MDA 2018 Clinical Conference - Highlights From The MARCH 11-14, 2018 | ARLINGTON, VA - Muscular Dystrophy Association
←
→
Page content transcription
If your browser does not render page correctly, please read the page content below
Highlights From The
MDA 2018
Clinical Conference
MARCH 11-14, 2018 | ARLINGTON, VA.
MDA’s 2018 Clinical Conference Is In The Books!
Unprecedented Times
The Changing Landscape In
Neuromuscular Disease It was a record-setting year for MDA’s annual conference series, with more than 700 attendees including MDA Care Center
multidisciplinary care providers, as well as medical and scientific experts from academia, private practice, government and
industry, gathering in the nation’s Capital to share best practices and learn about and discuss the latest scientific and medical
advances in neuromuscular disease research and care.
Participants representing:
CLINIC
Working together we’ll find treatments and cures
At MDA we know that progress doesn’t happen in isolation; nor does it move in a straight line. That’s why we’re committed
academic institutions MDA Care Centers biotech pharmaceutical companies to bringing together all the key stakeholders in the neuromuscular disease space at events like this. When the world’s top
neuromuscular experts share and compare information, tools, resources and results, everyone benefits — their teams, their
practices, their labs and their research and, most importantly, individuals and families living with neuromuscular disease.
2018 Clinical Conference by the Numbers If you joined us for those four jam-packed days in March, we hope you’ll enjoy this highlights report. If you weren’t able to
make it this year, check out some of the key takeaways and see what you can look forward to in 2019!
750+ Fourteen Four Communication • Collaboration • Networking
Unique
ATTENDEES Days
Sessions collaboration
networking
& exchange
FIFTY+
85+
Platform
Presentations
Poster
Presentations
60+
SPEAKERS &
PANELISTS
Up to 17.AMA PRA Category 1 Credits offeredAn Inspiring Keynote
“With clinical translation, you can achieve heights that you didn’t believe were
achievable,” said Jerry Mendell, M.D. “By and large, what we’ve learned from one trial,
we’ve translated to another.”
Keynote Speaker Jerry Mendell, M.D., founder of the Clinical Translational Gene
Therapy Program at the Research Institute at Nationwide Children’s Hospital, opened
the conference with an inspiring talk about incredible progress underway in the
development of gene therapy for neuromuscular diseases.
Mendell was the principal investigator on a November 2017 article on an SMA
gene therapy trial in which the experimental SMA gene therapy AVXS-101 showed
unequivocal efficacy. Of the 15 infants who participated in the trial, all have achieved
event-free survival for more than 20 months and many have achieved motor
milestones that they otherwise wouldn’t have been expected to achieve. Babies who
would have been expected to die before reaching 2 years old are now holding up
their heads. Sitting up. Breathing with minimal or no assistance. Even crawling and, in
some cases, walking.
“Our experience in SMA has gone a long way,” Mendell said, emphasizing that
learnings about gene delivery in SMA have direct application now to DMD and
perhaps, in the future, other forms of muscular dystrophy.
To Our Strength for Life Supporters…
Thank You!WHAT’S NEW
Amanda Haidet-Phillips
“It’s been so exciting to see
at this conference the results
of these therapies that have
been developed through basic
research,” MDA Scientific
Officer Amanda Haidet-Phillips
said. “It’s been a long road
working to understand, to
find the causes, the genetic
causes of disease, understand
how those genes lead to the
development of pathology,
understand how they’re spliced.
We have spent years trying to
figure out what’s the best way
to develop and to deliver genes
using viruses, what’s the best
virus to use and how to deliver
it. To see it all coming together,
all of these years of research,
at a clinical conference and
seeing these therapies being Kathryn Wagner James Howard
tested now in patients is really
incredible.” DMD MG
Kathryn Wagner, M.D., Ph.D., “This is a great time to be involved
noted: “We are potentially looking in neuromuscular disease,” said
at some game-changing strategies James F. Howard Jr.
for Duchenne.”
Charles Thornton James F. Howard Jr., M.D., FAAN,
Therapeutic strategies currently noted that over the lifetime course
DM approved or under development for of the MG, we are now able to
Charles Thornton, M.D., DMD include immune modulators, reduce incidence of early death
noted, “We are making rapid anti-myostatin drugs, antisense and improve quality of life.
headway” in DM research oligonucleotides and gene therapy
and care. approaches.
After the official welcome and opening remarks, monday’s morning sessions offered a roundup of the
latest news and advances in neuromuscular disease — including als (amyotrophic lateral sclerosis),
duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (fshd), spinal muscular James D. Berry
atrophy (sma), myotonic dystrophy (dm) and myasthenia gravis (mg). ALS
“The really profound answer to down SOD1 protein expression
what’s new in ALS is, everything is in the brain and cerebrospinal
new in ALS,” James D. Berry, M.D., fluid; AAV-delivered SOD1 gene
M.P.H., said. therapy to knockdown SOD1
protein; the mesenchymal stem
Promising therapeutic strategies cells secreting neurotrophic
under development include factors (MSC-NTF) cell therapy;
antisense oligonucleotides to immunomodulation; and
degrade SOD1 RNA and knock combination therapy.PERSONALIZED Murray Aitken
DIGITAL HEALTH
Leonela Amoasii
GENE EDITING IN DMD
MEDICINE
Murray Aitken, MBA, said it’s Leonela Amoasii, Ph.D., discussed
anticipated that digital health a vision for gene editing in DMD.
tools, including mobile health
apps and wearable sensors, Eric Olson’s lab is working to
will accelerate progress for permanently correct the most
neuromuscular diseases — common DMD mutations by
Grace Pavlath including in the areas of wellness using CRISPR/Cas9 to induce
and prevention, symptom onset exon skipping, in an approach
MDA MOVR DATA HUB and seeking care, diagnosis, dubbed myoediting. The team
“MOVR is designed to be the most condition monitoring and has demonstrated correction of
comprehensive registry in the treatment. a number of DMD mutations in
neuromuscular disease space,” muscle cells derived from patient
said MDA senior vice president iPS cells. It now plans to test the
and scientific director Grace strategy in mutation-specific mouse
Pavlath, Ph.D. “This is going to models, followed by preclinical
have a big impact on our ability to studies in large animal models and
transform healthcare and also to clinical testing in DMD patients.
lead to more drugs being tested in
individuals.”
In 2013, MDA launched the
U.S. Neuromuscular Disease
Registry to better understand how
neuromuscular diseases develop
and progress, and to identify
which treatments lead to the best
outcomes in ALS, BMD, DMD and Dennis Matthews
SMA. Now, MDA is expanding
the reach of the registry to 50 “The importance of accurate
sites and seven diseases. It diagnosis is even more
has partnered with IQVIA to important now because with
expand and enhance what is new treatments that are
now the MOVR (NeuroMuscular becoming available, it’s vital
ObserVational Research) that we know specifically what
initiative with a new infrastructure, the gene defect is, what the
electronic medical record data particular form of gene defect
capture, a digital health module, a is, so that we can personalize
user-friendly interface for sites to and tailor the medical
access their own data and state- treatments,” Dennis Matthews,
of-the-art security protection of M.D., said. “Personalized
data. medicine is actually what’s
coming in to the forefront where
your specific diagnosis — your
Learn more: mda.org/ specific gene defect — is the
Monday’s afternoon sessions focused on advances in personalized medicine, including the latest in precision services/neuromuscular- one that we’re going to be able
therapies, genomics, digital health, and MDA’s MOVR initiative. disease-registry to treat.”BEST RISE OF TECHNOLOGY AND
BIG DATA
TRANSITIONING FROM
PEDIATRIC TO ADULT-
PRACTICES
BASED CARE
An area of intense interest was
technology, with presentations Improvements in supportive —
in cognitive computing to help and now disease-modifying —
researchers pinpoint links treatments are helping people
between genes, diseases with neuromuscular disease
and drugs, and remote live longer. Now we must
Elizabeth McNally delivery of health-related consider: How can we help
services and information via individuals shift from pediatric
CARDIAC CARE telecommunications technologies care to adult care? Conference
“I can’t state this enough. (telemedicine). conversations centered around
The cardiologist and the how to help ensure individuals
pulmonologist have to work Tuesday afternoon the focus and families make a smooth
hand-in-hand in treating shifted to best practices in transition.
the neuromuscular disease newborn screening. Augmentative
patient, and that’s across all communication, pediatric-to-adult
the muscular dystrophies transition, respiratory health
and especially those that can and care coordination also were
affect the heart,” Elizabeth covered in parallel sessions.
McNally, M.D., Ph.D., said.
“That’s because fundamentally
what we’re dealing with is a
shortness of breath situation. If
you’re short of breath, that can
come from the lungs, that can
come from the heart, and it can
come from both. It doesn’t have
to just be one or the other. You
really need your cardiologist
and pulmonologist working
hand-in-hand.”
Lauren Elman Robert Griggs
“One of the things that can “…As we come to the stage
be very hard for a young where we have treatments for
NEWBORN SCREENING adult coming into an adult disease, and some evidence
doctor is that they’re leaving that the sooner you start a
Newborn screening (NBS) behind something that was treatment the more likely the
was explored through a half- very comforting, and very patients are to do well, then it
day workshop led by MDA helpful and was present for, becomes incumbent upon us
Chairman of the Board and maybe all of their life that to find a diagnosis as early
international expert in newborn they can remember. Coming as possible and introduce the
screening R. Rodney Howell, into a doctor who knows their treatment before the disease
M.D. It included disease- former doctor, and their former starts,” said Robert Griggs,
specific presentations as team, sort of helps make that M.D., FAAN”
well as an in-depth panel transition much smoother,” said
On Tuesday morning the focus was on best practices in cardiac care, as well as physical therapy, bone discussion about the current
health and nutrition as key components of the multidisciplinary approach to treatment of neuromuscular Lauren Elman, M.D. “I really try
and future state of NBS. to make it clear that I know their
disease.
old doctor and their old team
and that I’ve talked to their
doctor about them, and that this
is just the next natural step.”INSPIRATION
PROGRESS AND INSIGHT
Wednesday’s sessions focused on clinical trial updates, with presenters reporting encouraging results for a Networking, collaboration, and conversations around sharing ideas and brainstorming ways to address new
number of specific current, ongoing or completed studies including trials to test: opportunities and challenges were the essence of the conference.
• Autologous infusion of expanded Tregs to suppress neuroinflammation in ALS
• AVXS-101 gene replacement therapy in SMA1
• Domagrozumab, a myostatin inhibitor, in DMD
• ATB200, a next-generation rhGAA enzyme replacement therapy, co-administered with the oral
pharmacological chaperone AT2221, in Pompe disease
• Spinraza in presymptomatic SMA
• Golodirsen, a phosphorodiamidate morpholino oligomer (PMO), in DMD
• Omigapil in LAMA2 and COL6-related dystrophy
• The utility of glutamate dehydrogenase (GLDH) as a liver-specific biomarker in DMD
• AMO-02 (tigeglusib) in DM1
Barry Byrne Laura Hagerty
“I think it’s an important evolution of the MDA “There are a lot of new technologies that
care centers to include not only expertise in are being described at this conference,
clinical care, but full integration with research including new platform technologies for
opportunities for patients,” said Barry Byrne, making drugs, new ways for collecting and
M.D. “The usual clinical care activities have storing data, and a new push towards doing
to be excellent, but we also have to offer to genetic testing in patients,” said Laura
patients the opportunity to participate in cutting Hagerty, Ph.D., MDA scientific program
edge research studies that will really change officer. “And I think this is really exciting
the trajectory of their disease. So, I think the because the convergence of those, the
care center is now the nucleus of that activity maturation of those three different things,
and that’s why I’m excited that we have such really means that we’re going to be able to
an active group in both expert clinical care and push forward with precision medicines in a
research.” way that we haven’t before.”SAVE THE DATE 2018 CLINICAL CONFERENCE SUPPORTERS
The Muscular Dystrophy Association is grateful to the following companies for supporting the 2018 Clinical
We’re looking forward to next year and hope you’ll plan to Conference and continuing medical education program aimed at optimizing care and helping kids and adults
with muscular dystrophy, ALS and related life-threatening diseases live longer and grow stronger. Together with
join us for the 2019 MDA Scientific/Clinical Conference! our supporters, MDA is fighting to save and improve the lives of families living with neuromuscular disease.
2019 MDA Scientific/Clinical Conference
April 13-17, 2019
Hyatt Regency Orlando
9801 International Drive
Orlando, Fla. 32819
Please email Pam Bittner at pbittner@mdausa.org
for any questions related to this event
More information will be posted within the next few weeks. Stay tuned!MDA is leading the fight to free individuals — and the families who love them —from the harm of muscular dystrophy. ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America.
You can also read
