Spring / Summer 2020 | Research
5
Following the
Parkinson’s
Pipeline
Toward Cures
by KRISHNA KNABE
Genetics has cracked open entirely new avenues
of exploration and discovery toward nextgeneration
treatments with potential to slow
or stop the progression of Parkinson’s disease
— something no currently available therapy
can do. Today, several potential treatments
to slow or stop disease progression are being
tested in people with and without genetic
mutations. We expect to see some of these
results in the next 18 to 24 months. The
Michael J. Fox Foundation (MJFF) is tracking the
progress surrounding therapies in our priority
programs: alpha-synuclein, LRRK and GBA.
Alpha-synuclein
One of our most promising targets, this protein
clumps in brain cells of nearly everyone with
Parkinson’s. Today, there are now 13 therapies
targeting alpha-synuclein in clinical trials,
including vaccines, antibodies and traditional
small molecule drugs (e.g., drugs in pill form).
MODAG Neuroscience Solutions’ small molecule
anle-138b began Phase I testing in December
2019. Affiris, the company behind a vaccine
approach called Affitope PD01A, announced
plans to begin Phase II testing later in 2020.
Both programs received funding from MJFF.
LRRK2 and GBA
Therapies for two genetic targets — LRRK2
and GBA — are making strides. Denali
Therapeutics has two small molecule LRRK2
inhibitors in clinical testing. Biogen has
also started its trial of an antisense therapy
for LRRK2, which is a unique gene therapy
approach. Meanwhile, there are now five
therapies in trials targeting GBA, using small
molecule and gene therapy approaches.
Emerging Targets
In addition to these established targets, many
emerging targets have also reached clinical
trials. This progress is the result of MJFF’s
efforts to translate new findings about the
causes of Parkinson’s into therapies to treat
it, as quickly as we possibly can. Among these
are six therapies focused on inflammation,
which has been shown to contribute to cell
death in the brain, and three being tested
against mitochondrial impairment, which is a
failure of our brain’s energy system.
MJFF is hopeful that today’s diverse and
rapidly advancing pipeline of Parkinson’s
therapeutics will become tomorrow’s cures.