the network - Narcolepsy Network

THE
NETWORK
A Publication of Narcolepsy Network, Inc.
Fourth Quarter 2013
2013 is the Biggest Conference Yet!
The28thannualNarcolepsyNetworkConferenceinAtlantawasbig!Biginsize,
withmorethan400PWNsandtheirsupportersattending.Biginstature,withsix
ofthenarcolepsyworld’sbestresearchersanddoctorsmakingpresentations.Big
ininformation,withmorethan40sessions.Andbiginfunandfellowship,withold
friendsreuniting,newfriendshipsforming,andmanypeoplemeetingotherPWNsfor
theveryfirsttime.
TheconferencekickedoffFridaynightwithregistrationandtheopeningreception.
Saturdaystartedwithbreakfast,thekeynotepresentationbyDr.MichaelThorpy,and
anupdateonnarcolepsyresearchfromDr.JerrySiegel.
Conference-goersthenheadedtosmallerpresentationsthatmatchedtheirinterests.
Subjectsrangedfromsucceedinginschoolwithnarcolepsytoresearchupdates(turn
topage6formore)today-to-daylivingandsupport.
OnSunday,attendeescontinuedtoattendsmallgroupsessions,thengatheredfor
lunchandaresearch-focusedpresentationbyDr.ThomasScammell.
Throughouttheweekend,attendeeshadtheopportunitytobrowsetheN[Art]:
NarcolepsyArtexhibitfeaturingtheworkofmorethan20PWNs,andtomeetauthors
andartists.
Astheconferencewounddown,oldand
newfriendsmadeplanstomeetupagain,
eitheronlineoratour2014conferencein
Denver.Moreinformationondateswillbe
availablesoon.
Highlights from the
Keynote Address
Dr.MichaelThorpy,directoroftheSleepWakeDisordersCenteratMontefiore
MedicalCenterinNewYorkCity,was
thekeynotespeakerinAtlanta.His
presentation,Recent Advances in the
Understanding of Narcolepsy,covered
changestothediagnosticcriteriafor
narcolepsy,advancesinresearch,and
changesintreatment.
InMay2013,thelatesteditionofthe
Diagnostic and Statistical Manual of
Mental Disorders (DSM-5)wasreleased.
Thenewmanualincludeschangesto
thediagnosticcriteriafornarcolepsyand
introduceslevelsofseverity.
InJune2013,theInternational
ClassificationofSleepDisordersThird
Edition(ICSD-3)wasreleased.Itdivides
narcolepsyintotwotypes,Type1,
NarcolepsywithCataplexy,andType2,
NarcolepsywithoutCataplexy.
continued on page 9
Narcolepsy Community Shines at FDA Public Meeting byKarenRorie
RepresentativesfromNarcolepsy
Networkjoinedhundredsofothers
fromthenarcolepsycommunityatthe
FDA’spublicmeetingonnarcolepsy
In This Issue
• A Word from Our Executive Director
3
• Research Update
4
• Reaching Out to Educate Medical Professionals
5
• Conference Research Presentations
6
• Congratulations to our 2013 Award Recipients
7
• Voices From the NN Conference
8
• News from PWNs
10
drugdevelopmentonSeptember24.
Approximately150peopleattendedthe
meetinginpersonatFDAheadquarters
isSilverSpring,MD,withnearly700
moreattendingviaanInternetwebcast.
Amongthein-personaudience,67%had
narcolepsy.FDAoffi
cialshavedescribed
thestrongresponsetothenarcolepsy
meetingasunprecedented.
Perspectivessharedatthemeetingand
throughapublicdocketwillprovide
theFDAwithhelpfulinsightswhen
conductingbenefit-riskassessmentsfor
newdrugstotreatnarcolepsy.Patient
responsescouldalsoshowtheFDA
thattheyneednewwaystomeasure
theeffectivenessoftreatmentsbeing
reviewed.
Thereweretwomainareasofdiscussion
atthemeeting:“diseasesymptoms
anddailyimpactsthatmattermostto
patients,”and“patients’perspectives
oncurrentapproachestotreating
narcolepsy.”Foreachtopic,apanelof
fivepatientsmadepreparedstatements
followedbyafacilitateddiscussionwith
theliveandonlineaudiences.
Themeetingbeganwithremarks
fromFDAoffi
cialswhothankedthe
continued on page 2
1
Narcolepsy Community Shines at FDA Public Meeting
community for its outstanding response
and asked narcolepsy patients to shake
things up during the meeting. The
audience responded with four hours of
insightful comments.
Topic One: Disease Symptoms and
Daily Impacts
The first panel of speakers addressed the
symptoms of narcolepsy that most affect
them. Panelist and Narcolepsy Network
member Fran Rosen traveled from
Florida to attend the meeting. Like many
speakers, Rosen identified Excessive
Daytime Sleepiness (EDS) as the most
problematic symptom for her.
“At a certain point, I had to give up
control of my life to narcolepsy. It
controls how long I can shop, how long
I can read, how long I can work on my
computer,” Rosen said. “Can I go to the
party, will I be able to stay up during the
meeting, am I able to drive today?”
NN member Brandon Coonrod, a
15-year-old high school sophomore, said
that EDS was the most difficult symptom
for him. “If I’m sitting down in class, or
sitting pretty much anywhere, you can
count on I’m going to fall asleep,” he said.
Other frequently cited symptoms were
cataplexy, which a speaker described as
“being awake inside a corpse,” “brain fog,”
hallucinations, and difficulty sleeping at
night.
Topic Two: Patients’ Perspectives
Narcolepsy Treatment
Speakers on the second panel,
addressing current approaches to
treating narcolepsy, described the great
benefits of medications but also their
limitations – and for some the great
expense.
“I want to control narcolepsy and not let
it control me,” panelist Casey Thompson
commented. Around the room, many
heads nodded in agreement.
LaShun Ray, the mother of a 9 year old
who was diagnosed with narcolepsy at
5 said she worries about her daughter
taking three medications at such a
young age. One of them is approved for
use by adults, but not children. Without
medication, she said her daughter
would not be able to go to school.
2
continued from page 1
Even so, when she
returns home, she
is exhausted and
they have to decide
whether she should
go straight to sleep or
eat first.
“Her will is to eat
first, then sleep, but
her body’s will is to
sleep first. When she
does wake, she is
now battling hunger
and a headache. This
is a hostile battle
between the body’s
needs,” Ray said.
NN representatives Sara Kowalczyk, Suporn Sukpraprut, Mark Patterson,
Melissa Patterson and Patricia Higgins at the FDA.
Many speakers reported years or
even decades of incorrect diagnoses
and being treated for diseases they
didn’t have. Developing tolerance to
medications and dental problems were
other issues speakers identified.
Other common themes addressed
included general ignorance about
narcolepsy, and the difficulties of staying
in school and holding a job in a society
that is not very nap-friendly, and keeping
up home and family responsibilities.
Justin Greene of Leesburg, VA told how
narcolepsy had interrupted his promising
college education. He dropped out
because he couldn’t keep up the high
grades he had always achieved before.
“I want to be the last of the children to
have no choice but to sleep through a
bright future,” he said.
A Learning Experience
FDA officials were highly attentive
throughout the meeting and through
questions and answers were clearly
learning more about the disease. For
instance, speakers helped officials
understand that napping is a primary
effect of narcolepsy and not just a way
of coping with it. Other discussions
with FDA officials focused on the
pharmaceutical, behavioral, dietary and
lifestyle alternatives that patients try in
order to cope with limited medication
benefits, progressive aspects of the
disease, drug side effects or gradual
tolerance to the medications.
“All the panelists and speakers did
an outstanding job of representing
the many facets of narcolepsy and
sharing the challenges that people with
narcolepsy face,” said NN’s Executive
Director Eveline Honig, MD, MPH. “This
has been such a wonderful opportunity
for people with narcolepsy to be heard
and to influence the future of narcolepsy
treatment. We are very, very pleased with
the meeting.”
The FDA also collected information from
PWNs through a public docket, which
remained open for two months after the
meeting. The FDA is now in the process
of reviewing information gathered at the
meeting and via the docket and creating
a report on the meeting, which will be
shared with FDA officials who review and
approve new treatments as well as with
the public. The report is expected to be
released this spring.
Participants cited the need
for treatments for:
•
Excessive Daytime
Sleepiness
•
Brain Fog
•
Disrupted Nighttime Sleep
•
Hypocretin/Orexin loss
•
Treatments specifically for children
A Word from Our Executive Director
by Eveline Honig, M.D., M.P.H.
Dear friends:
This is our last newsletter in 2013. We
have had a very successful year with
the very well attended conference in
Atlanta as our wonderful highlight. It was
attended by over 400 people. We had so
many great sessions including a number
with well known researchers from all
over the country; we had a great hotel
with wonderful staff and excellent food;
everyone made new friends, and many of
us saw old friends as well.
The social aspect of our conference is
extremely important. Many parents told
me that their teens and young adults
had such a good time and made new
friends. For these young people, learning
about narcolepsy’s new research,
medications and ways to cope may not
be as important as meeting other young
people who struggle with the exact same
issues.
We at Narcolepsy Network are so happy
we can provide this weekend of learning,
fun, friendship and feeling “normal” for
a very reasonable price. We hope that
many of you will make plans to come to
Denver in October 2014. We are already
working on the Denver conference. Our
attendance has grown substantially but
we are intent on providing professional
NN membership benefits include:
• A subscription to The Network,
NN’s quarterly newsletter
• Online access to our newsletter
archive
• Discounted registration rate for
our annual conference, being held
in Denver in October 2014
• Invitations to NN events,
activities, and special networking
opportunities
direction and at the same time keeping
it “homey” with the idea that everyone
should feel they came “home.”
We were very pleased that our two
Researchers of the Year, Dr. Tom
Scammell and Dr. Jerry Siegel, came
to accept their awards and grants and
to share their expertise with everyone
who attended. With our newly created
research committee, we will follow
and assist in these and other research
initiatives.
My friend and former colleague,
Dr. Meeta Goswami, Director of the
Narcolepy Institute in the Bronx,
received the Mahatma Gandhi Pravasi
Samman Award for outstanding services,
achievement and contributions. On
October 4, 2013, the 144th anniversary of
Mahatama Gandhi’s birth, the Honorable
Baroness Sandip Verma presented
the award at the House of Lords in
London. The ceremony was attended by
dignitaries from around the world during
the Global Achievers Conclave.
The Mahatma Gandhi Pravasi Samman
Gold Medal recognizes people of Indian
origin for significant contributions in
their fields of expertise in the country
in which they live and in service to the
global community. As many of you know,
Dr. Goswami and her son after the award
presentation in London.
Dr. Goswami has been helping people
with narcolepsy in New York City since
1985 and she certainly deserves this
award. We are very proud of her.
Lastly, we plan to have an exciting new
year with many projects and programs.
We are again asking everyone to
contribute to National Sleep Awareness
Week. Please contact us for more
information. If you are interested in
organizing a fundraiser or would like
to set up a support group, please let us
know.
Wishing you a wonderful and peaceful
holiday season!
Warm regards,
Dr. Eveline Honig
It’s Time to Renew Your NN Membership!
2014 is just around the corner, and with it comes a new membership year at Narcolepsy
Network. As you prepare to turn the calendar to 2014, take a moment to renew your NN membership too.
Renew your membership by January 15, 2014
and you will also receive a QR coded medical
alert wristband. This wristband lets emergency
responders know that you are a person with
narcolepsy even if you are not able to speak for
yourself, and the QR code links medical personnel
to a webpage with information about narcolepsy
symptoms, medications, and treatment precautions. Having this information available to your caregivers in an emergency helps you get the safe and effective care you need.
Stay connected to your network. Visit www.narcolepsynetwork.org to renew your
membership today!
3
Research Update
Exciting New Drug
Development for
Narcolepsy
and coping
with symptoms
and improved
functionality of
daily life.
by Suporn Sukpraprut, MSc, MA, PhD and Anthony Tam, PhD
A phase 2b
clinical trial
was designed
to validate the
results from
the phase 2
trial in more
participants
and for a longer
duration. Phase
2b included 93
individuals with
narcolepsy, and
participants
were given a
treatment of
either placebo or
ADX-N05 for 12
weeks. Different
from phase 2a,
participants
were only given
one treatment
and not both.
On October 1,
2013, Aerial
BioPharma announced positive results
for the phase 2b clinical trial. Outcomes
of interest included the same three
primary outcomes from the phase 2a
trial (i.e. MWT, ESS and CGI-C) and also
included the number of cataplectic
attacks per week. These results are
encouraging in light of the September 24
FDA patient focused drug development
meeting. More companies and research
to develop new drugs to treat symptoms
of narcolepsy are always welcomed.
For a number of years Aerial BioPharma,
LLC, has been developing a new
stimulant drug, ADX-N05, to treat EDS
(excessive daytime sleepiness) associated
with narcolepsy. The phase 2a clinical
trial was conducted from 2011-2012 to
study the safety and effectiveness in 33
individuals with narcolepsy. Participants
were given a treatment of placebo for
two weeks followed by ADX-N05 for
two weeks or vice versa. This cross-over
design means that every participant took
both the placebo and ADX-N05 drug for
two weeks each, however participants
did not know when they were taking
each treatment.
The primary outcomes of interest were
the changes in scores from tests taken
at the beginning and at the end of
the study. The research indicated an
increase in the average sleep latency of
four Maintenance of Wakefulness Tests
(MWT), improved scores for the Epworth
Sleepiness Scale (ESS) and improved
scores in the Clinical Global Impression
(CGI) when comparing the ADX-N05 and
placebo test numbers, with statistical
significance.
The MWT consists of four tests with
two hours in between each test, and
is designed to see how long one can
remain awake–almost the opposite
of the MSLT (multiple sleep latency
test), which measures how long it
takes for one to fall asleep. The ESS is a
questionnaire that scores an individual’s
sleepiness during the day. The CGI is a
measure summarized by a clinician of
the patient’s global functioning, which
includes the patient’s history, symptoms,
behavior and the impact of symptoms
on the patient’s ability to function. The
results from the phase 2a clinical trial
indicate individuals on ADX-N05 saw
improvement in their ability to stay
awake during the day and decreased
daytime sleepiness, as well as an
improvement in symptoms, emotions
4
A clinical trials glossary
Clinical Trial (Interventional Study)4:
A clinical study in which participants
are assigned to receive one or more
interventions (or no intervention) so
that researchers can evaluate the effects
of the interventions on biomedical
or health-related outcomes. The
assignments are determined by the
study protocol. Participants may receive
diagnostic, therapeutic, or other types of
interventions.
Investigational New Drug4: A drug,
or biological product, that is used in a
clinical trial but has not been approved
by the FDA (the drug is either not
available for a doctor to prescribe or, is
available, but not approved by the FDA
for the use being studied).
Primary Outcome Measure4: The
planned outcome measure in the
protocol that is the most important for
evaluating the effect of an intervention.
Most clinical studies have one primary
outcome measure, but some may have
more than one.
Secondary Outcome Measure4: A
planned outcome measure in the
protocol that is not as important as the
primary outcome measure, but is still
of interest in evaluating the effect of an
intervention. Most clinical studies have
more than one secondary outcome
measure.
Experimental Arm4: A group
of participants that receives the
continued on page 5
Exciting New Drug
Development for
Narcolepsy continued from page 4
Reaching Out to Educate Medical Professionals
intervention that is considered to be
effective.
One of the missions of Narcolepsy
Network is to educate those who
come in contact with people
with narcolepsy (PWN). Previous
newsletters have discussed
the outreach to school nurses,
principals, and teachers, a vitally
important effort as these people
have significant daily contact with
youth who may have undiagnosed
narcolepsy. It is also very important
to educate physicians and other
health care providers about the
condition.
Placebo4: A substance that does not
contain active ingredients and is made
to be physically indistinguishable (that
is, it looks and tastes identical) from the
actual drug being studied.
Baseline Characteristics4: Data
collected at the beginning of a clinical
study for all participants and for each
arm or comparison group. These data
include demographics, such as age and
gender, and study-specific measures (for
example, systolic blood pressure, prior
antidepressant treatment).
Adverse Event4: An unfavorable change
in the health of a participant, including
abnormal laboratory findings, that
happens during a clinical study or within
a certain time period after the study is
over. This may or may not be caused by
the intervention being studied.
Randomized Allocation4: A strategy in
which participants are assigned to arms
of a clinical trial by chance.
New Drug Application (NDA) Key
Information2
• Safety and efficacy of the drug and
the benefits of the drug outweigh the
risks.
• Appropriate labeling and package
insert.
• Methods used in manufacturing the
drug and the control used to maintain
the drug’s quality are adequate to
preserve the drug’s identity, strength,
quality, and purity.
References:
1. U.S. Food and Drug Administration. “Code
of Federal Regulations Title 21”. www.fda.
gov. Revised 4 April 2013
2. U.S. Food and Drug Administration. “New
Drug Application (NDA)”. www.fda.gov.
Revised 21 Feb. 2013
3. U.S. National Library of Medicine. “FAQ
ClinicalTrials.gov - Clinical Trial Phases”
http://www.nlm.nih.gov. Revised 18 April
2008.
4. U.S. National Institutes of Health “Glossary
of Common Site Terms. http://clinicaltrials.
gov/ct2/about-studies/glossary. Revised
Aug. 2012
by Mark Patterson, MD, PhD
As many PWN report, there was
often a very long lag time between
the onset of their symptoms and
Dr. Patterson
their eventual diagnosis. One reason for this delay is the non-specific nature of by medical centers in order to provide
many of the symptoms of PWN. Another
for the continuing education of the
reason is that medical professionals can
medical staff. At this meeting, more than
only diagnose a condition of which they
40 members of the pediatric department
think. With an estimated prevalence in
were able to hear my talk on pediatric
the United States of 1 in 2,000, many
sleep disorders, with a special emphasis
physicians will encounter a limited
on narcolepsy. Many pediatricians are
number of PWN during their careers.
unaware that narcolepsy often has its
As such, it is essential that part of our
onset during the teenage years. The
awareness campaign is to target medical
attendees were very appreciative of the
providers to consider narcolepsy in a
lecture and invited me back to speak
symptomatic patient.
again.
As part of this effort, I had the
opportunity to give several
presentations to medical personnel
this year. I am a pediatrician in general
practice who also provides clinical
training to third-year medical students.
In March, I had the chance to tag-team
with a patient with narcolepsy and
cataplexy in giving a presentation to
second-year medical students at the
Virginia College of Osteopathic Medicine
in Blacksburg, VA. I discussed the medical
aspects of narcolepsy to the 25 students,
then my co-presenter discussed the
impact of the condition on her life and
coping mechanisms she has employed.
The students were very grateful for the
information and were excited to have a
face-to-face meeting with a patient.
In May, I was invited to give “Grand
Rounds” to the Pediatric Department of
the Carilion Clinic in Roanoke, VA. Grand
Rounds are an educational forum held
In August I was able to follow up on an
invitation to give a case presentation on
a PWN and speak about my involvement
with Narcolepsy Network at the Carilion
Clinic Sleep Center in Roanoke, VA. More
than 20 sleep specialists and technicians
were on-site, with more connected by
phone from satellite locations. They
were excited to hear about narcolepsy
(as opposed to their usual talks on
sleep apnea) and made me an honorary
member of their group! Partly as a result
of my talk, they are planning on starting
a narcolepsy support group for the
Roanoke, VA area.
So as you can see, your organization is
continuing in its effort to educate and
advocate on your behalf. Please do what
you can to support Narcolepsy Network
in all its activities. Additionally, talk to
your medical providers and encourage
them to help educate their peers.
Remember, we are all in this together!
5
Conference-Goers Hear the Latest in Narcolepsy Research
2013 was a big year for
narcolepsy research, so
research was a hot topic at the conference, with four noted narcolepsy researchers making presentations about
recent findings.
New Developments in Understanding
Narcolepsy and the Hypocretin System
by Jerry Siegel, PhD
Dr. Siegel, a professor of psychiatry
at UCLA, spoke about his recently
released research showing an increase
in histamine cells in the brains of people
with narcolepsy. Siegel theorizes that the
increase in histamine cells could correlate
to the loss of hypocretin cells, which
leads to narcolepsy. Siegel says that this is the first evidence
of an increase in the number of neurons
in any brain system, in any disease in
humans. This finding leads researchers
to believe that it may be possible to
therapeutically manipulate the number
of neurons to treat diseases, including
narcolepsy.
The finding on histamine is also
significant because, “there are now
two known major abnormalities
in narcolepsy. Before we thought
everything was due to the hypocretin,”
Siegel said.
Siegel also shared some information
about his current research on how factors
such as social interaction and light
exposure affect hypocretin and other
arousal systems.
The current hypothesis is that hypocretin
release is correlated with positively
motivated motor activity. The data is
consistent among five different species –
mice, rats, cats, dogs and humans.
The idea, Siegel says, “is that arousal is
not a volume control that’s up or down,
but it’s multidimensional.” 6
The Neuroscience of Narcolepsy
by Thomas Scammell, MD
Dr. Scammell, a professor of neurology
at Harvard Medical School, Beth Israel
Deaconess Medical Center and Boston
Children’s Hospital, reviewed what
has been learned about the brain
mechanisms of narcolepsy, including his
own research on the histamine system.
Scammell’s recently published research
on the increase in histamine in the
brains of people with narcolepsy was
a surprising discovery that opens a
number of possibilities about how the
histamine and orexin systems affect
each other. He theorizes that the
increase in histamine could play a role
in maintaining consciousness during
cataplexy and sleep paralysis. “We know that the histamine neurons
help keep you conscious so if that system
is now ramped up somehow, it may keep
you conscious even when you’re in a REM
like state.”
Scammell is also exploring the reasons
that strong emotions trigger cataplexy. In mice, the medial prefrontal cortex
of the brain is the area that produces
the emotions that cause cataplexy. By
turning off these areas in narcoleptic
mice, the number of cataplexy attacks is
reduced.
“So what this is showing is that part of
the brain is necessary for the production
of cataplexy,” Scammell says. “So now
this is another cell group that we think is
important for regulating cataplexy.”
What’s in a Name? Understanding
the Terminologies for the Family of
Hypersomnias
by David Rye, MD, PhD
Dr. Rye, professor of neurology at Emory
University, began his presentation by
discussing the meaning of the names
given to the class of sleep disorders
called hypersomnia. The roots of the
word Hypersomnia are hyper (above,
over) and somn (sleep), so the definition
of hypersomnia is too much, or excessive
sleep. In the medical diagnostic coding
manuals, narcolepsy is classified as a
hypersomnia. This presents a problem,
Rye says. Hypersomnia and narcolepsy
with cataplexy are two entirely different
things. In hypersomnia, sleep is
excessive in depth and duration, while
narcolepsy is a disorder of badly timed
sleep and sleep attacks, but not too
much sleep over a 24 hour period.
Searching for a way to treat patients with
excessive sleepiness who did not fit a
diagnosis of narcolepsy with cataplexy
led Rye down a path to a research
discovery, which was recently published
in the journal Science Translational
Medicine.
Rye and his colleagues wondered if
the problem might have to do with a
gain of something rather than a loss
of something. In exploring this idea,
they discovered that in patients with
hypersomnia, the gamma-amino butyric
acid (GABA) system is overactive. “In many patients that have been labeled
hypersomnia, idiopathic hypersomnia,
narcolepsy without cataplexy, in their
spinal fluid there is something like WD40 that makes their GABA system work
better. It acts very much like a natural
anesthetic or hypnotic,” Rye said.
There are still many unanswered
questions about this mysterious
substance, which does not have a name
yet, and many challenges, but Dr. Rye’s
research will continue to try to find the
answers.
Narcolepsy: Is There an Environmental
Trigger?
by Todd Swick, MD
Dr. Swick, the founder of Neurology
and Sleep Medicine Consultants in
Houston and assistant clinical professor
of neurology at the University of TexasHouston, offered an overview of recent
research showing the role of genetic
and environmental factors in the
development of narcolepsy.
“This is not a strictly genetic disorder,”
Swick said. There is a gene for narcolepsy,
continued on page 9
Congratulations to our 2013 Award Recipients
Researcher of the Year Awards:
Thomas E. Scammell, MD
Jerry Siegel, PhD
Dr. Scammell is a professor of
neurology at Harvard Medical
School, Beth Israel Hospital
Medical Center, and Boston
Children’s Hospital. In 2013 his
study “Increase of histaminergic
tuberomammilary neurons in
narcolepsy” was presented at the
SLEEP Meeting. Dr. Scammell’s
study provides surprising
evidence that people with
narcolepsy have an increased
number of neurons that produce
histamine, suggesting that
histamine signaling may be a
novel therapeutic target for this
potentially disabling sleep disorder.
Dr. Siegel is a professor of
psychiatry at UCLA. His laboratory
has made discoveries concerning
the loss of hypocretin cells in
human narcolepsy. In 2013, his
study “Greatly increased numbers
of histamine cells in human
narcolepsy with cataplexy” was
published in the online edition of
the journal Annals of Neurology.
Dr. Siegel’s research shows that
people with the disorder have
nearly 65 percent more brain
cells containing the chemical
histamine. Their research suggests
that this excess of histamine cells
causes the loss of hypocretin cells
in human narcoleptics.
Dr. Scammell
Dr. Siegel
Both Dr. Scammell and Dr. Siegel received monetary grants in support of their narcolepsy research.
Public Awareness Award:
Kevin E. Gonzales
Kevin is a senior studying sociology and political science at Oklahoma State
University. Inspired by his fiancée, Megan Phillips, Kevin organized the Dream Big
Concert in February of 2013. Following her diagnosis of narcolepsy in 2012, Megan
was worried that narcolepsy could cause a chasm in her relationship with Kevin.
To show her his love and support, Kevin decided to start a benefit concert to raise
awareness and funds for Narcolepsy Network. The concert was held on February
15 and was a huge success, with more than 250 people attending. Through
ticket and T-shirt sales and donations the concert raised more than $2,000 for
Narcolepsy Network.
Kevin Gonzales and his fiancée, Megan Phillips
Ruth Justice Nebus Volunteer of the Year Award:
Laura J. Evert
Laura has filled many roles including serving on the Board of Trustees, providing artwork for the
Creative Corner section of our member newsletter, and presenting sessions at many conferences,
including this year when she presented “Fear No Art: Dream Sleep and Creativity.” This year Laura
also coordinated the art show at our conference in Atlanta, and raised money to support the show
through an online fundraising campaign. Laura J. Evert
Laura is also the founder of N[Art], a grassroots effort to educate the world about narcolepsy and
the importance of sleep through continually evolving creative works and projects. Learn more
about N[Art] on Facebook.
7
Voices From the NN Conference
My First Narcolepsy
Network Conference
by Fran Rosen
When I joined Narcolepsy Network this
past spring I decided I would definitely
attend the conference in October in
Atlanta. I wish I could say that I based
my decision on something important
but it was really all about location. I felt
if it wasn’t an awesome experience–oh
well, at least I wasn’t traveling across the
country. Flying from Florida to Georgia
was not a big deal. Little did I know then
that attending the narcolepsy meeting
at the FDA and attending the conference
a few weeks later would have such a
profound and positive effect on my life.
Spending the weekend with 400 people
who are drowsy and in a fog like me is
sooooo refreshing. What a wonderful and
unexpected surprise. How liberating and
comforting it is not to be judged, stared
at, embarrassed or disapproved of for 48
hours! It was all about acceptance.
It was a weekend of total camaraderie,
bonding and making new lifelong
friends. For many who attend year after
year it was an annual reunion with hugs
and laughter. I can’t wait until next year
in Denver when I’ll see my new friends....
I was so touched by the many
“supporters” who also attended. There
were spouses, friends and siblings all
actively engaged. But it was the many
parents who came to support and be
there for their children that made me
the happiest. It is so easy to think that
someone with narcolepsy is really lying,
faking, and is just plain lazy especially
when you’re a teenager. Kudos to all
those parents who believe and believe in
their children.
I also learned so much about narcolepsy,
ongoing research and myself. For
example, when I was a teenager I couldn’t
understand why my parents every few
months would have me tested for either
mononucleosis or a thyroid condition. Well much to my surprise I learned
during a discussion so many people went
through the same experience. As silly as
that may sound learning that for me was
a major wow moment.
8
Probably the most important thing I
learned is that I’m really not alone.... Thank you Narcolepsy Network !!!
(By the way the hotel was lovely, the food
was great, the speakers were awesome
and the discussions interesting and
informative... and I had so much fun!!!)
Conference a Great
Experience for
Young People with
Narcolepsy
by Melissa Patterson
This year’s NN Conference had a lot
of great sessions for younger PWNs,
starting on Saturday with a fantastic
and informative session, Succeeding
Through High-School, led by Sharon
Link O’Shaughnessy, with some great
input from first-time conference
attendee, Bailey Wedelich. Some of the
information from that session is now
up on the Narcolepsy Network website,
for anyone looking for resources on
school accommodations. In the same
time slot, some of the older youth
found Succeeding through College and
Grad School, led by Anthony Tam, very
inspirational, though some people said
they had wanted to attend both the
high-school and college sessions.
After lunch, Sharon led another session
What’s Up With My Brain Anyway? Games
to explain the Narcoleptic Brain. There
weren’t many games involved, but
Sharon did have some great suggestions
for PWNs trying to stay organized, plus
some common sense advice on staying
healthy. She reminded people to take
care of their narcolepsy by taking care of
their physical health by eating healthy
foods and trying to stay active— good
advice for everybody!
Later that afternoon, Julie Flygare’s
session, Narcolepsy and Your Social
Life, was standing room only. Julie
talked about her personal history and
also offered advice for people feeling
pressured by friends to “be normal” and
for PWNs trying to juggle a social life and
sleep schedule that clash.
The rest of Saturday was pretty much free
time — personally, I enjoyed a fabulous
dinner at Maggianos with a handful of
other 20-somethings, then took the night
tour of Atlanta. Sunday seemed to be a little more
free-form, especially in the morning. I
hosted an informal session on college
accommodations and dorm living
(information coming soon to the NN
website!), and some of the other youth
used the morning to investigate the
tables in the lobby area.
Just before lunch, Katie O’Connell led a
great session called From Grief to Relief:
Coping with a Sense of Loss Post Diagnosis,
which gave people a chance to talk
about one of the more private aspects of
being diagnosed with narcolepsy.
After lunch, Julie Flygare led a session on
Communicating Narcolepsy in Everyday
Life. Some other popular sessions for
young PWNs included Nia with Sue
Dambrauskas, Time Management Tips
with Heather Rogers, and FEAR NO ART
with Laura Evert.
Making posters for Nicole Jeray’s
“Swinging for Sleep” campaign and
informal social gatherings were also
popular activities.
Some of the Swinging for Sleep posters
designed at the conference.
Overall, it was a great conference, with
lots to offer for the younger set — the
only problem was picking one, and then
finding enough time between sessions
to socialize.
Thanks to everyone who helped make
the youth-centric activities at the 2013
Conference such a success. Hope to see
you next year!
Highlights from the Keynote Address
In addition to Narcolepsy Type 1 and 2,
the new ICSD-3 also includes Idiopathic
Hypersomnia, Kleine-levin Syndrome,
hypersomnia due to a medical,
psychiatric disorder or medications, and
behaviorally-induced insufficient sleep
syndrome in its listing of Hypersomnia
Disorders.
continued from page 1
Both the DSM-5 and the ICSD-3 also
include different criteria for narcolepsy
in children.
While diagnostic criteria have changed,
advances in research and treatment are
moving forward, and Dr. Thorpy says
the future looks very bright.
The DSM-5 Criteria
for Narcolepsy
Under the DSM-5’s diagnostic criteria
a patient must have:
1. Recurrent periods of an
irrepressible need to sleep,
lapsing into sleep, or napping
occurring within the same day
occurring at least three times per
week over the past three months
2. The presence of at least one of
the following:
• episodes of cataplexy,
hypocretin deficiency
• nighttime sleep test showing
REM sleep latency less than
or equal to 15 minutes, or
a multiple sleep latency
test showing a mean sleep
latency less than or equal to
8 minutes and two or more
sleep-onset REM periods.
Thorpy noted that under DSM-5
criteria, cataplexy must be triggered
by laughing or joking.
ICSD-3 criteria for a diagnosis
of narcolepsy
Narcolepsy With Cataplexy
1. Excessive sleepiness for 3 months
2. At least 1 of the following:
• Cataplexy, and a MSLT test with
a mean sleep latency of less than
eight minutes and two sleep
onset REM periods.
• Cerbrospinal fluid hypocretin-1
levels less than 110 pg/ml
and a MSLT test with a mean
sleep latency of less than eight
minutes and two sleep onset
REM periods
Narcolepsy Without Cataplexy
Positive polysomnography/ multiple
sleep latency tests are met (mean sleep latency of less than eight minutes and two sleep onset
REM periods) but no cataplexy is present
The biggest change in recent years
is Xyrem’s move from a third line
treatment to the first line of treatment
for narcolepsy. This is because it is the
only drug that will treat all narcolepsy
symptoms.
In the future, orexin gene therapy and
treatments that target the histaminergic
system, both of which are currently
being studied, could make dramatic
differences in the treatment of
narcolepsy, Thorpy said.
DSM-5 criteria for the level of
narcolepsy severity
Mild
• infrequent cataplexy
• need for naps only once or twice per day
• less disturbed nocturnal sleep
Moderate
• cataplexy once daily or every few days
• disturbed nocturnal sleep
• need for multiple naps daily
Severe
• drug-resistant cataplexy with
multiple attacks daily
• nearly constant sleepiness
• disturbed nocturnal sleep
Conference-Goers Hear the Latest in Narcolepsy Research continued from page 6
called HLA DQB1*0602, but having the
gene does not mean you will develop
narcolepsy. An estimated 12-38 percent
of the general population carries this
gene and does not have and will not get
narcolepsy.
“But there are environmental issues
that are very significant if you have
the genetic predisposition to have
narcolepsy.”
One proven environmental factor is
exposure to H1N1 flu (swine flu) which
was a pandemic in 2009. Data from
Europe, China, and North America
shows an increase in narcolepsy cases
following exposure to either the flu itself
or a specific vaccine called Pandemrix,
which was used in Europe. Studies in
France, Canada and the US showed that
all patients who abruptly developed
narcolepsy after an H1N1 vaccination
had the HLA DQB1*0602 gene.
Exposure to streptococcal infection (strep
throat) is another strong environmental
factor that can trigger narcolepsy.
Some research shows that exposure to
environmental toxins such as second
hand smoke and insecticides can be
associated with narcolepsy, but these
findings, Swick says, “are on far shakier
ground to know whether there is a true
cause and effect.”
In light of these studies, Swick says, it’s
both nature and the environment that
contribute to the onset of narcolepsy in
those who have the gene.
9
News from PWNs
Narcolepsy by Anna Tyler
Editor’s note: Anna Tyler is 11 years old and
lives in East New Market, MD. She wrote this
essay for school, and her mom Barb shared
it with NN. Thanks to Anna and Barb for
allowing us to share it with you.
“Problems are not
stop signs they
are guide lines.”
Said, Robert H.
Schuller. Getting
a day time sleep
disorder may be a
curse, that never
goes away; but
now I think that I
am blessed. With narcolepsy it makes me
want to share my story about getting it
with you, hoping it will inspire you, and I
will inspire you. I wish to one day be able
to get the word out to a whole lot of people.
Falling asleep in class and having
your teacher wake you up is very
embarrassing; especially when it’s during
a test and your teacher used to be your
babysitter. I felt so tired and exhausted,
I didn’t know why. I went to bed on time
and fell asleep fast. Still very exhausted,
tired and starting to doze off, the bell
rings, waking me up and scaring me
out of my socks! My Heart beating fast, I
started rushing to get my things packed
up and ready to go. I was rushing down
the hall as a cute little first grader that is
just about getting trampled on by big
kids, with long legs and mean looking
faces. I finally made it to the bus, but as
soon as the bus started moving I dozed
off again. Almost making me miss my
stop, just in time, I woke up before the
bus doors closed and ran to get off.
Walking in the house, my mom is sitting
at the kitchen table and she asks, “How
was school today?”
I replied, “I don’t really remember, but
I fell asleep a lot in class today.” Mom
made an appointment with my
doctor; who suggested a sleep study.
A sleep study didn’t sound bad to me.
To me all I heard was the word sleep,
out of that. So it turns out I was wrong.
I had to take short naps, and then they
woke me up, even though I wanted
to go back to sleep. I had wires all
over my body and my hair had goo in
it with a rap covering it. I felt so tired
and my eyes were blood shot.
The narcolepsy had an effect on me
that changed my mood, it’s called
mood swings. This changed the
relationship between my mom and I.
I would get mean with my mom and
sometimes yell at her and make her
cry. But we always made up. Even
though it sometimes made us far
away, other times it made us closer.
Three years later. It was a Friday and
my mom was off from work. I was in
class at school, when I got called to
the main office all packed and ready
to leave. My mom was there waiting
for me, she had a big smile on her face
but looked like she had been crying. I
got in the car and asked her,” Why did
you pick me up from school early?”
She shouted relief, “We have an
answer; you have narcolepsy!”
So far in my life the day that my mom
and I got an answer to what was
making me so tired is the best day and
moment of my life. I am currently still
having a little trouble with my mood
swings that I sometimes take out on
my mom and dad. My mom, dad and I
are trying to get through this struggle
together. I have felt that what Robert
H. Schuller has said has had an effect
on me. Having narcolepsy is part of
God’s plan for me in my adventure in
life that is just getting started.
Mark Your Calendars For Narcolepsy Awareness Day!
Saturday, March 8, 2014 is Narcolepsy Awareness Day, also known as Suddenly
Sleepy Saturday. Once again this year, NN will use the day to help raise
awareness of narcolepsy. One of the best ways to increase awareness is to have
your city or state recognize March 8 Narcolepsy Awareness Day with an official
proclamation. Visit www.narcolepsynetwork.org for more information on how
to proclaim the day in your city or state.
10
Nicole Jeray recognized for
community service
Congratulations to LPGA
golfer Nicole Jeray who was
nominated for the 2013 KIA
Community Assist Award.
The award recognizes the
LPGA player who had made
an exceptional contribution
to a nonprofit organization
or program throughout the season.
As a narcolepsy advocate Nicole raises
awareness through her work on and off
the course. Nicole was one of nine players
nominated and although she didn’t win the
award this year, she is always a winner in our
eyes. Great work, Nicole!
Narcolepsy in Words
and Drawings
Solomon Briggs released
his first book, Expressions
of My Own Narcolepsy
With Cataplexy in October. Briggs shares his personal
experiences through his
hand-drawn illustrations
and text about living with narcolepsy with
cataplexy. The book also includes detailed
information about narcolepsy. The 60 page
book is available from Amazon.com.
Books for Kids of All Ages
Did your hand ever come
alive and start talking to
you? What would you say
to your hand if it did? This
is exactly what happened
to Nicole Cortichiato. She
was 16, not diagnosed yet,
when she started having
hypnagogic hallucinations. She turned
that experience into her first book, “The
Independent Hand.” She has followed it up
with another book, “What Kind of Bunny
Ears Do You Have?” Nicole is inspired by
her narcolepsy and lets the creativity flow
from all her crazy dreams. She hopes to
inspire others to use their narcolepsy as a
tool to create. Find more about Nicole and
her books at www.imagineart.net/nicolecortichiato-gallery.
MEDICAL ADVISORY BOARD
Emmanuel Mignot, MD, PhD, Chair
Stanford Center for Narcolepsy
Palo Alto, CA
Stephen A. Amira, PhD
Brookline, MA
Neil Feldman, MD
St. Petersburg, FL
Meeta Goswami, MPH, PhD
Narcolepsy Institute
Bronx, NY
Lois Krahn, MD
Mayo Clinic
Scottsdale, AZ
J. Gila Lindsley, PhD
Lexington, MA
Quentin Regestein, MD
Brigham & Women’s Hospital
Boston, MA
David B. Rye, MD, PhD
Emory University School of Medicine
Atlanta, GA
Thomas E. Scammell, MD
Beth Israel Deaconness Medical Center
Boston, MA
Lawrence Scrima, PhD
Sleep-Alertness Disorders Center, Inc.
Aurora, CO
Jerome Siegel, PhD
UCLA Neurobiology Research
Sepulveda, CA
Todd J. Swick, MD
Houston Sleep & Neurology Associates
Houston, TX
Michael J. Thorpy, MD
Sleep-Wake Disorders Center
Bronx, NY
PEDIATRIC ADVISORY BOARD
Narcolepsy Network, Inc.
National Office
129 Waterwheel Lane
North Kingstown, RI 02852
TOLL-FREE (888) 292-6522
(401) 667-2523 telephone
(401) 633-6567 fax
e-mail:
[email protected]
www.narcolepsynetwork.org
Editor
Karen Rorie
The contents of this newsletter are for
informational purposes only and are not to
be construed as medical or legal advice. If you
have questions, please consult your physician
or attorney.
This newsletter is published quarterly as a benefit of membership. No portion of this
newsletter may be reprinted for commercial
or noncommercial purposes without the
advance written approval of Narcolepsy
Network, Inc. Requests for permission to
reprint should be directed to:
Narcolepsy Network, Inc.
129 Waterwheel Lane
North Kingstown, RI 02852
or email
[email protected]
We welcome contributions and comments to
this newsletter. Please send them to:
Karen Rorie
c/o Narcolepsy Network, Inc.
129 Waterwheel Lane
North Kingstown, RI 02852
or email
[email protected]
Deadline for Submissions:
Submissions are reviewed on an on-going
basis. They will be used whenever possible, as
time and space permit.
Agnes Kenny, MD
Peru, IN
Suresh Kotagal, MD
Rochester, MN
Suzanne Moore-Darms
Marlton, NJ
Michael Eig, Esq
Chevy Chase, MD
Mali Einen
Menlo Park, CA
Michele Profeta
Atlanta, GA
The Network thanks
Jazz Pharmaceuticals and
Teva Pharmaceuticals for an
unrestricted grant that has partially
funded graphic design, printing,
mailing, and other
costs of publishing and
distributing this issue.
BOARD OF TRUSTEES
Sara Kowalczyk, MA, MPH
President
Boston, MA
[email protected]
Mee Warren, Vice President
New York, NY
[email protected]
Mark Patterson, MD, PhD, Vice President
Roanoke, VA
[email protected]
Rahul Kakkar, MD, Secretary
Pinehurst, NC
[email protected]
Louise O’Connell, Treasurer
Woburn, MA
[email protected]
Sarah DiDavide
River Grove, IL
[email protected]
Andrea Podolsky, JD
New York, NY
[email protected]
Suporn Sukpraprut, PhD
Lyndhurst, OH
[email protected]
Anthony Tam, PhD
Amherst, MA
[email protected]
Trustee Emerita
Niss Ryan, Founder
Rochester, NY
On the Back Cover
Creative Corner Artist, Jean Zack
Hi my name is Jean Zack and I am
a member of Narcolepsy Network.
I wanted to submit this poem into
the Creative Corner. I wrote it the
day after I experienced cataplexy
at work. I am a Special Education
Teacher at an elementary school.
The cataplexy occurred at the end
of the day during bus duty. It felt
very scary to me.
11
NarcolepsyNetwork,INC.
129WATERWHEELLANE
NORTHKINGSTOWN,RI02852
forwarding service requested — return postage paid
Creative Corner
CATAPLEXYbyJeanZack
Feelingweak,
Istarttosink.
Ihopethatitwillstop.
butpeopleseeandrushtome,
althoughIcannottalk.
Theygetmeadrinkandsitmedown
beforeIstarttofall.
Theytalktome,Icannotspeak,
althoughIhearthemall.
Isitawhileasthefeelingfades,
Ilookatthepeopleandsay“that I’m OK”.
WhenI’maloneIstarttothink,
asIdryawaymytears.
Iwishthatthiscataplexywouldgoaway,
formany,manyyears.