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Hello, I am Peter Childs, a Chancellor's Fellow in Biomedical Engineering
at the University of Strathclyde.
Today I will be speaking about the pre-clinical translation
of mesenchymal stem cell therapies.
My own research focuses on the development of bone graft materials
using mesenchymal stem cells.
In this lecture, I will discuss mesenchymal stem cells,
including their characteristics, and some examples of how we might go about
controlling their phenotype for clinical use.
I will then discuss some of the considerations when moving to manufacture
these cells for clinical use, and some of the testing strategies
which need to be considered at the pre-clinical stages.
Surrounding these topics are regulatory frameworks
which vary from country to country, and must be considered when seeking approval to
develop a clinical product, and enter into clinical trials.
Within Europe and the UK, stem cell therapies fall under the category
of advanced therapy medicinal products, or ATMPs.
These are regulated through a set of directives developed over the past 20 years.
The first directive of relevance introduced somatic cell therapy medicinal products
as a class of medicinal agent, along with gene therapies.
Refinements to the regulations later introduced definitions
for tissue-engineered products, and also combination ATMPs,
where cells are combined with a medical device
(for example, the use of a biomaterial scaffold as a carrier for the cells).
Within the USA, the FDA regulates the use of human cells as a therapeutic
under its HCT/P regulation, with two key sections being of interest
when considering mesenchymal stem cells.
Section 361 regulates products which are used in a homologous manner,
such as demineralised bone matrix which contains stem cells,
and then is applied for bone repair.
In practicality, the difference between these two regulatory categories
can mean the difference between a full pre-market review
or not drastically altering the pre-clinical development,
and the final price of the therapy.
Recent introduction of the RMAT designation allows for expedited development of
new therapies, where they tackle life-threatening conditions
or meet significant clinical needs.
In all cases, an early understanding of the appropriate regulatory framework will help
the development of a pre-clinical and manufacturing development plan,
even at the academic stage.