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0:00
My name is Dr. Mariah Lelos.
I'm a Reader at
Cardiff University,
and I'm going to
be talking about
cell replacement therapy for
neurodegenerative diseases.
0:13
During this talk, I'm
going to be discussing
both Parkinson's disease
and Huntington's disease.
For both conditions,
I'm going to tell you
why scientists are interested in
developing cell
replacement therapies.
I'm going to give
you a brief overview
of some key lessons
that we've learned from
clinical trials and
some key lessons
that we've learned from
pre-clinical models
of Parkinson's and Huntington's.
Then I'm going to
describe how close we are
to the clinic for each
of these conditions.
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Parkinson's disease is
the second most common
neurodegenerative disease.
The cardinal symptoms of
Parkinson's disease include:
tremor, bradykinesia, which
is a slowness of movement,
rigidity, and
postural instability.
But there are a wide
range of other cognitive,
neuropsychiatric, and
non-motor symptoms
that are also associated
with Parkinson's disease.
The key pathological features
that we see in Parkinson's are
a dramatic loss of dopamine
in the nigrostriatal pathway.
As you can see in this figure,
there is a cell body that
resides in the substantia nigra
and these are the
dopamine cells.
They then send projections
up to a brain region
called the striatum, where
they release the dopamine.
In Parkinson's disease,
we get dramatic degeneration
of the substantia nigra,
so these dopamine cells
in the substantia nigra,
and as a consequence, we get
a dramatic loss of
dopamine in the striatum.
Post-mortem tissue reveals
there's loss of
pigmented cells in
the substantia nigra and
the corresponding loss
across the entire what we
call nigrostriatal pathway.
The second pathological
feature of
Parkinson's disease is the
aggregation of a protein
called Alpha-synuclein,
which can be
found in these dopamine
neurons and around
other areas in both the
central nervous system
and the peripheral
nervous system.
