Stem Cells in A-T research

Most cells in the body have the ability to reproduce themselves. However, only a few, known as stem cells are able to generate cells of different kinds. This is like the very few cells in the embryo when a child is first conceived, which over time are able to turn themselves into all the different cells found in the body.

While these embryonic stem cells have the capacity to produce virtually any other cell,  the stem cells that remain in people once they are born generally only produce a limited range of cell-types related to the organ or system where they are found.

Stem-cell research has two principal directions:

A tool for research

Stem cells are used to produce living cells such as neurons (brain cells), which we could not otherwise have access to for research purposes. We can't take cells from the brains of people with A-T. However, we can take cells from their skin or blood and 're-programme' them to become stem cells. These cells are known as iPSCs (induced pluripotent stem cells) The next stage is to turn iPSCs into the types of brain cell, particularly purkinje cells, which are affected in people with A-T. When we can do this, it will help us understand what makes these cells vulnerable, and screen drugs to make them more stable.

The production of stem cells for research, is well advanced in a number of laboratories, with researchers using different cells as a starting point (skin cells, blood cells and in one case, cells from inside the nose).  However the big challenge is to turn these stem cells into neurons and other types of brain cell. While work is going on to do this, it is not easy to produce these complex cells in laboratory dish.

A means of therapy

We know that in A-T,cells in the cerebellum die off, causing ataxia and other neurological problems. If we could use stem cells to create new cells, we might be able to mitigate the effects of the condition. Stem cells in the brain also produce other substances that are protective of cells. This could be another means of helping brain cells survive better.

The therapeutic approach has a lot of problems to overcome before it can become a reality These include producing the right stem cells, getting them to the right place, ensuring that they produce only cells that are needed and so on.

Induced pluripotent stem-cells

Induced pluripotent stem cells (iPSCs)

 

A researcher with a test tube

Researcher at the Gurdon Institute, Cambridge