Category
Research
Research
News
Briefs
POSITIVE LRRK2
RESULTS IN HUMAN
STUDIES
In August, San Francisco
biotech Denali Therapeutics
announced that its
experimental LRRK2 drug
DNL201 was safe and met
its target goals in a study
in healthy volunteers. The
company is now planning trials
in patients with Parkinson's,
including those with and
without a LRRK2 mutation.
An estimated three percent
of people with Parkinson’s
have a LRRK2 mutation, which
increases activity of the LRRK2
protein. Recent findings from
a Foundation-funded study
showed that people with
idiopathic (i.e., cause unknown)
Parkinson’s also have increased
LRRK2 activity, meaning drugs
like Denali’s may work for a
widespread population — not
just those with the mutation.
GENE THERAPY
ADVANCES MJFF PARTNERS WITH
IBM ON DATA MINING
Two treatments that introduce
genes into the brain to
help boost what is lost with
Parkinson’s disease have hit
recent milestones. Voyager
Therapeutics started a Phase II
trial of its gene therapy to help
the brain convert levodopa
(Sinemet) to dopamine and
control movement symptoms.
Our Foundation funded early
development of this therapy at
the University of California, San
Francisco. And biotech Prevail
got a $75 million investment
to advance its gene therapy to
address GBA gene mutations,
a leading genetic cause of
Parkinson’s. A $1 million Foundation grant
to IBM Research will apply
sophisticated data analysis
technologies to look for
characteristics (e.g., genetic
mutations, demographics,
protein levels) shared in
common among different
Parkinson’s disease subtypes.
IBM is combing data from
the Foundation’s Parkinson’s
Progression Markers Initiative
study to create predictive
models of, for example, fast
progressors, or people who
have more problems with
tremor than with gait. These
models could help researchers
improve study design and
select appropriate participants.
6
The Fox Focus