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0:00
Hi, my name is Dr. Mariah Lelos.
I'm going to be talking about cell replacement therapy for neurodegenerative diseases,
with a particular focus on Parkinson's disease and Huntington's disease.
I'm an Associate Professor in Neuroscience at Cardiff University in the United Kingdom.
0:20
During this lecture,
I am going to be talking about why we're interested in using cell replacement therapies.
I'll talk a little bit about the background to the field,
where we've studied fetal and stem cell-derived cell therapy products,
and for the majority of the lecture,
I will be covering what we have learned from
clinical trials and from a wide range of preclinical studies,
mainly fields of Parkinson's and Huntington's disease.
Finally, I'm going to be exploring how close we
are to getting these therapies into the clinic.
0:51
Parkinson's disease is the second most common neurodegenerative disease in the world.
It can affect up to 10 million people worldwide.
The cardinal symptoms of the disease are largely motor,
so they include tremor,
bradykinesia, which is the slowness of movement,
rigidity, and postural instability.
But, in fact, there is also a wide range of
non-motor symptoms that are associated with Parkinson's disease,
which include autonomic nervous system dysfunctions,
dementia, cognitive changes,
pain, and a wide range of other symptoms.
In Parkinson's disease,
one of the main pathological events that occurs is
the degeneration of the dopamine neurons in the substantia nigra.
These dopamine neurons project to the striatum,
where they release dopamine.
When we have degeneration of these dopamine neurons,
we have a corresponding loss of this whole nigrostriatal pathway.
The second major pathological hallmark of Parkinson's disease
is the formation of alpha-synuclein-rich Lewy bodies,
which can be seen throughout
the central nervous system and the peripheral nervous system,
and they are highly abundant within the substantia nigra dopamine neurons.
There are a number of therapies that are associated with Parkinson's disease,
