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Research
News
New treatment for Pompe
Disease Approved
On April 28th 2006 the Food
and Drug Administration announced approval of Myozyme,
a new treatment for patients with Pompe disease, a rare
genetic disorder also known as acid maltase enzyme
deficiency. The condition is one of more than 40
neuromuscular diseases covered by the Muscular Dystrophy
Association (MDA), which sponsored early research in Pompe
disease and also helped support clinical testing of Myozyme.
"This is the first
definitive treatment for a genetic neuromuscular disease in
our program, and this important announcement represents the
culmination of many years of intensive research," MDA
Medical Director Valerie Cwik said. "Everyone who has ever
donated to MDA can take pride in their role in helping to
bring about this lifesaving achievement. This news also
offers hope to the hundreds of thousands of other people
affected by the diseases in the MDA program, because it
shows that support and research can lead to successful
treatments."
U. of Rochester Initiates
MDA-Funded
Clinical Trial of Insmed's iPlex for
Myotonic MD
Insmed Incorporated, a
Richmond, Va., biotechnology company, the University of
Rochester School of Medicine and the MDA announced today the
initiation of a Phase 2 clinical study investigating the use
of iPlex, a once-daily Insulin-like Growth Factor-1 (IGF-1)
therapy, for the treatment of myotonic muscular dystrophy (MMD),
the most common form of adult muscular dystrophy.
It has been known for
decades that MMD patients do not respond normally to
insulin. Recent research has identified an abnormality in an
insulin receptor protein as the underlying cause and IGF-1
as a potential remedy.
Myotonic dystrophy
affects an estimated 40,000 people in the United States and
causes progressive muscle wasting and weakness in the hands,
forearms, legs, neck and face. It often involves many other
systemic effects, including endocrine abnormalities,
especially with respect to insulin, a regulator of blood
sugar (glucose); neurological changes, including excessive
sleepiness and apathy; cataracts, usually requiring surgical
excision; gastrointestinal problems; and cardiac rhythm
abnormalities, often requiring pacemaker insertion.
The disease can lead to
severe disability, and death can result from respiratory
muscle weakness or fatal cardiac dysrhythmias.
At present, there is no
treatment to reverse the muscle weakness or wasting or the
defective insulin utilization in MMD.
The Phase 2 study to
investigate the safety and tolerability of once-daily
subcutaneous injections of iPlex in patients with MMD will
involve two sequential studies each involving 15 patients.
The first study is a 24-week, dose-escalation study of iPlex
to identify an optimal dose for the subsequent 24-week,
fixed-dose study. Both studies will evaluate a number of
safety parameters in a prospective manner, as well as
several key efficacy measures such as muscle mass and
strength.
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Local news
Dr. Marinos
Dalakas Comes
to OHSU
For the 2007
annual Neuromuscular Grand Rounds, The Neuromuscular Disease
Center was proud to welcome Dr Marinos Dalakas, director of
the NIH/NINDS section on Neuromuscular Diseases. Dr Dalakas
is a renowned expert in Inflammatory diseases and spoke to
the OHSU community on Stiff Person Syndrome. Following his
lecture, Dr Dalakas met with Residents and medical students
to advise them on the practice of diagnosing Neuromuscular
Diseases.
Dr. Stanley Appel
visits OHSU
The OHSU Neuromuscular
Disease Center was proud to host Dr Stanley Appel's visit to
Oregon in March. Dr Appel is a world renowned expert in
neuromuscular diseases, especially ALS. Dr Appel spoke to a
large group of local neurologists about his cutting edge
research in the treatment of ALS.
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ALS
Caregiver
Conference,
October 2005
On October 29th,
2005 the MDA ALS Division and the ALS Center of Oregon
presented the 2nd annual ALS Awareness & Wellness
Symposium at the Kingstad Conference Center in Beaverton,
OR. This symposium focused on symptom management and
caregiver support. Many of the regions top ALS researchers
were on hand to give presentations and answer questions. The
MDA and OHSU present many such symposiums for patients,
caregivers, and healthcare providers. For more information
on future ALS education opportunities please contact Sarah Johnson (johnssar@ohsu.edu
),
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Updates in
Neuromuscular
Disease
On Saturday January 21, 2006
physicians and healthcare providers from across Oregon,
Washington and California came to the Portland Hilton and
Executive Tower to learn the latest information on the
diagnosis and treatment of neuromuscular diseases. This all
day event was hosted by OHSU's Neuromuscular Disease Center,
and covered topics related to muscular dystrophy, ALS,
hereditary neuropathies, spinal muscular atrophy, and Pompe
disease, as well as general updates for treatments of
neuromuscular diseases.
Photos from the conference:
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