'Ground-breaking'
new drug gives hope in Huntington's disease
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[December 12, 2017] By
Ben Hirschler
LONDON (Reuters) - Scientists have for the
first time fixed a protein defect that causes Huntington's disease by
injecting a drug from Ionis Pharmaceuticals into the spine, offering new
hope for patients with the devastating genetic disease.
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The success in the early-stage clinical trial has prompted Roche to
exercise its option to license the product, called IONIS-HTT(Rx), at
a cost of $45 million.
Lead researcher Sarah Tabrizi, professor of clinical neurology at
University College London, said the ability of the drug to tackle
the underlying cause of Huntington's by lowering levels of a toxic
protein was "ground-breaking".
"The key now is to move quickly to a larger trial to test whether
IONIS-HTT(Rx) slows disease progression," she said in a statement on
Monday.

Ionis senior vice president of research Frank Bennett said the
protein reductions observed in the study "substantially exceeded our
expectations" and that the drug was also well tolerated.
However, experts cautioned that the results were still early and the
ability of the new medicine to improve clinical outcomes for
patients had yet to be demonstrated.
"The question is whether this is enough to make a difference to
patients and their clinical course, and for that we will have to
wait for bigger trials," said Roger Barker of the University of
Cambridge, who was also involved in the research.
Huntington's is a progressive neurodegenerative disease affecting
mental abilities and physical control that normally hits sufferers
between the ages of 30 and 50 years before continually worsening
over a 10- to 25-year period.
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There is currently no effective disease-modifying treatment for the
condition, with existing medicines focused only on managing disease
symptoms.
Ionis said Roche would now be responsible for all IONIS-HTT(Rx)
development, regulatory and commercialization activities and costs.
The drug uses an approach called antisense to stop a gene producing
a particular protein. The technique has already led to a drug for
spinal muscular atrophy that was approved last year.
Shares in Ionis rose around 2 percent in early Nasdaq trade as did
those in Wave Life Sciences, which is also working on antisense
medicine.
(Reporting by Ben Hirschler; Editing by Gareth Jones)
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