They have the unique ability to divide and produce copies of themselves or other kinds of cells, and, importantly, to replace damaged cells. This is why there is such interest in using stem cells in regenerative treatments and to treat diseases such as cystic fibrosis.
Initially, stem cells were derived from embryonic sources (eSC), which was very controversial and restricted their application. In recent years, new different types of stem cells have been identified and one of these is known as inducible pluripotent stems cells or iPS or iPSC.
Mature cells such as blood cells can be converted to iPSCs and then converted to another cell type, for example a liver cell. This means that any individual’s own blood cells can potentially be converted to the specific cells required for tissue repair. However, there is still quite a lot of research required to fully understand how to “programme” cells precisely.
Stem cell research for cystic fibrosis
The key for cystic fibrosis, then, is to correct the CFTR mutation in the stem cells, so that they can then replace the faulty cells with corrected versions.
The Trust is funding research to correct the mutation in these iPS stem cells. Achieving this in the laboratory is relatively straightforward; the big challenge is how to convert this to help people directly.
The Cystic Fibrosis Foundation in the US is also investing in research into stem cells.
Stem cell therapies hold enormous promise for the future, but the work is still at an experimental stage. If stem cell therapy emerges, it will need to be regulated by government agencies such as the FDA and EMA and be scrutinised as rigorously as any new treatment for cystic fibrosis.
In a special blog, Director of Research Dr Janet Allen takes a closer look at stem cell research.
Travelling abroad for stem cell therapy
There is a treatment being offered in the Dominican Republic that has received a lot of coverage in the media, but has not been approved by the regulatory authorities in the USA (the Food and Drug Administration) or Europe (the European Medicines Agency). There is currently no approved scientific evidence that the procedure is safe or effective, and the Trust does not encourage people living with cystic fibrosis to currently undertake any type of stem cell treatment in the UK or abroad.
Our Chief Executive, Ed Owen, has written a blog on the issues arising from this work, and why the Trust does not recommend it.
We’ve put together some Frequently Asked Questions about stem cell research.