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Printable Handouts
Navigable Slide Index
- Introduction
- Overview
- Parkinson's Disease (PD)
- Symptomatic therapies and procedures
- Alternative: cell transplantation
- Dopamine cells for PD?
- Fetal versus stem cell
- Pros and cons
- Parkinson's graft model
- Fetal grafts improve motor deficits
- Fetal grafts improve non-motor deficits
- First PD clinical trials - fetal grafts
- What do we do next?
- Generating stem cell-derived dopamine neurons
- DA neuron development
- Generating hPSC-dervied dopamine neurons
- hPSC-derived grafts improve motor deficits
- hPSC-derived dopamine grafts
- hESC-derived DA cells versus fetal VM (1)
- hESC-derived DA cells versus fetal VM (2)
- Overall differences and similarities
- Overview of hPSC strategies
- First-in-man clinical trials (1)
- First-in-man clinical trials (2)
- Huntington's Disease (HD)
- Genetics of HD
- Pathology and symptoms
- Why transplant in HD?
- Animal models of striatal repair
- Fetal grafts improve motor function in HD models
- Learning to use the transplant
- Clinical trials of fetal striatal grafts in HD
- Clinical trials of striatal grafts in HD: long-term
- Stratal transplantation in HD
- hPSC-derived medium spiny neurons (1)
- hPSC-derived medium spiny neurons (2)
- hPSC-derived medium spiny neurons (3)
- hPSC-derived medium spiny neurons (4)
- Summary
- References (1)
- References (2)
Topics Covered
- Parkinson’s Disease and therapies available
- Preclinical data supporting cell therapies for Parkinson’s
- Clinical data on fetal transplant trials in Parkinson’s
- Development of stem cell-derived dopamine neurons
- Huntington’s Disease and therapies available
- Preclinical data supporting cell therapies for Huntington’s
- Clinical data on fetal transplant trials in Huntington’s
- Development of stem cell-derived medium spiny neurons
Talk Citation
Lelos, M.J. (2022, January 31). Cell replacement therapy for neurodegenerative diseases [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved November 4, 2024, from https://doi.org/10.69645/ILYN7529.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Mariah J. Lelos has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
A selection of talks on Neurology
Transcript
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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,