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FDA Workshop on Drug Development for CFS and ME

Sasha

Fine, thank you
Messages
17,863
Location
UK
Yes she spoke. Something about ME/CFS like MS and Lupus all have immune issues. Also, Rituximab has 30% response.

I couldn't remember a word she said til I read what you wrote! :eek:

She made the point that MS, Alzheimers etc. don't have a known cause like ME doesn't but that doesn't stop heavy-duty drugs approved by the FDA for other conditions being approved for them (without further trials? some sort of fast-track/compassionate grounds thing?).

At least I think that was her - they're all jumbled up in my head now! It's a mess in my brain...
 

Sasha

Fine, thank you
Messages
17,863
Location
UK
Will the workshop be available for review?

I don't know. You mean on video?

They keep talking about 'the docket' - not too sure what that it but it sounds like a repository of everybody's documentation. I don't know if that will be public domain.

I hope Jennie Spotila (who is there and was on one of the panels) might summarise but I don't know if she's planning to. I'd like to see a transcript, especially of the last session.

Mindy Kitei made a statement - she wasn't on the list.
 

Ember

Senior Member
Messages
2,115
Has Mindy posted on Twitter? She wrote yesterday, “I'm going to the FDA tomorrow and Friday. If Internet connections and my Kindle cooperate, I'll try to post on Twitter in real or semi-real time. If not, I'll post when I return.”
 

JohnnyD

Senior Member
Messages
206
Hi All,

I managed to copy and paste the Public Testimony portion of the days (days end) from the translators box. These are two minute comments, among them Dr. Dan Peterson, Dr. Judy Mikovitz, Dr. Charles Lapp, Thomas Equels (vice chairman of Hemispherx), and a number of patients. I've corrected some of the transcriptions, but there are plenty of transcribing flubs still. I'll try to post it as a single message, but if it get truncated, I'll attach as a file. It starts with Anita Patton.

==========================================
>> Hello, thank you, a lot of cords over there. Thank you for
having this drug development meeting. I think it is incredibly
valuable and a pleasure to be here.

I would say so similar so many things about what so many patients
have talked about today, except that it naturally falls into
several different subsets like some people have sudden onset, slow
onset, some people have low n-k cells, high cytokines, those type
of things, doctors could get together, just an idea, get together
subset and really put their research, their science, their
experience with patients and over like some of the decades to
identify which people might most likely respond to much treatments
and my question about the pharmaceutical companies is how can we
draw them in if there is not a whole lot of representation here
today, how can we get that and how can we facilitate faster
treatments to help more people? To that ponent I would bet there
are a lot watching through webcast, they're certainly probably
hearing the input and some of them here.

>> The last thing of course I'm ampligen responder, 15 years,
long time on it and I'm disappointed that it wasn't approved and
hoping like is there any way to put some sort of another trial or
another type of investigation to say that the people who did
respond ydid they? How can we help hundreds or thousands of
people who could have a response if there was some sort of global
trial, another one that would be done.

>> Okay, thank you for the comments. The point of this is not to
respond, this is open public session, we're taking comments.

>> Thank you.

>> So next on our list, thank you, is Steven chillinski.
Courtney alexander. I'm sorry. I have a different list here.

>> Yes, it's been revised three times. Courtney, go ahead.

>> I'm not courtney, I'm standing in for courtney, Dr. Janet
Smith, I am on the board of timaron research. Dr. Daniel Peterson
foundation he foundd and what we're trying to do is scientifically
redefine CFS ME so we are strictly trying to do the science part,
but today what I would like to do is plead with the FDA and with
this panel to go along with the guidelines that just came out with
the alZEheimers with approving drugs, I would like to plead that
would go a long with looser rules for approving drugs for CFS ME,
no approved drugs, thank you. [ Applause ]

>> Thank you.

>> Okay. We'll try one more time, Steven chillinski. No?
Okay. So next we have Judy Mikovits. I know Judy is here.

>> Dr. Judy, my travel expenses have been covered by several
physicians and several patients. So I'm just going to read
because I want to make it quick, I can talk forever. We do not
know the causes of Multiple Sclerosis, parkinson disease, lupus or
ME CFS. All of these serious debilitating diseases have
abnormalities of the immune system and inflammation in common if
not central components. We made a handout and many of you have it
today, people who sponsored my travel. Of just a few of those
biological abnormalities, clinical lab tests, and the drugs with
potential for repurposing. The FDA has just approved (inaudible)
immune modulator antioxidant for treatment of Multiple Sclerosis.
The bright focus in drug discovery foundation just announced a
phase I clinical trial of (inaudible) an FDA approved cancer drug
which acts on RET noticed receptor like vitamin D and thyroid
receptors, both abnormalities on the test. No abnormality tested
in ME or CFS. In nor way, OVRNGologist just completed small
clinical trial of FDA approved cancer drug rituxam, with success
in 30% of patients. The logical next step to do in this trial and
with the ampligen responders and nonresponders is gene expression
and profiling and in fact immune profiling to determine the
difference between responders and nonresponders at the molecular
level. The FDA has demonstrated commitment by holding this
unprecedented meeting and we thank you. Drugs which are FDA
approved are generally safe in humans, of course everything has
risks. In serious diseases, without treatments, classification
FDA recently gave ME CFS the benefits far outway the risks. For
advocacy groups here today and those listening I encourage you to
fund these clinical trials with the drug companies, fund follow-up
studies, profiling responders and nonresponders to divide patients
that exhibit different levels of disease activity, prognostication
and possibly insight into patho physiology as foundations have
done. The technology and expertise exists. The absence of
cognitive agent should not leave this field floundering any
longer, it's a new era for ME CFS treatment. Thank you. [
Applause ]

>> Thank you, Judy. And I think we got the documents but it
would be good if you would submit those to the public docket so
that they are officially included in the record as part of your
presentation. Okay. Thank you. Next we have derek enlander.

>> Good afternoon. I'm from the Mount Sinai Medical School in
New York. About a year or so ago we actually received a million
dollar grant from one of my patients and the dean of the medical
school said, oh, that is very generous of you, let's actually form
an ME CFS center. And indeed we formed an ME CFS have at Sinai,
and our first project was to prove whether TET, was appropriate in
the disease. We actually have got undergoing the research in
post-exertional malaise and actually expect to look at 150
controls and 150 patients in this disease. We're actually going
to look at the usual array of cytokines and immune markers, but we
are also going to include genetic studies with a genet cyst Eric
Shaft, who seems to be actually well known amongst all genetic
people that I've spoken to. We've got actually a series of 39
pages of the JE nome study on our first patients. It is most
remarkable genetic study and we are now actually going to look at
whether in fact graded exercise therapy is correct method or
approach and we will actually prove or disprove the pace idea.
Thank you.

>> Thank you, Dr. Enlander. If there is additional information
related to a talk you want to submit to the docket, we would
welcome you to do that. Thank you. Next Gisela morales, I'm not
sure if I got that right, I tried to catch people whose name I
wasn't sure of before.

>> You are trying very well. Thank you for acknowledging that
because not everybody takes the time to ask the question. First
and foremost, thank you for putting this and make Thanksgiving
happen. Compassionate way today, I'm pleased and happy to be
here. I am Dr. Morales-Barreit, o, I am here today as caregiver
(inaudible) love dearly and have been sick for the last six years.
I'm not going to bother you with the symptoms, they are pretty
much what has been said all afternoon. He has suffered a lot and
we were blessed at some point in time to be -- to work and I heard
it on MPR the voice of Dr. Peterson. So he moved a year ago to
Incline Village and took him back a couple months ago, but he also
came back herself again in many, many ways. Being active server
of the symptoms that she has had has not been easy and watching a
person that move literally at the speed of light be limited for a
long time, to bed for days, months and years, really has been
demoraleizing and emotionally draining. (Inaudible) survivor and
when I had had cancer I had options for treatment. This
population has really (inaudible) not really approved by anybody
and guaranteed they are going to get cured. I'm here after almost
12 years and I know that if I get sick again God forbid, I can go
back and find treatments that have been approved. For me, the big
elephant in the room after everything that has been said is the
fact that we need pharmaceutical companies, FDA cannot do it
alone, and we need the pharmaceutical companies to come and play
the game. The only way I think as psychologist is to also invite
them with your approval, I think the FDA approval will open up the
door for the pharmaceuticals to say yes, we can. Yes, we can
help. So I saw the hand, but I really employ you to look into
this from that point of view. If you approve any drug with
whatever the name Ampligen will be nice, but if not, anything that
will support the patients and open the door for more research and
more pharmaceutical people coming into this. Thank you very much.
[ Applause ]

>> Thank you. And I skipped Mr. Thomas Equels. Close? Okay.

>> My apologys.

>> These don't work for up here and that one obviously not very
good at either.

>> Little tall for this one, too. First of all, thank everybody,
FDA staff and most of all the patients and clinicians that have
taken the time to come here today. I'm the executive vice
chairman of hemisphere biopharma and we have an experimental drug
that you have heard about, ampligen, I'd like to share a few words
about how hemispheric and ampligen got involved with CFS. We were
30 years ago asked by the FDA to get with I believe Dr. Peterson
and Dr. Lap with an unusual thing they had out there called the
Tahoe Flu. And to provide drug on experimental basis for a female
subject who had remarkable recovery. And I believe there were
about about 12 additional subjects that were approved that did
well. That was over 30 years ago and we're here today, and it's
been a long and difficult journey for small company such as ours,
but we're participating in this process because we know that there
are thousands of Americans that are disabled. We know from the
research that (inaudible) we believe we can contribute to that
process. And we responded when you asked us 30 years ago to come
to the table and work and we've toiled in this vineyard for many
years and we make our commitment that we will be 110 percent with
you and with you to make this happen. Now we know from the HIV
Aids epidemic that when there are no drugs the results are dire
and we believe that we're subsets can be identified where a drug
can be effective, whether ampligen or other drugs, where there is
unmet medical need there should be some form of expedited approval
applied. I'd like to mention --

>> You have 30 seconds.

>> Three articles that I think warrant a lot of study, causes of
death among patients with Chronic Fatigue Syndrome, which deals
with cardiac increases and shortening of mortality. Cardiac
toxicity in Chronic Fatigue Syndrome, which deals with effect of
you heard about 35, 40 different medications, all of which have
severe, some of which have severe side effects and the impact and
then a double blind placebo controlled randomized clinical trial
of tlr-3 agonist, that study has to do with the exercise issues
and how that may have an effect. Now we've expanded a COMBRAT
deal of time and money to get to where we are today and I just
want to say that we're prepared to enter into not a legal
partnership, but a real partnership with the FDA, with the
clinician and with the patients to bring relief for people who so
desperately need it. Thank you.

[ Applause ]

>> Thank you. And thank you for paying attention to the hand
signals. Mr. David Strayer.

>> Okay. Okay. Dr. Dan Peterson.

>> Thank you. I am here today representing myself as a caregiver
but of approximately 9000 patients over the last 30 years which
has given me -- this disease the frontlines, as they say O. Behalf
of the patients, their families and even their physicians, I
implore this esteemed gathering committee to not only device but
to execute therapeutic strategy which is much needed.

The Federal diagnostic criteria were established more than 25
years ago even though revised a few times. The CDC has identified
counted, surveyd and queried an estimated 1 million people in this
country suffering from the disorder. The attendance disability
and poor prognosis documented worldwide by families, physicians
and the patients. The estimated cost to our country 9 billion
dollars a year. Diagnostic marker would yield the company 250
million a year. A therapy probably in the billions of dollars,
yet 25 years later, we have no FDA approved drug for this
indication or therapy.

So the heterogenous nature of had disorder has been a problem and
I see as an invitation or a beg to study subsets in this disease
that have the same pathogenic mechanisms. Over this 25 year
period of therapeutic stagnation, there have been thousands of
pier reviewed articles published with respect to patho genesis,
but it's been difficult to connect the dots, I'll just mention a
few. Worldwide the number one immune logical marker is low MLK
function and abnormal by everyone who look at them. Speck scans
abnormal low stress testing is universal finding. Many clinicians
are already utilizing markers and end points in their own
practices as they treat patients to document efficacy or to teach
us more about the disease. Symptomatic therapy unfortunately I
think is useful in quality of life, but I have not seen it return
patients to full functional physical nor cognitive conditions. I
think targeted immune logical therapy has possibility to do that.
On behalf of clinicians worldwide, you may not know this, we've
already have established consortiums and collaborations worldwide
with multi-primary care clinics that are ready and willing to do
pilot projects, phase roam 1, II and III clinical trials and more
and I know we're all committed to doing this. So I implore this
committee to take some action to support the private sector, to
use the resources and Federal government, science, personnel,
computing capacity, to develop safe and effective therapies.
Thank you. [ Applause ]

>> Thank you. Mary silvey.

>> Eileen Holderman. James baraniuk. I'm sorry, Eileen? Okay.

>> Good afternoon, my name is Eileen, an independent advocate for
ME. Thanks to the FDA for hosting this conference and giving me
the opportunity to speak. I'd like to address a concern and that
is the concern of the name of the conference which doesn't
distinguish between myalgic encephalomyelitis and Chronic Fatigue
Syndrome. There is one million American men, women and children
suffering with ME, 17 million worldwide and unfortunately in 1988
the CDC renamed it Chronic Fatigue Syndrome, which is unscientific
and dismissive and it was further compounded by definitions that
included empire cal definition which just states one symptom
fatigue to have it and today you heard multitudes of symptoms that
really describe this disease. The results of the faulty
definition have caused muddied research, the inability to
replicate findings, no YOUN versal biomarker, drug development
without target audience, erroneous medical information and
websites, bad media and press, skewed public perception and most
importantly neglect and harm inflicted on patients who truly have
ME. The disease cost the nation billions in lost productivity,
tax revenue and medical benefits. Funding for this disease has
been low and it needs to be commiserate with the disease burden.
The solution is that all the government agencies, the scientific,
medical, academic, legal advocates and patient communities must
come together and adopt the Canadian consensus criteria and to
dismantle use of CFS and move research and treatment forward to
help the over one million Americans with this disease. Thank you.
[ Applause ]

>> Thank you. Dr. Baraniuk.

>> Thank you very much for letting me speak. I'm Jim from
Georgetown. I wanted to applaud -- crew for taking on this very
great challenge, all the best. I also want to tell you that it is
not all doom and gloom. We started publishing the results of our
studies from Gulf War illness, patients who also meet Chronic
Fatigue Syndrome criteria, only about half met Fibromyalgia
criteria, we've identified three separate dimensions of exercise
induced FRMI changes that we believe we may be able to apply to
Chronic Fatigue Syndrome. First off, we've identified white
matter abnormality that separates the GWICFS people from healthy
controls. We have two neutrally exclusive, bold blood flow
responses to exercise that subdivide into four neutral exclusive
groups and I think begins to address the issue of heterogeneity,
and these purely objective, you can't use any subjective criteria
to define them in advance. The simple message is all of these
subjective criteria that we're using for subjective syndromes
we're going to get rid of. We're going to have objective
diseases, we're going to have objective diseases that end up in
Harrison textbook of medicine. Little bit since I have 30
seconds, my complex message here is that what we identified is a
problem in the right interior frontal fACI culus, white matter
that connects prefrontal lobe here that deals with fatigue and
valuation of pain. It moves and connects with insula, which deals
with phantom pain, the emotional link of how much pain means to
you and it tracks posteriorly to involve the working memory, the
default network which is your mind wandering or daydreaming that
all of a sudden breaks up your thoughts, it also connects your
dorsal and ventral attention networks, which are the systems for
maintaining your focus. If you think about the cognitive
dysfunction, the exercise induced exertion exhaustion, that is
what our model is demonstrating abnormalities in and based on
this, we're hoping we don't get sequestered and we actually get
some funds to keep going. Thank you very much.

>> Thank you.

[ Applause ]

>> And again just reminder to you and anyone else, anything you
want to submit to supplement your public comment, please submit
them in writing to the docket. Next we go to Mr. Charles Lapp.
You have all proven me wrong, nobody has sat down, everybody came
up to the podium.

>> Thank you for this opportunity, I'm Charles Lapp, a physician
from Charlotte, North Carolina. I've been treating patients with
Chronic Fatigue Syndrome since 1985 and I've been using ampligen
since 1988. My expenses today will be reimbursed by hemispheric,
but I'm not here to speak for them, I'm here to speak for patients
and say in the 28 years or 25 years that I've been using ampligen,
we've had excellent success. I reviewed our records recently, and
shows over 50% of our patients have responded very nicely to
ampligen, and about 30% have significant improvements, which is
sort of disconnect from the data. We chose the end point from the
ampligen study many years ago and seem to be disconnect from the
end point we are using to measure the effect compared to the
patient global impression of change and the physicians global
impression of change. We see patients who are treated with
ampligen, who have seen remarkable improvement and return to
gainful occupation, school and work. I guess the most important
thick thing that I can say, two points I would make today, one is
that in all of the years I've been administering ampligen, we have
not had a serious side effect, not many drugs you can say that
about. The second thing is that I think history will show that
when a new drug is brought into a field, for example, when azt,
was approved for Aids or interferron was approved for
multimillionaire, many other players and FOORM suit cal houses
that got in the field and opened up treatment of these two
illnesses, we hope that perhaps ampligen will do that for Chronic
Fatigue Syndrome. Thank you for your time.

>> Thank you, Dr. Lapp.

>> Steven lempert.

>> I'm Dr. LECHLert, no financial interest. Approximately 70% of
patients tested in the 1994 study of Chronic Fatigue Syndrome were
positive by culture for hhv-6. There is subgroup of cfs patient
with active hh6 for culture. Characterization by method chain
reaction as indicated preNOM NANTly hhv-6 A, more frequently than
hhv-6 b. In the paper by Dr. Blushy titled ampligen inhibits
herpes, viral repcasion was inhibited by 46 to 98%. The inviTRO,
antiviral effect reported for ampligen translates clinically to
being effective inviVO in severely sick CFS patients. Mary was
(inaudible) positive before ampligen and negative on amplegen
three times. The drug converts hhv-6 infection into latent HERPes
virus with marked recovery of both patient health and functioning.
Patients severely afflicted with neurovigilent human HERPes virus
6 an occurs M subgroup with Chronic Fatigue Syndrome and
progressive multiple sclerosis have (inaudible) effective
antiviral such as ampligen, infusion is part of presumptive viral
trigger in subgroup of patients with CFS. Antiviral amp, needs to
be transferred to the FDA antiviral division and reevaluated now
rather than wait another 10 to 20 years. Thank you. [ Applause ]

>> Thank you. Mr. Dwight merriman.

>> Joan, I know she's here. Sorry, grobSTEin.

>> I'd like to sit down. Thank you. Hello, Dr. Joan, GROB, I'm
a physician and had ME for 14 years, I think I might have said 13
earlier, I've lost count. FDA faced with great responsibility to
encourage rapid development of treatment for this serious disease.
There are several important issues to keep in mind in this
process. It's important when evaluating drugs to make sure this
patient population is well defined. I strongly suggest that the
FDA require the use of Canadian consensus criteria for all
studies. In order to better define patient population FDA should
also make the validation of biomarkers very high priority. You
have heard today patients have many measurable abnormalities.
It's important to distinguish between therapeutic agents that
treat symptoms versus agents that treat underlying cause of ME.
Although the underlying cause is still unknown it's possible to
treat symptoms and also possible to treat associated infections
even if the infection is not the sole cause of the disease. It's
very likely that multiple agents will have to be used to treat
this multi system disease. This reality should be addressed in
the design and evaluation of drug trials. Outcome measures must
reflect the impact of ME on patients lives, small improvements in
function are extremely important to patients and we're willing to
tolerate risk to get improvements. Finally, the FDA should
demonstrate a sense of great urgency to evaluate therapies for ME.
As you know, there are currently no approved treatments, patients
are paying for expensive efKASHS treatments like amp, and rituxin,
out of pocket, causing financial harm on top of physical
disability. It is FDA's responsibility to remedy the situation.
Thank you.

>> Thank you, Doctor. Jeanett e Burm.

>> Okay. Nobody has paid for my expenses to be here. I'm here
to urge the FDA to play a more proactive role in working with
hemispheric toward approval of ampligen. Subgroup of ME patients
identified as result of being amp responders and tremendous amount
can be learn Friday that. One gets impression the aigence senot
interested in the drug.
It it is not even a topic at that drug development workshop.
Dr. Peterson, the physician with the most experience and success
in administering the drug and many other treatments, as well, has
not even been invited to one of the panels. I'm happy to see he
got two minute slot today. At the amp advisory committee, FDA
conceded no path for amp not accelerated approval process. No
explanation was given. The FDA recently developed new guidelines
for accelerated approval process for drugs for patients who are
not even sick yet. Why the drastic different standard I wonder?
Looking back at approval of azt, as first drug to TREEFT hiv and
aids it becomes clear the FDA does have discretion to adopt rule
if the circumstance is certaintied. At the end of last year the
FDA approved again under accelerated approval program a drug to
treat tuberculosis five times more likely (inaudible) standard
drug treatment for the disease without proof of inproved efficacy.
I'm not so convinced the FDA hands are bound when it comes to
approval of ampligen, it seems double standard compared to other
diseases and other drugs (inaudible) cliff hemispheric is running
out of money and the drug is going away potentially forever.
Thank you.

>> Thank you. And I have three more names that are not on the
slide, but Mindy katei, I'm sorry, I don't know if I got that even
close. Okay.

>> My name is Mindy and I'm a science reporter and blogger at CFS
central N. 1994, I wrote an investigative piece for Philadelphia
Magazine called Aids Drug no one can have about the experimental
drug ampligen, I'm here today to say it is vital that the FDA
understands one thing, much of the data on this disease is useless
because CDC and before that the NIH have not been studying
patients with ME. They are not studying patients with natural
killer cell defects, vo2 max abnormality or tilt table tests.
They are not studying patients who neat Canadian consensus
criteria for the disease or the international consensus criteria.
[ Applause ]

While the different definition can get incredibly confusing,
you'll need to know one thing, the kuda, and revised kuda known as
empire cal definition as well as oxford definition and the holmes
definition of this disease are not accurate. Yet CDC, your
disease definition instead of Canadian consensus and international
consensus which are accurate, Dr. Leonard Jayson of dePaul
University has shown in published studies that CDC in employing
empire cal definition is studying patients with major depressive
disorder, not patients with ME. That is like doing an hiv trial
and none of the people are hiv positive. As a result of studying
the wrong cohort doctors are misinformed. Dr. Lisa corbin said
she tells her patients that "Monday is for mending, Tuesday is for
ironing" I found her advice to be in a word clueless. But typical
of the help any patients receive. Imagine give Thanksgiving Hokie
advice to patients with hiv or MS, ME patients want and need
treatment not patronizing proMIEDs. Five of the patients I
interviewed in 1994 for the article have died, one a good friend
of mine, three in their 40s and 50s. Do you really think that
these patients would be alive today if only they had done their
mending on Monday? Thank you.

>> Thank you. Next is Mr. Don kalns.

>> Hi, I'm an owner of small company called hiperion
biotechnology. I am not really a CFS or ME person. We had
audacious idea in 2004 that we could measure fatigue by evaluating
changes in the composition of saliva. The army was very
interested in this topic because a lot of folks fighting over
there were profoundly fatigued. Since then we published a number
of papers about the use of this biomarker, there is several small
pep tides found in saliva that are quite informative. More
recently, we have published or given a paper at the American
association of clinical chemistry describing application of the
technology to CFS patients. I have to caution that these were
archival saliva samples, the Providence was not absolutely
certain, but the poster presentation got an award at this annual
meeting and pretty well attended meeting.

I guess why I'm here ultimately is to pose a question. I need
spit. I need spit from CFS patients, I need spit from control
patients or not control people. There is a lot of flaws and I'll
be the first to admit there is serious flaws with the SMOUL cohort
we looked at in CFS patients. I don't want to step on toes or get
any ruffled I'm very kog NIZent of the debockel, I would say that
if well are investigators out there that have protocols that are
ongoing, please talk to me. I'd want to get that saliva. I would
require that the saliva sample sent to me are blinded, I don't
want to have bias, if it doesn't pan out, it doesn't pan out. The
big market for us is in assessment of fatigue in healthy people,
not in sick people. But I really think this technology might have
a lot of applications in terms of evaluating new drugs, in terms
of efficacy, maybe in predicting crashes, there could be a lot of
potential here. But come talk to me, I'll be here tomorrow, as
well. [ Applause ]

>> Thank you. And Mrs. Dianne lewis. Is she -- you want
microphone Dianne?

>> I hope I can do this, lie laptop hasn't been working all day.
Dianne lewis, licensed certified social worker clinical therapist.
I'm here to tell you as a professional this disease is not -- does
not benefit well from CDT. But from a personal note, I want to
say that the distance between life and death is but one step but
the difference TWEEB living a daily death is life sentence with
this chronic disease. The experience robs you of who you are,
destroys your integrity, personality and stigma and discrimination
that comes with this disease is uncalled for. For every attempt
to step forward slow dying nature is no longer a step forward, but
stepping backward with each passing day. Not having medical
parity means we are nonexistent in life. I spend most of 75 to
80% of most days in bed at rest and it was easier for me as single
parent working two and three jobs to earn what Two master's degree
than to be sitting on the bedside. So today is a choice and I
know I will suffer from this and I want to let you know that when
I work I do four clinical hours of work because I stay within my
boundaries. I really, that is really taxing me. In order to do
that, I have to stay completely in bed rest, no contact at all
possible on Sundays. I will work my four hours Monday, it will
take me a long time to do my notes. Then when I come home, I am
in bed Tuesday and Wednesday unless I have appointments and if
that means I have appointments that extends that week. So I
actually am living to be able to help people, but I am on
palliative care plan, medications I get I've been told by our
hospital administrator, we don't treat that disease here. You
know, none of the doctors will consider this disease and so every
single doctor I see or get referred to will only treat just
symptoms that I would come in and say that was most disturbing. I
invest a lot of my energy and time and efforts into energy
conservation, knowing myself, trying to find out ways where I can
actually achieve something, but know that if I have to use the
cane, if anybody ever seen a social worker in a hospital we're on
the fast roll, I have to have the cane slowing me down otherwise
I'm going to burn out. I can eat a meal and my body will just
drain of energy.

>> You have 10 seconds left.

>> But I do use a lot of hydrotherapy, benefit from soaks, other
than that my medical interventions, you know, is mockery. I have
been told to do so many things and the best treatment that I've
gotten is because I have taken studies from Dr. Peterson and
Dr. Lapp, and taken them to doctors and doctors will respect that
research, then they will give me that medication I need otherwise
I am waiting for the last stages.

>> Thank you for your comments. [ Applause ]

>> And for agreeing to be the last speaker unless I think -- we
covered everybody who didn't get on this list, but had signed up,
is there any body who thought they were going to speak in the open
public session and didn't get a chance to? We haven't gone over
our time, so if there is anybody who didn't get an opportunity and
has a comment that takes less than two minutes -- one minute,
okay, that's it.

>> I would like to thank the FDA for agreeing to this meeting. I
would like to thank the advocates who helped to put this meeting
together. What I'd like to kind of reaffirm and reconfirm is that
FDA was willing in the days of hiv and aids to work with companies
and bend the rules, change the rules, alter policy, try to figure
out how to get a medication to people to save their lives. We now
have I'm referring to as alz, plan which I would ask FDA to allow
for ampligen. It's changing policy for patients who aren't
patients yet. It is willing to give people who are not actually
confirmed with Alzheimer's who knows what potent drugs that are
coming out to treat these people. But FDA is willing to bend, it
was mentioned that cituro was fast tracked December 31 of this
past year, and that this drug kills five times more patients than
placebo does, but somehow policy was altered because there is
unmet medical need. There is unmet medical need right here and
has been for a very long time. And so you know FDA is here and
ampligen sponsor is here, there is a gap, we got to figure out how
to bridge the gap. We need the right people in the room and
figure out how to do that, how do we bridge it. FDA, the
responsor, the expert clinicians you heard today and there are
other expert clinicians who have given amp, who could sit down and
help. Thank you.

>> Thank you. Okay. I think that closes the open public
session. You spoke during, we need to be fair here, this is I
think time now that I turn it over to Dr. (Inaudible) to close the
meeting and if there are comments that didn't get into open public
session or if you gave your presentation and neglected to add
something that you thought about later, please submit your
comments in writing to the docket. And I really applaud the
speakers for working with me and doing this without our timer.
This session facilitated that, we can't always do that in open
public meetings, but it was the right thing to do here and you
proved it and worked with me. Thank you very much.

[ Applause ]

>> Okay. I just want to close by thanking you again for being
here today. Thank you for sharing your experiences with what life
is like living with ME and CFS. You know, terrible cognitive and
physical impact you have been telling us about, the crashes you
experience and the fear of crashing and the consequences of you're
just trying to engage in daily life the way the disease has
constricted your life and managing physical pain and other
problems and challenges you have told us about it today. And
thank you for sharing your experience with what you have been
trying to do to treat the condition as best you can. And the
range of therapies that you have described and things you have
worked well and what has worked better than other things. I also
want to thank you for being so generous with your time. And after
spending a few hours with you today having a much better, I know I
have a better appreciation of what sacrifice you have made and
really how couragious you are to have come to the meeting today
knowing full well as I now understand that you are going to have
consequences for expanding the energy to be here and tell us how
you are doing, so I think that is just a tremendous courageous
contribution you are making to on behalf of this disease and other
patients who couldn't be here, I want to thank you so much. And
on behalf of this patient focused initiative, I thank you really
made me feel you set us off to a very challenging start because we
have to do our best to try to meet the level of contribution that
you have made to us. This is as I said a first meeting and this
is I think a first effort for us in this area to be trying to do
better with trying to capture and describe quality of life and
life impacts. As I said, we'll have to docket open to receive
comments until August. We'll be producing a summary report
capturing the information and having available a transcript and
raw material so people -- am I doing that? Maybe. And we'll be
posting that and sharing that with the reviewers and others and so
this has been a very rich and challenging start for us. Thank you
again and I hope that I know you'll have a very good day tomorrow
if you are able to be there. I hope you have a good night.

[ Applause ]

>>
 

JohnnyD

Senior Member
Messages
206
Before they closed down the webinar page, I copied the text out of the translator box that was on the webinar page. I jsut happened to think, -- I wonder if I can copy this text. Sure enough. It would have been available, but I just caught the tail end of the day (I work an the internet connection was timing out at times). I do have a bit of the discussion that happened before the Public Testimony - that I would be happy to post. Here it is, and then you've got all I've got. (Otherwise, you'll have to await the official transcription.)

==========================

it. It makes my liver markers sky rocket, which is ironic, of all
the other drugs I'm taking, but I tried cymbaltancombination with
antibiotic I ended up passing out (inaudible) we don't all have
Fibromyalgia. When you talk about pain and people with the
condition, it's not necessarily Fibromyalgia.

>> I might clarify, my question was not meant to imply people
with Chronic Fatigue Syndrome all have Fibromyalgia. That is not
what I was getting at, I was grouping the medications together
since they all have an indication for Fibromyalgia and pain
specifically related to that.

>> Sara Eggers: Let's go, we haven't heard --

>> I just wanted to say since this illness is so long-term, which
we didn't know in the beginning, but my daughter was 14 when she
got ill and by 18 doctors had put her on oxycodonePercocet, and
she had to take a lot of Advil, and wound up with eSOF GEal ulcers
after a few years of that and would have, she needs to go into
rehab or something to get off these medications, so I mean, I am
very angry that she was ever put on them. They wouldn't let me in
on the decision when I called the doctor and said why, questioned,
they -- what they were doing, they just told me I had no right
that she was an adult. I'm her caretaker, she has to live with me
because she's completely disabled, couldn't even be here, and it's
just a nightmare. 22 years of pain medication does a lot of bad
things and I'm getting older, I'm in my 60s now. I don't want to
take anything that is going to affect my cognition, which is
already, you know, poor from the brain fog.

>> Sara Eggers: You are making a good point, I want to make sure
we are capturing up here is that I think what I'm hearing is that
since the diagnosis wasn't made, wasn't easily made, that you were
trying, you and your daughter were trying a number of other things
and in the end if you knew what you knew now you wouldn't have
tried that.

>> If I knew 22 years --

>> Sara Eggers: Is this a shared experience with everyone? I see
a lot of head nodding. Yeah.

>> Like I said, she had hemorrhaging, esopHageal ulcers at 30 and
her pulse is now, she doesn't just have Chronic Fatigue Syndrome
or ME, she has ulcers and you know, just it's a nightmare, it's a
nightmare, I'm sorry.

>> Sara Eggers: No, if I can take, we'll go with --

>> I just wanted to add to Mary's point earlier, besides
Fibromyalgia, different types of pain in CFS. The definition
includes muscle and joint pain, there haven't been a lot of
research in to different pain in CFS. For example, some people
have nerve pain, that is related to like react VAGZ of HERPes
virus and some people reported they get better when they take
antiviral for that. There is stomach pain, you know, other types
of pain.

>> Sara Eggers: Dianne, right? She had her hand up.

>> I'm not on any pain medication at this time, although I
certainly would like to have some. But one thing that I I'm a
social worker, not a doctor, but I attribute a lot of my pain to
the fact I'm not getting enough oxygen and therefore my muscles
are really hurting and so you know, my lack of ability to deep
breathe, you know, my sleep issues, there's a lot of I think a lot
of reasons that cause the body to be depleted of oxygen and just
no way if a doctor doesn't believe that there is anything wrong
with you. I am on an extreme amount, 400 dollars a month in
copays for medications and none of it is working. It's all
palliative care, it doesn't get to the core of the problem. Ant
virals, pain medication, a lot of medications here, nobody has
mentioned proVIDual, it keeps me alert and it does really help and
I also use VyVA nse, instead of aderall. As far as the pain goes,
I think a lot of pain comes from lack of oxygen and your body is
just not -- nothing there for it to draw on.

>> Sara Eggers: Can I, I'm going to ask one question if we have
time. I caught up on something and I didn't write down who said
it. I would like to probe a bit deeper into the idea of masking
symptoms and causing by taking one medication to mask symptoms, I
think I'm sorry, I don't know which one, the idea that you maybe
push yourself a little bit more than you should and you would have
if you weren't on the medication that make you feel good right
now. Is that an idea --

>> I thought about that for a while. I think when I'm on it and
I can't speak in terms of the pharmocology, I just think my body
is on overdrive, even if I try to even pace myself, very hard to
pace because you are full of jitters, I find myself feel my body
running when I'm siting and not turning off. I feel like I'm a
car and the wheels are turning and I'm not moving and that is if
I'm lay nothing bed, that is just my side effect. She mentioned
Providual, I tried it, it made me jump out of my skin. I think
different people respond to different medications, you have to
find one that works for you. In terms of what you said, usually
because of medication, it lasts in my body longer and (inaudible)
not for me.

>> One comment, too. It's not actually related to your question.
My own personal perspective and not to go contrary to the purpose
of this workshop, we need medicines to help people with CFS and
but my philosophy is to try to take as few medicines as possible
and I can do that maybe because I had the illness for 16 years and
have a good sense, I've tried things and they haven't worked, I
discontinue them. I think we really as patients I hear people are
taking a lot of medicine and probably a lot isn't working.

>> Sara Eggers: I think the doctor has a follow-up to that.

>> Yeah, you mentioned that you thought one of the adjunctive
treatments that helped you most was physical therapy and the
stretching. Can you say a little more about what it was that
helped? How did it help you specifically?

>> Yeah, I only did that for a short while and I have to say that
was really probably the only thing that was good about the
treatment that I had in the UK. I think it was having a trained
professional who knew through conversations with me what the
limits were, basically at that time I could really only do lieing
and sitting exercises. But was able within that framework to give
me very limited exercises that I could do every day and I think
having that guide and none of us are specialists in this and often
don't know what to do. We want to increase our capacity as much
as we can and we just don't know the best way to go about it. I
mean the other thing I would say going back to pacing and I
disagree with some of what just from my own experience others have
said, I feel like I can tell when I'm overdoing things, when I'm
getting to a point where it is going to be too much. My life is
on a daily basis a very carefully collaborated system, you know.
I have a mat, I lie on at work. I've arranged all kind of
complicated work arrangements, I don't work a full day from the
office. I work from home much of the time. I think we really
have to figure out kind of by monitoring ourselves carefully what
works and didn't work. Of course some of us, I have a wonderful
husband who is there to help is and some people don't have that
kind of care and help at home. I think if we really can do things
ourselves that will help.

>> Sara Eggers: Would anyone, let me see if anyone wants to
follow-up on that and build on that?

>> Okay. Thank you.

>> Yeah, just quickly to build on what Tasha was saying I
maintain a yoga practice and which is kind of California physical
therapy, I mean, what is stretching, what daily or every other day
stretching does, releases endorphins and so there's a natural
release of natural pain control that comes from developing a
practice like that and when you first start you can barely do a
stretch or a posture but that is why I think Tasha's point having
access to a trained professional, they can help you build and
develop that so that it can have some effect.

>> Sara Eggers: Thank you. I will turn and see if my FDA
colleagues have other questions about particular treatment
approaches, things you haven't heard of about that you want to
probe a bit deeper? Dr. Michelle.

>> One thing that has been mentioned only in passing, but has
come up in the course of discussion of clinical trials is IV
saline I'm wondering how many of you have used that and if you
find it to be beneficial?

>> IV saline.

>> Let's go, next Dr. Kiser.

>> I'll tell you the abbreviated story. I flew down to Puerto
Rico with my grand solicit son, who didn't have the right paper to
get on the cruise ship and after many stressful hours we finally
got him on and I had a total crash. The next morning when I woke
up I said, I've got to go to the infirmery, I went in, my blood
pressure went down to like 65 and I fainted. The Belgian doctor
who understood what Chronic Fatigue Syndrome said I can either
send you home or give you a bag of saline. He gave me the saline
drip, I got up and went shopping. [ Applause ]

>> Sara Eggers: One in the -- one of you in the back. Did you
want to -- add to that? No? Okay. Come back up. Someone else
back there?

>> Yeah, IV saline helps, I consider it a treatment. Relatively
cheap one. It helps with the light headedness and I also tend to
get a little bit dehydrated when I have a crash and obviously
helps with that.

>> Sara Eggers: Yes?

>> My name is Joan, I would like to say you can also use oral
salt and water that can be very effective in an emergency.

>> Sara Eggers: Any other burning questions? We have five
minutes left and I just want to do what we did in topic for the
first topic which is see does anyone else have anything they want
to share perspective or something to share they haven't heard
shared by the comments from the panel or by others? Okay.

>> Joan again. I'd like to just say that in terms of pain
management, if somebody has a broken leg, it will be very painful
and it will be less painful as the leg heals. If you break that
leg repeatedly, it will get more and more difficult to control
that pain. So we have the equivalent situation here. We don't
know what the underlying cause of the pain is and so therefore we
cannot probably adequately treat many people's pain. So I know
that this is an FDA meeting, but I know there are members of other
agencies in the government here and I would like to put in a big
pitch for the fact that we need more research, more money to look
at the underlying causes of this illness until we find that people
are going to be taking drugs, having side effects from the drugs,
watching the drugs effects dissipate over time because we are not
treating the disease. I'm not saying we should not treat symptoms
because we should, we need to make everybody comfortable and we
need clinical trials period of measures, but what we really need
is research.

[ Applause ]

>> Sara Eggers: Here and then --

>> I have a topical pain medication, so therefore it doesn't
really affect my cognitive abilities, thank God. But what is in
it, (inaudible) lido cane, cycle, baclofan, I don't know how that
is pronounced, anybody is interested in percentages, they can see
me at the compounded prescription. It works beautifully.

>> Hello, Anita Patton. Will you please ask question of the
audience like you ask us to raise our hand, will you ask how many
people represented here today are from any sort of pharmaceutical
company that could help us in drug development? Do those --
would anyone care to volunteer themselves as being from there?

[ Applause ]

>> Any other final thoughts? I think we have time for two more
people.

>> I just want to -- obviously what we really need is approved
drug treatment and that is why we're all here and I think that's
the most important thing. I want to give big shoutout to the
research of Stacy Stevens and Chris Snell, I wear this heart wave
monitor all the time. I know what my threshold is and I don't
want to go over, I will crash. I brush my teeth, I sit down so I
won't go over.
That is valuable and I think that is something that is maybe
underutilized at this point.

>> Sara Eggers: Okay. Okay.

>> One more and then we'll come with your question. Yes.

>> One other thing that -- Janet Smith. One other thing that
hasn't been mentioned that goes along with physical therapy and
heart rate monitor is I've always asked myself why am I better
than a lot of patients as far as physically and I think it's
because the adaptation has come naturally for me. Like the shower
chair sitting down in the shower, I sit down whenever there is a
chair available. So I have a handicap sticker, since 1994, and so
if by using the handicap sticker I can do one more stop at the
store or see one more patient because I'm using those aids, that
come naturally for me, but other people they don't think about
sitting down and how to reserve energy.

>> Sara Eggers: Thank you. We have one more question from
Dr. KweedRESHGS.

>> It's a comment, I want to pick up on something that Joan said,
which is someone said it and might have been you, Joan, in the
last session about getting at the underlying cause. And I don't
want people to think that hearing about the symptoms and the
varied experiences of patients, even if it is common underlying
cause and everyone experiences it differently, is not a worthwhile
endeavor and I do think it is worthwhile in that it helps us
develop pathway research pathways to explore where we see some of
the commonality, where we get hints about potentially some of the
common patho physiology that those particular agents that are
helpful are addressing. Our goal is to -- that is one reason why
we want another researcher here and one of our goals is to
stimulate more conversation, more people smarter than me thinking
about how could we tie this all together pathophysiologically and
really begin to target therapy development so yeah, we're helping
symptoms, which may be first on our plate, but ultimately getting
to the bottom of this and understanding what that common root is
so that we can actually cure it. So your point absolutely well
taken and we are hoping ultimately this kind of exercise will is
one way to contribute to the building the map.

>> Sara Eggers: Thank you.

[ Applause ]

>> Sara Eggers: I think that is a good place to stop with the
discussion 2. Again, a fabulous discussion and there are --
excuse me, I made it this long. There are evaluation forms and if
you can't find them, I think they're in the back, come find me or
one of my colleagues and we'll find them. They are completely
voluntary, if you want to take one in and if you want to take it
tonight and think about it, we'll be here tomorrow. With that,
I'm going to turn over to Theresa Toigo who is going to close with
the open public comment session. I will ask the panel members up
here, you are free to go down, thank you again, so much. [
Applause ]



>> Theresa Toigo: I hope the panel will indulge me and change our
plan a little bit because it seems like we can maybe do a lot of
this from people sitting if they don't want to get up. So we'll
try something a little different here.
This is the open public comment session and both FDA and believe
believe in transparent process for information gathering so we
need to do a little formality here to ensure that we take care of
the process the requirements. We're going to invite the stake
holders that are preregisterd and confirmed to share their
perspective with us during this part of the meeting to to ensure
the transparency tis important to understand the context of an
individual's presentation, so you are encouraged to disclose at
the beginning whether or not any organization paid for your
expenses to attend this meeting and if you choose -- that is fine,
as well, that is important part of our open public process that we
explain that. So how is this session going to work?

We had originally intended to use a timer, but approximate you are
willing, if the speakers are willing to work with me, we have two
minutes for each person to speak and instead of timer, I've got my
timer and if you are willing, as I'm going to watch it, as you are
getting close to two minutes I'll let you know, I'll put my hand
up and then that way we can ensure that we get to everybody that
everybody is able to get their allotted time and that everybody
else gets out of here at the time they want. But I think we've
heard enough today it's not easy for people to stand. So to make
you come to the center to do it in the timed process is probably
not ideal. Are you all willing to work with me who will be
speaking in open public session? When I go like this, know you
have to get close to wrapping it up. Okay. That is how we'll do
it.

So I think what is important here is that we know that not all
patients speak with one voice and we certainly heard that today.
People are at different stages of a disease and therefore things
that matter to them are different. But the insight you can
provide us are going to be helpful to the sessions tomorrow and
they're also important as we think about the challenging issues
related to drug development in this particular disease area.

And so we want like I said to be fair, so you're willing to work
with me and I'm willing to change the system and hopefully my
panel here is going to allow me that, so give me some daggers if I
let the time go to go, let's give it a try. Our first speaker
it's not on there yet. Okay, the first speaker I have on the list
is Mrs. Courtney alexander. Mrs. Alexander here? Okay. So Mr.
Michael walser. Okay. How about Mrs. ANATa patton? Okay.
Anita, do you want to come up to the microphone? Okay.
 

Ember

Senior Member
Messages
2,115
Here it is, and then you've got all I've got. (Otherwise, you'll have to await the official transcription.)
Thanks! I missed the earlier part. Do we know when the official transcription will be available?
 

WillowJ

คภภเє ɠรค๓թєl
Messages
4,940
Location
WA, USA
What is the status of this version of the ICD-10-CM? Does Diagnostic Code G93.3 as shown below reflect the Coalition 4 ME/CFS proposal, submitted to NCHS in July 2011 requesting that CFS be reclassified? Is ME to be removed as a diagnosis and replaced with CFS?
The Coalition 4 ME/CFS provides no information, its website having been disabled.
again, the Coalition proposal dealt only with coding: changing from F codes to G codes.

The definition/description needs to be changed in a different way with a different committee (the coding committee they gave this proposal to doesn't do definition/description) and requires another proposal to someone else, which it's my understanding that the Coalition supports a clearer definition.
 

WillowJ

คภภเє ɠรค๓թєl
Messages
4,940
Location
WA, USA
They keep talking about 'the docket' - not too sure what that it but it sounds like a repository of everybody's documentation. I don't know if that will be public domain.

One can submit comments to the docket until August 2. jspotila says it is public and will be used to inform their documents. Someone (Kweder?) said the documents would be supplied to decision-makers at the time decisions were being made (which I found very encouraging). Link to the docket is at the end of her post here.
 

Ember

Senior Member
Messages
2,115
again, the Coalition proposal dealt only with coding: changing from F codes to G codes.
My question was about coding, not about definition/description. I had thought that the Coalition's proposal involved moving CFS from R to G. This version of ICD-10-CM has generated some concern that “Myalgic Encephalomyelitis...is to be removed as a diagnosis from the US version of ICD-10-CM, and replaced with CFS.”

Do you know why the Coalition's website has been disabled?
 

WillowJ

คภภเє ɠรค๓թєl
Messages
4,940
Location
WA, USA
My question was about coding, not about definition/description. I had thought that the Coalition's proposal involved changing CFS from R to G. This version of ICD-10-CM has generated some concern that “Myalgic Encephalomyelitis...is to be removed as a diagnosis from the US version of ICD-10-CM, and replaced with CFS.”

Do you know why the Coalition's website has been disabled?

I didn't know the website had disappeared. I don't think that changing the coding will make ME disappear. Almost no one diagnoses ME in the USA, and if they do they don't use the R code, which is what the Coalition proposes to scrap. (I have seen an argument for using what currently looks like a vague encephalitis code for ME, but I don't have any first-hand information about that - my only first-hand information is that it's not possible to diagnose ME under ICD-09-CM. Either way, it's exceedingly rare. There is little to no ME diagnosed as ME in USA anyway. So it's kind of hard to worry that something which is minimal to absent anyway, would disappear. That train already left. We cannot go back in time and fix that; we have to go on from where we are now.)

I think it would be dangerous to add a code for ME while leaving a code for CFS, because people would continue to diagnose both people with ME and people with random other diseases with CFS. It's better to (when we can) dismantle the concept of CFS altogether. We don't need it, IMO.

So there's really not too much we can do at this point, to make the diagnosis worse (other than moving to F40-F69). Hopefully we can make it better. Moving to G code should send a signal that it's a disease to be diagnosed more carefully and paid closer attention to, and have tests and procedures covered by insurance.

And if we keep at them to stop using especially the worst inclusions like Oxford and the Empirical approach but to assess those patients for other conditions (many of them have various other diagnosable conditions as their primary condition which would explain their illness) and to start with CCC or ICC and better diagnosis and then see who (if anyone) is left, and to subgroup so we can try to figure out if we are dealing with one disease or seven, then we will hopefully get somewhere.

I think this FDA conference might help, because then we'll finally have an official government document which explains the multiplicity of our symptoms and starts to get at the severity. And rather than just stick it on the record and ignore it, they are actually going to use it to help make decisions. So it's possible we could finally move forward maybe a bit faster than grass growing in winter.