Results of a phase II
clinical trial shows the drug dexpramipexole slows the progression of
amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease.
Currently, there are
only two drugs that treat ALS. The disease progressively causes death of nerve
cells in the brain and spinal cord that ultimately leads to paralysis and death
from respiratory failure.
Merit Cudkowicz, MD,
director of the Massachusetts General Hospital (MGH) Neurology Clinical Trials
Unit and ALS Center, lead author of the study said, "We need more
therapies to slow, halt and ultimately reverse the course of disease and also
therapies to treat the symptoms."
The two drugs available
for ALS treatment are riluzole, which extends life about 10 percent, and
Nuedexta, which treats the emotional instability associated with Lou Gehrig’s
disease.
Earlier this year,
researchers from Northwestern University announced a breakthrough in
understanding the cause of ALS, or Lou Gehrig’s disease. In all cases, the
disease occurs as the result of a breakdown of protein recycling in the brain
and spinal cord. The Northwestern
finding should also lead to new treatments within the next 5 to 10
years.
For the trial, patients
were tested in two phases. The first group of 102 patients was split into 4
groups; all had been recently diagnosed with ALS.
Patients in the study
were given either and oral placebo or dexpramipexole at total daily dosages of
50, 150 or 300 mg for 12 weeks.
After 12 weeks the
participants were given just placebo for four weeks; then split again into two
groups and given either 50 or 300 mg of dexpramipexole for 24 weeks.
The results showed 300mg
of the experimental drug slowed progression of Lou Gehrig’s 30 percent,
compared to placebo. The finding was the same in the second stage of the study.
"Since individual
participants could have been in different treatment groups in the first and
second stages of the study, seeing the same dose-dependent differences at both
stages gives us confidence in the data. In a way, this was two supportive
studies in one trial design," says Cudkowicz.
The drug is now in phase
III clinical trials. Dexpramipexole is licensed for development by Biogen Idec and was initially
developed by Knopp Biosciences of Pittsburgh.
Dexpramipexole seems to
work for treatment of Lou Gehrig’s disease by protecting mitochondria in nerve
cells from becoming dysfunctional. In the phase II trial, the drug was shown to
extend lifespan for patients with ALS by 30 percent
-November 22, 2011.
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