“What we need in CFS is what I call the development of an innovation supply chain.”

Bernard Munos

Munos stated chronic fatigue syndrome is barely on industry’s radar….but that if we build the infrastructure they will come – they do every time

Bernard Munos started off Day II of the FDA Stakeholders Meeting on Chronic fatigue syndrome (ME/CFS). Bernard Munos is not an FDA guy – he’s an independent analyst – really a gadfly of sorts –  with a long track record of calling for transformational change in how Pharma  develop drugs. Munos thinks drugs can be discovered and produced much more cheaply; in fact, he thinks pharmaceutical companies who  don’t change course are doomed to failure.

Munos was named one of the 25 most influential people in Biopharma. FierceBiotech calls him the “R&D revolutionary”. A former Advisor for Corporate Strategy at Eli Lily, Munos focused on what he called ‘disruptive innovation’ in radically redesigning their R&D model. More recently he founded  the InnoThink Center for Research in Biomedical Innovation. In short, the FDA brought in a key figure in innovation in drug development to present at  the ME/CFS Stakeholder meeting. He was a good choice.

Speaking extemporaneously, Munos laid out both the problems the field faces, and the solutions, in impressive fashion.

He described the ‘drug hunters’ in the pharmaceutical field who scan the frontiers of science  for new possibilities they can turn into drugs. Munos made it clear, though, they are not looking at  chronic fatigue syndrome and there are multiple reasons why.  The field, unfortunately…

  • Has a ‘dearth’ of translatable research; i.e. it has little research that easily translates into drugs
  • Is missing  infrastructure (data and tools) other disorders have
  • Has a fuzzy definition  and a heterogeneous population – both of which the industry abhors.
  • Has treatments that treat symptoms, not the core of the disease; i.e. there’s little agreement on what’s going on at the core of the disorder (which makes it difficult to devise a drug to treat it :))
  • Poor understanding of the disorder’s natural history (who it effects, how it proceeds, etc.)
  • Little understanding of endpoints

To make it really clear, he said for industry ME/CFS barely exists (!). (I took some comfort from the fact that he didn’t say we didn’t exist  – period – for them.) And it hardly exists for the mainstream research community. Researchers, he said, follow the path of least resistance and not many will tackle a disorder with so many issues. To really move forward, the field has to make it worthwhile for researchers to work in it.

big dtaa

Munos believes ME/CFS, in particular, needs lots of data to sort out its problems…,

One of the big problems is how heterogeneous the population is. That issue can be overcome; in fact, it’s imminently solvable, but solving it requires lots of data.  The ME/CFS field, Munos said, desperately needs more data. It’s got some, but it needs more: basicly, enough data to delineate the subsets in it.

 “So we need data, we already have some, but not enough and not in right format and not good enough. We need more and better data. We need tools to go along with the data. We need partners, we need money and lastly, we need leadership and passion, I should stress already we have quite a bit of that already, but we need to connect it a little bit better than it is connected today.”

Munos outlined a lot of information gaps but the news was not all bad. Munos believes each of the gaps can be dealt with, and he’s a big fan of filling them as cheaply as possible.  We’re simply going to have to become more innovative; we need to develop what he calls an ‘innovation supply chain’; that is,  we – the ME/CFS research and treatment field (which includes the patients) – need to get innovative at each step of the process.  As we’ll see, we definitely have some innovation leaders in our field.

Collecting good quality data is not always expensive. Munos emphasized that it’s now possible using apps and electronic data gathering tools to  gather data on many aspects of a disorder very cheaply. In fact, there’s some really good news on this end.  The Open Medicine Institute (and, it appears, the CFIDS Association ) is soon going to roll out a variety of integrated apps that will be able to gather all sorts of  data.   The apps will feed into the OMI’s database giving researchers the data and you the information you need to better manage your illness.  Munos said the FDA ‘loves’ this kind of data.

“You have got technology today that allows CFS patient to track mobility, track efforts with data, collected effort by bio sensors, cell phone, it could track how many stairs you climb per day and how many miles and when you actually do the activities; not only can you track the expense of the physical exercise, but also intensity, and all that can be collected effortlessly and  you get thousands of patients who are doing that at the same time, pretty soon you will have serious data for scientists to work on and all that hardly costs a thing.”

Apps to collect data for ME/CFS

The Open Medicine will soon be releasing apps that will ME/CFS patients and researchers to capture lots of data

Expect a blog on the Open Medicine Institute soon. You’ll be amazed at the breadth of Kogelnik’s vision;  he, the CAA,  Dr. Klimas, and probably many others, all recognize that collaborating and gathering scads of data to break down and figure out this illness is key.  That’s exactly what Munos was talking about.

On the topic of ‘cheap’ ways to gather data, Munos noted that the cost of a complete genotype for a person is down to 5K and is lowering  about 65% a year. (That would mean 2K next year, $800 in 2 years, $320 in 3 years and cost of fancy dinner in 4….???). In any case it’s going down rapidly and it should be around 1K pretty soon.

Infrastructure – If You Build It They Will Come

“The good news is if you build it, they will come.”

Munos said that if you build the infrastructure they will come every time, and they do every time, and he cited the example of the Multiple Myeloma Foundation established in 1998 for a far less common disorder than ME/CFS, which has raised 280 million dollars, conducted 45 trials and has 6 FDA-approved drugs.  Multiple myeloma is a very different disease with very different issues but their ability to raise money was impressive. What are the keys to an effective infrastructure that fosters drug development?

  • Networking and collaboration – and the infrastructure to allow for that
  • Open source  – allowing free access to data

Everything, he said, should be shared, and he referred to an Open Source Drug Discovery Platform for tuberculosis in India that runs on $2,000,000 that in 4 years has enrolled 6,000 scientists and now publishes 2/3 of the papers in the field.

“It happens all the time, it happens with diseases that are much smaller than CFS. So it can happen here, too.”

Our chief assets are our size and our passion. This is a big community, much bigger than smaller diseases that have got the job done. We’re also moving forward quickly on networking and he referred to the CFIDS Association.

Innovation From the Ground Up

ME/CFS practitiones

ME/CFS practitioners – ground zero for innovations in treatment

Munos felt the ME/CFS field is ripe for major discoveries and, for the first time I’ve heard, he emphasized how much innovation, in a diseases where the rarity of validated treatments causes physicians to experiment, flows from the physicians upwards. In fact, he called ME/CFS physicians possibly the most powerful source of drug innovation.  That certainly brought to mind Dr. Peterson’s work with Vistide, Dr. Shoemaker’s work with cholestyramine,  Dr. Chia’s work with oxymatrine,  Dr. Enlander’s work with GcMaF, Dr. Cheney’s many innovations over the years,  and, if you want to get really, really creative, Dr. Goldstein’s work with many drugs years ago.  Munos felt it was important to harvest the creativity at the physician level and in Suzanne Vernon’s presentation next we hear of a project designed to do just that.

Munos asserted that the patients also need to network and questioned, at least to my ears, where is everybody in this million person illness. We definitely need to find ways to find people with this disorder and keep them engaged.


Munos started off outlining the many gaps in this field, but by the end he was inspiring. With the CAA being a leader in this area,  Suzanne Vernon of the CFIDS Association of America was perfect person to follow Munos’ presentation.

Dr. Suzanne Vernon

“This is what is known as derisking science, making it attractive to industry in order for them to pick up the ball and  impact our patient community. This has been our innovation pathway over the past five years to really provide an infrastructure which allows that information flow, those discoveries that occur at the bedside to come across the way and be attractive investments for pharma.”

Dr. Suzanne Veron

Dr. Vernon was lock-in-step with Bernard Munos recommendaitons

Dr. Vernon stated that the CFIDS Association of America is committed to being a translational research leader; that is, they are committed to developing the tools and producing the research that translates into new treatments for people with ME/CFS as quickly as possible.  She spoke Munos’s language; the CAA is committed to ‘de-risking’ science to make ME/CFS as attractive to industry as possible.  Part of that involves developing an infrastructure that creates an information flow drug companies can easily utilize.

“We’ve also built a knowledge base and data analysis and data sharing platform, which you will be hearing more about in the coming months and this will be open and available so it will provide a tool source that is needed in our community to help with the aggregation and analysis and sharing of data that we generate and this will be open.”

 They’re definitely walking their talk.  They’ve built a sophisticated Biobank/Patient Registry that is easily queryable and Dr. Vernon stated the CAA will provide tools in the next couple of months to assist researchers with data aggregation and sharing. It’s not clear exactly what she means but if she means finding ways to put data into one big database so that everyone can use it – that’s one  of the critical missing components for this field.

“This has been our innovation pathway over the past five years: to really provide an infrastructure which allows that information flow, those discoveries that occur at the bedside to come across the way and be attractive investments for pharma.”

Drug Repurposing

Drug Repurposing could ntroduce new drug possibilities and suggest new biomarkers

Drug Repurposing could ntroduce new drug possibilities and suggest new biomarkers

A couple of years ago the CFIDS Association partnered with Biovista – a drug repurposing firm – which beats the bushes to find drugs that no one thought might work in a disorder but just very well might. They do this by throwing scads of drug (1,000s of drugs) and research information (symptoms, study results not just for CFS but from fatigue, etc. studies)  into a database and then popping in algorythms that identify drug possibilities….but not just drug possibilites – also biomarker possibilities.   Not only that, but Biovista will also highlight drugs that could harmful, and, guess what, Dr. Vernon stated that a number of the “baddies” Biovista highlighted are in common use in the ME/CFS population. (Ouch!).

So Biovista gives us a three-for-one result; new drug possibilities, drugs to stay away from and even possible biomarkers – that’s a pretty good haul.  Biovista turned up  at least two  serotonin-affecting drugs, which, when used together, appeared to be able to affect very many aspects of CFS.  (No word on their identities yet.)  Whatever they found was a bit shocking to them; Suzanne called the findings ‘counter-intuitive’ – which, come to think of it was exactly what we were looking for. Biovista was supposed to give us a new slant on this disorder and how to treat it and it apparently has.  (Besides, all the intuitive possibilities have been used up over the past 25 years.) These drugs fit what’s called the ‘Central Fatigue Hypothesis’. It’s going to be interesting.

“We are a partner with pharma.”

What the CAA found  was  compelling enough to them for them to form a partnership with a pharmaceutical company (apparently) to develop a proof of concept clinical  trial for these drugs.

Capturing Clinical Intuition

We have physicians with decades of knowledge using their insights and intuition to treat their patients. For every patient they see, there have be 1,000 or 10,000 who don’t receive expert help. With maybe 10 well-known ME/CFS specialists in the US and 1 million patients, that’s not a very efficient use of their expertise. Dr. Unger of the CDC has been visiting the clinics in her multi-center trials and apparently she’s come away, well . . . quite impressed  by what she’s seen, and she stood  up at one of those secret  Cold Harbor (sounds so Cold War) er Banbury ME/CFS researcher jam sessions and said “We’ve got to find a way to capture clinical intuition.”

If you thought  this is the same old mostly-closeted CDC, think again – things have obviously changed.) Dr. Unger’s statement  got the CAA thinking and they went to Biovista, and Biovista developed a program to capture  ‘clinical intuition’ which involves asking our leading physicians about drug efficacy and how they treat symptoms.

The CFIDS Association has also apparently used their FDA survey to identify three  clusters of  patients

  • One type had muscle pain, exhaustion, sickness, joint pain, malaise, fatigue, sore throat, brain fog.
  • Another was a pain subtype described by pain in all parts of the body  – which was very distinct from the others.
  • The last had exhaustion, malaise, weakness, spasms, sensitivity and sore throat.

Lastly – the CAA is expanding its Biobank for the proof of concept clinical trial.  To learn  about the eligibility criteria and what is involved in donating samples and clinical information, contact the Association’s BioBank Coordinator, Gloria E. Smith at (704) 362-2343, or biobank@cfids.org.

A Look at Our Infrastructure For Success

Now that the CAA’s given us their input, let’s see where we are on the ‘infrastructure for success’ front Munos talked about.

We know that both the CAA and the Open Medicine Institute are actively engaged in creating open sourced tools that will foster (demand?) collaboration.

Biobanks, Patient Registries – B

In just a couple of years the  ME/CFS research community has become surprisingly rich in stored samples. Dr. Peterson’s had his samples for decades, but in the last three years the CAA, the Chronic Fatigue Initiative, the NIH, the CDC  and the Whittemore Peterson Institute all have begun biobanks. So we’ve got a good start on the samples . . . What about really putting them to use by creating an open-sourced, collaborative network?

Networking, Collaboration and Open Source – D

Dr. Munos emphatically said all data must be open source. This field is so short on resources and big on problems that we need every good data point we can get.  (This, of course,  should be happening with every medical disorder; people are, after all, suffering…but it particularly applies to small, underfunded fields like ME/CFS.)

Open source

Munos declared that allowing reseachers access to data was critical to success in ME/CFS

My take on this was that a researcher should be able to look into a central database and find samples (all de-identified) that, say, have low natural killer cell functionality, and then open up his/her computer and do a ‘virtual’ study that determines whether they are associated with other immune problems, how ill that subset is relative to another, if high pathogen loads are associated with that problem, etc., etc.  These studies would collect no blood and analyze no samples but would simply use statistical methods a) to cheaply test theories, and b) to get enough data to apply for a grant.

Both the CAA and Simmaron Foundation are currently engaged in studies that provide the opportunity to do something like this. The Simmaron Foundation’s cerebral spinal fluid samples are being  tested for pathogens and immune factors by CFI and PHANU researchers, and three research groups are subjecting the same blood samples  at the CAA for epigenetics, viruses and auto-antibodies.

This seems so simple.  Want to know if epigenetic abnormalities are associated with increased pathogen loads? Simply check the data. Do people with high cytokine levels in their cerebral spinal fluid also have CNS infections? Check the data.  Imagine a database containing data from dozens of ME/CFS studies. Simply gather the data, do a statistical analysis on it and bingo  you’ve got at least preliminary information and a solid direction that would have taken years to get otherwise. We know that Schadt, for instance, is gathering scads of gene data in the Mt. Sinai trial. That’s a goldmine for other researchers.

If you combine the right factors – say an epigenetic problem that opens the door to infections in some patients – you could start to get to the heart of this disorder very quickly.

With some of the same patients appearing in the CAA, CFI, NIH, Simmaron and WPI Biobanks, the potential for an incredibly in-depth examination of a select group of very ill patients is huge.  The CFI  will have data on pathogens, the CAA will have data on everything ranging from epigenetics to brain scans to cardiovascular issues, and the WPI will have data on immune functioning and pathogens.

The really big question is whether the CAA  and/or Open Medicine Institute (or?) can come with the infrastructure and enroll enough researchers to allow this to really take off.

Let’s take a look at Munos’s ‘To Do List” now…

  • Translatable research – ?? The CAA’s Drug Repurposing Biovista study will immediately produce translatable research and the more molecular research being done the more the possibilities of translatable research grow.
  • Infrastructure – the Biobanks are there; what’s needed is to connect them and build an open source data repository.  Although we don’t know exactly what the CAA is doing both it and the Open Medicine Institute are  clearly  moving strongly in this direction.
  • Fuzzy definition – Lenny Jason is working to develop an empirically derived definition  and ways to operationalize it; i.e. ways to  gather good data to support it. He has networked with several groups to gather his own amount of big data.
  • Poor understanding of natural history –  the Open Medicine will soon announce a bagful of tools that will enable researchers to gather this information.

There’s movement in every area Munos outlined.  There’s definitely a  networking bug going around the ME/CFS Community; the CDC is networking with 7 ME/CFS specialists; the OMI has networked with five of them.  They are all gathering the same type of data. The Open Medicine Institute got almost thirty  prominent members of this field to support a course of action which included an An International Neuro Registry and Biobank that includes both data and samples.

building it together

Collaboration and sharing will be critically important in this field

A lot will depend on how good the CAA’s and the Open Medicine Institute’s toolsets are and how effective they are at enrolling researchers to enter their data in there.  These things take time and money but in the not too distant future we could have a foundation that will attract both researchers and industry and, of course, produce some real advances in ME/CFS.  It will take a degree of collaboration this field has never seen before, however, and patients can support that by demanding that the organizations they support join in.  For me, I would make it very clear to the organizations that I support that I expect them to engage in  these open source efforts or I would withdraw  my support .  That’ wouldn’t scare anyone in my case but others might have an impact.

I propose that we as a community should keep a close watch on and push for as open source an approach to this disorder as possible.

After all, what’s at stake is our future and the future of those who come after us.

“From that point on, I think you can start looking for partners, corporate partners, big companies, but also small companies that can take the tools that you have got, the data, the discovery that you have made and start helping you out in coming up with new drugs.” Bernard Munos

If you build it they will come….





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