Imagine that you’ve just been put in charge of the world’s ME/CFS research – yes, you – and you’ve got to decide what research you want. Come on, hurry up!
Erm, erm, erm… oh yes, well, of course, as a patient you want something that’s going to get practical benefit for you in the shortest time possible. You’ve been waiting decades for treatments: you want them fast. But you’re not some medical genius so how are you going to choose what research should be done? Well, you could get the best ME/CFS researchers in the world, shove them in a room together and not let them out until they’ve come up with a list of the best, fastest-payoff, top priority projects, and… hang on a minute, somebody’s just done that.
In June 2012, the brand-new Open Medicine Institute, founded by well-known ME/CFS clinician and researcher Dr Andreas Kogelnik, held a three-day conference of leading ME/CFS researchers from all over the world. Attendees and signatories were a roll-call of our best and brightest, including Drs Klimas, Fluge and Mella, Peterson, Singh, Lapp, Bateman, Montoya, Marshall-Gradisnik, Rowe, Alan and Kathleen Light, and many more.
OMI-MERIT Priority Projects
The remit of the conference was to come up with a list of research projects that would deliver practical benefits for patients in the fastest possible time. Here’s the list, in order of priority (if you can’t cope with this much detail, skip to the end of the list where I give you the highlights; conversely, if you’d like more detail, go here):
1. Large-scale, randomised, placebo-controlled trial of Rituximab and Valganciclovir (Valcyte) – $7.65 million (£5.1 million, €5.9 million)
This trial would be on a larger scale than the Haukeland study and would explore the possibility that it’s necessary to not only ‘reboot’ our immune system B-cells with Rituximab, but also clear the decks of viruses with Valcyte. As with any immune therapy trial, success here would both provide treatment for patients and help move ME/CFS into the medical mainstream.
2. International neuro-registry and biobank – $1.93 million (£1.3 million, €1.5 million)
A biobank of blood, cerebrospinal fluid, urine, stool, brain and central nervous system tissue and other samples will be collected both from patients and controls, along with data on symptoms. Cutting-edge tools in immunology, genomics and molecular biology will be used to establish clinical and biologic clusters in the population, paving the way for diagnostic biomarkers and cluster-specific treatments.
3. Protein panel in treated and untreated patients – $658,000 (£436,000, €509,000)
Specimens will be selected from the biobank based on expected yield from clinical data and subjected to protein analysis to identify bacteria, viral, hormonal, antibody, cytokine and other protein-based substances that might be present in patient specimens. Protein markers are key in identifying potential biomarkers, and many new advanced detection technologies that have never been applied to ME/CFS before will be used here. Discoveries will be confirmed in the larger patient population.
4. Treatment: Phase 2: Other mono- and combination-therapy pilots – $984,000 (£652,000, €761,000)
This project will assess, via pilot studies, the effect of other currently available treatments such as Ampligen, Etanercept, Rifaximin, Isentress and Famciclovir, and will establish immunologic and molecular parameters for measuring the efficacy of such treatments. This will help identify which drug therapies should be prioritised for further research.
5. Immunologic biomarker exploration studies – $447,000 (£296,000, €346,000)
These exploratory studies will examine B-cell, T-cell and Natural Killer cell responses in treated and untreated patients using comprehensive, rigorous methods, many of which have never before been applied to ME/CFS.
6. DNA genetics – $592,000 (£392,000, €458,000)
Advanced Human Genome Project technologies will be used to sequence key genome areas – including methylation genes – in a set of patients, controls, affected families and unrelated individuals. The goal is to establish or refute a role for genetics and potential heritable risk in ME/CFS.
7. Mass spectroscopy/environmental measurements – $232,000 (£154,000, €179,000)
This exploratory study will search patient samples for unknown compounds, toxins, proteins and other substances to see if nutritional and environmental factors are implicated in the genesis of the disease or otherwise contribute to immune dysfunction.
8. Comprehensive viral testing – $406,000 (£269,000, €314,000)
This study aims to establish a gold standard of clinical viral testing in ME/CFS. Testing will include blood, urine, saliva and other tissues where available for specific viruses such as EBV, HHV-6, CMV, Parvovirus, HSV-1 and HSV-2 as well as testing for novel viruses.
9. Advanced immunologic biomarker study #2 – $187,000 (£123,000, €145,000)
This second immune study will look at additional cell types that complement Project 5 above, such as monocytes, macrophages and dendritic cells.
10. Treatment: Phase 3: Natural and over-the-counter substances – $416,000 (£276,000, €322,000)
This project will be the first application of vetted scientific methods and molecular science to non-pharmacological substances that have had anecdotal benefits for people with ME/CFS, including Moringa oleifera, GcMAF, Vitamin B12 and Artemesinin.
From a patient’s point of view, that’s a very interesting list. On treatments, at one end it has studies on the heavy-duty immune drugs such as Rituximab, Valcyte and Ampligen: if any such study was successful it would not only help make those treatments available but would have a huge knock-on effect in validating our disease as an immune-system one. At the other end, there are treatment studies on non-prescription substances that we can get our hands on now, such as GcMAF and B12. All of these treatments are hot topics in the patient community.
The list also contains studies applying cutting-edge laboratory techniques to look at biomarkers, pathogens, toxins and genetics and includes all the major items of interest to patients – herpes viruses, NK cells, methylation, you name it. ‘Cutting-edge’ is a phrase that keeps coming up with the OMI – they want to use the most advanced technology to get at the heart of this most complex of diseases.
Plug in to the Network
They’re also keen on collaboration and opensourcing. Dr Kogelnik’s background is in bioengineering, molecular biology, genomics, immunology and bioinformatics – he’s well placed both on the science side and on how to get the most out of collecting, analysing and sharing the huge amounts of data needed to solve the ME/CFS problem. OMI will soon be opening Open Mednet, an online data repository where patients anywhere in the world, hopefully in their tens of thousands, can contribute to this research. We’ll be covering this in a future article: you can pre-register for the project now.
OMI’s collaborative, outreaching strategy has shown up not just in assembling the impressive panel of collaborators who drew up the priority research list, but as a networking approach that ranges from bagging a top-ranking researcher such as Dr Jay Levy – co-discoverer of HIV – to serve on the OMI’s board, to generously using OMI’s own Facebook page to support a patient, Dr Maria Gjerpe, in her fundraising efforts for a Rituximab trial in Norway.
The full whack to fund all of the studies is $13.5 million (£9.0 million, €10.4 million): peanuts for a government, a lot of money for ME patients to get together. Can the OMI raise it? Well, they’ve already bagged cash or pledges of $1.8 million (£1.2 million, €1.4 million). Although donations to the general pot go by default to the highest priority study, donors can specify where they want their money to go and two projects are already fully funded: the Neuro-Immune Disease Alliance have paid $40,000 (£27,000, €31,000) for a study on over-the-counter treatment Moringa oleifera (part of Project 10) and the Edward P. Evans Foundation have covered Project 6, the genetic study. An individual patient has donated $100,000 (£66,000, €77,000) to Project 1 – the Rituximab/Valcyte study.
The OMI are industriously tapping up every possible source of funding: foundations, individuals, corporations, pharmaceutical companies, diagnostic companies and the government. They’re starting to get traction on most of these and are taking ‘in kind’ donations as well as cash. For example, the OMI were just awarded a grant of both financial and consulting support by VMware, a virtualisation and cloud infrastructure company.
The OMI’s fundraising arm, the Open Medicine Foundation (OMF) are, incidentally, legit! A forum member asked a question on the boards a while back about their tax and charity status. The OMF are a legal 501(c)(3) nonprofit with effect from 31 July 2012 but since they had no activity for three years prior, the IRS revoked that status until they had activity and refiled. The Foundation are new so there is no 990 form yet. They can be seen on both the IRS and Guidestar sites.
So: big ideas, highly efficient use of samples and data, cutting-edge science and they’re not mucking about. Want to make a donation? The OMI do research on other diseases so when you go to their donation page, be sure to check the ‘ME/CFS Research (MERIT Initiative)’ option. If you’d like your donation to go to a specific project, say so in the ‘Message to honoree’ box.
You can keep in touch with the OMI’s activities via their free newsletter (sign up here). They have plenty more in the pipeline and are well worth watching!
Remember that Phoenix Rising costs money to run and needs your donations to support it. Please hit the button below and donate!
Health Rising article on the OMI’s ‘big data’ approach
Health Rising article on the OMI’s newly funded gene study
Review of medical evidence on Moringa oleifera
Phoenix Rising is a registered 501 c.(3) non profit. We support ME/CFS and NEID patients through rigorous reporting, reliable information, effective advocacy and the provision of online services which empower patients and help them to cope with their isolation.
There are many ways you can help Phoenix Rising to continue its work. If you’re able to offer your time, we could really use some more writers, proof-readers, fundraisers, technicians…and we’d love to expand the board of directors. You can even donate significant sums, at no cost to yourself, as you shop online! To find out more, visit Phoenix Rising’s Donate page by clicking the button below.