Medical Organization Says Biogen-Supported Study of Experimental Drug Shows Promise for Genetic Form of ALS
May 02 2019 - 1:48PM
Dow Jones News
By Stephen Nakrosis
An early stage trial of an experimental drug to treat
amyotrophic lateral sclerosis, more commonly known as Lou Gehrig's
disease, has linked the treatment to possible slower progression in
people with a certain form of the disease, the American Academy of
Neurology said on Thursday.
The study involving tofersen was supported by Biogen Inc.
(BIIB). Biogen licensed the global rights to develop, manufacture
and commercialize tofersen (BIIB067) from Ionis Pharmaceuticals
Inc. (IONS), the academy said.
The trial looked at a genetic form of ALS caused by mutations in
a gene called superoxide dismutase 1.
The study involved 50 people who had ALS with an SOD1 genetic
mutation. Participants received five doses of either the
experimental drug, or placebo. Researchers found patients on a
100-mg dose scored better on tests measuring breathing capacity,
muscle strength and how well they functioned at activities, when
compared to those given a placebo.
Researchers also found some of the patients given 100 mg of the
experimental drug had a 37% reduction of the SOD1 protein in spinal
fluid when compared to 12 who received the placebo.
"Lower concentrations of the protein in the spinal fluid suggest
that there were also lower concentrations in the brain and spinal
cord. Such reductions could lead to preservation of motor neurons
and slow progression of the disease, but more study is needed to
examine this further," said study author Dr. Timothy M. Miller.
--Write to Stephen Nakrosis at stephen.nakrosis@wsj.com
(END) Dow Jones Newswires
May 02, 2019 13:33 ET (17:33 GMT)
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