Dr. Suzanne Vernon and I talk the Solve ME/CFS Biobank, future research, the IOM Report, and more...
FULL TEXT TRANSCRIPT:
Dr. Craig: Thanks for tuning in to another episode of Spoonie Radio. I’m your host Dr. Courtney Craig. My guest today is Dr. Suzanne Vernon. Suzanne enjoyed a long career at the CDC where she first became interested in ME/CFS research.
Then in 2007 she became the first appointed scientific director at what was formerly known as the CFIDS Association of America. Now through the newly named Solve ME/CFS Initiative, she is spearheading breakthrough research efforts in the search for biomarkers and effective treatment She is on the show today to talk about these efforts, and how patients can get involved in moving us closer to a cure for ME/CFS. Welcome to the show Suzanne!
Vernon: Thank you very much for having me Courtney.
Dr. Craig: Since your appointment in 2007, the ME/CFS community has seen a tremendous transformation in terms of both research and advocacy. One major part of that has been the ME/CFS Biobank, which will be the main focus of our interview today.
So if we can start and tell us how did the Biobank come into being?
Vernon: So when I joined the organization in 2007, the first task at hand was to issue the then CFIDS Association, now Solve ME/CFS Initiative’s first formal competitive funding opportunity. So we did that, we got that launched in 2008, that was actually a million-dollar funding initiative and we made 6 awards in 2008 to the tune of around $650,000 to those 6 investigators. And those are 18-month awards that we saw all the way through.
And part of the process was really to try to-- part of this funding opportunity was to really try to establish a formal research network – a way for the investigators to use the same types of measures, share data, collaborate and so on to really again leverage each individual investigator’s expertise and knowledge as much as possible.
So we did that with the 2008 investigators, established this research network and in the process, we learned a lot about research networks and about what was needed in order to really accelerate research a lot more effectively, especially for ME/CFS, was an infrastructure component. It was clear that the investigators needed access to patients and access to healthy controls and not only access to their clinical information but also access to biological samples so that they could conduct their research.
Vernon: So that was really kind of what instigated us to begin to figure out how to put the Biobank in place for the organization and that really came to fruition in the end of 2009 – actually right on the heels of the Science publication describing XMRV in CFS patients and it was remarkable what happened when that publication hit the newsstands. Literally the next day there were pharmaceutical companies knocking on the door, ringing the phone to try to get access to patient samples in order to be able to confirm the findings and to validate XMRV as a potential biomarker.
We were fortunate because we had planned to put this Biobank in place again for researchers and as a way for patients to be active participants in the research process – really truly patient centered research. And that October 2009 paper really give us the opportunity to hit the ground running with our Biobank and we formed a partnership with GlaxoSmithKline (GSK). It was one of the largest pharmaceutical companies. It was very interested in trying to validate XMRV findings because they had a number of anti-retrovirals in their treatment portfolio that could potentially have an effect against XMRV.
And when that partnership was formed, they developed a protocol on the kinds of ME/CFS patients that could be eligible to study as well as healthy control And we had the consent process in place to be able to get our community to participate in research and more importantly, to sign up for the Biobank so that we could collect the samples from patients all over the country as well as controls, get them in the Biobank and then get them down into GlaxoSmithKline’s lab so that they can do the testing. So that was really how it started. That’s a long way about saying how it got started but it was fascinating once we identified the need for the Biobank and this remarkable paper came out in science and really kind of gave us the ground – the opportunity to hit the ground running when it comes to a Biobank.
And since that time we’ve had a number of publications using our written published on the Biobank samples and data that’s in there so it’s really just been quite an important resource for the ME/CFS community as a whole and in particular I think for making important progress in ME/CFS research.
Dr. Craig: Absolutely. Especially in the past couple of years, it’s really exploded. One thing that I found interesting as I am reading some of the research that’s coming out of the Biobank, is that most of these investigators are new to the field. They come from other fields of study and I think this is wonderful that we are getting some young, fresh talent in here that can look at this illness with a new set of eyes.
So is it the access to the Biobank that’s bringing in the new talent or what is it?
Vernon: I think that’s part of it. I always had this idea that if you build it they will come. And to some extent that’s true, but in reality you really need to market such a resource and I think really tap into your network in order for a resource like the Solve CFS Biobank to be used well.
And so what I have done since starting the organization is really tap into my network of scientific colleagues and I am constantly scanning the literature for research approaches and innovations that I think will be impactful and important and relevant in ME/CFS research. And then reaching out to those particular individuals that are on the cutting edge of many of these areas of research and seeing if they are game for kind of dipping their toe into the ME/CFS research domain. And because we have this resource available, it makes it a lot easier. It effectively lowers the barrier for these investigators to get in; both financially and from a timeframe perspective. They don't have to go out and identify patients and controls and collect all the samples and all of that kind of stuff.
And I think one of the other important things is that they don’t have to know a lot about ME/CFS in order to do that. A lot of people are reluctant to get into an area where they don’t know a great deal. They haven’t built up their portfolio to really understand what ME/CFS is. I mean that sounds kind of weird because scientists are a curious lot in general and I think that they are great at exploring new domains. But to do something... to again dip your toe into an area that is so complex and while there is a lot of opportunity, there’s also a lot of uncertainty from – is this worth my time? Am I going to be able to get a paper published? Am I going to be able to find something if I don’t understand really what it is that I am doing? It’s a risk right? – For the investigator.
So that’s why it’s also important when you have a resource like a Biobank, and in our case the Solve ME/CFS Biobank, to also have that backed up with some credibility, some scientific and research prowess that I bring to the Biobank and then it makes it a lot less uncertain, a lot less scary for investigators to venture in.
Dr. Craig: Right. And it also helps kind of bridge the gap between things we discover in the lab and things we can implement clinically right? – Because normally that transition takes a long time, even years to implement the clinical research into practice. So the Biobank is really helping to move things forward at a quicker pace then?
Vernon: Yeah. One of the things that I hope it will do more of in the future is this idea of validating biomarkers. Now it’s great for biomarker discovery and I think so far that’s what we really used it for. We have a number of publications that are currently in press that are using some cutting edge technologies and looking at things that haven’t been looked at before, so really biomarker discovery.
But the Biobank is also really good for validation. So take for example the paper that was just published in Science Advances by Mady Hornig and her team from the CFI initiative. So that is a very interesting and important finding that now needs to be validated right?
Vernon: So let’s use resources like this Biobank and maybe some other Biobanks to get out there and get those types of findings validated.
Same thing with other potential markers like NK cells. In the IOM report, the IOM report cited NK cells as being one of the potentially most important immune biomarkers for ME/CFS and it cited some of the strengths and some of the limitations of NK cells as a biomarker including that it hasn’t been validated as a biomarker for ME/CFS.
Again, let’s use these resources to get these potentially promising biomarkers out there and validated so that they can move to the next step. Once something is validated then it goes into the development pipeline for potential diagnostics.
Dr. Craig: Now you bring up the IOM report and one thing that I am interested to know regarding that, is that a new diagnostic criteria has been suggested. So how will this affect the Biobank and how you select patients for these studies? Do you plan to use the new diagnostic criteria or a combination of others that have already been used in the past?
Vernon: So what we have used was based on – so let me back up a little bit. The samples that exist in our Biobank, our current inventory, are still from the study that we did with GlaxoSmithKline in 2010. So we collected 340 I think, around there, samples from people that were diagnosed using the 1994 Fukuda criteria and the Canadian criteria by five ME/CFS expert clinicians including Dr. Nancy Klimas, Dr. Lucinda Bateman, Dan Peterson, Chuck Lapp and that’s it. That’s four right? Yeah that’s four. It is four, not five.
And so the partnership with GlaxoSmithKline (GSK) included working with these four clinical investigators so that they could do the diagnosing to make sure that the Biobank included samples from very well-characterized, clinically well-characterized ME/CFS patients and then that each of their patients were asked if they had a friend or a neighbor that they would help bring in to serve as a healthy control. So that’s how that whole initial inventory for that study was formed and it was effective.
And I mean what is remarkable is that from the blood samples that were collected from these patients and controls, after the GSK case study ended, we were left with a huge inventory of samples that were the components of these blood samples including plasma, cells that were frozen down and cryo-preserved again creating a very rich inventory for what we could do further research on. And they were attractive because the samples came from patients that were characterized by these ME/CFS clinical experts.
So as we go forward with the Biobank I think you may be aware that in November 2014, we were awarded a grant from Falk Medical Trust, about a half $1 million grant that was intended to work with Dr. Lucinda Bateman again a clinical expert in ME/CFS medicine. And we are working with her to recruit a whole new cohort of ME/CFS patients and healthy controls to not only conduct an epigenetic study on these samples to extend preliminary results from Dr. Patrick McGowan, but also to restock the Biobank with samples again from well-characterized patients and these patients will be enrolled using the 1994 and Canadian criteria again.
So when Dr. Bateman enrolls, these patients, she literally goes through a checklist for each of these criteria and the patients will have to meet both.
Now we are using the new IOM diagnostic criteria to form our questionnaires for this study. So we will ask specifically about post-exertional malaise in the study. We will ask specifically about function. We will ask about pain, we will ask about cognition and fatigue. So the study will be based actually on the criteria from the IOM – we would basically collect the phenotype information based on the criteria recommended by the IOM. So instead of a bunch of long questionnaires that might not be relevant to ME/CFS really focus our questionnaires in a way that I don’t think would’ve been possible without having this IOM report in hand.
So it’s going to be really, really cool I think to say, “Okay, here is patients that meets '94 and Canadian criteria,” and then, “Here’s how they respond to these questionnaire” I think it’s going to really help us further see some of the strengths and limitations of the '94 and Canadian criteria as well as whether or not or how the IOM recommendations can be used in a research setting because they are diagnostic criteria. They were not formed for research studies. They are diagnostic criteria so I think it’s going to be real exciting to see how we can begin to shape these so that they do accelerate the way and improve the way we do ME/CFS research.
Dr. Craig: That sounds like a wonderful opportunity. Now you talked about the collection of blood samples. Is there any plan to collect any other types of samples; from stool or tissue?
Vernon: Not for this study. I think what we are going to do with this particular award is generate some deep rich data that helps inform the kinds of samples and the directions of research that should be taken in 2015.
Again as well as using the samples to attempt to validate some of the findings that have come about in the past year or so. Maybe finding and hopefully get into looking at some NK cell stuff. So right now we’re focusing on blood.
Dr. Craig: So as far as joining the Biobank, who is eligible to join and sign up? You can sign up straight from the website right? I signed up with myself a couple years ago but is anyone else eligible that has a ME/CFS diagnosis?
Vernon: We do cast a broad net. So yes, anyone is eligible. And depending on how you answer the various questionnaires and surveys that we send out, determines your eligibility for various types of research. So over the past several months we have been sending out surveys that collect information about your diagnosis, about various symptoms, about function of late. And as research studies, that have specific types of eligibility criteria for example a duration of illness, certain types of medications that you are using, certain severity symptoms then we can go into the database portion of the Biobank and say, “Okay, this person meets all of the criteria,” and then we then collect the kind of sample that the investigator would require for the research. So yes, anyone can participate and then eligibility as we said is determined by the questionnaire
Dr. Craig: Right, right. So when you first hear about the Biobank, maybe you think it’s only for people who are currently sick. But I signed up for it myself even though I’m basically recovered, or in remission. So you never know, there might be studies looking at recovered patients one day, or there might be studies looking at family members of patients.
So would you encourage those that are currently sick as well as maybe recovered patients and family members to look into maybe signing up for the Biobank database?
Vernon: I do. And I think that recovered study is one of the most important studies that somebody has got to do, and maybe it will be us in the coming years but that is a very important study to do. It is interesting because 2 years ago when I was at the FDA I was giving a talk to the FDA on ME/CFS and during the question and answer period, one of the lead doctors asked, “Who is studying recovered patients and why aren’t you studying recovered patients?” And we discussed how important that kind of study would be to really understanding the risk factors and the factors that are important for recovery so that’s a great study.
Dr. Craig: Yeah, I hope to see it one day soon. I would love to see what would turn up from that kind of study and I would love to participate if possible. So a lot of the research that’s come out recently – last year you put out webinars for different studies that came out from the Biobank. You mentioned Patrick McGowan’s awesome study on epigenetics…
But for our listeners who maybe haven’t seen some of those webinars is there a way to go back and get the summary of these important discoveries?
Vernon: So our Solve CFS Chronicle that was published in December does actually have a summary of all of our funded research as well as our Biobank investigators so that’s a real nice overview and summary. And one of the other ways that you can get more information about some of the research that’s being done is on our Solve CFS YouTube channel so we had a number of our funded investigators as well as Biobank investigators give talk
One of my favorites was by Dr. Derya Unutmaz who was then at the NYU Langone Medical Center, is now moved to the Jackson Institute of Genomic And I mean he is a great example of lowering barriers to ME/CFS research because of the Biobank. He has submitted a grant application to the NIH to continue along this line of research looking at again, profiling and the main function in ME/CFS.
Again based on his results that he generated he is using the Solve CFS Biobank sample. It’s very, very exciting, very exciting resource and infrastructure that we provide to the research community.
Dr. Craig: Right. And all these videos are also available on your YouTube channel as well. I have watched most of them and they are all kind of geared for a layperson so they are not too overly complex. So I definitely encourage listeners to go subscribe to the Solve ME/CFS initiative YouTube channel so you can stay on top of those and new ones that will come out in the future.
Another thing that you are doing recently is a summary of the IOM report. You had a briefing in DC and you’re doing a breakdown of the report on ME/CFS initiative website. The report is what, hundreds of pages? So can you talk a little bit about what you’re going to be doing with the breakdown?
Vernon: It is 304 page. And that’s the small compared to ARC’s evidence-based report from the NIH P2P which I think is 477 pages so I am glad I am starting with the IOM report. And what I am going to do is really just to break it down into digestible portions – starting out by really trying to help people understand the difference between case definition and diagnostic criteria and then kind of highlighting the rationale that the committee has for the diagnostic criteria that they selected and how that would actually work in the clinic in a doctor’s office – the process that the doctor would have to go through to make that diagnosis and then kind of dive into each one of the criteria and substantiate why the evidence that was used in order for the committee to feel confident that that was an important criteria for the diagnosis of ME/CFS.
Dr. Craig: Such a thorough report. I have not read all 304 pages but I did read the abridged version for clinician. I definitely commend everyone who worked on that although there has been a lot of pros and cons raised, but in general it’s all been very positive. I would say. And I look forward to seeing where it goes in the future. And there’s been a lot of excellent pieces written up about the report in the New York Times and I think any media attention is good just like we saw with XMRV – media attention is always good for this illnes
One question that comes from all of that, I mean this half jokingly and have seriously, the Solve Initiative ME/CFS is a recent name change for you guys. Are there plans to change the name again to SEID Association or however you want to say it?
Vernon: I know that the board and Carol Head, our president and CEO are discussing how to handle it and so yeah, I think we will wait and see what happens.
Dr. Craig: Yeah well we will just have to wait and see if the name gets adopted. We kind of had the same thing with the introduction of the CFIDS was adopted by some but not universally so we will see and it is just too soon to tell how the name will pan out.
Vernon: Yeah. I wish – I’m a scientist so I like to stay focused on the research. And for me what would be most important is to just define it and name it based on the biology. And because we are moving into this era of precision and personalized medicine, if we can diagnose each individual based on what it is that ails them without having to name everything, I think we would benefit tremendously from that rather than getting bogged down with these names which as you indicate, are going to continue to change and evolve because none of them are spot on.
Dr. Craig: Right. I mean I don’t know what name I would give it so I can’t say anything about the new name really. It is what it is and we will see how it pans out.
So what else is on the horizon for the Solve ME/CFS initiative here in 2015?
Vernon: Well, we are gearing up. We just got IRB approval – that’s Institutional Review Board approval for getting the study that was funded by this half a million Falk award up and going. So we are getting the equipment in place, we are getting the clinic geared up, we are doing some preliminary studies with Dr. McGowan up in University of Toronto who will be doing the epigenetic component, getting those pilot studies done. So at least for 2015, this is really an exciting year hopefully furthering Dr. McGowan’s preliminary epigenetic findings as well as restocking the inventory of our Biobank so that investigators like Derya Unutmaz and Lenny Jason can further their preliminary findings on these next set of sample
Dr. Craig: Now Dr. McGowan is in Toronto. So will that mean that patient recruitment for the study will be open up to both US and Canadian patients or is it just going to be US cohort?
Vernon: So the samples that Dr. McGowan will be working on for this particular study are all coming from Dr. Bateman’s all being recruited into Dr. Bateman’s clinic because again remember what we’re trying to do is provide a set of samples from patients that are clinically characterized so that they actually had a doctor like Dr. Bateman lay hands on them and verify the presence of ME/CFS.
Dr. Craig: Right. They will have a stronger study there with better patient selection I am sure.
Vernon: That’s right and that’s really important especially to attract other types of researchers to the Solve CFS Biobank. Hopefully we will let’s say a year or two, have enough information available that we will be able to recruit and kind of classify ME/CFS patients from anywhere using maybe a questionnaire, a set of questionnaires and maybe a certain type of sample to say, “Yes indeed, this person has ME/CFS” But until we get to that point it’s really important to work with somebody like Dr. Bateman to get very, very well-characterized sample
Dr. Craig: Right. So for our listeners out there, how do you suggest that we become more involved or contribute either to the Biobank which is Solve ME/CFS initiative?
Vernon: Well certainly sign up for the Solve CFS Biobank and you can go to our website and there is on the homepage at solveCFs.org, a link that you can click on that brings you right to the page that gives the information of how to contact us to get involved with the Biobank and of course you can always donate. Research is very expensive.
For those of you that may have listened to the solve ME/CFS initiative’s event that we held in Washington DC today, that was what our president and CEO Carl had and Dr. Clayton who was the chair of the IOM and Morgan Fairchild said research is not cheap and so we could use all the help we can get. So you can go to the page and donate and you can also continue to advocate for increased research funding from the NIH and from all the federal agencies And this is a great opportunity, a great time to do so on the heels of evidence-based report from the NIH’s P2P workshop and now with the IOM report being published. It is just a perfect time to keep the momentum of awareness and the importance of research going forward.
Dr. Craig: Definitely. Especially with all the media attention we’re getting, let’s keep it on everyone’s mind
A little call to action for our listeners out there, last month marked a record high number of downloads for Spoonie Radio, close to 1500 listener. So I am thinking if 1500 listeners all go right now to sign up for the Biobank--it only takes a few minutes to sign up, it is very easy to do--then we can continue to build strength in numbers to move forward and hopefully find first a biomarker, and then a cure for ME/CFS.
Vernon: Here, here.
Dr. Craig: Thanks so much Suzanne for taking the time to talk to us and give us this information today. I look forward to seeing what else comes out of the Biobank.
Vernon: Thank you so much Courtney for this opportunity. Thank you so much for doing this. Spoonie Radio is way cool. It’s got a great name.
Dr. Craig: Yeah. Some people still don’t know what a Spoonie is but it’s coming around.
Vernon: Yeah. So I look forward to continuing listening and having the help you and all of your listeners solve ME/CFS.
Dr. Craig: Yes, and I encourage our listeners to subscribe to the show over on iTunes and if you like the show, feel free to rate it, would love to hear your feedback. And until next time, this is Spoonie Radio signing off.