How Genetic
Research Illuminates
the Path to Cures
Research
Category
An Update from Our CEO
At The Michael J. Fox Foundation, we are investing
tens of millions of dollars in genetic research. We
frequently report back to you on the progress this
science is helping us make toward breakthrough
treatments, and even a cure, for Parkinson’s disease.
But how does a genetic finding become a direction
to a new treatment?
with specific genetic mutations, and are working on
finding more of them.
But our great hope is that precision medicine
approaches eventually will benefit a much broader
population of people with PD, including the great
majority of patients — those without known genetic
mutations. Research undertaken with the help of
mutation carriers provides tremendous insight into
cellular function in everyone with the disease, offering
clues researchers follow from genes to other dysfunction
we may be able to target. This journey — from a narrow
to a broad population — is not uncommon in biomedical
research. Statins, for example, now taken by millions
to reduce the risk of heart disease, were first tested and
prescribed exclusively in people with a family history of
high cholesterol.
A brief history of Parkinson’s genetics: 25 years
ago, scientific consensus held that little to no
understanding of Parkinson’s disease was to
be found in our DNA. Ten years later, as the
Human Genome Project was drawing to a close,
this opinion had changed, with about 10 genetic
changes discovered to be linked to PD. As I write
this letter, more than 80 genetic associations have
been implicated in Parkinson’s. Every one of these
gives the field something critical: a starting point to
investigate cellular changes that mark disease — and
to search for new ways to stop them.
With your support and participation, Parkinson’s
research is in the midst of a genetics revolution. I look
forward to continuing to share news with you of the
advances you are making possible. Together, we will
change the course of Parkinson’s history.
Certain genes play such a major role in PD onset
and progression that they are drawing direct lines
to new treatments. Today, experimental drugs are
in human testing to target two of the most common
genetic mutations linked to PD — GBA and LRRK2.
Scientists call these “precision medicine” therapies,
because they precisely target and treat individuals
Thank you for all you do.
17 New Genetic
Links Identified 18,500+ Enrolled
in Fox Insight
In fall 2017, scientists identified 17 new
genetic changes linked to PD. These findings
can help researchers plot the biology of
Parkinson’s and perhaps find new ways to
stop it. Data for this and other genetic studies
are coming from MJFF-led programs such as
the Parkinson’s Progression Markers Initiative
(PPMI). Read more about PPMI on page 5. The MJFF-led Fox Insight study, in a
collaboration with genetic testing company
23andMe, is collecting data on Parkinson’s
genetics and daily life with disease.
Scientists can use this information to
discover new genetic links to PD and learn
more about those they’ve already identified.
Learn more at foxinsight.org.
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Spring/Summer
The Fox Focus
2018