DEBRA International Stem cell research and EB

DebRA Research

Stem cell research and EB – what does it all mean?

Everyone at DebRA knows that finding a cure for EB is the ultimate aim of all research. Everyone also knows that this isn’t easy, but it’s true to say that considerable progress is being made.

Although definitive “cures” may not be on the horizon yet, new strategies to develop better treatments are emerging, and DebRA currently funds several international projects that are focused on developing new therapies for people with EB.

These new treatments involve gene, protein and cell therapies, all of which are designed to restore or correct the primary faults that cause EB.

Of course, researchers, and everyone whose lives are directly or indirectly affected by EB, are always on the look out for better and more effective ways to speed up progress.

With that in mind, many people will have been aware of the recent media interest in “embryonic stem cells” and will be wondering whether or not this might be relevant to EB research?

At present, the gene therapy research that DebRA funds does involve work on stem cells, but this mainly involves skin stem cells – small groups of cells (keratinocytes) from within the epidermis or hair follicles that are capable of surviving and making generations of other skin cells that can go on to make new skin.

By targeting gene therapy to these stem cells, it is hoped that the benefits will last for a long time rather than just a week or two if any non-stem cell keratinocyte receives the new gene therapy. Other EB researchers are also studying stem cells in the dermis and in the bone marrow but, in essence, these strategies all involve attempts to manipulate “adult” stem cells.

The other type of stem cell, however, that researchers are also now focusing on, are the embryonic stem cells.

These human cells, which were first identified in 1998, represent part of an early embryo, known as the inner cell mass.

Their importance to modern biology and medicine derives from two unique characteristics that distinguish them from all other organ-specific stem cells identified to date. First, they can be maintained and expanded as pure populations of undifferentiated cells for extended periods of time, possibly indefinitely, in culture. Secondly, they are pluripotent, which means that they possess the capacity to generate every cell type in the body.

This implies that, given the right laboratory conditions, it will be possible to generate new tissues, including skin. At present, there is considerable research activity looking at the various chemicals and signals that can make the embryonic stem cells develop in different ways, such as making new blood cells, muscle, heart or skin.

Much of this work is currently confined to mouse embryonic skin cells, but the principles and lessons learnt will be directly relevant to human cells. Some of the emerging pathways of making different organs from embryonic stem cells are shown in the figure below (ES = embryonic stem cells)

stemcell.png (30808 bytes)

So far, generating blood cells and heart muscle has been studied in some detail, but basic research that explores how to make skin is also being tested.

Research on embryonic stem cells, therefore, may have enormous future implications for “cell replacement” therapies relevant to EB. For now, the topic is a research one, and one that is subject to stringent regulations and ethical debate.

Everyone at DebRA, however, will be keeping up-to-date with the latest information on both adult and embryonic stem cells, especially with how this research technology may help people living with EB.
31 May 2005.