2006 - A Very Good Year in MS Research
During 2006, rapid research progress
was made in the fields of science and medicine that impact understanding
and treatment of multiple sclerosis, an unpredictable neurological disease.
Significant advances have been made in both clinical and laboratory
studies in MS. In addition, more than 130 clinical trials are underway
around the world, and still other experimental drugs are in the pipeline.
Key highlights of the year include:
* Acorda Therapeutics (Hawthorne, NY) announced positive results of a
Phase 3, placebo-controlled clinical trial of Fampridine-SR, an oral
drug designed to provide symptomatic relief by compensating for lost
nerve conduction. In 301 patients with all types of MS, those on active
treatment showed an average increase in walking speed of 25% versus
those on inactive placebo. The company is expected to meet with the
U.S. Food and Drug Administration (FDA) to determine next steps needed
to apply for marketing approval.
* The FDA approved the return to market of Tysabri(R) (natalizumab,
produced by Biogen Idec and Elan Pharmaceuticals) to delay the
accumulation of physical disability and reduce the frequency of relapses
(clinical exacerbations) in those with relapsing MS. There is now in
place a mandatory registration program for patients and prescribing
physicians to minimize the risk of PML (progressive multifocal
leukoencephalopathy), caused by a common virus called the JC virus. The
drug is dispensed at registered infusion centers across the country.
Since Tysabri's return to market last summer, there have been no new
cases of PML reported.
* Members of the four Nervous System Repair teams from Europe and the U.S.
met to share progress being made in the Society funded Promise: 2010
initiative. The first clinical trials focused on protecting the nervous
system will begin shortly, and trials aimed at repairing damage and
restoring function in people with MS are expected to begin within the
next five years.
* In a first, Johns Hopkins University researchers reported that nerve
cells derived from mouse embryonic stem cells that were transplanted
into rats with spinal cord injury were able to connect with muscles and
partially restore function. While this work was done in a model of
spinal cord injury, it bears relevance to the potential use of cell
replacement to repair damage in MS.
* The Society will be sponsoring the first ever stem cell summit January
16-19 in San Francisco to explore the potential of the complete spectrum
of stem cell options as they might relate to MS research and treatment.
The by-invitation only meeting will bring together some 60 renowned
scientists from around the world to examine stem cell prospects and
strategies in MS. The results of this meeting are expected to set
research directions and priorities to best determine the potential of
all types of stem cells for treating this disease.
* Researchers from the University of California, Los Angeles reported that
administering Androgel(R) (testosterone gel applied to the skin) to 10
men with relapsing-remitting MS significantly improved cognitive
function and slowed brain tissue loss. This study was funded by the
Society's initiative on Gender Differences in MS and is expected to lead
to additional research involving larger numbers of patients to confirm
these early results.
* In another offshoot from the Society's initiative on Gender Differences,
UCLA investigators began the first large-scale trial of a sex hormone
for the treatment of MS. The two-year, controlled clinical trial of
estriol involves 130 women with early relapsing-remitting MS. If
successful, this clinical trial will lay the groundwork for a larger,
definitive trial that could lead to a new treatment option for women
with MS. Its results may also have implications for women with other
autoimmune diseases, such as rheumatoid arthritis.
* Several oral MS therapies continued to progress through the pipeline:
-- a phase II controlled clinical trial of oral fingolimod (FTY720,
Novartis Pharmaceuticals Corp.) in 255 people with active, relapsing
MS found that up to 77% of those taking fingolimod remained free of
relapses over two years; a large phase III trial is now underway;
-- oral cladribine (an immune-modulating drug by Serono), now being
tested in an international Phase 3 clinical trial, has been
designated by the FDA as a "Fast Track Product," which should
expedite its future review;
-- a multicenter, phase II controlled clinical trial of oral BG00012 (an
oral fumarate, Biogen Idec) led to a 69% reduction in active
inflammation on MRI scans in 257 people with relapsing-remitting MS;
-- in an open-label, 144-week extension study of oral teriflunomide (an
agent that may modulate T cells), those on placebo during the
original trial who switched to teriflunomide experienced up to an 85%
decrease in new, active areas of disease activity seen on MRI at week
144.
* Harvard investigators reported that individuals who showed signs of
significant exposure to the Epstein-Barr virus, which causes infectious
mononucleosis and other disorders, were twice as likely to develop MS up
to 20 years later. The study, funded in part by a grant from the
National MS Society, adds to previous evidence linking the virus to the
risk of developing MS, but does not prove that EBV actually causes MS.
Other recent studies have suggested that smoking cigarettes may
contribute to the risk of MS and MS progression, and that higher vitamin
D intake may help protect against developing MS.
* For the first time, the needs of children who develop MS-like symptoms
are being addressed through the Society's nationwide network of
comprehensive Pediatric MS Centers of Excellence, launched early this
year. The 6 centers have committed to sharing critical resources and
best practices such as MRI protocols and neuropsychological evaluations
so that all families can benefit from the collective knowledge of the
entire network. In addition to providing optimal care and support,
these centers will build a framework for research into this patient
population, which may also provide clues to adult MS.
* Two genes that may contribute to making a person susceptible to
developing MS have been identified by a group of European researchers
known as the "GAMES" Collaborative Group. MS involves an immune-system
attack on the body's own brain and spinal cord, and many genes are
thought to contribute to susceptibility. The two candidate genes were
singled out because they encode for a brain tissue component and an
immune component. This work was supported in part by the National MS
Society.
* Researchers at Stanford University have uncovered evidence they believe
may explain the role of a protein, osteopontin, in stimulating repeated
relapses and disease progression as well as inhibiting spontaneous
recovery from symptoms. This research, sponsored in part by the
Society, could lead to new therapeutic approaches that target
oseopontin's effect in the MS disease process.
* The National MS Society launched a new postdoctoral fellowship program
in MS rehabilitation research. The immediate goal is to recruit and
train talented clinician-scientists in rehabilitation research specific
to MS; the ultimate goal is to get more hands and minds working on ways
to help people with MS maximize their abilities.