and help answer questions that members of the Working Group had over the last year, year and a half, so next slide. Just a brief overview of the process. So as Dr. Breeden said, we started over a year ago and we started having monthly calls in September of 2018. We organized a time in December when all of us could get together face to face to you know really hash through some of the key questions that we needed to address over the next several months before making the presentation to council and so with these weekly calls and laid the groundwork and we divided into subcommittees so that we could divide and conquer right all the different that needed to be done and so some of the folks work just how do we gather stakeholder input and some worked on thinking about what might this structure for ongoing collaboration look like and then others dug into what are our real gaps in knowledge both around pathobiology of ME/CFS and what we know about it clinically and we had a group that thought about, you know, what's going on in research elsewhere and how do we loop them in to this research going forward. So I hope you can see from this that the Working Group was trying to tackle this from a lot of different perspectives with the goal of bringing together lots of ideas but also lots of people who could be committed to this in the long term, so next slide. I'll start addressing each of the charges of the Working Group and I start with this one first because I think it's really critical for the long-term success of increasing the focus on and the productivity of ME/CFS research and so that's how do we get enhanced cooperation and collaboration among federal agencies and of course other stakeholders in ME/CFS research and we talked about some various models and  there's one actually that is very active and very successful in the epilepsy field that we really ended up using as kind of a model of how this could work. That involves representatives from across NIH, involves representatives from other federal agencies like CDC, the Department of Defense, et cetera, who are doing research, and then it also involves national organizations and patient advocacy organizations. So this, it was really a way to bring all the folks together at the table and so we started with that as a concept, developed this concept of a ME/CFS collaborative group that includes the types of organizations that I just mentioned, really having the goals of sharing information but not for the purpose of just sharing it but for highlighting what's actually advancing and for discussing and digging into the gaps that still exist and the opportunities to fill the fill those gaps. The idea is and we've seen it work in the epilepsy space, is that it really drives collaboration among both the government and the non-governmental organizations that are a part of this and it allows, because everybody's at this table, it allows them to monitor the progress of the research field as a whole one. So we think this can serve as a good model for ME/CFS as well and the Working Group emphasized that the patient advocacy involvement is really critical for at least a few areas, and probably many more, but one is identifying the priorities of research because the patients who are living with this are the ones who need and know the priorities best and keep an eye on the progress and make sure it's moving in the right direction. And then the other, another, way that they can be involved is to address those barriers to participation in clinical research and clinical trials so you know the last thing that we want is for you know for researchers to undertake a large and expensive and long term clinical trial and you have it fail because it didn't meet the needs of the patients that they needed to be enrolled in this to have it be successful. So there are a number of reasons the patient advocates should be at that table. On the next slide, another of the charges to the Working Group was to develop some possibilities of ways that NIH can help attract and train a pipeline of new and young investigators and I wanted to make sure we understood why so I haven't put the data here again NIH staff helped us by pulling out some data and that's in the report and the presentation that was made to Council, but it turns out that there are a really a low number of ME/CFS related applications funded because there are a low number submitted and it turns out that the grants that have been funded over the last few years are primarily going to the same subset of investigators. So this really speaks to a need to increase the number of people who are aware of and interested in research related to priorities for ME/CFS and so that that increases the number of applications so NIH can increase the amount of funding and really broaden that you know broaden the experts that are being groomed for the next generation. So next slide please. So the approaches that the Working Group suggested for this is first of all the low-hanging fruit if you will is to increase the awareness of what already exists, increasing the awareness of the disease. Setting up folks with, so new or young investigators setting them up with mentors who know the ME/CFS field and then promoting the importance and the value of research on ME/CFS and the willingness of NIH to fund research proposals that that come to them, also promoting a more collaborative or a multidisciplinary approach and encouraging the sharing of resources. Sometimes barriers to getting new investigators in a field as it can be that they don't have access to the same samples or they don't have access to the patients because those are with the people who have always been in the field, so we felt that it was really important to emphasize that NIH take a you know, take actions to ensure that there are wide access to biosamples collected from people with ME/CFS so that researchers across the country and across the world can do novel and innovative research, that data are collected in consistent ways so that you can compare data from one study to another and really even on the you know the fourth bullet here helping to define certain case, you know cases, of ME/CFS and I don't mean individuals, but I mean you know, phenotypes. And again as you know ME/CFS is very different from person to person and so there are these different sorts of cases for lack of a better word that studies might use or definitions of ME/CFS different studies may use and so really having these more standardized and very well defined, it could help standardize research and therefore learn from you know for more studies learn more from more studies around the world. And then of course strategic planning which comes up next is probably best informed how do you get new investigators involved. Well one way is to bring them into the strategic planning process, bring some investigators who maybe haven't worked on ME/CFS directly but are perhaps in related fields, and bring them into the strategic planning process of the a they bring their sort of outside viewpoint into ME/CFS but they also might then get hooked into the challenges and the importance and the need in ME/CFS research. And so finally on the next slide and I want to stop talking so we can move on and hear about you know what next, but the third charge of the Working Group was to devise an overarching research strategy and of course we don't, the Working Group was relatively small you saw the names on the slide, so really qualified to develop a detailed strategic plan for ME/CFS but we know that one is needed and that and so we recommended to NIH that that be an undertaking because there are such large gaps in our understanding of ME/CFS. What are the underlying mechanisms, what are the triggers, how do you define the different stages, how do you distinguish ME/CFS from other syndromes that may look and how do we you know with that knowledge how do we come up with markers and medical models, whether cellular models and/or animal models and really stimulate the development of new treatments, particularly drugs that  might be effective so I hope this overview has been somewhat helpful and just summarizing in five or ten minutes what are the highlights that were presented through the council on behalf of the ME/CFS Working Group. And so next up on our agenda is Dr. Vicky Whittemore who will tell us some about how the implementation of Working Group's recommendations is expected to proceed and in some ways is already beginning.

Dr. Vicky Whittemore: Thank you very much Steve that was an excellent overview of the work of the Working Group and so we really appreciate you being on and thank you once again for your chairing of the Working Group. It was really a fantastic experience I think for everyone who was involved. What I want to do today is to tell you all about our initial discussions and how we're thinking about implementation of the report from the Working Group of council and just by way of explaining how and who will be working on this. The implementation of the strategies and recommendations in the report will be coordinated by the Trans-NIH ME/CFS Working Group and we are working with our NINDS Office of Science Policy and Planning as well as the Office of Communications to really help us with this effort going forward. So we had the initial meeting of the Working Group to discuss the implementation of the report in September and we really took a careful look at what was in the report and if you've looked at the report you know that there are many different strategies that were outlined in the report itself and so taking a look at those strategic research objectives we identified that really a lot of them are for a strategic planning process are already outlined in the report and what we need to do is identify areas that will need additional research and discussion. Just to go back to the report for a minute these are all of the strategic areas that were outlined in the report and the way we've grouped the strategies that were detailed. And first encouragement of research topics, and approaches, instruments and Common Data Elements, data sharing and biobanking, increasing collaboration and cooperation, outreach to scientific investigators, general outreach, conferences and workshops, research training, scientific review and then strategic planning. The way we've begun to look at all of the strategies in the report is that is to put them into three different buckets. And the first bucket is are those strategies that we actually are already implementing things that we're already doing and taking a look at, are we doing as much as we can for each of those strategies and/or are there additional things that we should be doing. The second is short term strategy, so low-hanging fruit, things that we think are easy or easier to implement are things that we can implement in a fairly short period of time and then the longer-term strategies that are a little more difficult or will require a lot more time to implement across the board. So what I'm going to do today is just to tell you about some of the things that are already in progress, some of our short-term objectives, and then the longer-term, which is clearly the strategic research planning process and I'll come to that last. So first of all under encouragement of research topics and approaches, there were many strategies outlined in the report and all of those are going to be included in the funding opportunity announcements or FOAs for abbreviation that will be released as PARs, or program announcements with special review. We've incorporated all of those strategies into those funding announcements and we'll do that for any additional funding announcements down the road. Under instruments and Common Data Elements, as many of you know we have Common Data Elements that were developed for ME/CFS and so we're going to certainly encourage their use and include that language in the funding announcements as they're released. We also have just formed an ME/CFS Common Data Element Oversight Committee that will be looking at any new additions, any revisions that need to be made to the Common Data Elements going forward and we're working with all of the individuals that were part of the Common Data Elements group that helped to put those Common Data Elements in place to submit a publication it's currently under review at the CDC and here at NIH and will be submitted for publication very soon. In terms of outreach to scientific investigators, we are going to provide information on ME/CFS to the NINDS exhibit materials at professional conferences. This is something that we've not done in the past but will be added to our exhibit materials and will encourage discussion with investigators about research and research opportunities within ME/CFS. And then general outreach. We are continuing to respond to requests for information and interviews from the media, from other nonprofit organizations, and this is something that Joe Breen and I and Barbara McMakin from our Office of Communications do on ongoing basis as those requests come in. So under data sharing and biobanking we have an ME/CFS biorepository in place at our contract site which we call BioSEND. It's a biorepository that is located at Indiana University and we have specimens there from the CFI funded study and we're just in the process of adding the clinical data and will very soon make those biospecimens available to the research community and we also have a proposed pilot to add additional biospecimens that we're working on right now that will greatly expand the number of biospecimens that are in BioSEND and will be available to investigators. The neuro-related Institutes at NIH came together and have what they call NeuroBioBank, which is actually several different brain banks and banking facilities that are funded through contracts where individuals can donate tissues at the time of surgery or at the time of death and so this is a resource to that we're working with the nonprofit organizations to get the word out about the availability of this NeuroBioBank and we currently do have two brains from individuals with ME/CFS who were donated at the time of their deaths that are available for research and hope to expand that as well. We're partnering with Solve ME/CFS Initiative to support the development of their registry, patient registry, and a research app that's being developed by Columbia University through their funded Center, ME/CFS Center, and we're also implementing what are called GUIDs, which is a way to identify an individual who may be participating in multiple studies, because what you don't want in research is to think that you're using say 100 samples from a hundred different people when in fact half of those individuals were participating in two different studies so you really are only using say 50 samples from 50 individuals. So it's a way that we're going to implement this through the network initially and then hopefully on a broader scale for ME/CFS research. And then last in this category we're working with RTI, which is the Data Management Coordinating Center for the ME/CFS research network, to develop what we're calling MapME/CFS, which is a really awesome database that they're developing as a way for investigators to share data and datasets. So initially this will be piloted and used within the network and then eventually opened up for greater use by the research community. In terms of outreach to scientific investigators, we have ongoing support to investigators who contact us all the time about interest in submitting grants on ME/CFS, they submitted a grant that was in review that was not funded, we work with them to improve and help them to revise their applications. We also are doing outreach as I said at professional society conferences and as soon as the funding announcements, the PARs, are ready to be released or are released we'll do an email blast to the ME/CFS research community and beyond. In terms of conferences and workshops one of the recommendations from the Working Group was that NIH hold regular conferences such as the one that we organized here at NIH on the NIH campus in April of this year and we are still considering how to implement that request or that strategy, but in the meantime we are supporting, NINDS will be supporting, a conference grant to Dr. Fred Friedberg for the IACFS conference that will take place in June 2020. And we will also have plans for participation in the Invest in ME conference that will take place in London in May 2020, including the Young Investigator Workshop that they hold just prior to their actual conference every year. So some short term strategies that we're working on is to increase collaboration and cooperation. Steve very nicely explained our plans for the Interagency Working Group so we're now looking at how to actually put that in place. And we will be doing that hopefully within the very near future because we see the great need for better communication and collaboration across the federal agencies as well as with other patient advocacy groups. And we're looking at ways in which we can coordinate research on overlapping conditions, for example with the Pain Consortium here at NIH and other groups as well. Under research training, we're continuing to work with Invest in ME to implement early career tools and activities and as I said we are going to continue to do and increase our outreach to other scientific investigators. So in terms of strategic planning as I said many of the research strategies were outlined in the report so we're pulling those together into the beginning of a strategic planning process and we're looking at ways to strengthen our ongoing activities as well as to identify gaps. We're looking for, to identify areas that require additional discussion and what we'll be doing for those likely will be to develop and have organized and hold workshops or think tanks to bring individuals together to really focus on those specific areas. As part of our strategic plan we're doing an overall planning for the process for the  research strategic planning and how we will actually roll this out, who will be involved, that's all currently under discussion and hopefully we'll be able to report on that in the very near future as well. So with that I will end and this is the link to the report from the Working Group of Council it's kind of a very long URL but it's also on the NINDS website under the council information, Advisory Council information, but I'll leave this up here for a while if you want to write it down. In addition these slides will also be posted on ME/CFS website NIH website together with the transcript from this call so you'll be able to find the link there as well. So with that I will, okay sorry I'm just getting my instructions here. So at this point we'll go to questions and so please use the raise hand feature on WebEx if you would like to ask a question and we will unmute you so that you can ask a question and one of us here in the room will attempt to answer your questions. So please raise your hand if you have a question to ask. So first we'd like to call on Claudia Carrera. Claudia your line is open.

Claudia Carrera: I am wondering about the timeline here for all of these issues because at the September NANDS Council meeting we heard that the council had approved program announcements for ME/CFS all the way back in May and so I'm really glad to see that those are you know that the implementation is in progress, but I'm wondering when exactly they're going to come out and can you commit to having them come out before the end of November which will be six months in advance of when we're funded. And I also am interested in knowing how much money would be included, if there would be guaranteed funding included, and how much it would be.

Dr. Vicky Whittemore: So first of all they're program announcements with special review, which means there is not set-aside funding so there's not specific funding associated with those PARs. It was a way to stimulate research as well as to direct grants to the ME/CFS Special Emphasis Panel because we're finding that ME/CFS grants were being reviewed in other study sections that really did not have the specific expertise. So they are in process. You can imagine it takes a lot to coordinate input from 24 different Institutes, Centers and Offices to put one of these PARs in place. So the concept was approved in May and we're close to submitting them for review here at NIH and I can't guarantee when they will come out because that really is dependent on the review process in the Office of Extramural Research, so we are working to feverishly to get them out and we hope they'll hopefully come out as soon as possible. So the next question is from DL. I'm not sure who that is, but you're unmuted.

DL: Thank you. The NIH clinical study uses experts to adjudicate patients in order to ensure that only appropriately characterized patients are studied. This is done in part to avoid the polluted cohorts that have been studied in the past and that make up such a distorted base of literature that gets conflated with ME. While the NINDS report talks about the need for a strategic plan, research case definition, et cetera there are no ongoing or scheduled efforts currently underway regarding development for these. The absence of an appropriate and a research case definition, please detail for us how NIH will ensure that all ME studies have the same rigorous adjudication criteria as a NIH clinical study thank you.

Dr. Vicky Whittemore: So I'll respond and see if Dr. Koroshetz also wants to respond. So one of the things that came up in discussion in our Trans-NIH Working Group meeting in September was the need to really bring together the experts to identify the case definition or diagnostic criteria that we will be using going forward and to really address clinical outcome measures that can be utilized in studies that are validated and specific for this community. So those are areas that we identified really do need for the research and discussion and are really were beyond sort of the tasks of the Working Group itself and as beyond the really what the Trans-NIH Working Group can do, so we will likely be pulling together workshops to really address those specific issues. Dr. Koroshetz?

Dr. Walter Koroshetz: Yeah I just add that you know that the study at NIH is looking at patients who fit previously established criteria for ME/CFS and we feel quite secure that the patients coming in do fit all those criteria, but I would say that they're also only studying a subset of those patients so I think we need to get at is a way of subdividing the multiple different types of ME/CFS that we may be dealing with. And so for instance the protocol at the NIH Clinical Center is just honing down on one subpopulation, which is those folks who are within a certain period of time of the onset and when the onset appeared to be related to an infectious-like illness, so it's an example of how when one studies ME/CFS, one not only has to be observant to the criteria that've already been established but also to indicate what subgroups of ME/CFS patients one might be studying.

Dr. Vicky Whittemore: We don't see any other questions. If you have a question at this time would you use the raise hand feature so that you can identify yourself as wanting to ask a question? I see a question from Charmain. Charmain your line is unmuted.

Charmain: My question is will pediatric studies be specifically included in the PAR announcements or mentioned specifically in outreach to investigators?  

Dr. Vicky Whittemore: Joe and I are looking at each other. They're not excluded but whether we specifically call them out I would have to go back and look at them. I'll turn the question back to you. Do you think that's a critical piece that needs to be included? Yeah we certainly can do that if we didn't. I don't think we specified adult or pediatric but I think we can specifically call that out. Any other questions, use the raise hand feature. I see a question from SS, your line is unmuted. SS are you there? No, okay I see question from Liz Burlingame, Liz your line is unmuted.

Liz Burlingame: Yes, during the announcement after you released this report sounded like Dr. Roberds said that ME is actually a disease of exclusion. I'm really worried about that characteristic. According to the 2015 IOM Institute of Medicine report we are our own disease. We're talking about the post-exertional malaise. Can you please speak to that?

Dr. Walter Koroshetz: I think that was, if I recollected, that was an acknowledgement of the inadequacy of our diagnostic criteria but not in any way reflecting on that it was not a disease. We think that, I guess, two points, one is that we need to develop positive diagnostic criteria that have, you know, biological markers with them, a blood test, an imaging test, a physiological test. Really important if you're going to screen patients, if you're going to subdivide different patient groups. And then I think also I'm not sure if Dr. Roberds was hinting at this, but we know say from the clinical study at NIH, it's incredibly important to identify other disorders that have a known pathophysiology but can present with symptoms of ME/CFS. Some of the discoveries the Clinical Center we're finding underlying diseases in people who had ME/CFS and were unknown to have these other disorders, oftentimes the autoimmune or chronic infections.

Dr. Steve Roberds: Thanks Dr. Koroshetz. I remember saying that and was really thinking of, I think the first situation, where it shouldn't be a disease of exclusion, we don't want it to be that, and that's I think one of the reasons that a clear research strategy and a lot more research in the area is needed to identify what we can use as a marker to definitively give a diagnosis rather than not being able to find any other diagnosis and then labeling it as ME/CFS and we don't think that's appropriate. We want to move research so we can get away from that.

Dr. Walter Koroshetz: Thanks Steve.

Dr. Vicky Whittemore: Okay I see a question from Courtney Alexander. Courtney your line is unmuted.

Courtney Alexander: Hi I'm hoping this works through the phone. I want to thank you all for your presentation and the detailed work you're doing on this disease and I wanted to particularly echo Dr. Koroshetz, your comments and I appreciate them, on the need to carefully subset patient groups for studies and in particular for movement toward treatment trials and I just want to raise the bar on moving this research toward the ability to try treatments, run treatment trials, run rigorous trials that will get us to approved treatments and urge this whole process to move as quickly as possible to helping lay out the foundation for ultimately approvable treatments for the disease. And I think the subsetting is essential but I also think NIH and NINDS can play a leadership role in starting to fund some trials that will bring us some of the response, you know the criteria, the characteristics the you know, that characterize response in subsets of patients that we're going to learn more on the road to subsetting very specifically if we start studying that worked for some and not for others and identify those the markers that can separate those groups and reiterate the need to move that on a parallel track with the research where you all have laid out and are pursuing and moving through the different Institutes and investigators but the parallel track is essential for the changing the dynamic that there are zero treatments for patients that are that even approved under the FDA. Thank you again for your work, it's well needed.

Dr. Walter Koroshetz: Thank you Courtney. I couldn't agree with you more and you know that is really what everyone's working for and you know I would just say that you know in a rational drug design one is looking for a biological target that a particular treatment will hit that is then thought to be beneficial and so there may be many multiple different targets depending on the subset of ME/CFS patients we're talking about or there could be you know for instance something that is symptomatic, a circuit that would improve you know say fatigue or post-exertional malaise, that symptomatic treatment might work for many different forms of ME/CFS so that's kind of the goal of the work is to identify these targets and go after them. Either things that will be what we call disease-modifying and potentially cure the disease but also things that are going to be a symptomatic benefit without a cure but can still really improve functional level for folks who are suffering. Well thanks for that.

Dr. Vicky Whittemore: Yeah, thank you Courtney. So next we'll take a question for Winston Christy-Blick, your line is unmuted.

Winston Christy-Blick: Hi there, thanks so much for your work to help us get our lives back, we so appreciate it. I'm wondering in particular how are the efforts of the Working Group will help us get around this catch-22 of being unable to secure funding because research being proposed wasn't sufficiently hypothesis-driven and getting kind of the background research we need to just get our footing in this space.

Dr. Vicky Whittemore: So I'd have to say that in the five years that I've been really focused on ME/CFS the research grants are becoming more and more hypothesis-driven. There is still the need for discovery science as well and I think that's the interesting thing about the Special Emphasis Panel and the reviewers really I think understand that we're at a phase in this research where we really do need to do a lot of research to understand the underlying mechanisms and just lay the groundwork for research. In terms of securing funding, typically the way things function at NIH, short of a congressional appropriation for specific funding for things like, for example, the BRAIN Initiative or the opioid addiction now or autism or Alzheimer's research, we go through a process which is what we're in the middle of now in terms of doing a strategic research planning process looking at where the research gaps are and then issuing, if needed, the request for applications, the RFAs, where there would be set-aside funding. So I think that we're driving that process and hopefully as quickly and efficiently as we can. Like I said I think it's very, it's not all that common to have set-aside funding other than through specific requests for applications here at NIH. Dr. Koroshetz do you want to comment?

Dr. Walter Koroshetz: Well I just say that so the issue of I think I understand what the question is so the chicken and egg question, that you need to have hypotheses to get the grant then on the other hand you need data to somehow support the grant that collecting the data is going to give you the answer. But I don't see them as this data collection and hypotheses as mutually exclusive at all. Basically what the investigator has to put forward to the reviewers is that collecting the data is going to answer some questions that are important for ME/CFS and I think that you know there are really smart people out there thinking about it that certainly can put in grants you know in that space and certainly if you look at what's going on now in the consortium, it's primarily data collection to get answers for particular areas, it's not focused on one mechanism or another mechanism. We're just not there yet. But all those grants are definitely collecting data trying to see if it's pointing in a direction but some of them are you know, there are hypotheses that you know are there potentially infectious agents that can be found in the bloodstream by different types of analyses that might have indicated that there was a trigger, so those kind of hypotheses are really still you know fundable hypotheses that will allow data collection and you know it's when we give grants out particularly these R01 grants, one can move with the data you're not forced to do exactly what you propose but you can go in the direction the data takes you.

Dr. Vicky Whittemore: Okay thank you Dr. Koroshetz. The next question is from Emily Chablet. I'm sorry if I mispronounced your last name. Emily.

Emily Chablet: Hello can you hear me all right?

Dr. Vicky Whittemore: We can thank you.

Emily Chablet: Great, I'm Emily Chablet. I'm the patient administrator for the Stanford in-patient groups and for Kaiser's ME/CFS patient groups. So I'm in sort of a unique position in that I hear the stories of 450 and more patients every day and what works for them. What I am most concerned with is what's working now, what are the best working protocols now, and we and part of, I understand the importance of the research and identifying the specific biochemical processes that are that underpin ME/CFS but I am most interested in a boots-on-the-ground approach. What are the best treatment protocols out there now? And I look at the disease maybe a little bit different from you folks in that I see component illnesses that already have treatment protocols and so what are the best protocols for those component illnesses and how do they inform an overall treatment protocol. And so my approach I would like to ask is there a way to put together what are the things that are really working right now that can really help patients right now? I have heard so many things from patients or let's put it another way I've had many patients tell me this really works for me and that has come up for a lot this so that I've seen patients regain functionality from say 40% to 70%, from 10% to 40%, and it seems like every little tweak that could be put into the best working protocol increases functionality by say a percentage of 5% something like that. Is there any effort being made to put together the best working protocol at this time and of course that would be updated as we get better research. I'm the most interested in how do we apply the best research we know now that's corroborated scientific research and apply to a working protocol.

Dr. Vicky Whittemore: So thank you for that question and I actually had the privilege of visiting Cindy Bateman's clinic, the Bateman Horne Center in Salt Lake City last week and had lengthy discussions with her about this. She has been heading up a clinical consortium that really has two goals and one is to do exactly that to compare notes across clinicians and input in terms of what is working for these patients or what absolutely doesn't work in this population and sort of getting a better sense of what treatments work specifically for components of the disease. For example her specific interest is orthostatic intolerance and so that clinical consortium is in process and is working together. Clinical care really in that vein falls outside the mission, the research mission of NIH but not to say that new information coming from the research can't inform and won't inform in the future those kinds of clinical protocols. But we have, I think, great interest in the Trans-NIH Working Group representatives are very interested in absolutely translating what we can from research and from those clinicians who are out there caring for individuals with ME/CFS to provide information for the best care for individuals with ME/CFS.

Dr. Walter Koroshetz: I would just add that the role of NIH in this space is to provide the evidence that will inform the doctors and patients on what to try and so we do that through what's called comparative effectiveness research. So these are clinical trials in which patients are randomized to one treatment or the other to see which one is best and that is generally how NIH has developed the evidence and informs doctors on what to try. I mean would you come up. what you're presenting is actually even more innovative than that and something we haven't done as much of but as important is to actually develop algorithms for treatment so when you see a patient for instance with epilepsy you may try drug A because the evidence says drug A is the best for a population of patients that was studied but your patient may not respond to drug A and then you would move to drug B, so that kind of stepwise treatment algorithm is also something that could be studied in comparative effectiveness testing, say one algorithm versus another one as we've done that in some senses with the treatment of status epilepticus in the emergency setting looking at one algorithm versus another, changing drugs to see which one is best. So those type of research questions are certainly the kind of things we'd like to see. That requires a consortium of investigators to kind of come together and you know put away their own personal favorites to test things in a kind of organized manner but that is certainly something we'd be interested in.

Alissa Gallagher: Okay at this time we have time for one more question on today's call and I know that there might be other people that we weren't able to respond to so if you can send any of your unanswered questions to braininfo@ninds.nih.gov or simply go to the NIH ME/CFS website and click on the “Contact Us” link to submit those questions. Now the last question for today.

Dr. Vicky Whittemore: We're coming back to SS, your line is unmuted if you have a question.

SS: Thank you. First of all just wanted to let you know most of us could not hear Dr. Roberds at all. We tried to get in touch through this format and of course there was no response. So just wanted you to know that was a complete waste for us. It's very unfortunate. Moving on in the previous meeting on September 4th Dr. Roberds announced that ME/CFS is a diagnosis of exclusion. We were horrified to hear this misinformation. The IOM report stated that ME/CFS is not a clinical diagnosis of exclusion. Could somebody brief Dr. Roberds so that NIH is not spreading misinformation? We were shocked at this sloppiness. Please do better.

Dr. Walter Koroshetz: So I think we addressed that question earlier that the comment was that is a testament to the inadequacy of our diagnostic test is not to mean that it's a diagnosis of exclusion is to mean that it should not be a diagnosis of exclusion, but in many clinics around the country that's kind of a so, that was a comment indicating that we really do need better diagnostic criteria to nail down this diagnosis so apologize for that. And for Dr. Roberds yes I'm sorry, we could hear him well here in our room so and we're on yeah, not sure what the problem was. Were you on a telephone or on the computer?

SS: A telephone. And also I understand what you were saying in your response but what I'm saying and I want you to understand this so please tell me you know I've had ME for forty one years so I may not be explaining this accurately but what I'm asking really what I'm saying here today about Dr. Roberds announcing that it's a diagnosis of exclusion.

Dr. Walter Koroshetz: No, he did not say, that is not what he meant.

SS: Let me finish. Let me finish. That the issue is for people who are listening in on this call who may not know the backstory or the other 85% of the iceberg they're hearing misinformation so when NIH speaks you need to be accurate and that's what we're asking for. We can't rely on you to have to come in and clean up misinformation afterwards. We're asking you to be accurate in what you say and that was inaccurate. Do you understand what I'm saying?

Dr. Walter Koroshetz: Yes, yes, I do but it was certainly not intended to be that way.

SS: But it was misleading and that's the issue and I don't want to fight or argue with you because I'm sick.

Dr. Walter Koroshetz: I understand.

Dr. Vicky Whittemore: Okay so at this time we're going to end the call and we thank all of you for being on and participating and for all of your questions and Alissa if you want to wrap up?

Alissa Gallagher: In closing today's call I just want to remind all of you about our listserv for updates from NIH. 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. Thanks again for all of your informative and thoughtful discussion and we wish all of you a good afternoon.

Dr. Walter Koroshetz: Thanks very much everyone.  

Dr. Vicky Whittemore: Thank you.





This page last reviewed on December 5, 2019