New Drug Shows Promise Against Huntington's Disease
Study suggests pridopidine eases symptoms, but doesn't attack origins of the disease.
MONDAY, Nov. 8, 2011 (Health Day News) — an investigational drugs called pridopidine look
like an effective and harmless treatment for people with the advanced movement
disorder Huntington's disease, researchers report.
According to the research
authors, this is the 1st drug exposed to improve patients' loss of ability to
move their muscles voluntarily. The only drug currently approved for
Huntington's is tetrabenazine, which treats only involuntary movements and can
cause serious side effects.
Huntington's
patients have an imbalance in the signaling chemical dopamine. The new drug
stabilizes dopamine signaling in areas of the brain that control movement and
coordination.
After 6 months of
treatment, patients taking the higher dosage of pridopidine showed improvements
in motor function — specifically in eye and hand movements, involuntary muscle
contractions (dystonia), and gait and balance — compared with patients taking
the placebo.
More than seventy percent of the patients taking the higher
dosage of the drug showed a significant benefit, according to the studies. Side
effects among patients taking the drug were similar to the placebo group.
The study included Four
Hundred Thirty Seven Huntington's disease patients from eight European
countries. The participants took either pridopidine (Forty Five milligrams once
daily or 45 mg twice daily) or a placebo for 26 weeks.
"Pridopidine
has the potential to complement available treatments by improving a different
range of motor deficits. Its lack of severe side-effects . . . suggests that
pridopidine might be useful even for those patients who are treated at sites
that are not centers of excellence for Huntington's disease," the
researchers concluded.
"There has been
a great deal of attention for this drug, as it is one of the few molecules that
in preliminary studies was found to have some efficacy in Huntington's
disease," said Dr. Alessandro Di Rocco, professor in the department of
neurology and chief of the division of movement disorders at NYU Langone
Medical Center in New York City.
One United States
neurologist agreed that the drug seems promising against a disease with few
treatment options.
"However, this
is the first large study to show evidence of a positive result in treating the
motor symptoms of this devastating disease. And, though the compound is
apparently well tolerated without significant side effects, the benefit is
modest and limited to the motor symptoms of the disease and it is unknown how
long the improvement observed could last," he added.
"Unfortunately,
Huntington's is a progressive disease and this drug is not a treatment of the
disease itself but only improves some of its symptoms," he explained.
"Nevertheless, it is a welcome addition because there is very little else
yet available to treat the symptoms.
The challenge for scientists and
clinicians is to discover therapies that actually slows or halts the
progression of the disease."
"A
well-tolerated drug that produces even small benefits for patients with
Huntington's disease would be a very welcome addition to the currently
available treatments for this debilitating disorder," Andrew Feign, from
The Feinstein Institute for Medical Research in New York City, wrote in an
accompanying commentary.
The study was funded
by European pharmaceuticals company Neuro Search A/S.
MONDAY, Nov. 8, 2011 (Health Day News) — an investigational drugs called pridopidine look
like an effective and harmless treatment for people with the advanced movement
disorder Huntington's disease, researchers report.
According to the research
authors, this is the 1st drug exposed to improve patients' loss of ability to
move their muscles voluntarily. The only drug currently approved for
Huntington's is tetrabenazine, which treats only involuntary movements and can
cause serious side effects.
Huntington's
patients have an imbalance in the signaling chemical dopamine. The new drug
stabilizes dopamine signaling in areas of the brain that control movement and
coordination.
The
results of the phase 3 clinical trial, conducted by Spanish researchers led by
Dr. Justo Garcia de Yebenes, of the department of neurology, Hospital Ramon y
Cajal in Madrid, appear in the November 8 online edition of The Lancet Neurology.
After 6 months of
treatment, patients taking the higher dosage of pridopidine showed improvements
in motor function — specifically in eye and hand movements, involuntary muscle
contractions (dystonia), and gait and balance — compared with patients taking
the placebo.
More than seventy percent of the patients taking the higher
dosage of the drug showed a significant benefit, according to the studies. Side
effects among patients taking the drug were similar to the placebo group.
The study included Four
Hundred Thirty Seven Huntington's disease patients from eight European
countries. The participants took either pridopidine (Forty Five milligrams once
daily or 45 mg twice daily) or a placebo for 26 weeks.
"Pridopidine
has the potential to complement available treatments by improving a different
range of motor deficits. Its lack of severe side-effects . . . suggests that
pridopidine might be useful even for those patients who are treated at sites
that are not centers of excellence for Huntington's disease," the
researchers concluded.
"There has been
a great deal of attention for this drug, as it is one of the few molecules that
in preliminary studies was found to have some efficacy in Huntington's
disease," said Dr. Alessandro Di Rocco, professor in the department of
neurology and chief of the division of movement disorders at NYU Langone
Medical Center in New York City.
One United States
neurologist agreed that the drug seems promising against a disease with few
treatment options.
"However, this
is the first large study to show evidence of a positive result in treating the
motor symptoms of this devastating disease. And, though the compound is
apparently well tolerated without significant side effects, the benefit is
modest and limited to the motor symptoms of the disease and it is unknown how
long the improvement observed could last," he added.
Still, pridopidine
does not get at the underlying cause of Huntington's disease, Di Rocco said.
"Unfortunately,
Huntington's is a progressive disease and this drug is not a treatment of the
disease itself but only improves some of its symptoms," he explained.
"Nevertheless, it is a welcome addition because there is very little else
yet available to treat the symptoms.
The challenge for scientists and
clinicians is to discover therapies that actually slows or halts the
progression of the disease."
Another expert
agreed that new treatment options for patients are sorely needed.
"A
well-tolerated drug that produces even small benefits for patients with
Huntington's disease would be a very welcome addition to the currently
available treatments for this debilitating disorder," Andrew Feign, from
The Feinstein Institute for Medical Research in New York City, wrote in an
accompanying commentary.
The study was funded
by European pharmaceuticals company Neuro Search A/S.
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