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MULTIPLE SCLEROSIS (MS)
Clinical
Multiple sclerosis is characterized by
multifocal CNS demyelination disseminated temporally and
anatomically. Acute symptoms are primarily due to inflammation
of white matter, but chronic disability is primarily due
to axonal damage and loss. MS affects 85,000 in the UK,
400,000 in the US and 2.5 million people worldwide. It
typically is diagnosed between the ages of 20 and 50. The
exact cause is unknown, although genetic factors and environmental
factors, possibly mediated through viral infections and
autoimmunity are believed to be the principle interactors.
The current therapies consist of steroids
and immunomodulatory agents. A short course of high dose
steroids speeds up the recovery of an acute relapse, but
doesn’t alter the eventual outcome. Immunomodulatory
agents such as beta-IFN or azathioprine decrease the relapse
rate by 1/3 in patients with the relapsing / remitting
form of the disease. Their effect on longer term disability
is limited and debated. Campath very effectively decreases
relapse rate. A trial to assess its effect on longer term
disease progression, if administered very early in the
disease history, is currently ongoing. All of the above
drugs have significant adverse side effects.
DanioLabs approach
DanioLabs aims to find compounds
which decrease the number of episodes of demyelination
and
accelerate remyelination. Most importantly the aim is
to enhance axonal survival, given that this is the major
cause of long term disability. Relapses and demyelination
can be stopped, but disability still ensues through axonal
loss.
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