CFSAC November 8, 2011, 2 pm – 4 pm

>>Nancy Lee:
The first person by phone is Joan Militello [spelled phonetically]. Hope I pronounced
that correctly.>>Joan Militello:
Hello?>>Nancy Lee:
Ms. Militello?>>Joan Militello:
Yes, speaking.>>Nancy Lee:
Yes, hi, this is Dr. Nancy Lee with the — excuse me, Chronic Fatigue Syndrome Advisory Committee.
Are you prepared now to do your testimony?>>Joan Militello:
Yes, I’m just trying to find an empty room. Okay, yes I am prepared.>>Nancy Lee:
Thank you so much. You have five minutes and we’re — your testimony is being broadcast
to the whole room as well as to anybody that’s on the phone.>>Joan Militello:
Okay, super. All right, hello everyone. My name is Joan Militello. I’m the mother of
a bright and beautiful 17-year-old [inaudible] whose life has been greatly affected by the
lack of knowledge in both the medical and education field regarding ME/CFS, fibromyalgia.
Janine [spelled phonetically] became ill in October of 2007, after a bout with shingles.
The pediatrician at that time misdiagnosed her because she’d never seen a case of shingles.
It was a doctor at an urgent care who immediately diagnosed and started treatment. Her pediatrician
sent her to a specialist because they did not accept the diagnosis.>>Nancy Lee:
Ms. Militello, can you speak a little bit more directly into the speaker?>>Joan Militello:
Sure.>>Nancy Lee:
Of your phone?>>Joan Militello:
From — okay.>>Nancy Lee:
Our –>>Joan Militello:
Did you hear the first part?>>Nancy Lee:
That’s better, thank you.>>Joan Militello:
Okay. This is only the beginning of the nightmare with medical professionals who did not have
an open mind to look outside the box. Janine was not able to return to school until February
and it was short lived. The doctors insisted she had school phobia as did her school. They
pushed it to the point that all she could do was cry. This was [inaudible] turmoil [spelled
phonetically] in our house as we argued about forcing her to go to school or letting her
stay home. Friends were put [spelled phonetically] to interject their thoughts. Needless to say
we lost faith in our doctors and turned our phones off to unwanted advice. After watching
Janine be forced to attend school for three days with threat of failure it was — it became
evident to our family that there was something seriously wrong with her. We decided then
and there that she was not going back until she was well. I spent many nights surfing the Internet for
someone to help my daughter. I even looked to the Center of Disease Control. They wanted
to speak to her doctor, but her doctors refused. I still wonder whether they would have been
able to help her or not. One pediatrician actually stated Janine has her own agenda.
We never gave up and went to see many specialists who really weren’t so special. She was told
by a pediatric rheumatologist that was supposed to be one of the best that she had fibromyalgia,
but she could not stay home from school and must go. We followed his advice for a therapist
who took no insurance and then after 12 weeks and no improvement we stopped. Upon receiving
copies of his letters to our pediatrician it was no wonder no one wanted to go to her.
It was filled with inaccuracies as to her illness. I have over 10 pages documenting
each doctor we have seen and treatments we have tried and to go into detail about them
would take a good day. I’m willing to share our journey which I update after each visit
without adding personal thoughts because those thoughts wouldn’t always be very nice. What I want to share with you is the need
for educating the medical professional about ME/CFS fibromyalgia especially in young people.
Most doctors who deal with young children and adolescents have no idea what is going
on in these children and teens. The frustration is so bad that there were times I would worry
about my daughter’s mental health and her ability to keep having people say it was all
in her mind. We have gone full circle and are back to the first doctor who nodded and
agreed it was chronic fatigue fibromyalgia and understood her symptoms and our frustration.
I didn’t want to hear that there was no cure and that there was no surefire way to make
her better. We have spent endless amounts of time and money trying to cure our daughter
and we’ve come to realize that it’s only by working with this illness that she can keep
sane. Janine has so many different symptoms that
we continue to seek out specialists who can help improve her quality of life. We’ve met
a few who’ve helped, but for the most part we get that look of being completely insane.
I implore you to create a center of excellence or at least make the funding available to
teach doctors and educators alike about this [inaudible] and debilitating illness. Janine is my hero. She always does the best
she can and makes no excuses for what she can’t. She doesn’t need to hear people tell
her what she can and can’t do. She knows her limitations, but will not let them keep her
from being the best that she can be. Her goal is to go away to college because she did not
have the high school social experience and her friends all left her when she was not
able to go out with them. We do not discourage her, but tell her to try and we will regroup
if necessary. She does not need others to tell her what she can and cannot do. She will
decide what is best for her. She is stronger because of this journey. Thank you for your time and your effort to
help all those who suffer with this terrible illness. A special thanks to Dr. Levine [spelled
phonetically] and the parents and teen members of Speak Up About ME for their support and
encouragement to allow Janine and our family to continue to face the challenges that are
placed before us and I beg you, please, have the funding available to enlighten all those
who are in the dark [spelled phonetically] about this illness. Thank you.>>Nancy Lee:
Thank you very much.>>Joan Militello:
Okay. [applause]>>Nancy Lee:
Now we’re going to have another testimony by phone, Janet Smith [spelled phonetically].
Dr. Smith? Hello? Try again. Like I said, we have to pick our battles.>>Janet Smith:
This is Dr. Smith.>>Nancy Lee:
Yes Dr. Smith, this is Nancy Lee at the Chronic Fatigue Syndrome Advisory Committee. Are you
prepared to give your testimony?>>Janet Smith:
I am.>>Nancy Lee:
Good. You have five minutes, please begin.>>Janet Smith:
Hello, I am Dr. Janet Smith. I’m a [unintelligible] patient and a practicing physician and a participant
in the only clinical trial for ME/CFS, Ampligen. I would like to thank the CFSAC Committee
for allowing me to testify via telephone. My neurological practice started in 1987.
I became ill with ME/CFS February of 1994, after a viral infection turned into pneumonia.
I’ve been one of the fortunate people because I can still function in a modified manner
but only with aggressive treatment and with the experimental drug Ampligen which I have
been on for almost 10 years. However, the last 72 hours has proven that each day is
a challenge. I felt very well Saturday morning, then in the evening I started with aches,
headaches, chills, et cetera, and by Sunday morning could not even get out of bed to go
to the bathroom. I was in tears, unable to make a phone call for help, unable to sit
up because the muscle aches were so bad. Then I realized that this is what most people
with ME/CFS experience every day without hope for the future and the feeling that life can’t
go on like this. Suicides are occurring routinely in the ME/CFS patient group. We need hope,
we need help, and we need it now. Not in five years, not in 10 years, but now. I am asking
you as a committee to convey this to all that ME/CFS is real and we need answers, diagnostic
markers, and treatments now. Physicians who have treated both AIDS patients and ME/CFS
patients concur that ME/CFS patients are much sicker than their HIV patients. In the last two years the ME/CFS community
has undergone tremendous highs and lows. We need to carry what energy that we now have
to say that we need help. There is new research for diagnosis and treatments that are occurring
in the world. Norway physicians are using a chemotherapy drug with good results. Researchers
of Bond University, Australia are studying natural killer [spelled phonetically] cells
in ME/CFS leading the way to a central immune abnormality. This is an international disease that needs
to be recognized and researched. There needs to be an ICD-10 classification for ME. There
needs to be a better way to keep track of biospecimens from people with ME/CFS. A grant
to the IACFS or the CFIDS Association could be made for an informational exchange of biospecimens
with contact information, what kind of specimens, et cetera. New nonprofit organizations are
aggressively asking for private contributions to fund research with many new ones in just
[inaudible] year. But the lack of funding in this country for research is painful. As
a result, the real advances are being made in Norway, where the clinical trials are happening
and in Australia, where experts are studying natural killer cells, the hallmark sign of
ME/CFS. There is also concern that the original pioneers like Dr. Peterson [spelled phonetically],
Dr. Klimas [spelled phonetically], [unintelligible], Dr. Crabernaugh [spelled phonetically], Dr.
Lapp [spelled phonetically], Dr. Bateman [spelled phonetically], etcetera, are all getting older
and there are not younger doctors to expand the field and fill their shoes.>>Nancy Lee:
Doctor, you have one more minute.>>Janet Smith:
Therefore regional centers of excellence which have been requested from this panel for years
continue to go likely ignored by this administration. These centers could accurately evaluate and
diagnose patients and return them to their local physicians for treatment. Fellowships
need to be funded. The old guard [spelled phonetically] of physicians need to be utilized
to pass on their unwritten knowledge to the next generation. My hope for the future is
that medical textbooks will have a whole section on ME/CFS with ideology diagnosis and treatments
and that all physicians are aware of ME/CFS and how to treat it. Thank you.>>Nancy Lee:
Thank you. [applause]>>Nancy Lee:
Now I’m going to call Dr. Kenneth Friedman [spelled phonetically].>>Kenneth Friedman:
Good afternoon.>>Nancy Lee:
Yes, Dr. Friedman?>>Kenneth Friedman:
Yes.>>Nancy Lee:
This is Nancy Lee at the Chronic Fatigue Syndrome Advisory Committee. Are you ready for your
–>>Kenneth Friedman:
Yes I am.>>Nancy Lee:
— testimony? And you’re going to have to speak into your phone directly to make sure
we can hear you. Excuse me, you have five minutes.>>Kenneth Friedman:
All right. Thank you. I wish to inform the Chronic Fatigue Syndrome Advisory Committee
and other stakeholders in the ME/CFS community of an opportunity to potentially restore ME/CFS
research education and related scholarly activities to one of this country’s most populated states,
New Jersey. The governor of New Jersey has formed a UMDNJ Advisory Committee to advise
him as to the future of the University of Medicine and Dentistry of New Jersey. UMDNJ,
the largest freestanding academic healthcare university in the United States could remain
intact or could be dismantled with its components given to other state institutions. The impetus
for an UMDNJ Advisory Committee may be the corroded image of UMDNJ caused by its purposeful
$35 million double billing of Medicare. However, of concern to the ME/CFS community
is the February 2010, university decision to ban ME/CFS research education and related
scholarly activity. According to that policy: one, Scholarly activity related to ME/CFS
can only be performed outside of regular normal business hours. Two, the University’s portal
to the Internet cannot be used for any ME/CFS related research. And three, the university’s
email client server cannot be used to correspond with anyone about anything related to ME/CFS.
UMDNJ controls all three of the state’s medical schools as well as the state’s only dental
school, School of Nursing, School of Health Related Professions, School of Public Health,
and Graduate School of Biomedical Sciences. Its ban impacts patient care in the Greater
New York Metropolitan Area as well as research and health care provider education and training
throughout the United States. If the ban persists, other institutions may
institute similar policies. UMDNJ’s actions are clearly an attack on academic freedom
which is the right of college and university faculty to pursue their academic interests
wherever they may lead and should be opposed on that basis. However, of particular concern
to the ME/CFS community is the failure of UMDNJ to honor the CDC’s ME/CFS policy as
articulated by its director in 2006, Dr. Julie Gerberding who stated, “We are committed to
improving the awareness that this, in brackets, ME/CFS is a real illness and that people need
real medical care and they deserve the best possible help that we can provide.” Why does the Department of Health and Human
Services continue to provide funding to UMDNJ when UMDNJ has decided that ME/CFS activity
is not a professional activity permitted of its faculty? Why does the Department of Health
and Human Services continue to give money to a university that knowingly and deliberately
violates this CDC mandate? The UMDNJ Advisory Committee has received employee testimony
expressing the belief that the university should be retained in its current configuration.
Stakeholders of the ME/CFS community may have a different opinion since dismantling UMDNJ
would remove the ban and restore ME/CFS activities to New Jersey’s health care centers.>>Nancy Lee:
You have one minute, sir.>>Kenneth Friedman:
Thank you. The CFSAC may wish to make a recommendation based upon the facts conveyed herein and its
own further investigation. Comments may be submitted via email to [email protected]
The window of opportunity for submitting comments is not specified. I would not wait long. Thank
you for your attention.>>Nancy Lee:
Thank you.>>Kenneth Friedman:
Thank you. Bye-bye.>>Nancy Lee:
Bye. [applause]>>Nancy Lee:
The next person to speak by phone is Gabby Klein [spelled phonetically]. Hello?>>Gabby Klein:
Hello.>>Nancy Lee:
Yes, is this Gabby Klein?>>Gabby Klein:
Yes.>>Nancy Lee:
Yes, this is Nancy Lee; I’m with the Chronic Fatigue Syndrome Advisory Committee. Are you
prepared to give your comments?>>Gabby Klein:
Yes, I am.>>Nancy Lee:
Thank you. You have five minutes. So that we can hear you well, please speak directly
into your phone.>>Gabby Klein:
Okay, thank you. Hi, my name is Gabby Klein. I’m a 56-year-old wife, mother and grandmother.
I’ve been suffering from the most severe, misunderstood, the ill-treated illness called
ME/CFS for the past nine years. I used to be an active member of society. I worked fulltime,
raised my children, took care of the household, and did charity work. All this came to a halt
in 2002, when I came down with a vicious virus that lasted for weeks. My health declined
to the point that I was forced to quit my job. My GP was clueless of what to do for me. His
medical training did not prepare him for this. All the other doctors I was referred to were
disbelieving. There were few tests that they were taking were coming back negative. They
thought I was exaggerating my complaints. Of course they couldn’t see my pain and suffering.
This is an invisible disease. At last, I was lucky to find a specialist who finally diagnosed
me with CFS. Unfortunately by then I was so ill with a heavy viral and inflammation [unintelligible]
that it was impossible to reverse the damage. Had my GP recognized the problem right away,
treatment might have helped me. At present, nine years after my diagnosis,
I’ve become mostly housebound. I feel like I have the flu all the time and like I just
hiked up a mountain for two days without sleep. I suffer from insomnia and constant severe
headaches. I can’t stand or walk for more than a few minutes. With this [unintelligible]
I suffer from cognitive problems which affect my memory and render [spelled phonetically]
me unable to think clearly or speak comprehensively. When I do try to do some minor activity, all
my symptoms become aggravated and I have to lie still in bed in a dark room without any
noise for a few days or weeks at a time. Can you imagine having seven grandchildren
and you can’t play with them because of your sensory overload? The noise causes sharp headaches.
This is my most painful reality. This is a dreaded disease that [unintelligible], an
AIDS researcher commented that CFS patients feels everyday significantly the same as an
AIDS patient feels two months before death. The name chronic fatigue syndrome coined by
the CDC is an insult and is denigrating. People tell me, “I am fatigued all the time too;
you should get out more and exercise.” Or, “You look fine to me.” [inaudible] suffering,
it is critical that this cruel name be changed if we want any advancement and improvement
for this illness. Because of the way CFS is currently coded,
it’s almost impossible to collect disability. [unintelligible] the National World Health
Organization has been coding this since 1969 as a neurological disease the new ICC agreed.
When will the U.S. catch up to the rest of the world? I belong to a CFS forum where someone
posed the question asking people, when do they believe that there would be treatment
for us? It’s very sad to read that the majority answered, “Not in my lifetime.” Our only hope
right now comes from the few doctors and scientists in the country who specialize and treat patients
with CFS. When private grants have to be given [spelled phonetically] to run sporadic studies,
for example I’m excited about the new ME/CFS Center at Mount Sinai in New York City, that
my specialist, Dr. Enlander [spelled phonetically], is part of.>>Nancy Lee:
You have one minute.>>Gabby Klein:
He received a matching grant for $1 million from one of his patients. These top specialists
are joining this research effort, Dr. Eric Schadt [spelled phonetically] and Dr. Ila
Singh [spelled phonetically] whose researched genomics [spelled phonetically] in diagnosis
of ME/CFS. [inaudible] treatment modality [spelled phonetically], Dr. David Bell [spelled
phonetically] has expressed interest in joining Dr. Enlander in this project [unintelligible]
treating patients. Can you imagine what they could accomplish if they would get government
funding? This illness drains the U.S. economy in the
amount of $27 billion annually, yet the NIH budget [unintelligible] year $69 [spelled
phonetically] for research this year. This makes no statistical sense at all if this
is what’s happening. The cost to the patients are running at the possible treatments [unintelligible],
insurance policies will cover very little if any of it, then patients become penniless
and homeless with no help from the government and health agencies.>>Nancy Lee:
Ms. Klein, I need you to finish.>>Gabby Klein:
Okay. [unintelligible] play fair, don’t hit people, say sorry when you hurt somebody.
I think it’s time for the U.S. health agencies to [unintelligible] neglect of this population
of very ill people, who can bring proper awareness, proper funding, and proper care. Thank you
for the opportunity to speak.>>Nancy Lee:
Thank you. [applause]>>Nancy Lee:
We will now have Martina Nickelson [spelled phonetically] in person. Is Ms. Nickelson
here? Okay, Robert Miller [spelled phonetically].>>Robert Miller:
Am I red? I’m red.>>Nancy Lee:
You’re red.>>Robert Miller:
Okay. Good afternoon, chairman and members of the committee. I’m Robert Miller. I’m here
today due to the drug Ampligen. Without it, I’m homebound. It’s not a cure for me, but
at least I’m not stuck home in bed. I must say there’s been a lot of changes for this
committee since the last meeting. Dr. Mangan’s [spelled phonetically] retired, Dr. Wanda
Jones [spelled phonetically] is gone, and half the committee members are new or about
to shift. There’s no live webcast for patients which should be a must, but we have to keep
from being distracted because more’s happened in ME/CFS since the last meeting. I’d like to thank the Blood Working Group
and other scientists for pursuing the truth about XMRV. The story may not be over yet,
but early on with it, I was hopeful that XMRV represented a real breakthrough. I also advocated
for scientists to pursue their research until we had the truth. I wanted a yes or no answer.
Lots of American science says it isn’t XMRV. So once again we know that ME/CFS or what
ME/CFS is not. Last week scientists in Norway published data showing that Rituxan, a B-cell
depleting drug used for lymphoma had dramatic results on a small group of ME/CFS patients.
It’s blown the lid off of science again because if these results hold up, a primary conclusion
is that ME/CFS is an autoimmune disease. I thank the Norwegians for doing their study,
but I want to point out that years ago an American CFS patient who developed lymphoma
had the same experience with Rituxan. I remember him telling me how he felt better having cancer
and getting treatments than he did with CFS. The higher incidents of lymphomas in ME/CFS
patients is not a coincidence, so I’m asking myself now, do I have to go to Norway to participate
in a promising drug trial? Why didn’t American scientists pursue the Rituxan experience years
ago in the U.S.? When will American scientists be funded properly to prove what ME/CFS is,
not just to prove what it is not? The answer is the NIH and CDC are neglecting
ME/CFS patients. NIH and CDC’s tiny budget for ME/CFS is going towards disproving XMRV.
Ten percent of NIH’s measly $6 million [spelled phonetically] a year budget is now going to
researchers at Stony Brook to test self-treatment models. Really? Conserve my energy and feel
better? I mean, really, again? I and other patients protested NIH and CDC’s neglect this
morning because we need real science to solve ME/CFS and we need clinical trials to learn
what works and what doesn’t. We don’t need GET or CBT. We’ve done that for 25 years and
it doesn’t work. We’ll need serious research, serious funding, serious clinical trials for
a serious immune based illness and if making us well is not reason enough to do it, then
do it so you can save $20 billion a year that it costs this economy. I’d like to end with a video from President
Obama, who actually made a statement and said it best. We in the patient population call
this the Obama Promise. It’s up to you and me to use our brains to figure things out
and I believe that we can figure this out. President Obama. >>Courtney Miller:
Syndrome is an illness very much like multiple sclerosis and –>>Male Speaker:
[unintelligible]>>Courtney Miller:
Mr. President, my name is Courtney Miller and I want to thank you for returning science
to the national priority. And I need to ask for some help for my family. My husband has
chronic fatigue syndrome, which is an illness very much like multiple sclerosis. And we
spend billions of dollars in this country on roughly a million patients for disability
and Medicare and lost tax revenue and lost productivity, and we spend less than $6 million
for NIH research on this illness. And I’m asking you for my husband and my kids, who
want their father to be able to go to their baseball games, if there’s a way to make improvements
on that?>>Barack Obama:
Well, let me, first of all, say that you are absolutely right that we’ve tried to put science
back where it belongs. [applause] You know, I am a Christian and a person of
faith, and I believe that God gave us brains to figure things out — [applause] — and that, you know, we’ve got to use science
to make life better for our families and our communities and this planet. That’s one of
the reasons why part of the Recovery Act was reinvesting in the National Institute of Health,
NIH, which is — does a huge amount of the basic medical research that ends up then creating
so many of the scientific advances that are making our lives longer and making our lives
better. Now, I will confess to you that, although I’ve heard of chronic fatigue syndrome, I
don’t have expertise in it. But based on the story that you told me, what I promise I will
do when I get back is I will have the National Institute of Health explain to me what they’re
currently doing and start seeing if they can do more on this particular ailment. Okay? [applause] >>Robert Miller:
Thank you.>>Nancy Lee:
Thank you. [applause]>>Nancy Lee:
Is Gabby Klein back in the room? Oh, no, I’m sorry, yeah, Martina Nicholson. We did Gabby
Klein. Speak now, this is your chance. Okay. Jennifer Spotila is by phone.>>Jennifer Spotila:
This is Jennifer.>>Nancy Lee:
Yes, Jennifer, this is Nancy Lee at the CFSAC Meeting. Are you prepared to give your testimony?>>Jennifer Spotila:
I am, thank you.>>Nancy Lee:
You have five minutes and make sure you speak directly into your microphone. Don’t use a
speakerphone because we’ll hear you better that way.>>Jennifer Spotila:
Thank you.>>Nancy Lee:
Sure.>>Jennifer Spotila:
I ask the voting members of this committee, do you think this committee is effective?
I believe that all of you serve with the best interest of patients in mind, but let’s examine
the track record or lack thereof for your own recommendations to the secretary. Regional
centers for research and clinical care? No. Health care provider education and training?
No. Use of the name ME/CFS across all agencies? No. Research funding commensurate with the
burden of this illness? No. National effort to arrive at a new consensus case definition?
No. No, no, no, no, no. No meaningful action. No meaningful funding. All your work, and what is there to show for
it? You should be angry or at least frustrated. Five years ago you recommended that NIH issue
a new RFA on CFS. It didn’t happen. Many patients expected new funding to be made available
after the State of the Knowledge Meeting. It hasn’t happened. You know there is an urgent
need for research funding and we need you to hold HHS accountable. Today I urge you to recommend that NIH issue
an RFA for CFS research backed by at least $10 million in funding and that this RFA be
issued in the next six months. This recommendation is specific, actionable, and measurable. Furthermore,
this money must be spent on CFS not related conditions and no more money should go to
CBT or other psychological approaches. We have begged NIH to fund research in pathophysiology,
objective diagnostics, and treatments. We have begged you to help us. I agree with Bob
Miller that for NIH to spend $600,000 on Dr. Fred Freidberg’s “commercially viable program
of illness self-management” is an insult to every CFS patient. At the State of the Knowledge Meeting, Dr.
Collins said that Secretary Sebelius had directed NIH to give CFS special attention. Today we
learned about a new HHS working group on CFS, but talking is not doing. I say we test this
alleged commitment to CFS. Make the recommendation that NIH issue a $10 million RFA for CFS research
in the next six months. At the next meeting of this committee, we can see if special attention
actually translates into meaningful action. I do not accept woe-is-me budget talk from
federal officials and neither should you. We know that money can be found for high priorities.
Dr. Fauci’s appropriation of $2 million for the Lipkin XMRV studies proved that. Money
is available, but the agency leaders are making conscious choices to spend the money elsewhere
on illnesses that are a higher priority. NIH’s fiscal year 2012 budget request is for
$32 billion. Can anyone seriously believe that there is no money in that budget to increase
research on CFS? Do you believe that this massive budget cannot be squeezed to find
a few extra million for CFS research? The current level of NIH funding for CFS research
is the equivalent of pocket lint. Do not dare to tell us that there is no money for CFS
research. The money is there, it’s just that NIH doesn’t think we’re worth it. While HHS
fiddles, our lives are burning. Physician education? We do that every time we go to
the doctor. Consensus case definition? Researchers and clinicians, not NIH and CDC, have participated
in devising case definitions based on true evidence.>>Nancy Lee:
You have one minute.>>Jennifer Spotila:
Thank you. CFS research? We fund that ourselves. Non-profits are funding pilot research that
only then can secure government funding. All these years we have done this work and we
will continue to do this work in cooperation with researchers and clinicians. But I say
to all of you, you are not off the hook. As long as we have to deal with CFS, you have
to deal with us. We are still here. Deal with it.>>Nancy Lee:
Thank you. [applause]>>Nancy Lee:
We have another person by phone, Melanie Pruitt [spelled phonetically]. Oh, wrong number.
Let me try this once more and then otherwise we need to see if we can find that number.
So — [off-mic discussion]>>Nancy Lee:
And in the meantime, Denise Lopez-Majano — are you, yes. Denise Lopez-Majano, Majano will
be reading testimony on behalf of her son, Alexander Lopez-Majano. Yes. Thank you. You
have five minutes.>>Denise Lopez-Majano:
Good afternoon. I’ve been asked about the biggest obstacles I face because of ME/CFS.
We all know about the paucity [spelled phonetically] of successful treatments. We all know about
endless problems finding knowledgeable medical professionals. Beyond that some of the biggest
obstacles are daily struggles for acceptance of the severity of ME/CFS that stems from
the erroneous perception that this is a psychological condition. ME/CFS is not a psychological condition.
It is a horribly disabling physical illness. I want more education. I want a profession. Since I can only leave the house two times
a week, since I can only study for 20 minutes at a time perhaps three times a week, it probably
will take a long time for me to obtain a college education. Unfortunately, it will probably
take a long time to then find a job. This should not be the case. There need to be successful
treatments for ME/CFS. There need to be many more medical professionals who know about
ME/CFS, respect patients, and who treat ME/CFS. We must move forward. You must define patients
uniformly. You must disseminate accurate information widely and often. Work hard. ME/CFS needs
to be solved soon. I want my life back and so does my brother. Thank you. [applause] [off-mic discussion]>>Nancy Lee:
Do you want to try dialing it for me since –>>Male Speaker:
Sure.>>Nancy Lee:
— I was not successful? Male Speaker:
Do I have to dial nine?>>Nancy Lee:
Yeah, dial nine and the number.>>Melanie Pruitt:
Hello?>>Nancy Lee:
Is this Melanie Pruitt?>>Melanie Pruitt:
Yes, it is.>>Nancy Lee:
Yes, this is Nancy Lee at the Chronic Fatigue Syndrome Advisory Committee. Are you ready
to give your statement?>>Melanie Pruitt:
Yes, I am.>>Nancy Lee:
Thank you. You have five minutes. Please make sure you speak directly into the phone and
don’t use a speakerphone because that way we’ll hear you better.>>Melanie Pruitt:
Okay, thank you.>>Nancy Lee:
[affirmative]>>Melanie Pruitt:
Hello, my name is Melanie. This December at Christmastime marks 10 years since I caught
the virus that led to this long road of illness that’s ME/CFS. My youngest child is 18, which
means he was — he’s lived more of his life with a very sick mother than not. I’ve been
a committed attendee to the CFSAC meetings, sitting up very early to listen and watch
online from my couch or bed here in the Pacific Northwest. First, I want to sincerely thank each and
every one of you for your continued interest and the heart you show for patients like me.
I have never added my voice to the public testimony before, but this time I’d like to
share with you two things that have become incredibly important to me. First, living
in a small town here in the Pacific Northwest means that I am very far away from any of
the great specialists that care for ME/CFS patients. Several years into my illness I
had to fly across the country to even get an official diagnosis. I have heard it said that if my doctor isn’t
familiar with ME/CFS or doesn’t acknowledge its existence that I should find another doctor
but that is not as easy as it sounds. No doctor’s office will tell me if they deal with ME/CFS
patients due to confidentiality rules. To meet the doctor I must go in for an initial
appointment, which I rarely have the energy for. I went for one such appointment and the
primary care physician that I had at the time immediately dropped me because I was quote,
seeing another PCP, unquote. It takes a long time to get to know a new
doctor and develop a relationship of trust. It takes a long time for her to get to know
me and trust me. Changing doctors over and over can give me a bad reputation. I have
also found that though a doctor may sound like they are willing to care for a patient
with ME/CFS, they may not actually know anything about it. I have yet to come across a PCP
who is willing and able to invest their time into fully understanding my illness. My first urgent request is that you continue
to address and pursue ways to educate primary care physicians on ME/CFS. I try hard to bring
straightforward, reliable information with me to my appointments without overwhelming
my doctor, but it would be so much better if there was one reliable place with current
up-to-date symptom and treatment information that I could direct my doctor to. PCPs today
need information to be efficient. We need an authoritative research resource for our
PCPs. Maybe it could be organized by symptom so it was quick and efficient. Then my PCP
wouldn’t have to learn everything about ME/CFS all at once, but to help me and learn piece
by piece. Even though there are no ME/CFS specialists in my area, I have come across
caring PCPs that would make use of such a resource. Currently the CDC website is completely
inadequate and as others have stated today, in fact it does us great harm. You all understand
the need for efficiency. You understand the need for information to be from a reliable
source. So I plead with you to remember this every day need of patients like me. Secondly, I’ve had the very great privilege
of traveling to be evaluated and treated by one of our top ME/CFS specialists. I am a
middleclass American, one that is lucky to have good health insurance coverage and yet
even with this high profile insurance company, I have spent eight months working with one
representative to get my visit with this specialist covered. Before I went, I was told by my company
that they would cover this visit as long as I had billing codes and told by the provider
that appropriate codes would be provided so I could get reimbursed and yet it’s taken
eight months for these codes to be coordinated. I’m still waiting on codes and items to be
processed. It seems that medical coverage comes down to billing codes and ME/CFS issues
and treatments are very difficult to fit into regular coding. Now I’m not trained in medical
billing codes. I don’t pretend to even understand all of this, but clearly if this very large
insurance company is struggling to coordinate codes with this wonderful provider, something
is wrong. I urge you to continue to address the issue
of insurance coding and ME/CFS. Aside from the expense and difficulty traveling with
ME/CFS, I find now that I may not be able to afford medical support from this specialist
for my ME/CFS. Even with one of the best insurance policies around, you can hardly imagine what
a hopeless feeling that is. Thank you for sharing my concerns today. Thank you very
much for caring.>>Nancy Lee:
Thank you. [applause]>>Nancy Lee:
After this discussion — yes, okay, go ahead.>>Male Speaker:
So we’re going to go in it’s — if Gailen, if you’d like to share your wisdom with us?>>Male Speaker:
When we start back [inaudible].>>Nancy Lee:
We decided that it was a little early for a break and that we’d — unless — because
we just had one for lunch and so we thought after this discussion, we could have a break.
Does that work for everybody? Yeah. Okay. [off-mic discussion]>>Nancy Lee:
And I know Gailen said he had some — a few slides.>>Male Speaker:
I didn’t want to give anybody a chance to escape Gailen. [laughter]>>Nancy Lee:
Especially his?>>Male Speaker:
Nor his. [laughter] [off-mic discussion]>>Nancy Lee:
Why don’t you stand over there and I can go advance the slides?>>Gailen Marshall:
Yeah, it doesn’t work. For some reason [inaudible]. Okay, we’re going to spend a little time talking
about the idea — strategies as it relates to future interdisciplinary research for ME/CFS
that’s going to require a variety of scientific disciplines. Nancy, if you go to the first slide. That’s
smaller than I thought, but I’m sorry about that. There are real challenges to interdisciplinary
translational research. I think most of the members of the committee may already appreciate
this, certainly those that do research. There are a few of the members that don’t and our
advocates and audience, many of them do not. So I’ve always felt that it’s always worthwhile
to have a little vocabulary lesson so people know at least what you’re talking about because
the first point of that slide is that the same scientific term can have different meanings
to different stakeholders in terms of how they interpret it. For example, those of us who take care of
patients, there’s a broad variety of use of the term psychosomatic. I suspect most of
our advocates, if I walked up to them and I said, “I think you have a psychosomatic
illness,” I would want to make sure I was out of arm’s length because the colloquial
use of that term in our society is that it’s all in your head and it’s not real. And I
think we’ve heard clear evidence with passionate folks in speaking and those of us that take
care of these patients are well aware that it’s not all in their head. Yet no one can
argue that there’s a single category of illness that does not affect one’s mind when one has
a physical illness any more than certain mental illnesses we know there are physical manifestations.
The mind and the body are inseparable. Having said that, unfortunately in our society,
anything that is real can be determined below the ears is worth treating, anything that
is above the ears is not worth treating or it’s not really important. That’s a tragedy
in our allopathic medical system and I agree with Nancy, on behalf of my profession I apologize.
It wasn’t always that way. A hundred years ago physicians would never dream of acting
like our profession acts to you all as patients. And there are those of us who believe that
that will change, not fast enough, but it will ultimately change. There are cultural and social influences,
and I’ve just described perhaps one of the major ones. There’s even clinical versus basic
influences. Trust me, working in an academic medical center, the basic science faculty
and the clinical faculty are at each other’s throat with some degree of regularity. And
they approach the same problem from a research standpoint from very different perspectives.
Personal professional bias perspective is true as well. The analogy I like is that old
Arabian proverb of the 10 blind men and the elephant. They could all feel, one could feel
of the tail, one could feel of the trunk, one could feel of the leg, and they all had
very different perspectives of trying to describe the elephant. The nice thing about that analogy
is that what they could have benefit from was an 11th person who could take the data
from the other 10 and synthesize an overall description which probably would have been
a very accurate description of what an elephant really is like. A natural conflict exists in the [unintelligible]
information between scientists and clinicians. Scientists are trained — and again just for
background, I was a basic scientist who went to medical school. And I went to medical school
with the intent of gaining a degree so I could go back to the lab and make more difference.
What I didn’t realize is they’d put a stethoscope in my life — in my hand and I fell in love
with taking care of patients as well. So I’m well aware of this dichotomy between the mindset
of how you’re trained as a scientist and how often times you’re trained as a clinician.
Scientists look for generalizable knowledge that may ultimately be applicable to people.
In contrast, clinicians care for the individual patient by balancing the components of the
illness that are generalizable, what we now call evidence-based medicine with our fundamental
responsibility to acknowledge and deal with the individual variability to make each one
of us unique human beings. So, truly translational research has to account
for both perspectives while remaining objective to the results derived from this study, in
other words the idea of following the data. The [unintelligible] understanding that no
single intervention works for everyone even with the same cause. If you don’t believe
that, remember that there are right now 67, six seven, different antibiotics that are
marketed to treat bacterial pneumonia because not every person responds exactly the same.
Now the difference is is that if they have bacterial pneumonia, they’re likely to try
to find an antibiotic, they’re not going to try to get them to do cognitive behavioral
therapy. So there’s a difference between the approach to it, but there may not be single
approach in the approach of acknowledging the underlying ideology of the illness. Next, next slide. There — many research efforts
are also discipline egocentric. The old idea that it’s not real if it doesn’t have the
P-value of less than .05 and in the other extreme of that is if it does have a P-value
of .05 or less, that makes it real. Neither of those statements are true. And then of
course using my methodology is the only correct way to look at this problem, is again how
we’re trained. It’s what we get used to and there are many different methodologies used
to address the same question. That’s a fundamental problem of science, particularly at the clinical
level, that many of us are dealing with in and outside of ME/CFS research. The reverse translational research approach
is basically the idea, and as was pointed out to me earlier today in conversations with
my colleagues, the classic translational research model is bench-to-bedside and back again.
You formulate a mechanism, you see if it might work, and then you come back and see how to
modify it from there. The reverse actually starts with the patient and it collects the
information from the patient. And it says, these are the problems that we’re seeing.
It’s almost by definition an epidemiology type approach and from that, you formulate
ideas that might have common mechanisms and common rationales for similar interventions.
Or at the very least by seeing the variability among the population that you would assess,
you can then try to account for the variability in response. Either of those would be in the
classic reverse translational — I won’t call it classic because it’s sort of a newer idea,
it shouldn’t be, but it’s sort of a newer idea of the reverse translational research
approach. And then finally approaches using biomarkers
themselves need very strong clinical correlates. Some are relatively clear. There are good
biomarkers for tumor burden. There are good biomarkers for renal function. When you go
on dialysis, you know that you’re kidneys are not working anymore. There’re good biomarkers
associated with survival and mortality. Unfortunately, many of the challenges with CFS research is
a lack of clear clinical correlates in terms of ideology or as I believe, plural ideologies,
variable symptom complexes, and then the idea of overlap syndrome. If you listen to most
people in — again of the folks that I care for and the ones that I’ve listened to others
that care for them as well, there are very few of them that say I just have seven of
the eight criteria and I have nothing else that goes wrong. People have other things that go wrong with
them. Some of them have to do with chemical sensitivity. Some have to do with things,
they’re labeled CFS fibromyalgia. Some are labeled because they have titers against Borrelia.
Some are given autoimmune ideologies. And what I think we have to understand as we approach
research is that are these truly overlap syndromes and that term — well actually comes from
rheumatologists talking about overlaps from different serological [spelled phonetically]
diagnoses, or are these modifiers, or are these co-factors? And I think addressing that
and understanding it is critically important as we address what we’re trying to do which
is to find biomarkers that have clinical correlates that can be measured in a large population
of patients. Next. So recommending the translational research
priorities basic science approach, what they do, or what we do I guess because I wear that
hat as well, is to identify the question, identify the population, identify the system
we’re going to use to study them, and then design the study with adequate controls with
goaling [spelled phonetically], again, is to only have one single variable. So if you
have for example, if we wanted to study ME/CFS fibromyalgia overlap and we wanted to study
the pain in ME/CFS compared to the pain in fibromyalgia, ideally what you would want
is to compare the study with the overlap, and this is my term it may not be the right
term for others, versus those who had pure “fibromyalgia” versus those who had pure ME/CFS.
Now the devil’s in the details with what that word pure means. Therein lies the problem.
That analysis plans [spelled phonetically] an ultimate conclusions and therefore applications
are highly dependent on how those things are done above, in interdisciplinary research
not multidisciplinary research. The 10 blind men and the elephant were the
multidisciplinary. The 11th person who could see and integrate what they had been told
by the others would be the true interdisciplinary researchers would be important to decide on
a definition of the illness. What is the essential criteria? And this would be independent of
having to come up with a new consensus diagnosis or consensus criteria. That’s a laudable goal.
That’s something that should be done, but it’s not something that we have to wait on
to do effective interdisciplinary research with ME/CFS patients, but particular if we’re
going to do an intervention, we’ve got to decide who we’re intervening for. What is
the characteristic of the population that we’re intervening for? Determining minimal data that are necessary
for comparative study, and Dr. Jason has led the group that I’ve been privileged to have
a little bit of part in that’s done an elegant job in writing a paper that discusses this
and will be discussed more tomorrow’s meeting. Careful constructed — careful construction
designed to optimize the answers to the research in question; very important to construct it
carefully and then strong consideration of the confounders and modifiers both psychological,
physical, pharmacological, et cetera. All of these are things that are just basic principles
to translational research. Next. And these are the different groups that
— and this is not meant to be an exhaustive list, but that’s a pretty big list all ready
of individuals that would be important for interdisciplinary research to be effective
in ME/CFS because each one of those both in terms of basic science disciplines on the
left, and clinical science disciplines on the right are individuals for which, even
at this meeting and again, those particular like Dr. Levine and Dr. Klimas who take care
of a much larger population than I, that they see these variations on a regular basis. So
getting input from these experts is extremely important for these interdisciplinary researchers.
So all this segues into the question for discussion. Next please, Nancy. Is this is a provocative
statement for discussion. Knock it down, it’s the straw man that we’ve put up here for discussion
and that is approaches to ME/CFS research should be focused on a reverse translational
model. And what I will do is nothing more than facilitate the discussion with your permission.
Just facilitate the discussion, and then maybe have a comment or two, and do we have until
4:00?>>Nancy Lee:
[inaudible] give an example>>Gailen Marshall:
For example — so — and Nancy suggests that I give an example, and I must give a very
clear one. Let’s use Rituximab [spelled phonetically] as an example because it’s such a neat one.
Here the — if you read the paper, the PLoS paper that was just published, the investigators
based upon the fact that they saw some observation that was mentioned was not a Norwegian observation,
was a in fact a repeat observation from people in this country that had been treated with
Rituximab in the past for a clinical indication, their symptoms improved. Now, there is a very interesting theoretical
reason to explain why, and Nancy and I’ve been going back and forth having a wonderful
science conversation about why you might expect Rituximab to work in certain patients, maybe
all patients, who knows, with ME/CFS because if these cells are a reservoir for a certain
types of latent viruses and you get rid of the reservoir, the viral titer is going to
diminish — the viral titer diminishes. The individual doesn’t have to respond to the
virus, the individual gets better. It’s very interesting logic, very testable hypothesis.
A classic reverse translational research. So that’s a good place to start and we’ll
just open it up for discussion. Dr. Jason, you look like you’d be ready to start. Go
ahead. Leonard Jason from DePaul University>>Leonard Jason:
Lenny Jason from DePaul University; and Gailen, you know, you kind of asked me to say something
before I raised my hand.>>Gailen Marshall:
I just saw the urge, Len.>>Leonard Jason:
But you’re right I was going to ask something. [laughter]>>Gailen Marshall:
Reverse translational question.>>Leonard Jason:
So, I guess the — you know, certainly you can have an intervention that affects multiple
illnesses and that could very well be the case with what the last example involved with
the variety of autoimmune illnesses. I guess the question that I have is, from all the
things that you’ve been talking about which is kind of selection of a group of individuals,
testing them, looking for potential biomarkers, developing treatment programs, isn’t there
some basics that you start with for example saying we’re dealing with lung cancer, or
we’re dealing with heart disease, or we’re dealing with AIDS or HIV, that in some ways
all those definitions have changed over time. HIV/AIDS, the criteria has changed over time.
So it’s not a question that criteria can’t change over time but that they do have a criteria.
And each of these fields has something that brings all the scientists together to say,
yes, this is it, this isn’t it, and that we basically at that point are able to kind of
build this pyramid, almost like a pyramid of cards, but it’s based on saying we think
these people have a particular illness or disease. And I’m wondering, isn’t that the
starting point for anything we do in basic research?>>Gailen Marshall:
No question about it. HIV is an interesting example, but I’ll go further back than that.
Go back to the example of real research in cancer. I grew up in Texas and the first research
job I actually had was at the University of Texas, MD Anderson Cancer Center, when I was
in high school, more years ago than I would like to admit. And in those days their understanding
of cancer was in many ways almost trivial by today’s comparison. If you looked at they
understood what cancer did, they thought of it as one disease. Cancer. And what we came to understand — and their
approach to therapy was really very uniform and over time with that single armamentarium
of interventions, which might have a surgical component, they recognized very early on they
couldn’t cut it all out though if you let — allow me a little license for a moment
and a pretty famous movie, John Wayne’s last movie called “The Shootist.” If you remember,
Wayne had what was most likely rectal cancer, colorectal cancer, and indeed if you go back
and read from near the turn of the century which was the timing of that movie, there
was a medical literature about how to treat it and it was purely surgical. They tried
to cut it out and they did it as well as they could and it always grew back and it always
killed the patient. Chemotherapy was a relatively later development.
Radiation therapy was an even later development, but it was still thought of this same disease
in different manifestations. Over time what we all have come to understand about cancer
is that while there’s a commonality of biological and clinical characteristics that define a
malignant cell and therefore a patient who has cancer, there are departments of every
organ system cancer in every major cancer centers and general oncologists now in private
practice tend to specialize in one group of cancers or another. There are breast cancer
oncologists in private practice, not just at MD Anderson, Dana-Farber. So that model is not an unrealistic model
to start in. The only objection to that, that I have early on, is that for example, Rituximab
because we started with that. That’s an extraordinarily expensive drug, number one. Number two, it
is not without side effects. Knocking out somebody’s B-cells in a drug that was designed
to get rid of malignant B-cells, is not trivial from an immunological standpoint. Number three,
it assumes certain things that may not be true, and that is for example the fact that
a B-cell is a reservoir for a virus does not mean that patient has an autoimmune disease.
I’m a T-cell biologist by training. There are T-cell mediated autoimmune diseases and
there are lots B-cell diseases that are not autoimmune in that they’re not making autoantibodies
that attack the body. So by looking at a criteria, and just even
from the standpoint of sorting them into those who have autoantibodies and those who don’t
in their serum, but they have a common clinical complex. And again that’s the fun part, get
20 clinicians in a room and let them argue about what is the common clinical presentation
of CFS, and I think you’ll get about 25 different answers. But once you come up and say all
right they have to be in this category, they have to be in this category, they have to
be in this category for this initial study, and again if you read the Norwegian study
they did that. They did that and they put them and they were common enough but — that
they put them in there but then you would divide them into people that have a biomarker
or the absence of it. They have autoantibody, they don’t have autoantibody. Would that be
a predictor of how they might respond to this? That is a reverse approach to translational
research that uses principles that are not new. These are principles that have been around
for 50 years, and — but it’s using them in a rational way where we’re finally moving
forward and methodically evaluating these. And as quickly and as rationally as we can,
begin to provide to the clinicians the idea of this is a group of patients that you would
be more likely to expect to respond to this compared to others. When they do clinical
trials in cancer trials, they don’t come up with a — they come up with a regimen they’ll
think that will work on the organ system that they’re interested in. In other words, breast
cancer versus prostate cancer versus brain cancer and they would not bring a prostate
cancer patient into a brain cancer protocol on the argument that that regimen would not
be expected to work, so they’re not going to submit them to that. We need to — with these powerful drugs we
need to get there as quickly as we can without systematically excluding someone else until
we know better. That early trial, they put colon cancer, they put brain cancer, they
put lung cancer more or less into the same group until they found out what was beginning
to work and what’s not. So it’s a balance between those two.>>Leonard Jason:
So Gailen, and I’ll let Nancy kind of jump in right after us. In a sense you’ve made
a great argument that there are many subtypes, even lung cancer, you’re not going to treat
all types of lung cancer the same way. There’s different types of lung cancer. So there’s
specialization that has occurred within cancer over the last 50, 60 years. But still they
basically can say we’re dealing with cancer and we are a situation where we’re going to
have subtypes too. There’re going to be individuals with ME/CFS that have immune or orthostatic
or other types of issues that possibly aren’t comparable. So there probably will be subtypes.
We think we all know people for example who never get a cold and some people get colds
all the time with this illness. So we know that there’s some clinical differentiations. So I guess the question I come back to is,
if we learn from history from some of these organ systems, from these other diseases,
haven’t most of those illnesses and diseases started with a paradigm involving people coming
together saying, “We are going to classify people this way”? It might not be right completely,
but at least we’re going to have an agreement on that so that we can compare samples across
laboratories and without that are we always going to be at a disadvantage to ever be able
to find uniform biomarkers?>>Gailen Marshall:
Well, I think you’re making the case for that statement and that is that you start with
the clinical complex and then you move to the biomarkers to help explain the clinical
complex because, candidly, that’s a really more cost effective way to do it and it has
the potential to begin to address the value of the interventions that we propose because
it forces us to think about what outcome marker do we want. I suspect again, if I could ask
each one of the folks out here who suffer with this illness, what would you rather have
me to show you I can do this therapy and it’ll improve my biomarker or it’ll make you better
able to function? They don’t care about the biomarker except as it helps them to be able
to function. And again, in our earlier conversation the
clinical research world is littered with a graveyard of biomarkers that people spent
billions on, well millions anyway, trying to show relevance because early studies showed
a statistical association and they didn’t get anywhere with it. So I’m simply suggesting
in this discussion is that rather than starting from some consensus diagnosis that people
that have ME/CFS meet this criteria is to go out and take the Susans and the Nancys
and the others who see these — and the Anns, I’m sorry — Ann, you’re here now so I’ll
— you’re in the group too — that see these patients all the time, people like me that
see them some, and Dane [spelled phonetically] and others who see them in different research
setting, and you yourself as you see them and get us in the same room and say, okay,
how would we categorize these people clinically? And then from that go back into the lab and
say, are the markers there, are they not there? Is there interventions that would be useful
and so on and so forth. That’s actually a very old school approach that I think is going
to be more likely to give us some ideas and get others outside of our community to take
these patients and this line of research more seriously. And Nancy had something she wanted
to say and then you’re next.>>Male Speaker:
Dane was actually waiting the longest, so.>>Dane Cook:
No. It was Steve, not me.>>Gailen Marshall:
Well, are you going to run this or am I? Just tell me. If you’re going to run it, that’s
fine.>>Male Speaker:
I’ll run it.>>Gailen Marshall:
Okay.>>Male Speaker:
[inaudible] Male Speaker:
And if you’re involved in the conversation, it’s difficult [inaudible].>>Gailen Marshall:
That’s fine.>>Male Speaker:
Go Steve.>>Steven Krafchick:
That’s really exactly what happened with fibromyalgia, because in 1987 or ’88 a group of x-people
[spelled phonetically] that were dealing with it got together and said, what’s the core
of what we have to accept to do research on this condition? And they did the study on
tender points and since then there is an explosion of research on that. And in chronic fatigue
syndrome, it’s always been for lack of a better term a kind of a mushy diagnosis with a lot
of disagreement and I’m sort of on Lenny’s bandwagon. At some point in time they’re — even
if you use a clinical approach to defining it, you need to define what we’re talking
about at some root level so that you can begin to identify your populations to study. To
get to your biomarkers or whatever else you want to study about it.>>Gailen Marshall:
Well, I’m on the same bandwagon. I’m simply saying we start with the clinical criteria
not with some consensus diagnosis definition no matter whose they are because not everyone
fits into there. Categorize the individuals on the ground in terms of what they’re dealing
with in their everyday lives. My guess is that if you ask clinicians that care for patients
on a regular basis with this illness, they’ll tell you they can sort of do some groupings
in their own mind of the kind of patients that they see. Start there and work backwards,
that’s the reverse translation model I had in mind.>>Steven Krafchick:
But why couldn’t you just start with the Canadian consensus and decide for better or for worse
we’re going to use that definition and then study the patients and see how it falls out.
And the problem is there hasn’t been any agreement as to whether you use the Fukuda criteria
or whatever to do the research and really push it forward. So it’s just a thought.>>Gailen Marshall:
I think — I understand what you’re going on. That would bite — that would be like
saying let’s study — let’s use a criteria where we’re going to study Fords versus Chevrolets
versus Chryslers. And if you talk about — and then we’re going to study trucks. Ford trucks
versus Chevrolet trucks versus Chrysler trucks. And then we’re going to study — we’re going
to do pickup trucks. The more specific you can get, the better off you’re going to be
as you begin to ask questions backwards about saying which one’s going to be able to pull
such and such a load better or not. Which subpopulation of patients are going to respond
to an intervention? If all you’re doing is saying, I’m going to categorize them based
upon what is fairly broad categories to start out with. Maybe this approach, this reverse
approach would work fine that way, so it leads back into a consensus diagnosis — or a consensus
criteria. Certainly the people that designed those didn’t design them in a vacuum; they
used clinical information to do it.>>Steven Krafchick:
Right, but they all sat around and talked about what they were seeing in their practices
and some saw widespread pain, and some felt they had tender points or trigger points.
They then did a study of the tender points because that seemed to be something everybody
was talking about. So which ones of the 30 or 40 of them on the body are we going to
use that give us a specificity and reliability to diagnose a significant number of people
versus anything else? They were able to identify a group that they later found their way into
clinical practice has produced another problem because they’re leaving people out of the
diagnosis whose tender points go away over time. But that’s a problem you can deal with.
What you really need to do is have a commonality of group and I just throughout the Canadian
consensus criteria because they exist, they’re accepted by a lot of people. You could say,
okay let’s start there. Let’s use those and let’s move forward with their research and
as the research develops maybe you refine them more, but it just — I’ve been listening
to this kind of discussion off and on for as long as you folks have. I mean, it’s been years and years and years
and the difference between the fibromyalgia community and the chronic fatigue community
is simply the agreement on a case definition and classification criteria and I think had
that happened in chronic fatigue syndrome at least the studying would be farther along.
My thoughts.>>Male Speaker:
Nancy.>>Nancy Lee:
So I’ve two different points and one I have to just mention. Talk about case definition
for a moment because we use them for such different reasons and it’s really important
that in all this conversation that you don’t lose sight that researchers frequently attempt
to narrow things down to the most unique homogenous group possible at the expense of maybe even
80 percent of the rest of the population if need be, to get at the guts or the core of
what you can get from that core group. And we have to be careful in case definitions
that we acknowledge that that was merely for research purposes because when we accept more
narrow definitions, including the ICC, we are throwing out vast numbers of patients
and it will have a very real impact on their ability to get disability and so on and so
forth because they no longer meet the case definition. So a research case definition, clinical case
definition, two different animals, be careful. The first HIV case definition was men 18 to
65 who were dying of a small series of infections and you know, now we know the iceberg thing.
You know, that it wasn’t that, it was — we needed to do that to get at the virus that
caused that illness but –>>Male Speaker:
But you could call it classification criteria.>>Nancy Lee:
— 25 percent of people of people with AIDS, dying of AIDS didn’t meet the case definition.
Twenty-five percent in the first years and I was there, I remember those things. So I
would be careful that we don’t tie ourselves into a brand new knot in all these case definition
debates. Be careful, be careful. But that’s not why I wanted to say something.
I wanted to talk about I love this concept of what you’re calling translation models.
Like it’s so confused by the changing language of science, you know. Because two years ago
that was N of one studies, right? So N of one meant that you saw something interesting
in somebody and it made you think, right? And it made you reframe and rethink and it’s
very important that we don’t lose sight of those. I think one of the best n of one people
I know is John Chia [spelled phonetically]. John Chia, I don’t know how he does that in
his clinical practice but he comes up with the most astute observations that he acts
on and within a year or two is changing the standard of care. It’s phenomenal and, you
know, we were talking about standards of care that are ephemeral in our group because we
don’t write them down and all act the same way, but when John gets up and says something,
everyone’s writing it down, you know, because the man has got some tremendous belief in
what he sees and an astute way of thinking. And I think every clinician in this area needs
to pay attention to that because we learn so much from a patient who got better or from
a patient who got worse on a therapy that was completely unexpected. It’s just tremendously
important and then we have very few ways to categorize this. The few journals that would
accept these kinds of observations, The Bulletin — The IACFS Bulletin certainly would and
it may not — it’s a peer review and it’s not, you know, got a giant whatever score,
but it — by gosh it’s a place to categorize these things and get them down. So if people
would be thinking about that in their clinical practices. A case study, a single [unintelligible]
case report, let’s call them case reports, but we should be doing case reports, more
and more case reports of interesting things.>>Male Speaker:
Okay, we have a hand up. Sorry, Susan.>>Susan Levine:
Hi. I wanted to agree with Nancy that I do agree that good clinical observations are
very much needed in this field and I think the one area I disagree with Gailen is that
chronic fatigue syndrome unlike some of the other illnesses that are listed you know,
HIV, lung cancer we’re talking about, they seem so restricted compared to the vast number
of areas that chronic fatigue, you know it involves the nervous system, the endocrine
system. How do we rein in all the specialists you were talking about in the so-called interdisciplinary
study? I mean, how do we get the geneticists involved, the endocrinologists involved? You
know, I’d like to sort of address that aspect of it.>>Gailen Marshall:
I can tell you as a clinical researcher the — what always gets them interested are biomarkers.
So the geneticists who won’t get involved in doing gene, you know, they won’t be doing
microarrays to start out with looking at their whole genome and see how a ME/CFS patient
regards to that but, you know, Susan, you and I both know to clinicians what the genetic
link means. It’s called family history and we all know there are too many cases where
siblings get illnesses, parents get ill, and then the children get ill. Now could it be infectious? Sure, but the
fact is is that — unless if they’re different people with genetic background, similar genetic
background in the family, some are getting sick and some are not, that begs the question
of the genetic link and when you talk to a molecular geneticist or a molecular epidemiologist
about them, it usually gets them interested. Same argument with endocrinologists, the postural
hypertension subcomponent that they’ve talked about as the potential adrenal component;
well, when you invoke the adrenal gland, then you’ve got to invoke the pituitary gland then
you’ve got to invoke the hypothalamus, and you know this as well as I do. There’s your
endocrinologist. I think you do that by beginning to — as they begin to say there’s a potential
input for that. Does everyone need Florinef [spelled phonetically]? No. But are there
some who might benefit from Florinef? Possibly. The ones that you’d be most likely to want
to figure that out seems to me would be you’d start out with the ones who do have postural
hypertension versus the ones that don’t. I’m just being naīve about that. But as you design those ideas what you’re
doing is you’re engaging the different groups around. You don’t go and pull them all in
and say, “We’re all going to get in the same room and we’re going to talk about it.” You
start with candidly those who would do the primary care of these patients. If you are
going to do an intervention you’d want to bring some expert in who had some expertise
in that intervention since there’s not yet anything been designed specifically for ME/CFS
patients. It’s going to be from the outside coming in and then you’d grow it from that.
That’s how I would see you would engage the interdisciplinary team. You would not arbitrarily
[spelled phonetically] go out and find 20 people and get them in the same room.>>Susan Levine:
I guess and I just want to make this one quick point. I think it’s clear from the last 20
years of doing this that I’ve had to be kind of a jack-of-all-trades so to speak in my
practice and I feel that the complexity of the disease warrants almost becoming a new
discipline such as, you know, rheumatology is its own discipline. Like there should be
for instance a chronic fatigue fellowship or that because of the, all the different
aspects of it that one person just has to be trained in all the different — you can’t
just be an infectious disease doctor anymore and understand chronic fatigue. You can’t
be a rheumatologist. I think that would — I think education in terms of medical education
has to make room for this as a whole new area because of the evolving complexity of it.>>Gailen Marshall:
I certainly agree with that and I think that speaks to the fact that what I think I hear
you saying is that ME/CFS is a primary care disease, it’s not a specialty disease yet.
And I think that may be true but I think there may be — there may come a time when subtypes
of ME/CFS patients will be cared for by subspecialists because they’re primary clinical problem.
I still think whatever the common mechanism is that explains the symptom complex and the
physical manifestations of ME/CFS, I don’t for a moment believe it’s a single pathway
to get there. I think there’re different pathways to get there and therefore like many other
illnesses we know, you treat the underlying problem and then the central mechanism begins
to resolve. The problem is right now we don’t really have
a clue as to what the central — we’re beginning to get an idea of the central mechanism, we
want to have a clue of what the underlying pathways are in individual patients yet. That’s
still — therefore that’s why in my mind it’s very much a primary care illness, and you’re
right. In the context of primary care, internal medicine, pediatrics, family medicine, ME/CFS
should be a — there should be a rotation to ME/CFS clinics. By the way let me put a
plug into this, that there’s centers of excellence, there’s going to be a more natural way for
that to occur.>>Male Speaker:
Deborah.>>Deborah Willis-Fillinger:
So I’m listening to the conversation. I love it by the way, and I’m trying to map sort
of my mind, sort of draw some of the things that you’re talking about doing and I don’t
see any dissonance between, Steve — I don’t see any dissonance between your different
approaches with the case definition and I love what you said, Nancy, about being, you
know, the warning to not try to force ourselves into little boxes yet because I think over
time these things will kind of — they’ll play themselves out. You mean, if you start
Marshall’s, you know, Gailen’s approach and eventually you’ll meet someplace in the middle. I was just thinking about the idea of as you
allow — as you work on the definitions, the clinical definitions that you want providers
to focus on that help them think through and look for when they’re meeting — when they’re
working with their patients. It — I can’t help but — I keep thinking of the concept
of Bayesian, the Bayesian, when we were talking about Bayesian theories before where you’re
looking for the groupings. You know, you’re looking for the larger percentage of folks
who have a particular expression and then you sort of go, “Hmm, let’s go and see what’s
going on in that group that explains their particular expression.” And then you have
another group somewhere else, you know. And then you try certain therapies on them and
they group out again, you know and it helps you to focus really quickly on what work — on
the groups that are going to be most responsive to what, you know, to different therapies. So this fits a little bit with that kind of
— and I think it’s I mentioned before that there’s sort of Bayesian approach to researches
and even I believe the FDA it’s starting to bubble up, fix with this model and if you
could draw it, I think it’d probably end up being something that there may be some nibbles.>>Male Speaker:
Well I think we’re all — you know, researchers have this tendency to come up with their own
little paradigm and work from that. But I think the concept of this, and the question
would be for the — what would the value be? Going back to what Nancy said about being
careful about the research and clinical consensus. Everybody remember what the Fukuda criteria
were designed for. They were not clinical criteria; they were research criteria. And
they ended up being clinical criteria. So we have a unfortunate, very real example of
what can happen in that context. And I’ve talked to a lot of clinicians who express,
again, like everything else, medicine is composed of human beings. And there are bad apples
in everything that, in all endeavors. And there are people who, for whatever reason,
forgot the reason they went to medical school and the compassion there. But I think that
doesn’t describe the majority of clinicians. But what I think does describe, whenever they
hear ME/CSF, is total confusion. What is it? And what can I do about it? And it’s not that
they’re not interested in doing about it. They just feel helpless, just like everyone
else. So if you can put something in their hands that says, this is what — these group
of people are going to tend to respond in this way, these — Bayesian, just like you
said. So there’s a value right there. The second value is that, and I’ll let Theresa
speak for herself, but for her own agency, I guess that’s probably big too, but the idea
— yes, well congratulations. [laughter] But the idea, seriously, is that the FDA,
when we come to them and say, “We want to” — and we go with a sponsor, and we want to
form, we want to do an IND to look at the applicability of this agent, and it would
be a pharmaceutical agent most likely, or I guess a device. I can’t think of a device
example right now. But a pharmaceutical agent to treat an ME/CSF. They’re going to want
to know, okay, well what’s your endpoint? What are you going to use? Dane pointed out
in our earlier conversation that for fibromyalgia that they use three different measures. But
the three different measures were for the assessment of one clinical outcome, that being
pain, that they could show that that intervention was sufficiently better according to the criteria
that the FDA has set, that they got a clinical indication for that. And then when you extend that to the things
that Steve and others like him deal with, which are getting these things paid for, that’s
the process in our society that’s going to be necessary. And we can talk until the cows
come home about how it shouldn’t be that way. And I would be the first one to talk about
it. Or we can try to work within the system to get our patients some relief in a timeframe
that would be of value to them.>>Male Speaker:
Eileen.>>Eileen Holderman:
Yeah. Well, I think this is a fascinating discussion. But I think that we are already
there. We have the biomarkers. If you were talking about, that’s the way to draw a multidisciplinary,
interdisciplinary team to treat ME/CFS, we have the biomarkers. So what’s the obstacle?
No offense here, but it has been political. We’ve — from our government agencies, on
the websites, in the printed materials, they keep saying, “We don’t have biomarkers. We
don’t have biomarkers.” But we do. We have HP access [spelled phonetically], low natural
killer cells, RNAs. We have the biomarkers. I remember Nancy Klimas going on camera on
Kim Snyder’s film saying, “Biomarkers? We have biomarkers.” So what — we have those.
We just need our, I guess the health agencies and everyone, to acknowledge that we have
them, and to move forward. The science is here. Why can’t we do that?>>Male Speaker:
Well, as researchers, we have the bio — [applause] — as researchers we have the biomarkers.
As clinicians, we do not. Therein is the dichotomy. The dichotomy is that natural killer cells,
let’s use this as an example. Natural killer cells, we still are not absolutely sure what
the natural role of a natural killer cell is. There are very few patients that have
been born that are deficient in natural killer cells, which is always a — in humans that’s
how you figure out, somebody’s born without something, so you have an idea what they have.
In animal studies that they’ve done where they’ve done natural killer cell knockouts,
that is variable. We know, for example, that there are lots of things that are, there are
a lot of things that will affect natural killer cells that have nothing to do with the complex
of ME/CSF. And there are ME/CSF patients that don’t have abnormal natural killer cells.
And I’m beating up this, because I know this is a favorite of Nancy’s. [laughter] But I’m saying simply because it is, in someone
who is a super, super specialist like Nancy, following natural killer cells, in terms of
things that she does to patients, has a certain value, because she has an expertise. She works
with a lab that, first of all, the variability of quantitating natural killer cells from
lab to lab is pretty substantial. So she has, within the context of what she does. But for
the generalist out there to say an NK cell is a good biomarker for, let’s just say, disease
activity of an ME/CSF patient, on a population basis, a research basis, that’s true. On an
individual basis it’s not true yet. And that’s one of the emphasis that needs
to be done is to take those biomarkers that are candidates, just like the therapies that
are potential candidates, and pull them into the clinic and show their variabilities and
show their values for an individual patient. And I could go through a hundred different
lab tests where that’s where they needed to do. And we’re not there yet with most of the
biomarkers for regular, generalist care. It takes people that are highly skilled that
are able to do that. We’ve heard — we even heard in testimony today, there’s so few of
folks like that. And I don’t even put myself in that category at all. There’s so few people
like that, not everybody can get to them. There’ve got to be people that are out there,
general, internist, pediatricians, family medicine, emergency medicine people that can
do a biomarker, say this means I need to do this for this patient. That’s that reverse
translational approach. That’s the component of what I’m talking about that needs to be
done.>>Male Speaker:
Lenny, last question and we’ll take a break.>>Female Speaker:
[inaudible]>>Male Speaker:
Oh, I’m sorry. [off-mic discussion]>>Theresa Michele:
Just wanted to follow up with Dr. Marshall’s comments. And bravo. You said a number of
things that the FDA, and our group in particular, would resonate with, that biomarkers need
to be tied to feels, functions, and survives, which is how products are approved. I imagine
that many of you, I could tell you that biomarker X is now normal. Congratulations. And you
would say, “Okay, I still feel awful.” So that doesn’t help you any. So the biomarkers are wonderful to explore.
But eventually we need to get to a point where those biomarkers mean something in a clinical
trial. And certainly, this field is so early on that you have to look at a lot of things
in a clinical trial, because we have no idea what the right endpoint is. But eventually
we’ll get there. And just a reminder that whatever endpoint we do get to has to be tied
back, as Dr. Marshall said, to something that is clinically meaningful to the patient. Thank
you.>>Male Speaker:
Chris, can I go after Nancy, because –>>Male Speaker:
Okay, sure. Because he’s going to anyway. [laughter]>>Female Speaker:
He’s going to anyway. What you say about biomarkers if very real. I think where we are right now
is we know that patients are sick and they have poor function. And we can measure function.
Okay? And I don’t think it’s very hard. And I think that the Ampligen study’s been using
exercise tolerance. There’s many different instruments that measure function. We’re using
the actigraphs [spelled phonetically] and other kinds of things. But there are ways
to measure function. Now a biomarker in my mind’s eye, we’ve been
using biomarkers more to direct therapy, to better understand the mediators of illness
and attack the mediator. And I think that’s a phenomenal use of a biomarker. And then
it’s also a test of concept, because you can measure all of the parameters that affect
that mediator over the course of an intervention and see if your intervention was actually
effective on the system you were trying to intervene upon. And so that’s, I think that’s
a very exciting thing. But right now, I don’t think there’s anything to stop us from doing
clinical trials using functional markers. And we shouldn’t be sitting around on our
hands waiting for the magic biomarker, while we have very reasonable interventions to put
into play, so — [applause] But I wasn’t going to attack the NK cell,
which by the way reflects the side attacks [spelled phonetically] of T cell, which by
the way is one of Gailen’s favorite things. And by the way, that’s very measurable in
a reproducible fashion using a [unintelligible] assay. But okay, I didn’t say that. [laughter]>>Male Speaker:
Well, what she just said is absolutely true. But they don’t do those in hospital labs.
They do those in research labs. What they could do, and what they do do are two different
things.>>Male Speaker:
Can I just say, I had to give Nancy that chance to follow, because –>>Female Speaker:
[inaudible]>>Male Speaker:
— this has really been, I mean, I’ve been on this committee for four years. And truthfully,
I want to thank Gailen for just opening things up, giving us some time to think and to play
around with ideas. And we haven’t had enough of that. And I think this is exactly what
we need to have at each of our meetings, some time where we can just sort of think, and
sort of think out of the box. One thing I’d like to sort of throw a couple out of the
box ideas is that since this meeting has occurred, I’ve been tabulating how many times people
said CFS and CSF. [laughter] And I would just like to suggest that because
we can’t seem to pronounce the acronym right, why don’t we get rid of the acronym? [applause] But on a more serious note, we had a really
interesting thing about case studies, where one of our long-term 10-year follow-up study
with patients with ME indicated that someone gained enough weight so that they had ME initially
and then 10 years later they had a BMI that was no longer classified as ME. So it’s very
interesting situation, I think, that we’re talking about. Researchers can use certain
case definitions for our purposes. But we can have clinical case definitions that can
also be used too. There’s no reason that one obviates the use of the other. But the key thing that I wanted to kind of
sort of say is that there needs to be an outcome for these types of discussions, because I
think they’re so critically important. And I think, and I would like to disagree with
some of the clinical observations that people are making, I think it’s really statistical
methods and neural networks that are ultimately going to derive this conversation. And I think
there are methods that are being used in the sciences that we can bring into our field
that can help solve some of our problems: artificial intelligence being one of those
ways that can really help us think through this, that can have computer models really
help us in ways that we cannot. So I’m just going to throw out the last point.
And that is right now we have an Oxford criteria, a London criteria, a Fukuda criteria, Canadian
2003 criteria, an empiric variation of the Fukuda criteria, an Oxford criteria. Our science
is not going to be given legitimacy as long as we’re always using different criteria.
No one’s going to take us seriously. We have to deal with that issue at some point. [applause]>>Male Speaker:
So one thing that we could do is recommend that HHS convene a group of technical experts
in this area to come up with an agreed set of criteria for research and publishing.>>Female Speaker:
You’re the experts.>>Male Speaker:
I’m a lawyer [laughs].>>Female Speaker:
You guys are the experts.>>Male Speaker:
Well, there’s a paper that’s coming out. [off-mic discussion]>>Male Speaker:
That’s really the segue for Lenny’s discussion tomorrow. And that is is that I think that
that’s exactly what’s going to happen. And my personal belief is it is not going to happen
inside the government; it’s going to happen outside of it. And it’s going to come from
the scientists and the clinicians that will exert the pressure in the literature. If we
call this a evidence-based thing, I think if you give the evidence — so the HHS secretary
convenes that and we have a nice big argument, and they get a proceedings out of it, and
they’re no farther along five years from now than they are now. I’m with Nancy. Let’s get
together and come up with a set of criteria that are evidence-based. And this was purposefully
provocative. And I would think it would be appropriate to summarize is that this is not
going to be an either-or. It’s going to be a both-and. I think we need to have a clear component
of reverse translational research as we ask the questions, because that’s going to help
us formulate the questions. But I think we have to have a orderly, scientific way that
involves all of the things that you were mentioning, Lenny and others, to allow us to do it. This
is the whole idea of interventional research. And that is is that you’ve got to be able
to ask the right questions before you’ll ever get the right answers. And then you’ve got
to approach it with a methodology that calls in all the different things we can do. You
just set up and say, “All right.” You get a group of people together and say, “This
is how we’re going to define ME/CSF” — I’m still calling it that. I slobber so much you
can’t tell whether I’m saying it right or wrong. We get together and we say that. And
we go to the FDA, we ask to do a clinical trial. Or we go to the NIH and we get a grant
and examine something within the context of that. We publish those data with scholarly
literature. We follow the data. But we do that in a timely fashion that’s go a application
to it to help our patients as quickly and as rationally as we can. Remember the first dictum of medicine is:
above all, do no harm. We don’t want to hurt people. But we can use that to sit around
and do nothing for years. But I think we can use it to go. And we can go forward; we don’t
have to wait for an agreement in criteria. And I appreciate you listing those, because
I was trying to do that a while ago and I missed two of them, of how many different
criteria that are out that each have their own advocate for, each saying this is the
correct criteria. And that most of us are kind of scratching our head and say, “Which
one do we really want to use?”>>Male Speaker:
That’s exactly what went on with fibromyalgia in the ’80s before they did that study.>>Male Speaker:
And I think, Steve, I think your point of bringing fibromyalgia to the forefront as
a model for us to understand is really a very, very, very important thing to do, because
there’s no reason to reinvent the wheel if we don’t have to do that.>>Male Speaker:
They just reinvented that fibromyalgia wheel, by the way. [laughter]>>Male Speaker:
No, I mean the original wheel, not the current wheel.>>Male Speaker:
No, those are proposed. Proposed, proposed.>>Female Speaker:
Just one more. I know we’re supposed to be calling it, but just if you could –>>Male Speaker:
I can cancel break if you want to continue.>>Female Speaker:
There’s another advantage to what you’re talking about with the reverse translational research
is the rapidity with which you’re able to refine your hypothesis. And so as you draw
that model, including those loops, I think, will be really powerful. And I’m just saying
you draw it. I mean, you don’t have to draw it for me. But when I think, once again, about
things like the Innovation Center and some of the things they’re interested in, it might
be good to include that.>>Male Speaker:
You’re on.>>Female Speaker:
Can I just add a quick point to that? Is one thing that we haven’t talked a lot, or at
least when I’ve been here, is about the HHS’s initiative around electronic health records
and how transformative that could really potentially be in a lot of arenas. I know the Institute
of Medicine has been doing a lot work on the role that that can play in just really advancing
comparative effect of research and really taking it to a whole other level. Because
all of a sudden there will be this vast depository of data, potentially, that can be mined in
a way that we’ve just never had before. And that that can really feed into all types of
research. So I just wanted to point that out as well as another piece of the puzzle.>>Female Speaker:
Go ahead. No, you go.>>Male Speaker:
I don’t want to [unintelligible] our break. But I just wanted to tag onto what you were
saying that, going back to the beginning of the discussion, I don’t know how easy it is
to get experts to agree on a single definition. Again, I hate to bring that up, but I know
a little bit about the activities of the map that’s going on in chronic [unintelligible].
And I think bringing together a group of experts had actually taken four years to agree on
a definition, which is not there yet. But maybe using the electronic data and record,
as you were mentioning. You can probably start with the list of certain things that certain
people agree on and then maybe construct a certain set of criteria that you can look
at. And then maybe look at a greater number of symptoms or signs and try to see how the
dielectric cluster around, based on what Lenny was saying. And maybe just see the patterns.
And just going again to the Bayesian networks, maybe you can just look at different subsets.
But probably have like at least a set that you can start off with, which are clinically
based. And maybe then look at biomarkers in those same subsets. I think, going back to the Rituximab example
that you gave at the beginning. I remember back in the days when most of the tyrosine
kinase inhibitors came around for lung cancer. I think that there was a lot of excitement,
so there was a mechanism that was found finally. And then when the drug was actually tried,
it worked only for 10 percent of people with lung cancer. So then if you actually go back
and test all the people with lung cancer for that specific mutation and only treat the
people with the mutation, then you actually get 90 to 100 percent effectiveness. I think
that’s what we kind of need to do. We probably need to start off with a clinical definition,
then look for biomarkers which can probably substratify patients, and then base the treatment
based on those biomarkers.>>Male Speaker:
But I think what that does into the design idea is that now you don’t have to look for
60, 70, 80 percent response.>>Male Speaker:
Sure.>>Male Speaker:
A 30 percent response, compared to a placebo response of five percent, should be plenty
to get you — that should be enough of a signal to get you interested and say this could be
a good therapy for the subgroup. The natural thing, again, because the community has felt
so disenfranchised, appropriately so I would agree, is that they want to play homerun derby.
They wanted to hit it over the fence. And then the skeptics say, “Well, this only worked
in 30 percent of the individuals, so it’s not useful.” I think we have to stand up and
say, “That’s crazy.” Because if it works in 30 percent of the individuals, that’s 30 percent
compared to a third of that or a fifth of that in the placebo group. There’s a signal
there. We need to pursue that signal. And that’s something that this committee, I think,
can have some impact on back to the secretary as a recommendation.>>Male Speaker:
Okay. But that speaks to large sample sizes, which typically we don’t have in CFS –>>Male Speaker:
Well, if there’s 1.7 million active patients and maybe as many as four times more than
that that are not diagnosed, I think that’s enough to study if you get them together.>>Male Speaker:
Get them together [unintelligible]. Ten minutes break. Thank you.>>Male Speaker:
Thank you.

9 thoughts on “CFSAC November 8, 2011, 2 pm – 4 pm

  1. Patient Mom testimony: "We went to many specialists who weren't too special." Haha. all too true and typical for ME patients.

  2. excellent patient testimony, including by Jennifer Spotila, CAA director. Thank you, Jennifer! I agree 100% with everything you said… except, you validly lambast CFSAC for not ensuring HHS comply with its recommendations; you specified the recommendation that all govt agencies use the term "ME/CFS." How can you be mad at that if you and CAA only call the disease "CFS"? Why has CAA said that it will only start using anything but "CFS" after CDC does (even though NIH already uses "ME/CFS")?

  3. Why doesn't CAA make basic free and low-cost changes that will help us and have been requested over a year ago by a number of patients such as all of the following (except re Rituxan), How about calling the disease ME? How about requiring patients in the biobank to have ICC and CCC ME? What about making it clear that while XMRV proper is dead, HGRVs may have just been born (pending further published evidence)? Why doesn't CAA work to make changes on the content of it's website info?

  4. The following isn't low cost, but crucial: why not provide or raise emergency funding for a Rituximab trial? Why are the salaries of the execs still stratospheric while their performance is not?

    CAA has made marginal improvement, but we need you to speed up these important and/or low-cost measures!

  5. "CSF", "chronic fatigue", Ugh! Come on people! You are some of the top experts and you can't even get the name right? Get it together. Just call it ME; I don't think you can mess that one up.


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