Chronic Fatigue Syndrome specialist Dan Peterson, MD, sat down recently with Phoenix Rising (PhoenixRising.me) founder Cort Johnson, to explain a few of his new insights and projects - some of which just may turn the tides for ME/CFS diagnosis & treatment.
In particular Dr. Peterson wanted to talk about a high-priority research partnership that his Simmaron Foundation has in the works. Following are excerpts, reproduced with kind permission,* from Cort’s news-packed report on their discussion – “Dr. Peterson Talks!”
Dr. Peterson Talks!
On Ampligen, Autoimmunity, Pathogens & a New Partnership
Cort Johnson, July 13, 2012
One of the few physicians providing Ampligen® over the past 15 years, Dr. Peterson intimately knows what Ampligen can and can't do. He made it clear that while the drug has its flaws, it's effective… at times stunningly effective.
In fact it's the most potent tool he has in his drug toolkit; it's so potent that he told Corinne [Cort’s friend & a long-time patient of Dr. Peterson] that if Ampligen becomes available for her it'll replace everything she's taking - the IVIG, Procrit, saline solution, amino acid drips, CoQ10, etc., all will disappear and she'll simply be taking Ampligen.
On July 11th, in a move that renewed hope for Ampligen, the FDA informed Hemspherx BioPharma, Ampligen's producer, that it would not require (prohibitively) expensive new studies before it would review the drug.
Instead, Hemispherx will be allowed to present an analysis of old and new data to the FDA in the third quarter of this year with the opportunity for approval coming six months following that. [See sidebar below: “Major Move From FDA Puts Ampligen Back on Track.”]
It's Complicated - Just Like ME/CFS
The thing, though, is that the drug works. Dr. Peterson would love to see the drug approved and he's not clear why it hasn't been.
Certainly there have been problems. Ampligen has moved so slowly in the FDA's pipeline that it has been shifted to at least five different divisions, but hundreds of patients later the safety data is clear and it's shown to be more effective than other FDA approved drugs in improving exercise-related endpoints.
• The FDA can engage in a process called 'accelerated drug approval' for disorders with unmet needs (check!)
• In which the drug, which has shown to be safe (check!), may be tentatively approved for use - giving patients access to it and allowing a company to recoup its costs and collect data for final approval.
Why Ampligen hasn't been given this opportunity is a head scratcher given the presence of a large patient community with no FDA approved drugs….
[To read about the different experiences of three of Dr. Peterson’s Ampligen-treated patients, see the “Interlude” section of “Dr. Peterson Talks!” at Phoenix Rising.]
Rituximab (Rituxan) and Autoimmunity
In an interview with ME/CFS Alert Co-host Llewellyn King, Dr. Peterson noted that he's seen the same pattern of improved health in lymphoma patients of his with ME/CFS that Fluge and Mella saw in Norway.
A monoclonal B-cell antibody drug may work very well for some patients, but Dr. Peterson was concerned that simple market economics may keep Rituximab from being the drug for ME/CFS.
Rituximab's soon to be generic status means Roche has no incentive to undertake expensive studies on its effects in CFS, and without those studies there's little hope insurance companies will cover the cost of the drug.
Some people will be able to pay the out of pocket costs once the drug goes generic and its manufacturer does provide steep discounts for lower income people. But it may be a next generation drug of this type - and Dr. Peterson noted that several are under development - that ultimately benefits the ME/CFS community the most.
Dr. Peterson will be administering the drug to patients in Dr. Andreas Kogelnik's Open Medicine Rituximab study getting underway.
Dr. Peterson doesn't know if CFS is an autoimmune disorder per se, but he finds that as the years go on more and more patients have positive antinuclear antibody tests. (Given that perception, it'll be interesting to see if longer duration patients tend to benefit more from Rituximab.)
He noted that a common finding in his practice, autoimmune thyroiditis [Hashimoto’s disease], is often virally induced.
Dig Deeper...Check Out:
• Phoenix Rising's Rituximab Resource Center
• “Rituximab: the Drug - From the Beginning to Now” by Jorgen Jelstad
* * * *
Chronic Fatigue Initiative Pathogen [‘Lipkin’] Study
Dr. Peterson runs one of the centers participating in the Chronic Fatigue Initiative's Hornig-Lipkin pathogen study. I asked him how definitive he thought the study would be.
• If pathogens are present in one of those four tissues (blood, feces, tears, saliva) would [Dr. Ian] Lipkin find them?
• Will the herpesviruses that Dr. Peterson at times finds show up?
Calling the study “extremely complex and rigorous,” he said absolutely they would.
With its four tissue samples the study is already far more comprehensive than any others, but Dr. Peterson, noting that pathogens found in the spinal fluid often don't show up in the blood or elsewhere, wanted the CFI team to sample spinal fluid as well.
When I talked with him in March he was negotiating to do that; now according to his recent Llewellyn King interview that's been done - so add spinal fluid to Lipkin's list.
Dr. Peterson stated that Dr. Lipkin is interested in ferreting out subsets, which suggests that simply the appearance of any virus will be significant enough to warrant attention.
Again my understanding was that if Lipkin finds a virus that means it's active; these are not the difficult-to-interpret antibody tests that we're used to with herpesviruses; it sounds like a found virus is a serious virus and a cause for potentially setting a group of patients aside.
It's hard to believe that some positives will not be found; it’s the percentage that is the question. Dr. Peterson, of course, finds positives in his patient population, but with his focus on patients with Natural Killer cell dysfunction, he probably sees a group of patients more likely to have them. When asked to take a guess he took a conservative guess - something in the 10% to 20% range perhaps.
• He felt that finding even small percentages, though, would make a big difference, since that should get different patient groups segregated from each other in research studies.
• His guess was that Lipkin will not find new pathogens. (In our recent ME/CFS professional survey most [6 out of 8] felt the same.)
• He noted this kind of study from this kind of researcher could have profound treatment implications including justifying the use of off-label drugs for CFS (presumably to doctors not currently willing to do so).
Kudos to the Hutchins Family Foundation and the Chronic Fatigue Initiative for putting this study together. A comprehensive pathogen study should have been done years ago, but with the pathogen arrays improving all the time, it also would not have been nearly as powerful…This is a great time to do this study.
A Proving Ground
The XMRV Lipkin study was a proving ground, so to speak, for Dr. Peterson and Simmaron because it gave them access to a high-level, rigorous, research environment. From the beginning of Lipkin's NIH XMRV study, they were eager to position themselves as a group Lipkin could trust and is comfortable working with. With Simarron adding another element (spinal fluid) to the CFI study, they've clearly done that.
It probably helps that Gunnar, Dr. Peterson's research assistant, has a competitive streak a mile long. His goal was to be the first center in the CFI Lipkin study to get their samples into Lipkin's hands and they were.
* * * *
Jonathan Morse, Director of the Simmaron Research Foundation, talked of the exciting work done on the immune system and Simmaron's focus in that area.
A major part of his job, as he sees it, is to ferret out and bring new researchers into Simmaron's orbit, expanding research opportunities for ME/CFS. To that end Simmaron has created a roadmap of investigations they feel will shed vital light on the connection between CFS and the immune system, autoimmunity, viral reactivation and cancer.
Simmaron has formed several partnerships with ME/CFS research groups, none with more potential than a relatively little-known but rapidly emerging group in Australia called PHANU (Population Health and Neuroimmunology Unit).
The relationship began, as so many in the research world do, at a scientific conference in Australia in December 2010. Both groups have something to give each other; Dr. Peterson has a large and well-characterized patient population and decades of experience; PHANU has a fantastic lab with cutting-edge technologies that is only going to get better.
PHANU's director, Dr. Sonya Marshall-Gradisnuk, PhD, is on Simmaron's Scientific Advisory Board. In a recent interview with Phoenix Rising she said:
“I along with the CFS/ME research group that I lead have the greatest respect for Dr. Peterson. He is a true gentleman and an amazing clinician. I met Dr. Peterson when he came to Bond University in December 2010 when he was invited to an International Science Symposium for CFS/ME that I was leading with Dr. Staines from Queensland Health.
"Our meeting was organized by the Alison Hunter Memorial Foundation - a wonderful group of dedicated people. I am very proud to say that Dr. Peterson is a significant collaborator on a number of large national grants for CFS/ME that I have.”
Spinal Fluid Breakthrough?
Simmaron's top priority at this point is a joint Simmaron/PHANU study designed to look for evidence of immune/autoimmune activity in the spinal fluid.
In our last interview Dr. Peterson said he believes:
• Immune activation in the brain is probably driving many of the symptoms present in ME/CFS,
• And the spinal fluid is probably the closest shot (short of taking a biopsy) to figuring out what's going on there.
Simmaron's been focusing on gathering 'pre-pilot' data, and their pre-pilot spinal fluid data blew researchers away and helped inspire the Mason Foundation to give PHANU one of the largest ME/CFS grants ($830,000) ever.
But the grant came with a condition - a $220,000 pilot study had to be completed first.
Thanks to the generosity of patients (with special thanks to the Linda Tanenbaum of the Neuro-Immune Disease Alliance) a third of that is in the bank, leaving just $150,000 to go.
One In… Five Out
You can't get a much better deal than this: Each dollar you donate will, with the Mason Foundation's help, bring in 5 more research dollars. But we have to get that money in there first.
Long time patient, advocate and supporter, Rich Carson of ProHealth, recently launched a personal appeal for funds stating "There has never been a better time or a better opportunity."
This study has been in the works for over a year.
Gunnar, Dr. Peterson's lab director, described the lengths to which the groups went to [ensure they’ll] get the spinal fluid safely from Dr. Peterson's lab in Incline Village across the world to Australia. [See Llewellyn King's new interview with Gunnar here.]
• First there was an overnight flight to China with a rep meeting the temperature controlled and digitally monitored 'dummy package' at the plane and then the trip down to Australia, where it was immediately retrieved.
• That process worked, and the [actual ME/CFS patient] spinal fluid will be winging its way down to Australia should funds permit.
Simmaron is so enamored with PHANU's technologies that they've gone so far as to send selected patients - not samples - all the way down to Australia to take advantage of them.
Bond has been very good to PHANU, and Bob Miller, one of three patients traveling to Australia, described a rigorous lab - the patients’ blood being whisked away as soon as it was taken for workup, and a large and well-equipped lab - the largest ME/CFS lab he had seen.
With their sophisticated lab, PHANU had a good gig going at Bond but they're moving on. As interest in their work has grown suitors have come calling, and recently Griffith University, a much larger university, made Dr. Marshall-Gradisnik an offer she'd couldn't refuse - a substantially larger lab in a new wing they're building.
The lab is a quick skywalk to the hospital providing easy access to medical facilities and, of course, they're hooked in the academics as well. It’s a true Center for Excellence down under - the largest and most sophisticated ME/CFS lab in the world.
Things are converging for Dr. Peterson and Simmaron....
Major Move From FDA puts Ampligen Back on Track
Cort Johnson, Jul 11, 2012
[Is Hemispherx “sitting on a multi-billion dollar chronic fatigue syndrome market worldwide”?]
Reversing a 2009 decision some commentators felt was potentially crippling to Hemispherx BioPharma, Ampligen’s producer, the FDA today stated they would not, after all, require an expensive 300 person study to assess Ampligen’s effectiveness in chronic fatigue syndrome (ME/CFS). (Safety concerns appear to have been taken care of.) Instead they would allow Hemispherx to use new analyses of data it has already collected. Hemispherx must be breathing a huge sigh of relief.
A 300 person, double-blinded, placebo controlled drug trial was far beyond Hemispherx's financial capacity, whose assets, Reuters reported, fell from $58.1 million in late 2009 to just $3.6 million in March of this year.
The FDA imposed some major conditions on Hemispherx in 2009 but it could have rejected the application outright. Instead it chose to give the company more and more and more time; essentially keeping the door open. With this [July 11] decision, which occurred after a recent meeting between the company and the FDA, the FDA opened the door a bit wider. Reuters reported that Hemipherx’s stock price soared 50% on the news.
It’s not clear what circumstances prompted the FDA to change its mind but it has been under fire for low rates of drug approvals over the past five years; in particular for drugs to treat chronic illnesses.
With about a million people with ME/CFS in the US and government-funded studies suggesting as much as $20 billion a year in economic losses, ME/CFS may be the only ‘major’ disorder without an FDA approved drug.
In its report MarketWatch called ME/CFS a ‘chronic, serious debilitating disorder.” (The FDA recently agreed to a Stakeholder’s Meeting to discuss its approach to chronic fatigue syndrome.)
In March an analyst noted that although the economics of ME/CFS treatment are unclear, Hemispherx could be sitting on a multi-billion dollar chronic fatigue syndrome market worldwide. Even if just a portion of ME/CFS sufferers were treated with Ampligen the potential upside for the company and its shareholders is large indeed.
MarketWatch reported that upon news of the FDA’s decision Hemispherx immediately began hiring more staff, consultants and independent contractors to prepare for the work ahead.
A 2010 study suggested Ampligen could take the place of drugs that expose some ME/CFS patients to cardiac toxicity. Other studies have shown Ampligen can significantly increase exercise tolerance.
The best drug never to get approved for ME/CFS suddenly appears to have a real chance.
Hemispherx reported it will file its response in the fall of this year. The FDA typically takes up to six months to produce a decision.
Nothing is assured but with Hemispherx finally getting its day ‘in court’ and Rituximab trials ramping up, the treatment possibilities for ME/CFS appear quite a bit brighter.
* ©Phoenix Rising, 2012 (http://phoenixrising.me). All rights reserved. This information is excerpted from a longer article, first published Jul 13, 2012. To read the full article, which covers more subjects of interest to Dr. Peterson, click here.