Transcript What is NMO? - Guthy-Jackson Charitable Foundation

Neuromyelitis Optica
NMO
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What is NMO?
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Neuromyelitis Optica (NMO) Spectrum Disease
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Originally known as Devic’s Disease
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Eugene Devic was a French
neurologist who summarized
the features of the condition
in 1894
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Uncommon disease syndrome of the central
nervous system (CNS)
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Autoimmune Disease
What is NMO?
Optic Nerve
NMO affects the optic nerves & spinal cord
Demyelinating Disease
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NMO leads to loss of myelin – a fatty substance
that surrounds nerve fibers and helps nerve signals
move from cell to cell
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What is NMO?
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Immune system cells and antibodies attack and
destroy myelin cells in the optic nerves and the
spinal cord
NMO Symptoms
Individuals with NMO develop
Optic Neuritis (ON)
• Inflammation of the
optic nerve
• Eye pain
• Vision loss
Transverse Myelitis (TM)
• Weakness, numbness and
sometimes paralysis of the arms
and legs
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Sensory disturbances and loss of
bladder and bowel control
NMO Attacks
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After the first attack, NMO follows an unpredictable
course
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Most experience clusters of attacks months or years
apart, followed by partial recovery during periods
of remission
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Relapsing form of NMO primarily affects women
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Female to male ratio > 4:1
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NMO Attacks
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The onset of NMO varies from childhood to
adulthood, with two peaks, one in childhood and
the other in adults in their 40s
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NMO-IgG Antibody
• The discovery of an antibody in the blood of
individuals with NMO gives doctors a reliable way of
determining if someone has NMO
• Antibody is a kind of protein
in the blood = NMO-IgG
(NMO Immunoglobulin G)
• Antibody seems to be present
in about 70% of people with
NMO
• Not present in people with MS
or other conditions that can
present with NMO-like illnesses
NMO-IgG Antibody
• Doctors can diagnose
very early (probably after
the first attack of ON or
myelitis) people who are
at risk to develop further
symptoms that would
lead to a later clinical
diagnosis of NMO
• Doctors can consider
preventive measures, to
reduce the chance of
relapse or severity of a
second neurological
attack
Astrocytes located in
brain & spinal cord
Check the Test!
NMO-IgG Antibody
Is the NMO-IgG necessary for diagnosis of NMO?
• No
• 30% of individuals with NMO, for reasons currently
not understood, do not have this antibody when
tested
• However, when present it helps to make a more
confident diagnosis
• There may be undetetectable antibodies that
resemble NMO-IgG
• Testing methods are still being refined
MS / NMO
What You Need to Know
• Neuromyelitis Optica is NOT Multiple Sclerosis
• Until recently, NMO was thought to be a type of
Multiple Sclerosis
• Recent discoveries indicate NMO and MS are
distinct diseases
• Traditionally spinal cord lesions seen in NMO are
longer than MS but this is not always the case
MS / NMO
What You Need to Know
• NMO & MS share many symptoms
• NMO can sometimes be confused with MS or other
diseases including
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Sjögren’s Disease
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Acute Disseminated Encephalomyelitis (ADEM)
• NMO and other diseases are treated in different ways
• Early detection and treatment help ensure best
outcomes
Treatment Therapies
Some MS drugs can
exacerbate an NMO attack
Treatment Therapies
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NMO has no cure - yet
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Therapies exist to treat an attack while it is
happening:
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Reduce symptoms
Prevent relapses
Initial attacks of NMO usually treated with
combination of:
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Corticosteroid drug (methylprednisolone)
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Stop the attack
Immunosuppressive drug (azathioprine)
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Prevention of subsequent attacks
Treatment Therapies
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Plasma exchange (plasmapheresis)
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Separates antibodies out of the blood stream
Used with people who are unresponsive to corticosteroid
therapy
Pain, stiffness, muscle spasms, and bladder and
bowel control problems can be managed with
the appropriate medications and therapies
Why do people get NMO?
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No one knows – yet
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Why this is predominantly restricted to these two
locations is a mystery
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Possibly a unique protein in these structures or the blood vessels
in these regions, makes them vulnerable2
NMO Is a Rare Disease
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Rare diseases affect fewer than 200,000 people
in the United States
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About 7,000 rare diseases exist that affect more
than 25 million Americans and their families
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Rare diseases present a significant health care
concern
NMO Is a Rare Disease
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Patients with rare
diseases are frequently
misdiagnosed or are
undiagnosed
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Few drug companies
conduct research into
rare diseases since it is
difficult to recover costs
of developing treatments
for small, geographically
dispersed populations
National Institutes of
Health Response
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In response to the Rare Diseases Act of 2002, the
NIH established the Rare Diseases Clinical
Research Network I (RDCRN I) to address the
unique challenges of research on rare diseases
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In 2009, the NIH funded 19 continuing and new
clinical research consortia to strengthen the
ongoing clinical research in rare diseases
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NMO is not included in the consortia
The Guthy-Jackson
Charitable Foundation
Dedicated to funding basic science research to
find answers that will lead to:
• NMO Prevention
• NMO clinical treatment programs
• Potential cure for NMO
The Guthy-Jackson
Charitable Foundation
Founders
Bill Guthy
Victoria Jackson
Their daughter was diagnosed with NMO in 2008
The Guthy-Jackson
Charitable Foundation
Funding NMO Scientific Research at:
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Brigham and Women’s Hospital, Harvard, MA
Johns Hopkins University, Baltimore, MD
Mass General Hospital, Boston, MA
Mayo Clinic, Rochester, MN
Mount Sinai School of Medicine, New York, NY
Stanford University, Stanford, CA
University of California, San Francisco, CA
University of California, Los Angeles, CA
University of Colorado Denver, Aurora, CO
University of Texas Southwestern Medical Center, Dallas, TX
More on the way!
NMO “Blood Bank”
• GJCF Biorepository for NMO
• If you or someone you
know has been
diagnosed with NMO,
please donate blood
samples to the
NMO Blood Bank
• Blood samples are vital for NMO research
• Accessibility to NMO biosamples by our
research scientists is an urgent need
NMO “Blood Bank”
• We need NMO blood samples
• You can help us meet this critical need by donating
your blood samples and raising awareness of our
repository program
• Donate again and often!
• Ways to donate
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Repository Nurse will travel anywhere in the USA
NMO Patient Day
Donate Blood Samples at LabCorp NMO blood draw sites
NMO “Blood Bank”
Ways to donate blood
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Repository Nurse will travel anywhere in the USA
If you have NMO (or you know someone who has NMO) and you are willing to donate data or
samples to our cause, please contact our Repository Nurse at 858.333.1704 or email at:
[email protected].
NMO “Blood Bank”
Ways to donate blood
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NMO Patient Day
GJCF hosts a group draw at NMO Patient Day in Los Angeles, CA.
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Location, Los Angeles, CA
Check website for future dates
NMO “Blood Bank”
Ways to donate blood
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Donate Blood Samples at LabCorp NMO blood draw sites
The Guthy-Jackson Charitable Foundation is expanding its biosample draw sites. Working with
Laboratory Corporation of America (LabCorp), NMO patients will be able to donate their
blood samples at 59 new sites in the United States. Details are coming soon. Please visit our
website for the launch announcement of the GJCF - LabCorp NMO blood draw sites.
Donate to NMO Science
Many ways to donate to NMO science
• Direct donations via GJCF website
• Get creative
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Hold Fundraising events like:
• Bake Sale
• Walks / runs / relay races
• Auction
• Become inspired at:
http://www.nmotion.guthyjacksonfoundation.org
Sources
2. Office of Communications and Public Liaison National Institute of Neurological Disorders and
Stroke National Institutes of Health Bethesda, MD 20892
http://www.ninds.nih.gov/disorders/neuromyelitis_optica/neuromyelitis_optica.htm
3-6. The Walton Centre.
http://www.old.thewaltoncentre.nhs.uk/services/devic_s_disease/information_on_nmo.asp
7. Weinshenker BG. Neuromyelitis optica: what it is and what it might be. Lancet
2003;361(9361):889-90.
Office of Communications and Public Liaison National Institute of Neurological Disorders and Stroke
National Institutes of Health Bethesda, MD 20892
The Guthy-Jackson Charitable Foundation. http://www.guthyjacksonfoundation.org/nmo-mswhat-you-need-to-know/
Image Credits
Smith’s Science. http://1.bp.blogspot.com/__y-lXOrOC8A/TN6zQ8RBQXI/AAAAAAAACZ0/F3RNORK9lYY/s1600/myelin.jpg
Healthy vs. Damaged nerves: Elements for Health.
http://www.elements4health.com/statins-could-have-a-negative-impact-in-multiple-sclerosis-patients.html
Mayo Clinic. http://www.mayoclinic.org/medicalprofs/enlargeimage5723.html