Chemistry Reference
In-Depth Information
manufacturing/regulatory pathways. Somatic cells (stem cell derived) are
alternatives to current recombinant cell lines.
It is now possible to develop disease-specific stem cells and rapidly generate
panels with ranges of genetic phenotypes. Consequently, a more accurate
prediction of how a drug will behave across a mixed population is possible.
Another important potential application of stem cells lie in the discovery of
regenerative drugs that could promote cells to repair lost/diseased tissues for cases
such a heart failure and stroke.
Currently, difficulties in directing routinely stem cell differentiation
in vitro
,
hampers development of their full potential. In addition, there are difficulties
associated with high pure populations, high yield, large scale, reproducibility and
cost effective challenges.
The study of disease progression and pathology
in vivo
should soon be routinely
possible. This should be enabled by the ability to generate disease specific iPS
cells (reprogramming adult somatic cells to a pluripotent state).
Current drug screening methods face many challenges/difficulties. They rely
largely on usage of recombinant transformed cell lines that express the target of
interest. The use of physiologically relevant primary cells is desirable. These are
difficult to obtain and it is challenging to obtain sufficient numbers for high-
throughput screening applications. There is also the problem of variability
between donor and preparation of cell batches.
An attractive alternative is offered by stem cells, which can be propagated for
prolonged periods, be cryopreserved and can differentiate to relevant cell types.
Large batches of cells can be generated for use in experiments and screens. iPS
cells can also yield disease specific somatic cells. They can yield panels of stem
cells with different genetic phenotypes. Genetic effects on drug performance can
be investigated.
HTS screens have reported cells differentiated into specific receptors and
pharmacologically responsive to specific compounds. Lineage selection
technology is then used. As cells differentiate, drug selection can be applied such
