November 2, 2016

NIH ME/CFS Advocacy Call



Moderator: Marian Emr
November 2, 2016
2:00 pm CT

Coordinator:    Welcome and thank you for standing by. At this time all lines are on a listen only mode until the question and answer session. At that time if you’d like to ask a question, you may do so by pressing star then 1 and recording your first and last name. 

Today’s call is being recorded. If you have any objections you may disconnect at this time. I would now like to introduce your host for today’s call, Ms. Marian Emr. You may begin. 

Marian Emr:    Good afternoon, my name is Marian Emr and I’m the director of Communications and Public Liaison at the National Institute of Neurological Disorders and Stroke. 

On behalf of the NIH, I’d like to welcome you to this afternoon’s teleconference and to thank you for your interest in participating in this discussion with us today. 

Here at the table this afternoon we have Dr. Walter Koroshetz, director of the institute, Dr. Vicky Whittemore, Dr. Joseph Breen, and we are hoping to soon be joined by Dr. Avindra Nath, who is in D.C. at a ceremony honoring one of NINDS’ physician scientists this afternoon. 

Dr. Koroshetz will introduce the speakers, each of whom will make some brief remarks. After which we’ll open the phone lines for your telephone questions. Please be patient with us through this process. We will try to get to as many questions as we can in the time we have available to us this afternoon. Now, Dr. Koroshetz. 

Dr. Walter Koroshetz:     Hello everyone and welcome to the second telebriefing on the work that NIH is doing in ME/CFS. And I want to just start off by just briefly describing what NIH does and how it relates. 

The National Institutes of Health is the country’s investment in biomedical research. And our mission is generally seen as a two-part mission. One is to seek fundamental knowledge about the nature and the behavior of living systems. And to learn about diseases and apply that knowledge then to enhance health, length in life and reduce illness and disability. 

From the ME/CFS side of things, that’s really what NIH is trying to do, to learn much more about this mysterious illness in a very methodical, fundamental and rigorous way. And then to use that knowledge to try to reduce the disability and the burden of illness due to ME/CFS. 

Now NIH, as you may or may not know is not a monolithic organization, but it’s made up of 27 institutes and centers with their specific research agendas. And these centers and institutes receive their budgets from Congress. But in actual fact, NIH is really the community of investigators who are outside the walls here at Bethesda, but at the university’s medical systems throughout the United States. 

So we’re a funding organization. And we - most of our funds go out in grants to investigators outside of NIH. The exception being the intramural program here at NIH, which we’ll talk about their ME/CFS program shortly. 

Important to note is that, as I mentioned, the budgets go to the different institutes. And I think we’re very fortunate that we have a very vigorous Trans-NIH ME/CFS Working Group. 

And this has representatives from over 24 institutes and offices and centers across the National Institutes of Health. So these are people with different types of expertise in different areas, but it’s a very broad range of expertise that we can bring together at the Trans- NIH ME/CFS Working Group. 

And in addition, those representatives have the ability to go back to their institutes to bring in funds from their institutes to the work on ME/CFS. So I think we’re in a good situation with a very good Trans-NIH Working Group. 

And with that I want to turn over to Dr. Breen from the National Institute of Allergy and Infectious Diseases. And he’s going to talk to you a little bit about the generic process in which the NIH funds go out to investigators. So Joe, why don’t you give the group an overview there. 

Dr. Joseph Breen:    Super. Thank you Walter. So my name is Joe Breen and I am the National Institute of Allergy and Infectious Diseases representative to the NIH Working Group. 

And I’m a member of this group because in my group, we are in charge of the immunology grants, particularly immunology of chronic fatigue syndrome. In NIAID we also have a number of grants that have to do with looking for the etiology of chronic fatigue syndrome. 

So there are a number of these opportunities and applications that are currently being supported. So I want to tell you a little bit about some general info about NIH grants as it pertains to chronic fatigue. 

I want to remind you that the majority of research grants that are funded are actually not in response to specific calls for applications, or RFAs, but are investigator-initiated projects. 

And as always, applications for ME/CFS research that score well during peer review will be funded. Researchers do not need to wait for a disease-specific RFA before sending in their applications. 

And applications that score well anytime throughout the year - and we evaluate them three times a year - regardless of the disease area, will be funded. Ultimately this is the way to build up the research field in any disease area, particularly ME/CFS. 

This is where advocacy organizations can help. Research-based organizations can provide seed money to fund pilot projects and allow investigators to collect preliminary data, which can be the basis for a future NIH application. This has been a successful model in multiple diseases, including Lyme disease and epilepsy. 

In addition, advocacy organizations can help in this effort by spreading the word that NIH is looking for projects to support. We’re really committed to solving the mystery of ME/CFS through research and better understanding. 

We know there are scientists and clinicians out there who may have really innovative ideas on how to tackle ME/CFS, but we simply can’t fund them if they don’t submit a grant application. 

All applications undergo rigorous peer review to ensure meritorious projects receive funding. In fact, that’s by law that all applications must undergo peer review. 

Applications related to ME/CFS are reviewed by a Special Emphasis Panel of experts assembled just for that purpose. We encourage researchers, especially young researchers and those new to the field who are considering putting in applications to first contact the program director from an institute or center involved. 

And you can find program directors on the NIH website for chronic fatigue syndrome. There’s a list of all those who are participating. And you can contact people on that list. And if they’re not the right person, they’ll find the right one for you. 

Putting together a grant application can take considerable time and effort. And our job here as program directors is to help investigators and guide them through the process. 

So I wanted to take a second to follow up about the International Association for Chronic Fatigue Syndrome ME meeting that happened last week in Florida. Dr. Vicky Whittemore and I had the pleasure of attending the meeting. 

And it was a very productive meeting and we enjoyed meeting many researchers. And NIH was proud to have been a cosponsor of the meeting and provide travel grants for young investigators to attend the meeting. 

And during that meeting Dr. Whittemore, myself and other NIH officials hosted a grants-writing workshop to help researchers navigate through the grants application process. It was well attended with a vigorous question and answer session. And we hope that the participants found it useful. 

Any investigators considering submitting applications should contact a NIH program director for guidance in the process. Again, that’s our job here is to help and to really build up the basic infrastructure and understanding of this disease. I’ll turn it over back now to Walter. 

Dr. Walter Koroshetz:    Thanks Joe. So that was I think a good overview of how NIH works on a day-to-day basis. And we emphasize the point that while funding goes through a peer review system, which is a very competitive system, which is kind of the hard part for investigators to stay in the game is the fact that our ability to fund research is heavily resource limited. 

So at our institute, NINDS for instance, only the top 12 percent of the grants actually get funded. So the process is a fairly harsh one because it’s so competitive. 

Now that being said, I would say that going around the world and seeing what goes on in other countries, there is tremendous advantage I think to our system because it is competitive and it’s also open. 

It’s completely open, so the funding decisions are not top-down, you know, this much money goes here or there. But it’s really much more bottom-up generated by the investigators. 

Now I’m sure everyone on the phone is thinking the same thing, which is what we need to get those kind of investigators so we can compete for the ME/CFS funds to move our science forward. 

And that’s actually very, very true. And so in the instances, particularly with regard to ME/CFS, but not solely ME/CFS, NIH does try and think about how investments can be seeded or can really change the research landscape in particular disorders. 

And particularly ones where the research really needs to be tuned up multiple notches. And so that’s what the Trans-NIH ME/CFS Working Group has really been thinking about now over the last couple months. 

And so I’d like to turn it to Vicky who will explain to you a little bit about what the Trans-NIH Working Group has been thinking. And then what we hope to do is to tell you about a really exciting program that we hope to roll out for two reasons - one, is it’s exciting and, two, is that I think the people on the phone can really help make it successful. 

Without the input of the patient groups, it’s very - it’s really impossible to do really good clinical research. So we’re hoping that we can build a partnership in this new program going forward. But let’s turn it to Vicky and she can talk to us a little bit about the work that the Working Group has been doing, particularly to energize our research in ME/CFS, so Vicky. 

Dr. Vicky Whittemore:    Thank you Walter. So the Working Group, as you may know, has been working hard since last fall. And we’ve been outlining short-term, intermediate and long-term goals. 

And we’ve accomplished several of our short-term goals already, which includes stimulating research through a notice for the availability of administrative supplements which went out. 

And this is a way in which NIH can provide additional funding to currently funded NIH researchers. And so through that mechanism we were able to award seven administrative supplements, five from NIAID and two to researchers funded by NINDS. 

In follow-up to the P2P workshop that was held in December 2014, we held a Federal Partners meeting to follow up on the recommendations that came from that workshop and to develop next steps and ways in which the Federal agencies can partner and collaborate to move research in other areas of ME/CFS forward. 

And a summary of that Federal Partners meeting is in preparation now. So we also are working to identify research priorities. And many of you participated by submitting responses to the Request for Information we put out. 

We received many excellent suggestions and comments from ME/CFS stakeholders that we’re going through and really trying to prioritize now and look at the areas that are the most fruitful for research in this area moving forward. 

One of the things that we’ve just initiated, and if you were at the conference last week, you may have attended the evening session we held to discuss our Common Data Elements project. 

And we feel this is a really important step in developing common ways - essentially a common language for collecting clinical data on ME/CFS such that studies can be done using the same data collection tools and language so that we can compare across sites and across studies to really foster research. 

And we had a lot of really good input during that meeting we held last week in Fort Lauderdale. And many individuals have stepped forward saying, you know, we already have something. We want to participate. We want to harmonize all of these data elements across the different research groups, both here in the United States and in Canada, and in Europe as well, which is really, really terrific. 

And in terms of international collaborations, we really are hoping to foster those international collaborations going forward. And are already in discussions with our colleagues in Canada. 

I’ve been contacted by folks in Europe and the UK, as well as in Australia. So we think there’s really opportunity for tremendous collaboration to really build the research infrastructure. 

In terms of intermediate goals, we have been developing an extramural research program, and this includes the RFAs that Walter mentioned. And before I discuss those though, we’re also, as Joe mentioned, we’re trying to encourage individuals to submit grants through our regular funding mechanisms because we can’t fund grants we don’t receive. 

And so we need to get those grants coming into the system and being peer reviewed. We’re also encouraging young investigators to submit postdoc grants and clinical fellowships. These are avenues that are open to any investigator during their training and their career development phases to submit grants that utilize these mechanisms specifically for training. 

So moving to the RFAs, we issued two notices that we intend to publish RFAs. And the notices went out in late October. And one notice was announcing that we intend to publish an RFA for multidisciplinary, multi- site, ME/CFS collaborative research centers. 

And then the second is for a data management coordinating center that would work with the centers to coordinate their activities, their research as well as data collection and analysis. 

So we hope that these centers would help to foster the use of the common protocols and Common Data Elements across sites, as well as to really enable and support research at academic centers in the United States. 

So we’re hoping where applicable that the investigators will also partner with the NIH funded Clinical Science Translational Awards. This is a program funded through our translational center here, NCATS. And they can provide additional training and infrastructure for the ME/CFS centers as well. 

And we definitely encourage involvement of young and new investigators, both basic science and clinical, through these centers. And an important component will be that they will require community engagement and involvement to ensure success of the center program. 

And as I said, discussions are underway for additional partnering and collaboration with other countries and other networks that currently exist. 

So in terms of long-term goals, clearly our goal is to continue to support and expand ME/CFS collaborative research. And to support the development long-term in new therapies and treatments for ME/CFS. 

We hope that one day we will be able to encourage and support clinical trials for ME/CFS and ultimately the end goal is to improve the quality of life for all individuals with ME/CFS. I’ll turn it back to you Walter. 

Dr. Walter Koroshetz:    Great. Thanks. So I just want to just emphasize the main point that Vicky ended up with, which is that the Trans-NIH Working Group feels that the most important thing we could do at this time is to incentivize a coordinated approach to research in ME/CFS but again, it’s not top-down. 

It’s going to be investigators coming in with their best ideas, coming in with a commitment to work together. And to really try and go after what are the most important problems that need to be solved that will enable better treatments for patients. 

This process I think has worked in many other areas at NIH. So many of the institutes have research consortia that work on trying to solve problems in their Institute. 

There’s one which we were very interested in looking at, which is the Rare Disease Consortia that runs out of NCATS, which Vicky mentioned. And that’s been very successful at getting investigators together to start research in an area where there hadn’t been much research in the past. 

The other thing we’re hoping is that this coordinated approach will bring new people in to the research area of ME/CFS. It’s going to be critical. And this is clearly going to be the case that these consortia will all be working with patients, working with physicians who take care of patients with ME/CFS. 

And so building those alliances are going to be really essential to the success of the consortium. As I mentioned, we’re not going to force them to do Project A or B. That’s really not the role of NIH. 

The role of NIH is to listen and try to allow people’s creativity to come through. And to fund the most innovative, high impact research. And that comes from the investigators themselves. 

So I think most importantly, now is the time to try to drum up interest on the part of investigators who were very interested in ME/CFS, and maybe investigators who may not know much about ME/CFS, but know how to run clinical research networks. 

And to form the alliances between the patients and the physicians and the investigators who will be interested in competing for these awards. And I think, you know, we’re committed to this in the long-term. 

So these are all multiyear projects. We’re going to be very sensitive to make sure the timing’s right so that we can’t at this point say, you know, how many there are going to be. It’s going to depend on what comes in. 

Oftentimes at NIH the first attempt has got problems, and people have to go back and kind of re-calibrate and come back in with a second attempt to get funding. So all these things could come into play. 

So it’s pretty hard to know exactly what timing is going to be and what the finance is going to be. But we’re committed in the long run to build a high impact consortia to move ME/CFS research forward. 

I’m sure there will be questions about it. Before we go there, I wanted to ask Dr. Avi Nath who runs the intramural protocol for ME/CFS to give an update on where that is at this point, so Avi. 

Dr. Avi Nath:    All right, so we have been working very hard in getting this protocol up and going. It’s a very complicated protocol with lots of different arms to it. So it’s taking us a little bit longer than we thought to get started. But nonetheless, we have now and it’s open for enrollment. 

We have brought in two healthy volunteers to the Clinical Center for that purpose. One is currently here at the moment and one we had the last week. And then there are a few more lined up to come. 

And then starting early next year sometime we’ll start getting the patients with chronic fatigue syndrome, ME/CFS patients. We decided to go with the healthy volunteers first because we wanted to make sure we ironed out all the kinks before we bring in patients who are actually sick. 

And so things are working out well so far. We are pretty pleased with the way the study is rolling out. And so we’ll see from here. 

Dr. Walter Koroshetz:    And in terms of engagement, I understand there’s also been some focus groups that have been set up to talk to patients who have ME/CFS about what is the best interview tool to use to get at the post-exertional malaise. 

So a lot of outreach to patients as well guiding this protocol at intramural NIH. 

Dr. Avi Nath:    Yes so, you know, we have received a lot of input from the community and other researchers around the country. And we welcome that input and it helped us shape our study in many ways. 

And we continue to seek their participation in many different forms. The one is the focus groups that Dr. Koroshetz just mentioned. The other thing we also have the seminar series. 

And we’ve been bringing in people there that way too. And that allows us to bring in experts, various aspects of the disease. And they give us a seminar. They spend the day with us. We have discussions with them and show them what we are doing and receive feedback. 

Similarly we formed the committee of experts who will provide us feedback on the patients that we are recruiting to make sure we get the right people that meet our inclusion criteria. So we’ve done our best to involve and inform ourselves as we go forward. 

Dr. Walter Koroshetz:    Great. Well thanks everyone for the updates. And so just to summarize, we talked a little bit about NIH’s strategy as it applies to ME/CFS both trying to encourage applications from across the country. We are always open for applications. They come in three times a year. And all good ideas are sent in for peer review. 

And then we talked about the particular focus targeted initiatives to stimulate ME/CFS research. And especially the new program to develop a research consortia in the U.S. to really work on trying to develop better knowledge and better treatments for patients. 

I would say that that consortia also makes it easier for us to collaborate with similar groups that are amassing say in Canada or in England. That if you can imagine how networks can coordinate their activities once they’re established. Harder to do for the smaller individual grants, but easy to do for consortia. 

And again, not to overemphasize, because it can’t be overemphasized that we really need the patient community to play a strong role in ensuring the success as we roll out these research consortia. 

So with that I think we can open up the lines for questions. We’d be happy to answer questions as they come in from the group. So thanks again for your attention. Look forward to the questions. 

Coordinator:    At this time we’d like to begin the formal question and answer session of the call. If you’d like to ask a question, please press star, then 1 and record your first and last name. 

To withdraw your question, you may press star then 2. Again to ask a question, please press star, then 1 and record your first and last name. One moment for the first question please. 

Dr. Walter Koroshetz:    Is somebody on mute? 

Marian Emr:    Could you open up the line for the first question please Lauren? 

Coordinator:    Our first question comes from Robert Miller. Your line is open. 

Robert Miller:    Yes hello. Can you hear me? 

Dr. Walter Koroshetz:    Yes, very well. Thanks Rob. 

Robert Miller:    Hi so Robert Miller here. Long-time patient and patient advocate with ME/CFS. I appreciate the get together here Dr. Koroshetz, Dr. Breen and Dr. Whittemore and Dr. Nath. 

I’d like to make what are two key points. One is that it’s great to hear about there being RFAs put up to try to attract researchers, but I’ve heard, since those two RFAs have been produced is that the research community is really not going to, and I’m talking about the more experienced researchers here, are really not willing to come to the table and put forth their time and energy until there’s a dollar amount that’s placed on these RFAs. 

There have been many long time researchers that have done, the time and energy put into being able to put in for studies that have just been repeatedly turned down over and over again. 

You know, they’re just not willing to commit the time and energy to do it unless they know that there’s going to be a specific dollar amount that’s attached to this so they can kind of figure out exactly what it is that they should be applying for. 

 I had one researcher recently basically say that there was discussion at the IACFS in regards to the clinical centers. And there is talk of maybe I guess a dollar amount of $2 million was spoken about. But they didn’t know if that meant per center or if it was $2 million to stretch out amongst various centers. So I think that it’s key that, you know, that that comes about, but there’s a dollar amount put on there. 

 And again, you know, reminding NINDS, and again I hardily appreciate that NINDS is taking the lead on this. But you guys have to remember that NIH is decades behind the curve for treating patients with this illness or doing research on patients with this illness. 

 And so, you know, we need you guys to really come to the front. And I keep hearing - I heard the term several times of this has got to be a bottom-up situation. And I completely disagree with you. 

 If you put the money there, the researchers will come. And if you don’t, they won’t. And if you get researchers, then you’re going to get what you’re asking for, which is people new to the illness that really don’t know a whole bunch about it. 

And once they get into this labyrinth of this illness, they won’t come back. So that’s one point. The other point is I’m chained to my house again because I cannot access Ampligen. And I have asked previously for the NIH, specifically for NINDS to do a clinical trial of Ampligen and basically determine, you know, how it works in patients because once that’s done it will certainly open up, you know, a purview of ability to try to determine potential biomarkers for this illness. And even bring other pharma in, you know, into the illness and the disease. 

And with that, of course, comes money. And that’s what this all boils down to, is money. And if there’s not money there, pharma doesn’t come. And with that money being there, the researchers - specifically the big name researchers just will not be there. So thank you for taking my question. 

Dr. Walter Koroshetz:    Thanks a lot Robert. So let me just start out by saying that the funding for the centers is, you know, is significant. We can’t give you a dollar figure at this moment because we only have what’s called an intent to publish notice out. 

But when the actual RFA comes out, it will have indications of the ballpark figures. But they are comparable to the consortia we have for other diseases. So this is not trivial whatsoever. This is a significant investment on the part of multiple Institutes. 

And I would say, I mean I should have said right from the beginning is that the funding is coming from multiple Institutes. So this is not NINDS carrying all the weight. NIAID and all the other Institutes are contributing. 

So I think that the, you know, you never - at NIH the secret is we never give as much money as it takes to actually get the work done. We rely a lot on the altruism of the investigators to push things forward for all the diseases. But this is going to be equivalent to any other centers that we do. 

And so I think that that’s not going to be the issue. In terms of people applying I, you know, I do understand that as I mentioned before, the process of being funded by NIH is a competitive one. 

And that’s, you know, that’s the process that we’ve always had at NIH. And it means that people get rejected all the time. I can tell you the stories of when my grants got rejected. And I’m sure everyone has those stories. But it’s persistence that pays off. 

And so to have people who are real champions for ME/CFS that are going to persist in this process because we’re talking long-term here. 

We don’t need ten, we need hundreds of people who are going to be working in the ME/CFS field. So that persistence is absolutely critical for people to be able to access NIH funding. But the RFA makes it, you know, it’s - this is only for ME/CFS research. 

So this is not, people outside of ME/CFS can’t compete in this world. This is a targeted initiative for people interested in ME/CFS alone. So I think this is got a good chance of working if the patient community can align themselves with these high quality investigators. 

But if that happens, then I think we’re - that’s, you know, it’s almost like starting a bonfire. You have a match, but if we can get this going, this could really spread across the country. So I understand your hesitation based on the past experiences. But I’m very hopeful we can turn that around with this new initiative. 

And with regard to any particular thing that’s going to be tested, I think the group is going to come together and they’re going to decide on what is the most important thing to test among all the consortia members. 

So again, there are a number of different ideas out there that different people think are the most important. And they’re probably all right, but we can only go after one at a time. So we’re going to - we’re going to ask, really force and challenge the investigators to come in with a proposal for a high- impact research study that they can do across all the research centers that come in here. 

So thank you very much. How about next question. 

Coordinator:    Next question comes from Eileen Holderman. Your line is open. 

Eileen Holderman:    Yes thank you to Dr. Koroshetz and Dr. Nath and Dr. Whittemore. My question is involving funding. How much has been allocated for the NIH intramural ME/CFS study? And what is the start-to-finish date and the publication date? 

And then my second question involves Dr. Walitt’s participation. I recently attended the IACFSME conference in Fort Lauderdale and I heard some experts saying that, you know, Dr. Walitt, they met with him. So could you just clarify his role in the study? Thanks very much. 

Dr. Walter Koroshetz:    The thing, I think, you know, it’s really hard to put a number on what an intramural NIH study costs for a variety of reasons. One is that - the most easy one to explain is it occurs at the NIH Clinical Center, which is a research hospital. 

And that cost is never factored into any of the protocol costs. So we have protocol costs that are, you know, for the items that get done in the Clinical Center. But then there are a tremendous amount of resources and really a research hospital that comes in to do the work. So the patients would be in-patients do you think? 

Dr. Avi Nath:    Yes so that is correct. The patients are admitted for a week. Their costs are all covered, all the costs for their investigations are covered, this is a substantial amount of cost. 

Now for example, my salary and a number of other researchers already hired by the government, so our salary is already paid for, right. So it doesn’t matter what study we take on, we don’t get any extra payment on top of that. 

So those costs are already factored into it. Which is very different from an academic center. When I was at Hopkins I wrote grants there, we would budget in our salaries or say 10 percent is my effort for this. I’m going to ask NIH for 10 percent of my salary. 

If I need a research nurse, I need to budget this out. But the research nurse is already employed, and we’re redirecting our efforts to the study, then we’re not budgeting per salary any longer. 

Well for the RFA, when people write grants, they will be able to and they will ask for every dime that they’re going to spend. And the intramural program, you don’t ask for those kinds of things. 

For example, routine care. Say I wanted to do a complete blood cell count on a patient, or I want to do a urinalysis on a patient and check them for diabetes or whatever, but those things are already covered by the Clinical Center. I don’t ask for extra dollars for that. 

But yes, I do get extra dollars to pay for the patients’ travel or their specialized tests that I would do, so we’re going to do some, you know, specialized microarray work or look at RNA analyses that are research things that are not available to the Clinical Center. 

And then I have to pay for them. And so those costs are definitely protocol costs. So we do have a budget that was allocated to us for this purpose. And that covers those additional expenses. 

And you said publication date. Research never works by publication date. So I never look at research in those terms. What you want to do is you want to do the best research once the study is finished. Then you analyze them. 

And then you look and see what is publishable and what is not. And try to get the data out. And so it won’t be a single paper. There will be lots of papers emerging out of it. Probably the first one large paper that will describe the cohort and whatever findings we have. 

And then subsequently there will be lots of papers on each of the individual parameters that we collect on them, including ways of standardizing how to examine these patients, how to work them up. So there will be lots of things that will come up. It’s hard to put a timeframe on it. 

And then Dr. Walitt, yes. So Dr. Walitt is a, you know, delightful individual, very experienced. I have full confidence in him. And so he’s doing a superb job putting this protocol together. He has done all the work in helping me. 

You know, there’s so much paperwork that has to go in to get these things approved. If I really didn’t have his help, I don’t think I could take this on at all. 

And then coordinating day-to-day activities, bringing the patients that need to, you know, blood needs to get drawn at some point in time. And MRIs to be done and something goes wrong. Scanner’s not working, whatever. Things have to be done. 

So a lot of day-to-day activities that you really need somebody like Dr. Walitt to really do it. He’s very methodical. He’s very compulsive. He’s very organized. So, you know, those are qualities that are hard to come by. 

And so I think he is ideally suited to help me out. I’ve developed a good working relationship with him. And so I think he’ll do a great job. I have no reason to be concerned about him. 

Dr. Walter Koroshetz:    So in terms of some of the info on the question. So the cost, you know, if you just think about bringing someone into a hospital for seven days, you know, that gets you $70,000-$100,000 per patient. 

And then the additional cost that Dr. Nath is receiving for testing, you know, puts the project certainly over a million dollars a year. It might be even closer to 2 million a year. But those numbers are very hard to pin down because as we said, the costs are paid for out of the general NIH budget, and not just the particular protocol budget. 

And then the protocol itself is planned for three years. Is that right?

Dr. Avi Nath:    So we have about 80 patients. And you bring in one a week is about as fast as you could possibly do. Plus there’s the screening things, you know, and then the holidays. And some patients cancel. We obviously have to factor those kinds of things out. 

So you can see that it will take a good three years to really recruit all these patients. 

Dr. Walter Koroshetz:    Okay great. Okay can I have a next question? 

Coordinator:    Next question comes from Brian Vastag. Your line is open. 

Brian Vastag:    Hi. Thanks for taking my question. You mentioned the Special Emphasis Panel that I guess has been around for quite some time. And I know there’s been a lot of concern in the patient community that in the past when grants came into the panel, you know, the comments that came back with grants that were turned down tended to just - they suggested to the researchers that the people on the panel maybe didn’t understand this illness very well. 

And so I’m just wondering if the panel has been changed recently or if you guys have tried to refresh it or update it or tried to get people who are maybe, you know, a little more, you know, who are closer to the illness and maybe know a little bit more about it? 

Dr. Joseph Breen:    Hi Brian, this is Joe Breen. Thanks for the question. I think it’s true that there have been people with concern about the panel. And I think the panel has evolved, and panel does evolve actually. 

It’s a Special Emphasis Panel that is - it’s not a standing group because it’s put together specifically for whatever group of applications that’s in for that particular round. 

So it definitely has changed. The composition of the panel is actually a public record. So one could look on the website for the Center for Scientific Review here at NIH and you can see the people that are on it. 

And I think that the staff at the center work really hard with program directors like myself and others to try and get the most accurate and fairest review as possible. 

And I can say that we are supporting a number of projects each year that come out of that. So there are projects that make the payline that Dr. Koroshetz mentioned each year that have been reviewed. So I think the process is working. I think there’s always room for improvement. 

And the other part of that is that I think as a program official, we try and work with the potential applicants to help recruit the kind of expertise that’s needed. In fact there’s a form now that’s built in to the way the applications are put in where an applicant would suggest the type of expertise that they need. 

Of course, they can’t name someone because that would disqualify them. So we try and make it so that it’s the ideal type investigator. Here’s the kind of expertise I need to evaluate my application. 

And I think that it’s better than it was. And I think we’ve learned, just as the field has evolved. And I think actually, you know, I mentioned at the meeting last week, I think there’s a lot of exciting science. 

So I think that this group is the place to evaluate it here at NIH, which by the way would be separate, a panel would be convened separately for a request for applications. 

This group that you mentioned is pulled together every, three times a year. But there are exceptions that come in directly from investigators not related to a specific call. So it’s ongoing. 

And it does change, I guess is the short answer. And I would encourage you to look because it is public information. 

Dr. Walter Koroshetz:    And I guess the other thing I’d add is just the honest truth is that because NIH doesn’t have the resources to fund a high percentage of the applications, most of the applications do get rejected. 

And there’s always, you know, a criticism of the review when you get rejected. It’s natural. And it’s not anything wrong. So you’re going to hear that. 

And everybody here at NIH hears it every day because unfortunately that’s just the way the finances play out. But I think our main thing is persistence by getting these reviews, getting the criticisms, trying to reshape the proposal to come in again, with those critiques answered. That’s the recipe for success. 

Dr. Vicky Whittemore:    This is Vicky, and I’d just like to add that as Walter was saying, every grant application that comes in is discussed and reviewed. And the investigators receive a summary statement. 

So the grants are accepted and reviewed. So there isn’t a process where a grant will be sent back unless it doesn’t meet the guidelines for the actual logistical forms of and information of the application itself, but grants come in and are reviewed and receive reviews. 

 So that everyone can improve then on their next application based on the critiques and comments that are received. 

Dr. Walter Koroshetz:    And the system is kind of like our justice system. It’s jury by your peers. So it’s never perfect. But if you look around the world, it is the most democratic and it, you know, most countries see it as the crown jewel of how money is apportioned in the U.S. But it is a very harsh system, that’s for sure because it’s so competitive. If we could go to the next question. 

Coordinator:    Next question comes from Mary Dimmock. Your line is open. 

Dr. Walter Koroshetz:    Ms. Dimmock, are you there?
Mary Dimmock:    Sorry, I was on mute. Thank you. Thanks for this teleconference today. Both the 2011 State of Knowledge report and the 2015 Pathways to Prevention report highlighted the need to reach consensus on the research case definition. 

NIH’s Common Data Elements is important but it does not address this need because it does not specify required inclusion criteria like PEM. What is NIH doing to help achieve consensus of this case definition? 

Dr. Vicky Whittemore:    Hi Mary, this is Vicky. As I pointed out, within the Common Data Elements, there are general core elements that are required for studies to use. And those are things like demographics,  so sex, location, age, et cetera. 

And then there are disease-specific core data elements that are agreed on. And until the group comes together and discusses what those core required disease elements are, we can’t say that PEM is not going to be included. 

In fact, I think it likely would be, and probably the community would agree, would be one of those required disease elements. So it’s not meant to focus on any specific criteria, but to collect the important clinical data that will be needed to carefully characterize the patients, either in a clinical study or eventually in clinical trials. 

Dr. Walter Koroshetz:    And can we have the next question please? 

Coordinator:    Next question comes from Diane Bean. Your line is open. 

Diane Bean:    Hi. Thank you for taking my question. I am on the Board of Directors of the Solve ME/CFS Initiative and the parent of a patient. And I have a question. I’m curious as to why the National Cancer Institute is not on your TransNIH Working Group, given that the Cancer Institute itself found an increase in lymphoma back in 2012 among ME/CFS patients. 

And also, my daughter who is a patient, participated in the big XMRV study that was started back in 2011. And then once the XMRV, the original paper was withdrawn, it seemed like, you know, I know that the study was completely abandoned, but there was never any follow-up with her. 

And I know that she and a number of other patients have a lot of data in your database. So I’m just curious as to why NCI isn’t on the Trans-NIH Working Group and if you plan to change that? 

Dr. Vicky Whittemore:    So in fact they are. NCI is on the Working Group. And they’re now, if you look at the listing for the Trans-NIH Working Group on the website, their representative is listed there. 

Diane Bean:    Okay thanks, my mistake.

Dr. Walter Koroshetz:    It’s a recent addition. And yes, I think it’s a good point. And that was something that we talked to them about. And they then have responded. And they do a lot of work in fatigue in cancer, for sure. So we’re looking across all the different diseases where - and trying to get clues as to what is the cause and the characteristics of ME/CFS. 

And as Vicky mentioned, it’s different from a diagnostic criteria that would be used for, you know, coding or billing or things like that. 

We’re really interested in breaking things down into pieces and trying to understand piece by piece so we can put the big story together. Great, can we go to the next question? 

Coordinator:    Next question comes from Donna Pearson. Your line is open. 

Donna Pearson:    Hi, thank you very much. I appreciate this update. And I just want to ask you, I think Dr. Koroshetz said that you’re asking the patient community to align with high quality investigators. And you stated that we need champions. 

And we do have champions. And they’ve been working for years. And the patient community is kind of tapped out. And it has been stated over and over again, you know, in all the recent government sponsored reports, there’s an urgent need for research. And there’s an urgent need for treatments. 

And as Bob Miller commented, the bottom-up approach really doesn’t result in prompt and timely results. We need the NIH to become the champion, at least in my opinion. What can be done to truly respond to the call for urgency? 

Perhaps by thinking outside the box specifically for this disease so that we can have an impact on the current generation of patients. 

Dr. Walter Koroshetz:    Well, I think that’s a good point. And I must say that one of the hardest things about coming to NIH is just how many diseases we don’t have good treatments for and how slow the process seems to be. 

And so this is a generic issue that I think NIH is always trying to break through and move things faster. I think at this point we still believe here that trying to empower our consortium in the U.S. is going to be extremely important. 

And that that is, hopefully, you know, starting a little army that can then build over time, hopefully quickly over time, to move the science forward faster. 

I think in the past I think one, you know, analogy might be that we have soldiers in many different areas kind of fighting the battle on their own. And this is now an attempt to kind of bring the groups together and form a coordinated attack on the problem, which I think is if you’re going to move things forward, I think in almost every disease that’s what you need to move it forward. 

Until you have that group that has an identity, a social structure, that has connections with the patient and the advocacy groups, you’ll always take in little shots. But once you have a consortium that’s really successful both, in terms of the research side and the alliance with the patient groups, then things can move very, very quickly. 

So I think that’s what we’ve seen in the past. And that’s what we’re hoping with every disease. And that’s what we’re hoping will happen for ME/CFS. Can we go to the next question? 

Marian Emr:    Lauren I think we have time for one final question. I know that there are others of you waiting to ask questions of us. And please, send us your unanswered questions at We’ll do our best to respond to you in a timely manner. Now please, one last question. 

Coordinator:    Last question comes from Lily Chu. Your line is open. 

Lily Chu:    Good afternoon everyone. This is Lily. I had two questions, but I’ll just ask you one. In the letter that Dr. Francis Collins wrote in response to the House of Representatives asking about ME/CFS efforts at NIH, it says here that the ME/CFS interest group is going to be open to all intramural and extramural investigators interested in learning about ME/CFS. 

So my question is, are there going to be some ways that people can watch some of these seminars online, or possibly even, you know, participate in them, live, in order to reach out to extramural investigators? And this is important because they cannot only inform NIH about their efforts and about new ideas, it’s also important for new and younger investigators who can learn from NIH and also from more experienced investigators. Thank you. 

Dr. Avi Nath:    So these seminars are not set up that way, but I do think there’s merit in that kind of interaction. II think those things are probably better done in a setting of a conference or symposium or something like that. We’ll have a better, bigger room and more time. 

And so the goal of this really is to have a one-on-one interaction with that individual and learn from them and share what we are doing. Start expanding the role of the small group and it gets out of hand. 

So I think better to do it in a more symposium type setting I think. That would be more useful and more productive. But I value your opinion and I think that’s useful. 

Dr. Walter Koroshetz:    I would say that I think that’s a very good idea in terms of trying to do outreach and particularly to young investigators. And so for some of the consortia that we have, that’s actually done by the consortia folks. 

And so I think that’s something that’s a very good idea, and maybe we’ll try to encourage our consortia folks to do that. And to say that also the intramural program will, you know, hopefully be coordinated with the consortia and be, you know, another site in the group. But very good idea. 

So I’m sorry, we still have lots of questions out there. So as Marian mentioned, you can email them back. But we’re going to plan on trying to do these telebriefings on a regular basis. Marian has put a hold on a February 1 date and then a May 16 date in the afternoon for the next teleconferences. 

And I really appreciate the thoughtfulness of the folks on the call. And I just want to emphasize that the group here at NIH has been thinking hard and is really looking forward to trying to get these new research initiatives out. 

And again, we apologize that things just seem to go so slowly. But there is, I think a method to the process which has kind of been at NIH and refined over time. And the product is usually, benefits greatly by the process. 

So I apologize for how long it takes, but I’m very looking forward to seeing high quality research coming in. And so thanks very much to Joe and Vicky and Avi, appreciate you coming and Marian for setting this up. So talk to you again next time. And send in your questions. 

Marian Emr:    Please remember to send your questions to And for those of you who aren’t near a computer or a pencil and paper, you can go to the main NIH ME/CFS website and simply click on the Contact Us button at the bottom to submit those questions. 

You can also use that button to add your name to our list serv for future news and information. If you didn’t receive an email from us directly about today’s conference, then you might want to do that. 

And you can also watch the website for news and information in general. Thank you for participating this afternoon for a very productive discussion, and remember to watch the NIH website for the transcript of today’s call, which will appear sometime early next week. Thank you very much. 

Dr. Walter Koroshetz:    Bye now. 

Coordinator:    This concludes today’s call. You may disconnect at this time. 


This page last reviewed on November 10, 2016