February 1, 2017

NIH ME/CFS Advocacy Call - February 2017



Moderator: Marian Emr
02-01-17/2:00 pm CT
Confirmation # 2531113

Coordinator: Welcome and thank you for standing by. All participants will be in listen-only mode until the question and answer session. At that time you may press Star 1 to ask a question. Today’s conference is being recorded. If you have any objections, you may disconnect at this time. I would now like to turn today’s meeting over to Marian Emr. You may begin.

Marian Emr: Good afternoon. My name is Marian Emr and I’m the Director of Communications at the NIH National Institute of Neurological Disorders and Stroke. On behalf of the NIH, I would like to welcome you to this afternoon’s teleconference and thank you for your interest in participating in this discussion with us today. Here at the table this afternoon we have Dr. Walter Koroshetz, Dr. Avindra Nath and Dr. Joseph Breen. Dr. Vicky Whittemore is on the phone joining us from Chicago where she’s attending a meeting with the Epilepsy Foundation. Dr. Koroshetz will introduce the speakers, each of whom will make some brief remarks. After which we’ll open the phone call to your questions. We’ll try to make our remarks brief so that we can answer as many questions as possible in the time we have available to us this afternoon. Now, Dr. Koroshetz.

Dr. Walter Koroshetz: Good afternoon everyone. Thanks for getting on the line. So we are hopeful that this is a useful exercise. We’d like to let the community know about the research opportunities as they roll out from NIH. And we have an exciting one to talk about today, which is a Data Management and Coordinating Center along with multiple, hopefully, Collaborative Research Centers that we’re hoping to set up in the U.S. And as people know this was in the works for quite a while and was just recently released for investigators to apply to. But it was announced months ago, so people knew it was coming. And hopefully people have been preparing and that we’re very much looking forward to this new research opportunity to hopefully make a big difference in ME/CFS research. And Dr. Joe Breen from NIAID will be talking about that.

As you know, almost a year ago now Dr. Collins challenged the Intramural NIH, which is the laboratories and clinics here in the Bethesda campus to focus on ME/CFS research. And Dr. Avi Nath from NINDS, along with many other investigators from multiple other institutes, have put together a program. And he’ll talk to you a little bit about where that is now. And then we’re very much interested to hear from the community and get your feedback. And so we’re going to try and make our remarks a bit shorter this time to give us more of an opportunity for the open discussion and questions. And so with that, I’m going to ask Avi if he could tell us a little bit about where the Intramural ME/CFS Research Program is. Avi?

Dr. Avi Nath: It’s a real pleasure to have this opportunity to talk to you. And we are making excellent progress in the Intramural Program. Our team is now all in place. So we have a nurse, a nurse practitioner, a patient coordinator and undergraduates who are called IRTAs [Intramural Research Training Award]. And so they’re all trained. Everybody is in place. We have protocols, all the amendments that we made have all been approved and we’ve started recruiting individuals to the study. So far we’ve had six healthy volunteers come through. Two of which, interestingly, turned out to have some abnormalities on their MRI scan. So we have four that I think meet all our criteria.

So we’ve put them through all the extensive testing that we were planning to do. And I’m glad to see that everything actually went very smoothly. Usually you expect an extensive study like this to have some things fall through the cracks, but I think everything went on really well. So we’re very encouraged. We have now screened a large number of patients as well, for potential candidates for the study. And so there are five that I think meet our criteria. And the first of them is going to be here in early March. And then we’ll be taking it from there. So that’s where we stand at the moment.

Dr. Walter Koroshetz: Great, thanks Avi. And as people may know that there’s been interest in bringing in experts in ME/CFS to talk to the Intramural Program. And I understand that there’s plans for, to continue that. Is that…

Dr. Avi Nath: Yes. So we have a line of speakers. So we have Ian Lipkin who will be coming from Columbia University and talking about the immune profiling and all the viral studies that he is planning on doing, including a microbiome study that he is involved in. So we’re looking forward to that. We have Paul Levine coming to talk about cancer epidemiology and then Nancy Klimas who is going to be talking again about immunology. And then Xinnan Wang who will be talking about mitochondrial dysfunction. So we have the next, these will be our next four speakers that we lined up so far.

Dr. Walter Koroshetz: Great. Well thanks a lot. And now I’d like Dr. Breen from NIAID to talk to us a little bit about the new request for applications for our center program.

Dr. Joseph Breen: Great thank you. My name is Joe Breen. And as Dr. Koroshetz mentioned, I’m from the National Institute of Allergy and Infectious Diseases. And I’m a member of the Trans-NIH Working Group that developed these two requests for funding announcements. And I’m part of this group because I’m in charge of a number of grants in this area that are assigned to NIAID and in our mission area. And it’s because of the post-infective connection there, it’s very important and actually we have a long standing commitment in this area.

We’re very excited about the opportunities that should really come out of an initiative such as this. With significant input from the community, with RFIs, national meetings and in fact, quite a number of in-person meetings, the committee developed the RFAs. They were actually released last Friday. At the core, the research centers, the RFAs are designed to establish a network of collaborative research centers to better understand the cause and discover improved treatments for chronic fatigue.

Importantly, we see this as a starting point for increasing the research infrastructure necessary to address this complex challenge and really to build the field up. We do have investigators as I mentioned that have been in the field that we’ve been supporting all across NIH including NIAID for some time. But we think we need more and even better efforts. The idea of these centers is really to develop collaborative basic and/or clinical research centers that include longitudinal studies really within each center and across centers to build a true network that we think will synergize and help build, move the field forward.

And importantly these were also seen and hope to be centers of information that will be useful for clinical researchers, basic researchers, academic and practicing physicians, healthcare professionals, patients and the lay public. We’re hoping that the collaborative and synergistic nature of these centers will really increase the overall efficiency of the research side but also provide real leadership across the country and expertise in this area as we try and really better understand and then eventually develop better therapeutic options.

While this is an exciting opportunity I don’t want the point to go by that we want people to stop applying with our regular grant mechanisms. We have three opportunities every year for people to apply for research questions in any of the areas that are of interest to different institutes in the NIH. And that’s a really important stream of information and hypothesis testing and generating. And we don’t want that to stop. And it’s really independent of this new opportunity with the RFAs.

We have, as I mentioned, we have long standing mechanisms to support that kind of work. And the last point about the RFAs is that there is a webinar that’s planned for prospective applicants on February 13 at 3 o’clock Eastern. And more details about how to sign up for that if you’re a prospective applicant, which was the intended audience for that webinar, will be available on the Trans-NIH website.

Another quick opportunity I wanted to talk to you about was, we are trying to build up the research field. One way to do that is to talk about ME/CFS at meetings where it might not be discussed previously. This summer we’re actually hosting, NIAID is helping to host an immunology forum called Insights into the Immunology of ME/CFS where we have invited four nationally recognized speakers. And I think it’s actually on June 14 at the Foundation for Clinical Immunology Society meeting in Chicago. We have a half-day session dedicated to really talking about what’s known about the current immunology regarding this disease. Then hopefully we can translate that to developing collaborations with a host full of clinical immunologists who will be in the audience. That’s an example of where we’re trying to build the momentum that this RFA will generate in the community to try and really address this problem from as many perspectives as we can. With that I wanted to turn it over to Vicky who I think is going to talk about the Common Data Elements project.

Dr. Vicky Whittemore: Yes. Good afternoon everyone. This is Vicky Whittemore. And I’m at NINDS. And I just wanted to give you a quick update on where we are at with the Common Data Elements program. We have 55 individuals, clinicians, researchers, and patients and advocates who are participating in this project. And we’ve had one training call and we’ll have a second in another couple of weeks. And from there we’re going to begin to have the subgroup or the different working groups who will begin to really dig into the work of developing common data elements for all the different domains within chronic fatigue syndrome within ME/CFS. So, post- exertional malaise, sleep dysfunction, et cetera, et cetera.

So we’re very excited. And the people who have volunteered their time to help us with this, it’s greatly appreciated. The founder and I are working actively to put the working groups in place and to really kick off this project. So I encourage everyone to stay tuned and we will certainly, the process is that once the Common Data Elements are developed they will then be released to the public for a public period, for a time of public comment. So we will keep you updated as the process moves forward. So I’ll turn it back to you Walter.

Dr. Walter Koroshetz: Let me just step back a second for folks on the phone to make a couple of points. Common Data Elements I guess is the first thing to define. I guess the easiest analogy is a common language. So what happens in research oftentimes is that if an investigator is studying the same problem but using different terminology and it’s very hard to compare, that really lessens the value of the research. So what we’ve done across NIH now is to try to encourage the investigators to come together and agree upon a common language, which allows the studies to be compared to each other. And then also in a similar vein I’d say that the center program of the consortium that we’re hoping to put together also stresses coordination among investigators.

It allows for a lot of flexibility of thought and ideas. But it has a data coordination center where all the data will come together. And the idea there is that the data will be valuable for everyone in the consortium and that the data will be comparable. In addition somewhat an innovative stream here the centers program is going to challenge the investigators who are finally chosen to actually come together and design a project that they will do together. So again, another sign that this type of coordinated attack on ME/CFS is really in demand at this point in time and the new programs will support that. So I think we’d be ready now to answer questions. I’m sure people have questions about the items that were mentioned here today. So Vicky, and Avi, and Joe and I are happy to entertain your questions or comments.

Coordinator: Thank you. And to ask a question please press Star 1. Please unmute your phone and clearly record your name at the prompt. To withdraw your request please press Star 2, one moment please for the first question. The first question is from Courtney Miller. Your line is open.

Courtney Miller: Hi. Good morning or good afternoon Dr. Koroshetz, Dr. Breen, Dr. Nath, Dr. Whittemore. I wanted to first express our continued support and excitement at new initiatives at NIH on our disease. You know, the NIH clinical trial and now RFA, is the first RFA in what’s at least a dozen years, are a strong initiative. Even as we need, you know, much more funding and much more research to make the most out of those initiatives and to move our disease understanding forward.

My question really to you is related to the third leg of a stool, which is the complete absence of treatments and clinical trials in our disease. And one that I believe and we’ve had discussions about and I want to ask you how we can move this timeline forward. But the NIH and FDA need to do something dramatic to get us out of our catch-22 of no trials, no pharmaceutical investment and no FDA-approved drugs. And I say this as, you know, the one small clinical trial Ampligen constricts even further and puts some small number of patients at, in a position of not even getting that. And so as NIH funds 20 clinical trials in other diseases, you know, I really want to ask you how we can take, you know, the progress of these first two initiatives and add support for clinical trials in the really near term not, you know, three to five years from now. And again thank you for the work you all are doing.

Dr. Walter Koroshetz: Well, certainly we feel the urgency from the people who are suffering now. And this is a constant tension at NIH. And so I think you have, we have to think about short-term and the long-term and be quite realistic about the chances of success going into clinical trials. And there is I would say, you know, from experience, failed clinical trials are quite a disappointment when they don’t show what people hope for. Multiple failed clinical trials actually can destroy an entire field. So I can tell you in the stroke field right now after many failed clinical trials in the 90s, industry is not at all interested in stroke as a field.

So one has to, you know, keep your eye on the goal and prepare as best you can to try and understand biology that you’re trying to influence in any clinical trial. You want to gather the information that’s necessary to create a steppingstone where you can get across the river of doubt and the river of a lack of knowledge, without falling into that river and get to the other side with an effective treatment. So you have to take every instance of opportunity and explore it. And you have to develop ways in which you can test interventions for their biological effects and move from, you know, Phase 2 trials, which look like they’re actually hitting the target towards a Phase 3 trial that’s going to be successful.

And so that process is unfortunately a long process. And it’s something that we I think for the diseases that we care for most of the diseases have no treatment and they’re oftentimes fatal diseases and there have been many failed trials. So I think a kind of long-term approach is clearly the one that’s going to have the highest probability of success in the end but it takes a long time. There may be instances where things pop up that look like, you know, it’s definitely worth the opportunity to try something even with, you know, limited knowledge of the effects of the intervention. Those are tricky to do but we definitely do those because we have this short-term commitment to try and help people who are suffering now, not just the people who are suffering in the future.

And those clinical trial applications can come in. They always come in to our regular announcements. And those are things that we are always open to bringing in, putting before peer review. We think that the center program that we are putting out now will help in the long run because we think that it will if things go well develop the army of investigators that are going to be needed to launch a successful clinical trial. So we are hoping that this new center program will bring in new people into the ME/CFS field, that it will bring in seasoned clinical trialists into the ME/CFS field. We have in the language collaborative, collaborations with the Clinical and Translational Science Awards, which are the large infrastructure across the country to do clinical research and clinical trials. And although the program itself is not designed to do clinical trials, it is and it’s stated explicitly, that it is to do work that’s preparatory to clinical trials.

So that pathway is open. And we just need to grease that path with the knowledge that’s necessary for a successful trial. So a long-winded explanation but it kind of reaches into kind of our sense of how we need to operate to eventually be successful in ME/CFS.

Marian Emr: Melinda, we’ll take the next question.

Coordinator: From Leonard Jason, your line is open.

Leonard Jason: Thank you. Once again as Courtney said, appreciate very much this opportunity to hear about these exciting center grants. I have a couple quick technical questions. The $1.2 million per year, is that direct and indirect costs? A second question is the $200,000 set aside each year for five years, which is approximately I guess a million dollars is going to be funding the collaborative study. Can you give us an idea of how we can write that section without knowing who the collaborators might be and what types of advice you might give us? And a third quick question is, two case definitions are proposed, one the Canadian criteria, the other the IOM. Is one basically recommended as a clinical case definition and another as a research case definition? Thank you.

Dr. Joseph Breen: So I’ll take the first question. Its $1.2 million direct costs.

Dr. Walter Koroshetz: So for people on the line, the total cost of a project is a combination of the direct cost which goes to actually fund the science. And then universities or contracting organizations have a fixed called indirect cost where they have a percentage, which is additional to the direct cost, goes to the institutions to provide for things like the building, the IRBs, and equipment and things like that. So right, the numbers that are in our budgets are the direct costs and in general about 50% of the direct cost is going to be added on to give the total cost.

I guess with regard to the project, the common project, we’re interested in what people’s ideas are, but that is something that we are going to convene the different collaborators once they are identified and have them then put together that project. So it’s not something that’s going to be required for the grant itself. And it sounds like you may be interested in applying so that webinar February 13 is probably the place to go for that. And you’re right about the two hundred in each of the centers for the common projects. So if we get three centers it would be about I think $600,000 for that project. Then again that’s the direct cost. I think those were the main questions.

Dr. Joseph Breen: What was the third question, the diagnostic criteria?

Dr. Walter Koroshetz: Oh.

Dr. Joseph Breen: Vicky, are you on? Can you address that?

Dr. Vicky Whittemore: Yes. I think what we’re looking for is that those are meant to be the case definitions by which individuals are identified for studies. So we will work once the centers are funded, we will work with them to make sure that the case definition across the centers is consistent and that we’re using similar criteria for recruitment of individuals for any studies are being done within and across funders.

Dr. Walter Koroshetz: Good point.

Dr. Vicky Whittemore: Good.

Dr. Walter Koroshetz: Okay, next question.

Coordinator: Cynthia Johnson, your line is open.

Cynthia Johnson: Thank you. I’m in the Portland, Oregon area. And thank you so much for your time. I have several questions. And I should let you know that I just lost my house and everything. I’m in a motel and homeless as a result of CFS discrimination and bad hospital experiences. So one of the things I’m wondering is, I live in a major metropolitan area and none of the hospitals, I don’t know if you have any way of getting your research information out to or if you’re trying to the CEOs and the medical directors of the major hospitals because I’ve had a very bad experience, even in the neurological area where they don’t understand the difference between CFS and a neurological problem.

I’m fortunate enough to be in the Stanford Program but I haven’t been able to get down there. So technically I wonder if any of your centers for research will be up in the Northwest, where it’s very much needed. And also I’m a cancer survivor. So I’m very interested in you working, I noticed you mentioned immunologists. Are you doing any more work further I know in studies originally there was some connection between cancer and CFS are working with oncologists as well as with immunologists. Was that too much? So can you understand me?

Dr. Joseph Breen: No. I can answer. Well first let me say I’m sorry to hear about your personal circumstances.

Cynthia Johnson: It happens to a lot of people. And I want to thank you for saying that. But you should know that they give you psychogenics is what they do. And a lot of CFS patients, I worked in healthcare committees for the governor here, but they do they say it psychogenic, where it’s neurological. And they can’t distinguish between their CFS movements or lack of thereof at times and the other. So it’s pretty horrifying. So thank you, go ahead.

Dr. Joseph Breen: So we don’t know yet what, where the centers will be located geography-wise.

Cynthia Johnson: Okay.

Dr. Joseph Breen: So I think but I, your question lends me to write down some notes to talk about to remind me about how we want to disseminate information once we do know who those are. The second part is, there are a large number of active research grants trying to look at that connection between immunology and cancer certainly.

And there are a few grants, but not that many granted, looking at potential connections between CFS and cancer. So there are those projects that are ongoing. And I think that’s still, the connection is not well understood which I think maybe Dr. Nath could speak to. But there is a research portfolio in that area at NIH. And those grants are publicly searchable to see what those projects are, through a vehicle called the NIH Reporter if you wanted to look there.

Cynthia Johnson: Okay. Thank you. And yes I actually gave a CEO of Legacy Hospital years ago when I served on that committee, a subcommittee of his. The primer for practitioners on CFS. She was interested but somehow it never trickled down. So, you know, that’s I think it really big thing they need to get you, you know, your information out so they understand the legitimacy of the disease. The physicians know nothing about it. Yes is there anything I can do to help with that? Even though I’m not in a great position but yes is there anything I can help to do with that? Patient or locations like the Northwest, it’s really kind of a desert here right now.

Dr. Walter Koroshetz: So I think, you know, the issue of the kind of big gaps across the country for care is clearly a problem that a lot of the patients have faced.

Cynthia Johnson: Yes.

Dr. Walter Koroshetz: The NIH is a research organization. And so we don’t affect that directly. But indirectly, for instance we would expect that those centers programs will all have if they don’t have them already will put in programs where they’re seeing patients at not, a proportion of which may be enrolled in research protocols but usually the larger number are patients being cared for and serve as the catchment group for the research. So there is a somewhat sideline expectation that this NIH program will raise the view of ME/CFS among some of the major hospitals and universities and provide the evidence that will, you know, then get back to the doctors and lead to a change in practice.

Cynthia Johnson: Great. Yes, thank you. There’s a lot of great research going on here so thank you.

Dr. Walter Koroshetz: In terms of what people can do I think it’s important to know is that the centers themselves are mandated to have collaborations with patients and patient advocacy groups. So this is truly a joint project between the investigators, the NIH and the patients. So when these begin, the investigators will certainly be looking for collaborators in the patient community to help them not only with their application but also with their research.

Cynthia Johnson: Okay. Okay great.

Dr. Walter Koroshetz: Can we move to the next question…

Cynthia Johnson: Okay great. Well maybe they can take my name somewhere else off line. But I’d be happy to send all my services in the paper so it mentioned CFS a lot. And so if you…

Dr. Walter Koroshetz: Okay. If you can send me an email I don’t want to, yes I think you should stay anonymous, but if you could send me an email that would be great.

Cynthia Johnson: Okay. What is your email address? Who is this? I can’t tell who I’m speaking to right now.

Dr. Walter Koroshetz: Dr. Koroshetz. I’m easy to find, just NINDS Director.

Cynthia Johnson: Oh okay.

Dr. Walter Koroshetz: All right thanks, next question.

Coordinator: Matina Nicholson, your line is open.

Matina Nicholson: Hello. Can you hear me?

Dr. Walter Koroshetz: It’s very faint.

Matina Nicholson: Okay let me see, I’m in New York. How about now as I’m going outside? Can you hear me now?

Dr. Walter Koroshetz: Better. If you really shout I think we can hear you.

Matina Nicholson: Okay. Once again, thank you. But my question is I really, you know, using the two centers for them as investigators to control the research. I think you should use another approach that was used by the Children’s Oncology Group for research. They allow all experts to be involved in terms of designing and using the case definitions. And I think if you can have that design and then have them work with the two centers of excellence because I think we need to involve all our top experts in the process of what we’re going to realize as the methods of the case definitions, et cetera.

Dr. Walter Koroshetz: So to reach out to more of the experts is the point to that right?

Matina Nicholson: Yes have a group. I don’t know if you ever looked at by NIH, but on the pediatric side the Children’s Oncology Group is all the leaders, all the experts in that consensus. And they kind of made all the determinations and answers of how to be able to move forward. So I think if we try to do something similar because if you’re using another two I don’t think it would be advantageous because we are so behind. And we really need this research because we have a lot of people with severe ME that are almost, just waiting to die. I mean we’re not dying fast enough but we’re dying from a long term process. So I think you need to get people in the early stage. Like me, I live, this is the first time I’ve been able to see my doctor in six months. And I’m ready to (unintelligible). So basically, I know its hard work and I’m happy for it. And I’m just adding some positive feedback that help us kind of set up for the next phase.

Dr. Walter Koroshetz: Okay well thank you very much.

Matina Nicholson: Thank you.

Dr. Walter Koroshetz: Yes. Can we go on to the next question? That was a good comment to try and clearly we don’t want to have a closed shop. We want to always be open to folks who want to work in ME/CFS. And the centers oftentimes can expand and branch out and pull people in. And so it’s broader than a few people for sure. Okay next question.

Coordinator: Johanna Kaiser, your line is open.

Johanna Kaiser: Yes thank you. First I want to express my deep gratitude for all that each of you are doing and I mean this. I am alarmed by the lack of urgency and the low level of funding that’s been put forth. I know you’ve worked hard on this. But I have been a patient for almost 40 years. And that’s 40 years of my life that have been taken from me by not the disease but the inaction of NIH. And I don’t think that I and people like me should be further punished by plodding along at a snail’s pace.

I personally know two ME patients, one who is in and out of the hospital constantly, and one who is currently fighting for his life and losing. His mother is a pediatrician. And because the Kaiser Hospital doesn’t have any ME expertise, I hope you all realize that there are no more than a dozen clinicians for 2.5 million ME patients in all of the United States. His mother has been staying with him at the hospital day and night while he’s dying.

So I understand that you have a responsibility to proceed with care but the funding level isn’t enough. You have to really change this. It’s barbarous. This is not a humane way to treat people like us. And I urge you to reconsider. We have been told that you need more investigators. We’ve been told by investigators how their grant applications have been turned down. These are not third rate people, these are world-renowned researchers. So patients are caught in between what NIH says and what these world-renowned Nobel Laureates, members of the Academies of Sciences are telling us. You’ve really got to quit this and get moving because this is murder by neglect. I don’t mean to insult you but you’ve stolen 40 years of my life from me. And thank you for letting me have this opportunity to speak.

Dr. Walter Koroshetz: Yes. We all hear the pain there. And we feel terrible that we don’t have…

Johanna Kaiser: Well do something. Please change it for God sakes.

Dr. Walter Koroshetz: Yes.

Johanna Kaiser: I can’t go on like this and neither can most of us. You give us words but not action. And the increase in funding is just a kick in the face. You really, you people are not doing enough. And, you know, my family, most of my family died in the Holocaust. They wanted to come to America. If they saw what happened to me they wouldn’t believe it. This is not what America is about. So please enough with the words. We’ve heard too much. It’s all hollow. You’ve got to up the funding and get moving and save our lives. That’s it.

Dr. Walter Koroshetz: Yes, we totally agree, absolutely.

Johanna Kaiser: So will you move with urgency then? I mean, thank you for really from my heart, thank you for saying that. Can we expect then a change sooner rather than later because we’re dying. So I really do appreciate what you’re saying. Will that translate into urgent action in the near term? I’m desperate.

Dr. Walter Koroshetz: I’m hoping yes. That’s our plan. And that’s what the center program is trying to do.

Johanna Kaiser: But that’s five years. I’m not going to make it five years. I’ve had this for 40 years. I can’t go another five years at this pace. This is my point.

Dr. Walter Koroshetz: No, we need more answers, absolutely.

Johanna Kaiser: But you’ve got to fund the, you know, the people like Ron Davis, the Lipkins, the Solve ME/CFS Initiative that was just, their grant application was rejected. You know, NIH says one thing and does another. And we’re and, you know, we want you to know that we’re seeing it. This is, you know, words aren’t enough. The actions have to change. The money has to change. You know, people are committing suicide. And you guys are going at a snail’s pace. I don’t know how else to make it clear. But you’re…

Dr. Walter Koroshetz: Well, we’ll be looking at every opportunity.

Johanna Kaiser: …but NIH is being stubborn and letting our lives burn to the ground.

Dr. Walter Koroshetz: That’s certainly not our intent here. We really would like…

Johanna Kaiser: But that’s the result. And you must acknowledge reality.

Dr. Walter Koroshetz: We’re very knowledgeable about reality. The question is, where are the answers? And that’s sometimes not so easy to get to.

Johanna Kaiser: But you’re not funding the people who want to research this. Good people...

Dr. Walter Koroshetz: Well let me explain, let me just explain so that at NIH most of the funding comes in because people write grants, not RFAs. So almost, most of our funding goes out not in RFAs, but in people sending in grants.

Johanna Kaiser: And they’re being denied.

Dr. Walter Koroshetz: Now let me just finish. So the competition at NIH, because of the funding situation, is such that only 12% of the grants get funded. So that means that 88% get rejected. And we get about ten ME/CFS grants a year. So that, even with those numbers, that’s one grant a year. What we need to do is to get 100 times more people interested in working on ME/CFS. And that’s what the program is that we’re putting out is trying to do.

Johanna Kaiser: But let me ask you something.

Dr. Walter Koroshetz: So I know, I wish I could move things faster. And I will do anything I can to do that if I can see an opportunity there.

Johanna Kaiser: But NIH did this for AIDS. And, you know, grants from people like Ron Davis. And, you know, people, I mean, Norway is making more progress than the United States is. You know, so grants by people, from people like Ron Davis are being turned down. And then we’re being told that low-quality grants are being submitted. We just don’t buy it.

Dr. Walter Koroshetz: Well, we don’t say low quality grants. Lots of high quality grants don’t get funded.

Johanna Kaiser: In the CFSAC meeting in 2015, that’s what we were told. I can’t remember her last name, Susan something. She actually said that. The audience was incredulous.

Dr. Walter Koroshetz: Well, we are trying to turn things around. And I hope for this program we’ll get some really, really strong research going. And that it just kind of exponentially expands.

Johanna Kaiser: How do we survive these next five years then, the patients who are so desperately ill?

Dr. Walter Koroshetz: I think, you know, just like in medicine we are stuck with always trying to do the best for patients with what’s known but knowing that we need to know more. And I think that’s the problem with ME/CFS, is how, and with many different diseases unfortunately, and it’s quite sad.

Johanna Kaiser: But our disease has a larger patient population than almost any other. So there really isn’t a comparison. And what was done for AIDS is not being done for us. There was an urgency there, eventually. It didn’t take 30 years – 30 plus years like it is for us.

Dr. Walter Koroshetz: Right.

Johanna Kaiser: There are things that should happen that aren’t. Thank you.

Dr. Walter Koroshetz: I agree with you. Okay, thank you very much. Can we go on to the next question?

Coordinator: Nancy Klimas, your line is open.

Nancy Klimas: Thank you. Boy that was hard to hear because that would just, I understand that urgency. And I understand…

Dr. Walter Koroshetz: Nancy, it’s hard to hear. Could you talk a little bit louder?

Nancy Klimas: It was hard to hear the last speaker because I understand her worries. And I wanted to say something, not my question, that the two consortia that are out there right now funded for Gulf War illness has financed more than twenty additional applications for funding in their first two years. So this consortia idea is a great idea to put some momentum behind the R01 and R21 and R15 programs that are already available for funding. But that’s not what I wanted to say when I pushed the Star 1 button. But I wanted to reassure the advocates on the line that this is a really cool mechanism for bringing more people and momentum to the field.

Anyway, what I was going to ask was more to the point of two things in the application and maybe it’s more appropriate for the webinar. But one was that you’re funding two to three projects per year. Why that cap if you think you could do additional projects under the budget cap? And the other is in the instructions there was quite a bit actually towards the end of classical areas of interest, things about like probiotic use and so on. And I don’t see how some of those objectives can be met without clinical trial. They seem like they’re not, you know, animal or laboratory models but very precise objectives that require clinical information or clinical intervention.

Dr. Walter Koroshetz: Right. Well Nancy, important to know is that clinical research is definitely in. So that, you know, and the hope would be that there might be some clinical research that would be a stepping stone to an actual effectiveness clinical trial. But the clinical research is clearly, I think, going to be a major component of the centers.

Nancy Klimas: You know, and I love that idea of across consortia and mutual projects, giving the possibility of developing a data and a biorepository could be extraordinary.

Dr. Walter Koroshetz: And if the group can form across multiple centers and that’s usually what’s needed to do clinical trials so that, so we’re hoping that this is kind of a stepping stone to really, you know, the next level for patients. With regard to the number of projects, we don’t usually limit the number. But, you know, if the project, the rule is if the project, if there’s so many projects, it may look like it’s not feasible to do them all but we’ve just made an estimate terms of, you know, what the cost would be for a project and then that’s how we determine the numbers. And also it’s, you know, it’s difficult to do many things well and but that was more of a guide then a, you know, imprimatur.

Nancy Klimas: Well I appreciate that. That’s very helpful. But in terms of clinical trials and again and a little word of reassurance to the advocacy community. You don’t have to wait five years for work that’s going on now to spin off into trial. But I think what you’re really asking is, you know, is there a mechanism within the NIH that could fund Phase 1, Phase 2, Phase 3? And, I like, many others have voiced the concern that we really don’t have a mechanism.

And we, I mean there are mechanisms within the NIH to go and have clinical trials in any subject under the sun. I mean this is a broad clinical trials mechanism. But there’s not one that specifically is focused on chronic fatigue. And there’s a worry that those protocols when submitted through those existing mechanisms would not be reviewed by the Special Emphasis Panel. Is there any way to look at that to know whether or not the SEP would be the reviewing body for clinical trials that might be spun off earlier than this five year timeline that is being put forward?

Dr. Walter Koroshetz: Yes, I would say that certainly NIAID and NINDS have mechanisms that we would bring in clinical trials there. You know, they’re throughout NIH. I’m sure Joe can mention, but at NINDS those are heavily managed because of patient protections, the cost of the trials and so they’re, they are usually what’s called cooperative agreements. And those mechanisms exist. In terms of the review, who reviews them, you know, we haven’t really thought that through. But that’s an interesting suggestion. And certainly you want, the key thing on the review panel is you want folks with the expertise in clinical trial design to be sure that the product, and the effort and time that the patients put in and actually the risk that the patients take is going to have, you know, value in the end. And similarly for the investment on the part of the taxpayers, but we could certainly look into where things get reviewed for sure.

Dr. Joseph Breen: Nancy, so at NIAID outside of a standing network of an investigator initiated clinical trial would be reviewed in a panel that would be convened for that purpose. There’s probably one or two at one time, that would be it.

Nancy Klimas: Okay great. So there is a current mechanism. And there is no need to wait on an idea that has sufficient pilot data to go forward.

Dr. Joseph Breen: Right, right.

Nancy Klimas: That’s good to know.

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

Coordinator: Carol Head, your line is open.

Carol Head: Thank you. Thank you to all of you. This is Carol Head from the Solve ME/CFS Initiative. Joe, a question for you, I want to make sure I understand the comment you made about there being clinical care involved at these collaborative research centers. As we know we have a crisis at clinical care, so many patients who can’t get diagnosed and/or get any care at all. Might this open up more opportunities for patients to get some clinical care through your collaborative research centers?

Dr. Joseph Breen: Well, I think what I meant to say is that this would be, the site would be a place where clinical research would occur. NIH doesn’t pay for clinical care per se in this kind of setting. And since we’re not supporting clinical trials it’s under this particular FOA or RFA, that’s sort of what it is. What I meant to say is that we wanted to build a bridge between the basic researchers and clinical researchers. And then incorporate some of the structure that Dr. Koroshetz is talking about with the clinical and translational centers which are already ongoing and contain valuable expertise and tools that could be brought to bear. So I didn’t, I’m sorry if I misled you that there would be clinical care associated with that.

Dr. Walter Koroshetz: I think Carol, in many other diseases as I mentioned, the research participants are usually coming from a pool of patients that are seen in the clinic and receiving their clinical care there. So sometimes it’s, although it’s not the primary goal, it turns out to be a consequence of setting up these type of research centers around the country. And maybe you could comment because I understand that you’ve joined the UCLA CTSA which is, you know, a major force in trying to move clinical research in the L.A. area. And we’re hoping and we actually, you know, we should’ve mentioned this right from the beginning that the consortia is being funded by nine different institutes at NIH which is really quite impressive. And that includes the National Center for Advancing Translational Sciences, which funds those CTSA programs. But hopefully you’ll be able to convince the UCLA folks to make a strong effort in this project. But do you have any comments on your experience with the CTSAs and how they might help in ME/CFS?

Carol Head: You know, I’m new enough to it Dr. Koroshetz that I don’t think I could make any definitive statements. But, you know, as you say, you know, I joined it because I do want to increase their interest in or even awareness of ME/CFS. So I hope to be able to answer that question in the future but, you know, it is a good effort. It’s certainly a wonderful effort occurring here in California. And, you know, we’re doing as you’d expect what we can to push forward.

Dr. Joseph Breen: So Carol did I answer your question? It’s an important one. I want to make sure that….

Carol Head: Yes. Joe, you did. I of course, you know, I was optimistically hoping for slightly a different answer. But yes, you answered my question. And it sounds as though there may be some instances in which some folks enter into the research and get some care they might not get otherwise. But I understand that’s not the primary role of your research.

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

Marian Emr: So we’re three minutes to the hour. It looks like we have time for one more brief question.

Coordinator: Ronald Davis, your line is open.

Ronald Davis: Well thank you very much. We’ve been putting together a consortium just to work together for some time now. And the problem is from this point of view of this RFA is that we have about 15 scientists in that consortium at six different institutions. And they could easily become part of this application. And then how do we structure it, because you’re certainly not going to take that amount of money and put two projects and divide it all up amongst those 15 different researchers.

So is it okay to have people involved that don’t actually get any funding? Because if you divide it up by 15 you don’t have much money per person. This is certainly how the group grant worked at GM and Ron Tompkins ran one on trauma. And in fact Ron Tompkins is going to be part of, he is a part of this consortium because he knows how to run one well. And we had a lot of people there that were just, we called them advisors. They were critical but they actually didn’t do any experiments.

Analysis of the data and what to do about it was the most difficult thing. And that required a lot of people. They don’t really need much in the way of funding except maybe travel funding to come together for a meeting. Is that possible, would that would fit here?

Dr. Joseph Breen: I mean this is my opinion Dr. Davis. I think that’s certainly possible. And that model has worked. I think you just need to be careful that, that the peer review group understands what the contribution of an unpaid contributor is so that they feel comfortable that they’re still contributing to the project.

Ronald Davis: Well that is my main concern, yes.

Dr. Joseph Breen: Yes. But sometimes they add tremendous expertise and that can be a real strength.

Ronald Davis: Yes.

Dr. Walter Koroshetz: And I think the other thing is that, you know, the hope is that a center can spin off many other grants besides just the center grant. And so to have that level of firepower would certainly…

Ronald Davis: Well that’s the idea.

Dr. Walter Koroshetz: …would be impressive yes.

Ronald Davis: That’s mostly how those get groups together that can work together. And most of these people are on the West Coast, but not all. The other thing I would recommend is you might consider how the Genome Project was launched. That got off to a very good start. Jim Watson was really largely responsible for how that got off to a really good start. And he actually went around and solicited people to apply, which is a little unheard of, but he didn’t care about rules. And he also urged the reviewers not to nitpick proposals, but focus on getting the job done, and getting people involved in the project that could get the job done and to take a bigger picture look at it, as opposed to the minutia where you often criticize a proposal because they’re using the wrong buffer concentration or something.

And I would urge you to do something in that regard too. This is a big effort. AIDS did something similar to try to get it off to a real start fast. And given the one person talking here, this disease deserves that kind of an effort. I’m a little worried about the amount of money being put up at the beginning. I’m pretty confident that I could use ten times that amount of money just in our own group of people that we put together that could very effectively do good research with that amount of money. Of course we’ll do the best we can with the amount that we get. Thank you.

Dr. Walter Koroshetz: Okay. Thank you very much.

Marian Emr: Thank you. In closing today’s call I want to remind everyone that a recording and a transcript will be posted on the NIH ME/CFS website sometime next week. I’d also like to let you know that we’ve created a new listserv for updates from NIH. If you received a message about today’s call directly from the NIH ME/CFS Working Group, then your email has already been added to the listserv. If you received notice from a friend or a colleague, you might want to go to the NIH ME/CFS website and click on the Join our listserv at the bottom of the left sidebar.

Also sometime tomorrow we’ll be sending out a call for patients to participate in Dr. Nath’s DIR Intramural Trial. Please watch for that. And our next telebriefing will be on Tuesday, May 16. Thank you all for a great discussion today, a very thoughtful and informative discussion and good afternoon.

Coordinator: Thank you. That does conclude today’s presentation. You may disconnect at this time.


This page last reviewed on February 13, 2017