Presentation of Prof. Dr. Catherine Verfaillie

Amyotrophic Lateral Sclerosis: Can stem cells contribute something?

Prof. Dr. Catherine Verfaillie, Head Interdepartmental Stem cell Institute Leuven (Catholic University Leuven Gasthuisberg)


Professor Verfaillie’s lecture was split into a number of parts:

• Definition of stem cells

• Type of stem cells

• Applications of stem cells in neural diseases

• How does stem cell therapy become reality?


Definition of stem cells

A stem cell is a cell which is able to differentiate into one or more other cell types.  Stem cells always start with a blank slate.  Their daughter cells are identical copies that differentiate into kidney cells, brain cells…


Type stamcellen

Embryonic stem cell or ES cell

ES cells are primitive or non-differentiated cells that come from an embryo of 5 days old (fertilization of the egg-cell happens in-vitro, In Vitro Fertilization (IVF) techniques).  These ES cells, which originate in the blastocyst after the fertilization of the egg-cell, develop into a variety of specialized cell types (skin, brain, blood, heart, lungs …)

Human embryonic stem cells

It is stated that these cells also develop in humans into different cell types.  This offers the perspective that a lot of diseases could be treated. 

But soon you will encounter problems, namely:

1. When these are implanted in humans, a tumor develops

2. Rejection: methods must be found to prevent this

3. Ethical problems and legislation, such as destruction of surplus fertilized egg-cells 

“Adult” stem cells or AS cells

An adult stem cell is a non-differentiated cell that appears in differentiated tissue and is capable of renewing itself by giving existence to specialized cell types of the tissue from which it comes.

Examples of these AS cells are:

- Neural stem cells from the brain tissue

- Blood forming stem cells from the spinal cord

- From the heart for heart muscle cells

But do these AS cells also develop cell types from a different type of tissue than the one they themselves are coming from?  And adult stem cells, are they as potent as embryonic stem cells?  This research is still in its infancy, but could offer a lot of perspectives for many diseases, such as ALS.  At the same time these AS cells would raise fewer ethical questions than the ES cells.


Applications of stem cells in neural diseases

Prof. Verfaillie first explained how you get to an ES cell line with a genetic disease.  It allows you to gain insight in the pathogenesis of ALS.

Then she described how you make ES stem cell lines that develop neural cells.

Everybody knows EPO that is used in the world of sports.  EPO are in fact stem cells that produce red blood cells.  In medical science EPO mainly finds its application with cancer patients.  In fact it should be explored how to make a similar EPO that grows to liver cells, brain cells, heart cells …

For research into the neural diseases the MAP cells qualify because they are able to produce neurons and other nerve cells.  With ALS one should check how they can correct and cure the affected area of the neurons.  Besides that research should be done on how they can regenerate motor neurons.  The latter will be the hardest.

Hopefully research on fruit flies and zebrafish deliver fast results because once we have good results there, we can quickly move on to tests on humans.


How does stem cell therapy become reality?

A lot of work still needs to be done.  We have to check:

1. Which cell: embryonic versus adult stem cells? Differentiated or not?

2. Tests on small animals

3. Tests on big animals

4. Large-scale production

5. Phase 1 : clinical tests

To realize this, we need to work together, because the University of Leuven cannot do this on its own.  Both within our country as abroad, universities and research centers will have to join forces.

Yet there was also warned for the stem cell therapies already available on the market, because to date no approved therapies for humans are available.   So go with caution to the reporting from China or other companies and clinics.



Most questions are about the funding of the research and the ethics towards stem cells.

As a conclusion we can say that obtaining funds for research varies from country to country.  In the United States the research entirely relies on private funds.  In Europe this is different.  The EU does not fund the production of ES stem cells, but she makes resources available from the beginning of the cell lines.  For what concerns Belgium, there is funding under strict conditions.

Ethically it is clear that the Vatican is completely against stem cells.  They even don’t recognize in vitro fertilizations.  The United States standpoint is that IVF can be done and that the surplus of fertilized egg-cells must be destroyed, but 70 to 80% says that research must be possible.  The whole discussion is about “when do you speak about a human?”  According to the Vatican this is as from the conception.  In Judaism this is from the moment the woman feels the first movements in her womb.  Science talks about a human from the moment that no identical twins can develop anymore or approximately 14 days after the conception.


Translation: Tina