Published on March 31, 2015 at 1:52 PM
A patient's very own skin cells may
hold the key to new treatments and even cures for devastating neurological
diseases. A generous $1 million donation from Mr. J. Sebastian van Berkom, and
critical partnerships with Brain Canada, Laval University, Marigold Foundation
and the FRQS-Réseau Parkinson Quebec are driving an innovative, iPSC (induced
pluripotent stem cell) research platform that will transform research into
Parkinson's and other neurological diseases.
Millions of Canadians are affected
by diseases of the brain such as ALS, Parkinson's and brain tumours, for which
there are limited treatments and no cures. By 2020, neurological conditions
will become the leading cause of death and disability. "Everyone's lives
are touched in some way by neurological disease, says Mr. van Berkom, President
of Van Berkom and Associates Inc." In creating The van Berkom Parkinson's
Disease Open-Access Fund, I hope to change lives and support new research that
will lead to new treatments and one day cures. The iPSC platform is a new
paradigm for neuroscience research and as one of the world's great neuroscience
centres, The Neuro is the place to drive it forward."
"This is the ultimate bench to
bedside paradigm, from patient to the bench, back to the patient," says
Dr. Guy Rouleau, Director of The Neuro. "With a unique interface between
fundamental and clinical research, The Neuro is uniquely positioned to be a
central hub in the iPSC platform. Partnering with Mr. Van Berkom, a generous
and visionary philanthropist, propels The Neuro toward the goal of
significantly deepening insight into disease mechanisms with unprecedented
efficiency."
Patients' skin cells will be
reprogrammed into induced pluripotent stem cells (iPSCs) at Laval University,
under the leadership of Dr Jack Puymirat, and then differentiated at The Neuro
into disease relevant cells for research. For example, in the case of Parkinson's
this could be dopamine neurons. The cells can also be genome-edited, a
state-of-the-art technique that can introduce or correct disease associated
mutations - creating the most accurate disease models. These iPSCs will be made
widely and openly available to researchers across Quebec for neuroscience
research. This open-access approach exponentially increases the likelihood of
breakthroughs in neurological disease.
"The unique and exciting
aspect of this platform is that we are creating the most specific cells for
studying disease using the patient's own tissue, which has distinct advantages
over using generic cells or animal models," says Dr. Edward Fon,
neurologist and co-Director of the Quebec iPSC platform. "Disease models
using human samples are increasingly shown to be far more efficacious in
trials, as they much more accurately mimic the disease condition. In the iPSC
platform, not only can specific mutations be introduced but, cells are from
patients' whose specific clinical history and genetic profile are known, a
first step on the road toward neurological personalized medicine. The Neuro has
access to a large and well-characterized patient population, who can help
create a rich clinically-and genetically-derived registry and biobank. The initial
targets in the platform will be ALS and Parkinson's disease (PD), using
dopamine neurons for PD and both motor neurons and astrocytes for ALS."
Source:
McGill University
http://www.news-medical.net/news/20150331/Patients-own-skin-cells-may-hold-key-to-new-treatments-for-neurological-diseases.aspx
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