The job of Patient Advocate came upon me uninvited. I did not apply for this job, nor did I have any qualifications for it. I am a sculptor, not a doctor or a researcher. My daughter became sick with a mysterious fatigue illness and I was the obvious person to fill the job. Learning the job of a PA unfolds over time and there is no instruction manual. Certain ideas and thoughts can be transferred from former jobs and former lives, but much has to be learned from scratch. It is helpful in doing the PA job if you have a lot of time and a lot of money, as the solution to this disease takes a great deal of both. It would also be helpful to have an education in bio-chemistry, of which I have none. The most important qualification that a Patient Advocate needs is persistence and discipline. A PA also needs to remain objective and detached, even under the most extreme conditions. Every Patient Advocate has a patient. My patient is my daughter. The objective of this particular Patient Advocate is to make his daughter better. How to set about it is another matter, and turns out to be a complex and sustained set of illusive problems. While most doctors look at a broad and confusing set of symptoms and try to attach treatments to an entire cohort of partially differentiated patients, the PA’s problem is slightly different. The Patient Advocate, by job definition, is obliged to help one person - in this case, his daughter - his patient. Thus the PA is looking at one narrow and confusing set of symptoms, which makes his problem slightly easier.
I am the patient advocate of my 40-year-old daughter. She is housebound in St. Paul MN with CFS/ME. This blog presents reports from several lectures or conferences. It also attempts to define, in my own way, the role of a Patient Advocate. The premise is simple. I make my observations in hopes that they might be beneficial to others, in the same spirit of generosity that so many others' comments have been useful to me. These entries are presented for information only purposes. In no way should they be taken as medical advice. I am not a doctor and I do not want to be one.
Dr. Joan Grobstein has testified four times at the CSFAC meetings in the last two years. What she has to say is very important and the government knows this. They are afraid of her and for good reason.
The text of the original written testimony follows:
Here is Dr. Grobstein's written testimony, which she read at the 2009 CFSAC. It is different than the online version. It is a well known detail that those presenting patient testimony have to submit an sanitized version. At the presentation they then say whatever is on their minds.
CFSAC Oral Testimony October 2009
"Hello.I’m Dr. Joan Grobstein.I’ve been a physician since 1977, last working at Children’s Hospital of Philadelphia Division of Neonatology.I’ve had Myalgic Encephalomyelitis/Chronic Fatigue Syndrome since 1999.I’m a doctor and a patient. I‘m going to talk about science and ME/CFS.
To be blunt, scientific research on ME/CFS is a mess.Given how little time I have, I’ll focus mainly on the worstoffender, the CDC.The CDC has underfunded and underinvestigated this disease since their initial involvement in the mid-‘80s.They’ve also failed to correctly define the disease.In 1994, they created the Fukuda definition, which is flawed but which has been used to define the CFS data set for fifteen years, resulting in a significant body of research.However, oddly, in 2005 the CDC redefined the data set.Perhaps they noticed research using the Fukuda definition seems to suggest physiologic explanations for ME/CFS symptoms.One wonders.In any case, using the new Reeves 2005 “empirical” definition, their estimate of the number of people withCFS in the United States suddenly jumped from one million to 4 million people. Basically, they created a new, unverified definition which defined a new, much larger data set, and they still used the name CFS for this very different data set.This is outrageous!This isn’t science--it’s a shell game.
Dr. Peter White was involved in a similar definitional misadventure in the ‘90s, also muddying the research waters.He helped develop the Oxford definition, which was actually a description of Idiopathic Chronic Fatigue, which is not CFS.Putting a prestigious name on a definition does not necessarily give it a useful meaning.
As Dr. Mikovits and her colleagues have shown so brilliantly in the past month, when researchers look at patients that meet the Fukuda & Canadian consensus criteria, they can quickly begin to discover potential mechanisms and possible treatments for this severe illness.
So how do we find our way out of this mess?We need to tidy up the literature, so we know when we’re talking about apples and when we’re talking about oranges.I suggest the following solution:Until we have a better name, call the cohort of patients who meet the Fukuda criteria:CFS-Fukuda;the Canadian Consensus cohort:CFS/ME;the Ramsey cohort:ME;the Oxford criteria cohort:Idiopathic Chronic Fatigue;and the Reeves definition cohort:Reeves’ disease.After removing CFS-Fukuda, CFS/ME and ME from the Reeves cohort, Reeves’ disease will probably consist of a group of people with Idiopathic Chronic Fatigue, various other undiagnosed conditions, and some, but not all, people with major depressive disorder.These people deserve study and treatment, but they do not have ME/CFS.
It is very important that any ME/CFS study published states in its abstract which group is being studied.A retrospective review of all previous CFS studies should be funded in order to determine what group of patients were actually studied.Research on Idiopathic Chronic Fatigue is not relevant to ME/CFS.
I suggest the following recommendations to Secretary Sebelius:
1.No taxpayer dollars should be wasted on ME/CFS research which uses the Reeves definition.All federally-funded research should use the Fukuda criteria & the Canadian Consensus Definition.
2.Abandon the CDC’s current proposed 5 year plan.Ensure that this Committee’s previous recommendation for a change in the CFS leadership at the CDC actually happens.The new leadership should propose a new 5 year plan which should then be reviewed by anunbiased panel.Meanwhile, make the taxpayer-funded data that the CDC has already collected available to all researchers to analyze.
3.If the XMRV connection to ME/CFS is confirmed, initiate a congressional inquiry into why Elaine DeFreitas’ research into retroviruses and ME/CFS was not pursued in the early ‘90s. Many people may have been harmed by this decision.
Finally,
4.Increase funding for ME/CFS research.Patients and doctors need more information.Designated funding for a collaborative trials network is imperative, as is the retrospective review previously discussed.
I could say much more, but my time is up.I have submitted written testimony.Thank you."
I want to consider Dr. Joan Grobstein once again - as what she says is so important. I don't pretend to be able to articulate issues as well as her - and I don't think there is a finer mind in analyzing the current situation with ME/CFS. Dr. Grobstein was a pediatric physician at CHOP in Philadelphia (forced out of work by ME/CFS), one of the finest hospitals in the world (I know firsthand.). Despite being sick for twelve years, she can still reach out in any situation and put her finger precisely on the pulse. It is unfortunate that she is so ill - as the good guys could use more of her insights and expertise. For the past few years Dr. Grobstein has struggled to make semi-annual trips to DC to testify before the CFSAC committee. (I tell her not to waste her time and energy. When was the last time that a committee - particularly a government committee - accomplished anything? The short answer is - never.) Dr. Grobstein persists in the face of this known reality, and here are several of her testimonies.
The video of her October 2010 testimony can be found here. On this HHS website Dr. Grobstein is identified as "speaker 2" - an Orwellian reduction of this very smart woman. This video is worth watching. Being a government website, it, of course, takes forever to work, if in fact it does work.
Here is a written statement of Dr. Grobstein's 2009 presentation. At the moment I cannot find the video although I am still looking for it.
ME/CFS is presented to the world, especially by the press, as an astounding set of confusions. The issues are not as difficult as they are made out to be. If one is seeking clarity, one can start here - with the Canadian Consensus Criteria. This 2005 CCC criteria gives an unambiguous basis by which ME/CFS patients can be identified. Who can't see this - and why?
The Patient Advocate listened to the public testimony at the CFSAC, many of which were inspired presentations.These testimonies are available on the internet. The ME/CFS patients and advocates are articulate, passionate and focused - each in a different way. Their collective testimony is the very best and strongest part of these meetings.
The Patient Advocate would like to highlight one testimony and make a suggestion. Let's get rid of all the people on the CFSAC committee and put Dr. Joan Grobstein in charge. Let her pick the new members. Dr. Grobstein, 11 years ill with ME/CFS, can run things from her couch in her home. The HHS can hire as many assistants as necessary - and they can be trained to deal with the new ME/CFS committee head in a manner that is conducive to her illness.
Watch how much clarity and direction that Dr. Grobstein packs into the measly five minutes that they give her.
Dr. Grobstein is "Public comment - Speaker 2". Take the time to watch this. (Hopefully it will be posted soon on youtube for more easy access.)
The Patient Advocate also presents Dr. Grobstein's written 2010 testimony, which includes some important suggestions not included in her oral presentation. It is worth reading.
CFSAC Written Testimony October, 2010 Joan Grobstein, M.D.
I am a former neonatologist who had to retire from practice in 2000 due to illness. I have had ME/CFS since 1999. Before retiring I worked at the Neonatal Intensive Care Units of Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania.
At this Committee’s meeting a year ago, I spoke about science and MyalgicEncephalomyelitis/Chronic Fatigue Syndrome, or ME/CFS. Specifically, I addressed the negative impact of the CDC’s “empirical” definition of CFS on scientific research into the causes of and potential treatments for ME/CFS. A resolution was passed at that meeting stating that the “CFSAC rejects the empirical definition” of CFS. Despite this recommendation, the CDC has published 3 papers in the past year using the “empirical” definition, including a paper which was unable to find any evidence of XMRV in blood from a cohort of patients with “empirically” defined CFS or in healthy controls, and another paper which discussed personality features and personality disorders in the same group of patients. At the last CFSAC meeting six months ago, Dr. Unger stated that she stood by the CDC’s estimate of the number of people affected by CFS in the United States, a number based on the “empirical” definition. There is no evidence that the CDC has paid any attention to the expressed views of this Committee about the “empirical” definition. Communication with the leadership of the CFS program at the CDC about the definition and other issues has been next to impossible for other professionals involved with ME/CFS as well.
At the same CFSAC meeting six months ago, I confined my comments to the
Committee’s charter, at the Committee’s request. At that meeting, an amendment was made to the Function section of the charter to include the statement “the current state of knowledge about the epidemiology, etiology(s), biomarkers, and risk factors relating to Chronic Fatigue Syndrome and identifying potential opportunities in these areas”. The words etiology(s) and biomarkers are not in the current version of the charter on the Committee’s website.
It must be frustrating to members of the Committee to have their recommendations ignored so blatantly. Taxpayers are spending a reported $130,000 each year on this Committee. We do expect effectiveness. I’m sure the dedicated experts who take the time to serve on the Committee also expect that their recommendations will be implemented, especially the ones that are revenue-neutral.
These are big issues: (1) that the Center for Disease Control of the United States of America is continuing to use a discredited definition in publications about CFS, and (2) that the charter of this Committee ignores the importance of a search for the cause of a disease that affects one million Americans and their family members. But today I want to move on from discussions of science and bureaucracy and talk about medicine.
Medicine is an applied science. Doctors take scientific facts and apply them in real world situations in order to improve the lives of real people. Part of the reality of medical practice is that sometimes doctors don’t yet have all the information we need to make a fully informed decision. And yet we must act. For example, a patient arrives in the emergency room in shock and we may not initially know why. But we have to act. We draw tests to clarify the cause of the patient’s low blood pressure at the same time that we start IV infusions to treat the low blood pressure. We may give antibiotics for the most likely infections before we know what the infection is, or even if infection is the cause of the patient’s condition. We act.
At the present time, there is no action for patients with ME/CFS. We know, for example, that many patients have low blood pressure and orthostatic intolerance, yet there is no recommendation to treat the low blood pressure, or even to do tests to establish its cause, in the CDC’s CFS “toolkit” for professionals. We know many patients have chronic viral infections, yet there is no recommendation to treat those infections. The CDC “tool kit” for CFS does not recommend testing for any infections. We know that many patients have abnormalities of immune function yet there is no recommendation to treat those abnormalities. We know a lot about Canadian Consensus Criteria-defined ME/CFS, but we don’t apply that knowledge to treat the disease.
In the past year we have learned a lot about MLVs, including XMRV, which present an attractive, although as yet unproven, hypothesis about the underlying cause of ME/CFS: a newly identified family of retroviruses may affect the immune system causing patients to be susceptible to various new or re-activated viral infections, as well as other possible infections, and perhaps themselves are causing much of the diverse symptomatology of the disease. It is time to act. Despite the incompleteness of our knowledge, we can treat. We must treat.
We know from past clinical trials that some patients improve, if not recover completely, when treated with antivirals appropriate for the viral infections that they have as demonstrated by appropriate lab tests. These clinical trials include, but are not limited to: enteroviruses, as shown by Dr. Chia, various herpesviruses, as shown by Dr. Lerner, and HHV-6, as shown by Dr.Montoya. Other infectious agents that have been shown to be common in ME/CFS patients include chlamydia, mycoplasma, and parvovirus, among others. I propose a randomized controlled trial of XMRV positive patients in which patients are randomly assigned to two groups: half the patients receive treatments for the infections that they are shown to have, and the other half receive treatments for the infections that they are shown to have and, in addition, are given the three antiretrovirals that have been shown by Dr. Singh to be active against XMRV: raltegravir, zidovudine and tenofovir. The primary outcome measure should be Karnofsky score, i.e., patient functionality. This is what patients care about: what they are able to do in their daily lives. Appropriate measures of the other, non-retroviral, infections should also be followed as secondary outcome measures. There is no currently accepted measure of XMRV activity, but it is not necessary to follow viral counts or to have an accepted immunological marker to establish efficacy of anti-retroviral treatment. Improved patient functionality or a more rapid eradication of other infectious agents will establish efficacy. Raltegravir, zidovudine and tenofovir have been used in thousands of AIDS patients, and their safety profiles are well understood.
I’m sure some will argue that there should be a third group in this trial, a group that receives no treatment for the infections that they have. I personally think that it would be unethical to include a group that receives no treatment. The word Tuskegee comes to mind. Although, ME/CFS patients were not deliberately infected, unlike the men in the Tuskegee experiment, it is, in my opinion, unethical not to offer treatment to patients with known infections. However, I do realize that the current standard protocol for ME/CFS at this time is not to test for other infections, and, even if found, not to treat them. As a physician, this is unacceptable to me.
One could also argue that a third group should receive antiretrovirals alone. I am not opposed to this. I suspect all infections will need to be treated, but, since this is not documented, it is a reasonable question to research.
People with ME/CFS have been extremely patient with the medical and scientific community. However, our patience is not inexhaustible. Despite the cautionary mumbling of some physicians and scientists, people want to be treated for this serious, debilitating illness. We read the scientific papers on the internet. Despite our intermittent brain fog, we are not stupid. We will seek treatment. We will not wait for scientists to satisfy their intellects and establish who is “right”. If clinical trials are delayed too long it may be impossible to find untreated patients to enroll. At a certain point, it will also be impossible to claim therapeutic equipoise, because the mass of anecdotal evidence will be impossible to ignore.
Research cannot be done without funding, and funding for ME/CFS research at the NIH has been abysmally low. The NIH website gives an estimate of 5 million dollars, or about 5 dollars per patient, for 2010. ME/CFS received no ARRA funding for 2010. It is time to establish designated funding levels for ME/CFS.
I have spent my life working primarily as a clinician, not a researcher. I am therefore not particularly familiar with different types of grants or the mechanism of organizing randomized controlled trials with NIH funding. I have neither the time or the energy to get that information now, and it is not my job to do so. A request for proposals should come from the NIH for the necessary projects. More research dollars are urgently needed for this significantly underfunded, serious illness. It is time to act.
Many thanks to all the people who serve on this Committee and support this Committee. It is my hope that the future will soon be much brighter for people with ME/CFS.
References:
http://www.cdc.gov/cfs/toolkit/diagnosis.html
http://www.cdc.gov/cfs/toolkit/treatment.html
Switzer WM, Jia H, Hohn O, Zheng H, Tang S, Shankar A, Bannert N, Simmons G, Hendry RM, Falkenberg VR, Reeves WC, Heneine W., Absence of evidence of xenotropic murine leukemia virus-related virus infection in persons with chronic fatigue syndrome and healthy controls in the United States., Retrovirology. 2010 Jul 1;7:57.
Maloney EM, Boneva RS, Lin JM, Reeves WC, Chronic fatigue syndrome is associated with metabolic syndrome: results from a case-control study in Georgia. Metabolism. 2010 Sep;59(9):1351-7. Epub 2010 Jan 27.
Nater UM, Jones JF, Lin JM, Maloney E, Reeves WC, Heim C, Personality features and personality disorders in chronic fatigue syndrome: a population-based study, Psychother Psychosom. 2010;79(5):312-8. Epub 2010 Jul 28.
John K. Chia, Andrew Y. Chia, Ribavirin and Interferon-a for the Treatment of Patients with Chronic Fatigue Syndrome Associated with Persistent Coxsackievirus B Infection: A Preliminary Observation, The Journal of Applied Research, Vol. 4, No. 2, 2004
A Martin Lerner, Safedin Beqaj, James T Fitzgerald, Ken Gill, Carol Gill, James Edington, Subset-directed antiviral treatment of 142 herpesvirus patients with chronic fatigue syndrome, Virus Adaptation and Treatment 2010:2 47–57
Singh IR, Gorzynski JE, Drobysheva D, Bassit L, Schinazi RF., Raltegravir is a potentinhibitor of XMRV, a virus implicated in prostate cancer and chronic fatigue syndrome., PLoS One. 2010 Apr 1;5(4):e9948 http://report.nih.gov/rcdc/categories/
I have looked further and carefully for the video testimony of Dr. Grobstein of the October 2009 CFSAC meeting. It is no longer online. Many of the other testimonies and committee discussions are still available. (leelaplay supplies this: found it! http://www.hhs.gov/advcomcfs/meetings/index.html Look under CFSAC Oct 29-30 Day 1. I think you need Real Player (and a free down comes up if you don't have it) Dr. Grobstein is the 5th speaker at 3h 35 min. Thanks leelaplay!)
On a related matter - sitting near Dr. Grobstein at the CFSAC October 2010 meeting - I could not help noticing that Wanda Jones would not recognize Dr. Grobstein for a question.
Here is Dr. Grobstein's written testimony, which she read at the 2009 CFSAC. It is different than the online version. It is a well known detail that those presenting patient testimony have to submit an sanitized version. At the presentation they then say whatever is on their minds.
CFSAC Oral Testimony October 2009
"Hello.I’m Dr. Joan Grobstein.I’ve been a physician since 1977, last working at Children’s Hospital of Philadelphia Division of Neonatology.I’ve had Myalgic Encephalomyelitis/Chronic Fatigue Syndrome since 1999.I’m a doctor and a patient. I‘m going to talk about science and ME/CFS.
To be blunt, scientific research on ME/CFS is a mess.Given how little time I have, I’ll focus mainly on the worstoffender, the CDC.The CDC has underfunded and underinvestigated this disease since their initial involvement in the mid-‘80s.They’ve also failed to correctly define the disease.In 1994, they created the Fukuda definition, which is flawed but which has been used to define the CFS data set for fifteen years, resulting in a significant body of research.However, oddly, in 2005 the CDC redefined the data set.Perhaps they noticed research using the Fukuda definition seems to suggest physiologic explanations for ME/CFS symptoms.One wonders.In any case, using the new Reeves 2005 “empirical” definition, their estimate of the number of people withCFS in the United States suddenly jumped from one million to 4 million people. Basically, they created a new, unverified definition which defined a new, much larger data set, and they still used the name CFS for this very different data set.This is outrageous!This isn’t science--it’s a shell game.
Dr. Peter White was involved in a similar definitional misadventure in the ‘90s, also muddying the research waters.He helped develop the Oxford definition, which was actually a description of Idiopathic Chronic Fatigue, which is not CFS.Putting a prestigious name on a definition does not necessarily give it a useful meaning.
As Dr. Mikovits and her colleagues have shown so brilliantly in the past month, when researchers look at patients that meet the Fukuda & Canadian consensus criteria, they can quickly begin to discover potential mechanisms and possible treatments for this severe illness.
So how do we find our way out of this mess?We need to tidy up the literature, so we know when we’re talking about apples and when we’re talking about oranges.I suggest the following solution:Until we have a better name, call the cohort of patients who meet the Fukuda criteria:CFS-Fukuda;the Canadian Consensus cohort:CFS/ME;the Ramsey cohort:ME;the Oxford criteria cohort:Idiopathic Chronic Fatigue;and the Reeves definition cohort:Reeves’ disease.After removing CFS-Fukuda, CFS/ME and ME from the Reeves cohort, Reeves’ disease will probably consist of a group of people with Idiopathic Chronic Fatigue, various other undiagnosed conditions, and some, but not all, people with major depressive disorder.These people deserve study and treatment, but they do not have ME/CFS.
It is very important that any ME/CFS study published states in its abstract which group is being studied.A retrospective review of all previous CFS studies should be funded in order to determine what group of patients were actually studied.Research on Idiopathic Chronic Fatigue is not relevant to ME/CFS.
I suggest the following recommendations to Secretary Sebelius:
1.No taxpayer dollars should be wasted on ME/CFS research which uses the Reeves definition.All federally-funded research should use the Fukuda criteria & the Canadian Consensus Definition.
2.Abandon the CDC’s current proposed 5 year plan.Ensure that this Committee’s previous recommendation for a change in the CFS leadership at the CDC actually happens.The new leadership should propose a new 5 year plan which should then be reviewed by an unbiased panel.Meanwhile, make the taxpayer-funded data that the CDC has already collected available to all researchers to analyze.
3.If the XMRV connection to ME/CFS is confirmed, initiate a congressional inquiry into why Elaine DeFreitas’ research into retroviruses and ME/CFS was not pursued in the early ‘90s. Many people may have been harmed by this decision.
Finally,
4.Increase funding for ME/CFS research.Patients and doctors need more information.Designated funding for a collaborative trials network is imperative, as is the retrospective review previously discussed.
I could say much more, but my time is up.I have submitted written testimony.Thank you."
The CFSAC committee met again on October 3-4, 2012. Public testimony can be seen in the above youtube clip and an accompanying one. As usual the testimonies of patients and advocates were varied, heartfelt and stirring. Over the years these presentation have done a great deal to publicly give a face to ME/CFS and to elaborate, in the most personal and often harrowing terms, the toll that it takes on patients. Collectively these testimonies are the strongest evidence of the case against the government for their weak response to this medical emergency. This year there is some slight evidence that the CFSAC committee and the federal government itself, principally the FDA, is waking up from their long slumber and beginning to take ME/CFS seriously.
Many years ago I came to realize that the testimonies of Dr. Joan Grobstein are particularly articulate and insightful. Dr. Grobstein has the capacity to distill various problems down to their essential matter - and to cut out the crap. Dr. Grobstein has repeatedly clarified the central issues and constructed a path that will lead us out of the quagmire impeding research and treatment into this illness. It has always been my opinion that this woman should be given a position where she could "make things happen". Certainly her background as a physician gives her a unique and important perspective. When concerned people are gathered to design a plan of how to attack this illness, Dr. Grobstein's name should be first on the list.
Dr. Joan Grobstein's oral presentation at the October CFSAC meeting begins at 38:50 on the youtube video . Previous testimonies of Dr. Grobstein are available elsewhere on this blog or on youtube. Each of them is noteworthy.
In no way should this focus on Dr. Grobstein be seen as diminishing the myriad fine efforts of all the participants in the public testimony. Their testimonies do us all a very great service and I thank these individuals for the special time and effort that it takes to give testimony.
Two important items of Dr. Grobstein follow: The first is a transcript of Dr. Grobstein's oral statement delivered at the CFSAC meeting. The second is Dr. Grobsteins' five-page written submission.
"Hello. I’m Dr. Joan
Grobstein. I’m a physician.
My topic is responsibility.
Recently we’ve seen accomplished scientists honestly and
courageously admit that their original findings about XMRV were mistaken. They took responsibility for an
error. At its best, this is how
science works: form a hypothesis,
test the hypothesis, revise the hypothesis. We would like to see the same honesty and courage from
scientists at CDC about the empiric definition. The hypothesis that the empiric definition defines the same
disease as either Fukuda or the Canadian definitions has been tested and found
to be wrong. Scientists at CDC
must take responsibility for this error.
The original paper on XMRV in ME/CFS patients has been retracted. CDC
should retract all papers using the empiric definition or, at least, rename
them to indicate that they are studies of Idiopathic Chronic Fatigue and major
depression, not ME or CFS.
NIH must also take responsibility: fund studies that will clarify the definition controversy,
as it did with XMRV. Because we
saw money appear quickly to study XMRV, we know that funds are available. It’s clear that CDC’s new study about
the definition is unlikely to be impartial. They are vigorously defending their position. During a recent phone contact with
patients Dr. Unger said CDC could be “lumpers one day and splitters the
next”. This is a remarkably
unscientific statement. The
scientific method demands that we rigorously define the conditions of our
investigations. Without
understanding this, how can Dr. Unger be trusted to undo the harm to ME and CFS
patients that the definitional conflict has caused? Leaders at CDC must take responsibility and ensure that
science done at CDC is impeccable.
There are biomarkers for Canadian-defined ME/CFS
patients. Does CDC wish to conceal
that these biomarkers are not found in empiric-defined CFS patients?
Turning now to the responsibilities of the voting members of
CFSAC, its staff, and its ex officio members, we’re all aware that very few
recommendations made by this Committee have been implemented.
For example, CFSAC made three specific revenue-neutral
recommendations which have been
ignored. The first, from 2009,
condemned the use of the empiric definition. Despite this recommendation, CDC continues to publish
studies using the empiric. The
second recommended removing the Toolkit from the CDC website. The third recommended making
IACFS/ME’s Primer widely available.
These last two recommendation were made 4 months ago. Today, the Toolkit remains on the CDC
website. Meanwhile, CFSAC’s own
website has a new link to the Toolkit and no link to the Primer.
Who’s responsible for the lack of action? Committee members have fulfilled their
responsibilities. It says on the
website they “asked” that these three things be done. On the contrary, they are experts, and, as such, they made
recommendations to the Secretary.
Yet neither the ex officio members nor the staff have apparently worked
on implementation. Dr. Koh’s
comments gave no indication that he is aware of the importance of these
recommendations. In a letter to
advocates explaining his refusal to meet, Dr. Koh said, “CFSAC provides a
mechanism to ensure stakeholders are engaged and have the
opportunities to
offer input.” Instead, it appears that
CFSAC provides a mechanism to ensure stakeholders are contained and have
opportunities to offer input that will be ignored.So
what is the point of CFSAC? If it
cannot accomplish these small, high impact tasks how can it accomplish what we
really need: significantly
increased funding for research, better education and improved clinical
care? CFSAC costs $235,000
annually. That sum could fund a
study of natural killer cell function in Canadian-defined patients, or create a
mechanism to identify an undiagnosed low-income patient, or educate a few more
physicians. I’m a taxpayer. I’m concerned that we’re spending
almost a quarter of a million dollars each year on a committee whose
recommendations are routinely ignored by DHHS.
Here’s a suggestion.
Ask ex officio members to take responsibility. Ask staff members to assign each recommendation to the
agency or agencies with responsibility for implementing that
recommendation. Ask each ex
officio member to report on activities to implement each recommendation at
every meeting. If nothing is
happening ask for explanations from higher levels within the agencies.
We need to see progress.
[Please read my written testimony which contains other
suggestions for progress.]
If you fund it they will come.
Progress in our understanding of ME and CFS has been hampered by a lack of funding for good research. We have been told by the NIH that funding levels are low because very few high quality applications are received addressing these diseases. Yet we are told by researchers who have had repeated success in obtaining NIH funding for research in other areas that their applications for funding for projects concerning CFS have been repeatedly rejected. Funding for clinical trials has been rare. This has resulted in a vicious cycle: few people enter the field out of fear that they will not be able to obtain funding, and thus there is a smaller pool of high quality applicants and applications. It is time to break that cycle with dedicated funding for ME and CFS research.
Following the model that resulted in Requests for Applications issued on September 21, 2012, by the National Cancer Institute, I suggest that several Institutes at the NIH develop a list of provocative questions (PQs) about ME and CFS that then lead to Funding Opportunity Announcements, using RO1 and R21 mechanisms. The total funding available in the NCI announcements was $64-84 million. In addition, $21 million was made available for a Clinical Trials Network, something that is also desperately needed to further our knowledge of ME and CFS. The relevant institutes that should be involved in developing the PQs are: National Cancer Institute (increased incidence of early malignancy), National Eye Institute (multiple ophthalmologic problems), National Heart, Lung, and Blood Institute (orthostasis, post-exertional malaise and altered blood volume), National Human Genome Research Institute (increased incidence in family members), National Institute of Allergy and Infectious Diseases (multiple co-infections, possible infectious etiology, immunological abnormalities), National Institute of Arthritis and Musculoskeletal and Skin Diseases (joint and muscle involvement), National Institute of Child Health and Human Development (pediatric ME and CFS), National Institute on Deafness and Other Communication Disorders (hyperacusis), National Institute of Diabetes and Digestive and Kidney Disorders (associated irritable bowel syndrome), Nation Institute of Environmental Science (associated chemical sensitivity), National Institute of General Medical Science (basic processes of cellular energy allocation), National Institute of Mental Health (adjustment of patient, family and community to chronic disease), National Institute on Minority Health and Health Disparities (no access to diagnosis or treatment for minorities and low-income individuals), National Institute of Neurology Diseases and Stroke (cognitive dysfunction, sleep disorders, autonomic nervous system dysfunction, possible disorders of glymphatic system), National Institute of Nursing Research (need for nursing care for homebound and bedbound patients, quality of life in a chronic illness), National Center for Complementary and Alternative Medicine (patients forced to use alternative medicine because no mainstream treatments available), NIH Clinical Center (clinical studies of bedbound patients), and National Center for Advancing Translational Sciences (need for rapid implementation of bench science in these diseases).
Many of these divisions are already represented on the Trans-NIH ME/CFS Research Working Group. This group should be tasked with formulating the Provocative Questions with the input of both expert clinicians, who have been working with affected patients for years, and also expert patients. Because patients have been largely abandoned by the scientific and medical communities, many have developed considerable expertise in pathophysiology and available therapeutics. We know which Questions are the most Provocative, because we live with these questions every day.
The Working Group is currently reported to be meeting monthly. An accelerated schedule will be necessary to develop and prioritize the Provocative Questions, so that RFAs can be issued as soon as possible. A high priority should be given to studies of potential therapeutic agents, because there are no therapeutic agents currently approved for the treatment of ME or CFS. Similarly, there are no validated biomarkers to use for diagnosis or to follow responses to therapies. This is also a very high priority. Of note, expert clinicians are using therapies that they believe to be effective and also use biomarkers to evaluate those therapies.
Unlike cancer, which has been well-funded and extensively studied for decades, ME and CFS have largely been ignored. Although there are over 5000 published studies, most are small and many have not used rigorous criteria for inclusion of subjects. The required funding for this first initiative is therefore larger than in the cancer initiative described above. It is, of course, impossible to know how much funding is needed to advance our knowledge of ME and CFS to the point where patients’ lives will be measurably improved. However, it is possible to make an educated guess based on current NIH funding for Multiple Sclerosis, a disease which causes a similar level of disability and may well be a related or overlapping condition. MS is estimated to affect approximately 400,000 Americans and receives $121 million per year from NIH for research. ME and CFS are estimated to affect at least 1,000,000 Americans. It therefore seems reasonable to allocate $302 million annually to ME and CFS research.
A Barrier to Productive Research Besides Funding
The biggest problem for ME and CFS research has been a somewhat esoteric one: there have been multiple definitions of the disease, some of which have been overly broad, have not focussed on the most important and unique features of the diseases, and have been shown to include large numbers of patients with other disorders as their primary diagnosis. This has resulted in wildly varying results and has made it appear that there are no consistent biomarkers or responses to therapeutic agents. It has also given an erroneous picture of the diseases. For example, because large numbers of patients with major depressive disorder were included in cohorts defined by a definition developed by the CDC (the empiric definition) and probably also by another definition developed by physicians in the UK (the Oxford criteria), it has appeared that ME and CFS could be psychological disorders which may respond to cognitive behavioral therapy and a program of increasing exercise intensity (called graded exercise therapy or GET). However, even in this diluted population the benefits of these therapies are quite minimal. There is evidence that GET is actually harmful to patients with ME and CFS defined by more stringent criteria preferred by expert clinicians. It is therefore extremely important that research funded by the NIH use an appropriate definition during patient selection.
The International Association for Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (IACFS/ME), an organizations of clinical and research experts, has recommended using the Canadian Consensus Criteria (CCC) to define ME/CFS in its CFS/ME Primer for Clinical Practitioners. Patient groups also support this definition. Thus, it seems reasonable to use this definition in NIH research.
Most (but not all, as noted above) research in the past has used the Fukuda definition, developed at the CDC in 1994. The problem with Fukuda is that it does not insist on post-exertional malaise, which is probably the cardinal symptom of ME. It also does not give a higher priority to the presence of other very important features of ME: orthostasis (not even mentioned in Fukuda), cognitive dysfunction and sleep disorders. Until the issues in the definition are clarified, it will be necessary for every study funded by the NIH to also classify patients using Fukuda and, in addition to standard data analysis, to separately analyze the data for patients who meet Fukuda criteria and those who meet Canadian criteria (many will meet both). Thus, over time it will get clearer if there are distinctions between groups defined by these two criteria. It seems likely that CCC defines ME and Fukuda defines a less-specific group that has been called CFS. However, only consistent data analysis will provide this clarification.
There was recently an effort among researchers, including the CDC, to establish a minimal data set for ME and CFS research in order to begin to answer some of the questions about differences between definitions and also basic information about the diseases. However, I have recently heard that this effort has stalled, for unclear reasons. This is unfortunate. This could be one of the Provocative Questions: what data should be collected in every study in order to make patient cohorts uniform (or at least clarify when they are not) and enhance our understanding of these diseases? An NIH-funded review of the 5000 studies already done to see whether the definition used correlates in any way with the results would be very helpful.
The only study that is currently being done about the definition is a study by the CDC. This is problematic, because the CDC is the originator of both the Fukuda and the empiric definitions. They have continued to publish research using the empiric definition as recently as this year, although it has been condemned by CFSAC (see below), and most researchers now agree that it is overly broad and that research using the empiric definition is biased toward demonstrating psychopathology in CFS. Although it is possible that the next CDC definition will be an improvement, this seems unlikely. In a recent question-and-answer session with patients, the head of the CDC’s CFS program, Dr. Elizabeth Unger said that she thought the CDC could be “lumpers one day and splitters another” (lumpers referring to more inclusive definitions and splitters to more precise ones). She also said that she thought that heterogeneity has been the barrier [to moving forward in our understanding of ME and CFS]. It is unnerving that she thinks that heterogeneity is the barrier, and yet she is unwilling to improve consistency by promoting a splitter-definition over a lumper. She may feel more of an allegiance to past failed efforts of the CDC to appropriately define the diseases than she does to rapidly advancing research that will help to alleviate the suffering of patients.
It is obvious that more research must be done to specifically address the problems that the multiple definitions have created. NIH should be actively promoting this effort. A possible solution is an NIH-funded symposium whose task would be to address the definition problem and the resulting methodological challenges with the goal of creating standards for research within a year.
It is important that outside groups either validate or challenge CDC’s efforts. However, this should not delay other research, especially in the areas of biomarkers and therapeutics. Patients should not have to wait for therapies because researchers are squabbling over the small details of definitions. We have two serviceable definitions: Canadian and Fukuda. For now, the best solution is probably to recruit only patients who meet both sets of criteria, but it may also be possible to use a study design that incorporates patients who meet one and/or the other. It is also possible to improve Fukuda by requiring post-exertional malaise. In any case, it must be very clear what definition is used, and it will be important to analyze data collected in such a way that it can be leveraged to clarify the definition problem. It is vital that the definition used be stated in the abstract to the article, so that clinicians and researchers can rapidly ascertain the study’s relevance to their patient population. Continuation of the effort to delineate minimal data sets is also very important.
Yet another barrier: will
To members of the patient community it does not appear that HHS is really interested in promoting research or other solutions to the problems and challenges that American citizens with ME and CFS face. We have asked to meet with high level officials in the Department and have been told that our only avenue of communication with the Department is the Chronic Fatigue Syndrome Advisory Committee (CFSAC). Patients have attended meetings of the Committee with great difficulty and at great personal cost. The Committee has made some excellent recommendations to the Secretary, but these recommendations have mostly not been implemented. Most recently, three revenue-neutral recommendations have been ignored: a condemnation of the empiric definition (2009), a recommendation to remove the CDC’s clinical guidelines from their website (2012--see Mary Dimmock’s testimony about the Toolkit), and a recommendation to post the IACFS/ME Primer instead (2012). There have also been multiple recommendations to increase funding for research and for centers of excellence for research and clinical care. NIH research funding for CFS is currently $6 million per year. This level of funding is 220th out of 232 diseases. There is no funding for ME, i.e., no funding that requires using the Canadian definition. There are no centers of excellence.
No HHS official has given an explanation as to why the three important, revenue-neutral recommendations (empiric definition, toolkit and primer) have been ignored. To date, Dr. Koh’s comments have given no indication that he is aware of those recommendations or of their importance. At this point, many patients are asking, what is the point of this committee? If it cannot accomplish small tasks that probably only require a single bureaucrat’s signature how can it ever accomplish something more substantive like significantly increased funding for research, education and clinical care? What, then, is the purpose of this committee? CFSAC costs $234,532 annually. This money could fund a study of natural killer cell function in Canadian Consensus Criteria-defined ME/CFS patients and/or a study to determine appropriate dosing of rituximab for ME. As a taxpayer, I am extremely concerned that we are spending nearly a quarter of a million dollars each year on a committee whose recommendations are routinely ignored by HHS. We have begun discussions about this problem with our legislators.
We feel that CFSAC, as it is currently functioning, is not meeting our need for an urgent, coordinated and fully funded federal response to ME and CFS. Here is a link to the reply we received from Dr. Koh, Assistant Secretary for Health, when we asked to meet with him and key deputies to discuss our concerns: http://bit.ly/TnoClG . The full text of the letter can also be read in testimony submitted to CFSAC by Denise Lopez-Majano. Of note, in response to a request for patients to have direct input on the Ad Hoc workgroup Dr. Koh stated, “While the [Ad hoc] workgroup and its charge are inherently internal to HHS, some of the members are also ex officio members of the CFSAC where they hear the stakeholder perspective. CFSAC provides a mechanism to ensure stakeholders are engaged and have the opportunities to offer input.” I would suggest that CFSAC keeps stakeholders engaged in activity that consistently leads to no results and gives the opportunity to offer input that is consistently ignored. This has to change.
If CFSAC is to become a more effective avenue for cooperation between the patient, clinical and research communities and the federal government that produces substantive action to rapidly improve the lives of citizens with ME and CFS, we need to see some changes in how it functions. We need to be able to question the ex officio members and Dr. Koh directly. We need frequent updates on the activities of the Ad hoc workgroup (we have had none to date). We need to see the promised report from the NIH the State of the Knowledge conference 18 months ago. We need to discuss many other important issues. We need to see substantive recommendations implemented. We need to see progress. If CFSAC continues to be ineffective and its recommendations are ignored, it should be disbanded. A more productive process must be implemented. We need to meet with Dr. Koh and key deputies from HHS to discuss these issues.
At the beginning of the NIH State of the Knowledge Workshop, Dr. Mangan emphasized that the Workshop was “not designed to prioritize or establish an agenda for future initiatives.”But we need to do this.In the absence of leadership from the NIH, the CDC, this committee or any other agency in the Department of Health and Human Services, I’m going make suggestions that should be implemented within the next six months.
First, I'm going to present a case.
I have Pat Fero’s permission to talk about her and her son, Casey.Pat’s acute onset ME/CFS started in 1980 with a viral-like syndrome.Casey was born prematurely in 1982.He was first diagnosed with ME/CFS at age 9 and worsened at age 15.He died suddenly at age 23.An autopsy was done.The pathologist told Pat that Casey’s heart tissue was “loaded with viruses, inflammation and fibrosis”.The University of Wisconsin lost the heart tissue blocks.The viruses in Casey’s heart were never identified.
What does this case tell us about priorities and an agenda for future initiatives?
First, it tells us that it is very likely that ME/CFS is transmitted within families. Establishing the mode of transmission should be the highest priority.As far as I know, the CDC has never investigated family clusters.It also hasn’t investigated geographic clusters for more than a decade.ME/CFS should be a reportable disease, so that clusters can be identified.To make it possible for physicians to report the disease, they must have a precise case definition.The best definition available is the Canadian Consensus Definition.It should be adopted now and disseminated to all physicians. Until we know who has and doesn’t have the disease we cannot study it.Epidemiologic studies should start immediately.Special attention must be paid to identifying the sickest, housebound patients, many of whom aren’t receiving any medical care.This is a disgrace.
Second, Casey’s disease progressed over time and resulted in death.We don’t know the natural history of either untreated or treated ME/CFS, although many people with the disease are not only untreated but undiagnosed.The CDC should conduct observational, longitudinal studies of Canadian Consensus-defined ME/CFS.
Third, it’s very likely viruses are involved in ME/CFS, and myocardial infection is possible.Japanese researchers have identified “small heart syndrome” in ME/CFS patients, which may reflect cardiac infection.Of course, other organ systems, including the CNS and Gi tract, may be infected.Sites of infection may vary from person to person.It’s urgent that associated viruses be identified and treated, if possible.New antivirals may need to be developed.The NIH and the CDC should work together to identify viruses that are associated with or cause ME/CFS.This is a very high priority.
Fourth, medical schools don’t take ME/CFS seriously. The NIH should convene a meeting of medical school leaders to educate them about the seriousness of this disease.
Finally, Casey was not treated successfully.Treatments for ME/CFS exist.Expert ME/CFS clinicians are using antivirals, dietary supplements, sleep medications, and treatments for orthostasis among other therapies, and they are improving the lives of many patients.The NIH should convene a meeting of expert clinicians to formulate guidelines for diagnostic testing and treatment.
The CDC website is inaccurate and misleading.It needs updating immediately. For example, the CDC says that tilt-table testing for orthostatic hypotension is experimental.This is incorrect.The test has been used for 15 years at Johns Hopkins, and orthostasis is a frequent and treatable finding in ME/CFS patients. The website also says testing for viruses is not indicated, yet several clinicians are having success treating viral infections.The website should cite the Canadian Consensus Criteria as the correct definition for ME/CFS.Many people visit the CDC website for information about ME/CFS.It must be accurate.
A small book with the improbable title The Sound of a Wild Snail Eating was published in the late summer of 2010. It was written by an ME/CFS patient using the pen name Elisabeth Tova Bailey, who, by the nature of her illness, is forced to limit her contact with the outside world.
The Patient Advocate first saw this book in the hands of Dr. Joan Grobstein. As a result I have read it, and given it to various people for the Holidays. Every one of the recipients has expressed admiration for Bailey’s work.
A quote from Rainer Maria Rilke’s Letters to a Young Poet begins Part 1 and sets the tone for the book:
"Try to love the questions themselves as if they were locked rooms or books written in a foreign language. Don't search for the answers, which could not be given to you now, because you would not be able to live them. And the point is, to live everything. Live the questions now."
The Sound of a Wild Snail Eating is disarmingly simple but has a great reach. It is a book about observation and its implications - about gathering information from a small, seemingly insignificant source and looking for larger meaning.The work, while appearing to be the modest effort of a severely disabled individual, instead is comprised of powerful feelings and observations, proving that a story or poem or a piece of music does not have to be large to be emotionally profound.
The author of The Sound of a Wild Snail Eating was given a small woodland snail by a friend. At first mystified as to what possible purpose this gift could have, the bedridden author became curious about this other living being who was now a resident of her isolated world. Knowing nothing whatever of the nature of snails, she began, through observation, a "relationship" with the snail. The result is both an investigation of the world of snails and of chronic illness. The author cites various literary sources on snails as well as relying heavily on obscure nineteenth century scientific books on gastropods (stomach feet). She delves into dusty volumes, recording the habits of snails - their eating habits, locomotion, amorousness, strength, disguises and defenses, reproduction, and many other aspects of snail life.
Throughout the book the author's curiosity and thoughtfulness construct larger meanings from the simplest of events - the wonders of a snail in a terrarium. This book is part biography, part memory-play, part journal, part observational record, and part disease description as Bailey interweaves observations of her own physical state with gastropod lore. For a period of months the author describes, with self-deprecating modesty and humor, her snail observations and discoveries. Many of these cloak larger profundities, as the author draws parallels between the tiny habitat of the snail and the larger world. We learn all sorts of particulars about snails and their habits, as well as terms that we have never heard of: schneke, gastropods, radula, dextral, sinistral, pedal mucus, foot drinking, estivation, slime plates. Along the way the reader learns all one needs to know about slime.
While this book is ostensibly about a snail (or snails in general), it also includes finely expressed feelings describing the strange debilitating nature of this nasty illness, ME/CFS. To those unfamiliar with ME/CFS, these insertions of the disease reality might seem jolting, but to the initiated they will read as authentic and familiar . Alternating between elegant and humorous write ups of her observations and research, are the author’s personal revelations of the very nature of this dispiriting, restrictive illness and her methods for living with it.They are among the very best written. Bailey’s description of the process of receiving visitors for someone with ME/CFS, for example, is particularly moving and convincing.
Several other elements of The Sound of a Wild Snail Eating are worth noting:
This erudite book is laced with small, significant quotes from Rilke, Billy Collins, Patricia Highsmith (Strangers on a Train), Edgar Allen Poe, John Donne, Elizabeth Bishop, Emily Dickinson, Florence Nightingale and many others, thus revealing the author as a reader of great breadth and scope.
Bailey’s book is the product of a rigorous writing process: - paring down, compression, editing, and reduction make it a very powerful statement. At the end the author acknowledges the editorial help that she received. The result of this honing is a finely wrought, compressed and readable story line that contains just the essentials
On the back cover, in extremely small print, is this statement: "Author and publisher will donate a portion of the proceeds from the sale of this book to the Whittemore Peterson Institute and to a national conservation organization".
I highly recommend this book, and further recommend that the reader follow the advice of Thomas Mann of what to do when one finishes a good book: “Read it again.” The PA particularly recommends it as a gift for someone who knows little or nothing about ME/CFS, but is receptive and open to learning more. It is a splendid book.
It is always of interest to read about the ineptitudes of the functionaries who work for the United States Government. We must thank Dr. Joan Grobstein for her letter to Wanda Jones, outlining the abuse dished out to those ME patients who can actually move and attend a CFSAC. With these recent incongruities we are entering a zone of total absurdity and contempt. Something needs to change at the highest levels of the federal government in relation to this ME disease. They have gotten a free pass for too long and they take advantage of it with continued abuse toward these patients.
Consider for a moment the reality of this illness and of those who suffer at the very bottom. This is where the nuts and bolts of this illness presents itself and where research needs to be directed. Natalie Bouton has done a great service for the rest of us and for the uninitiated by giving us the InvestinME sponsored book "Lost Voices" which documents the most seriously ill ME patients. Soon we will be able to see an astonishingly powerful video produced by Natalie and her son Josh. It packs a wallop and presents this illness at its ground zero moment.
Another powerful articulate and sustained voice of the desperately ill has emerged recently and can be viewed here.
In rooting around for Dr. Joan Grobstein's 2009 CFSAC testimony, I came upon once again Laurel's powerful video testimony from October 2009 CFSAC meeting. Perhaps there are a few new or uninitiated persons reading my blog this weekend. This is for them.
Also please watch this video on ME/CFS made by Laurel, ill and housebound with CFS for 13 years. It gets the major points across in a powerful, concise and beautiful manner. Laurel is the very best! Learn from her. Both these films are on youtube.
