Cord Blood Stem Cell Therapy for Cystic Fibrosis
	Professor Robert Williamson Professor of Medical Genetics University of Melbourne Australia

Background to Research

Cystic fibrosis (CF) is the most common severe inherited disease affecting children in New Zealand, Australia and Northern European countries such as Scotland and Ireland.  Approximately 1 baby in 2000 has cystic fibrosis through inheriting the mutation from both parents, and one adult in 22 is a healthy carrier of one copy of the CF mutation (there are about 100,000 healthy CF carriers of this genetic time bomb in New Zealand).  Children with CF require daily physiotherapy to keep lungs free from chronic infection, and have to take pancreatic enzyme supplements to aid digestion.  In spite of great improvements in care, and a life expectancy averaging 30 years or so, CF is still a life-threatening disease that becomes more severe during early adult life.  Although the gene (CFTR) was identified in 1989, attempts to use healthy copies of the gene for therapy have been unsuccessful to date. 

What are your research objectives?

Our objective is to show that cord blood stem cell therapy for cystic fibrosis is ethical and safe and will be effective in restoring lung function.  If the therapy is useful for CF, it may also help others, both children and adults, with lung diseases such as asthma or COPD.

How will you conduct your research?

We will firstly collect cord blood from women who volunteer to help cystic fibrosis research.  Studies on cord blood stem cells are considered ethical, and we have approval from our Research Ethics Committee.  We will study whether stem cells from cord blood can suppress immune rejection of a cell transplant, as would be predicted from the data for cord blood stem cell transplants for leukaemia.  We will then determine whether this suppression continues if the stem cells are pushed down the lung pathway, by mixing cells from the cord blood with white cells from the baby, from family members and from random unmatched individuals.

Are there others in your field in NZ or globally conducting similar research?

Many research groups are trying to improve care for those with CF, or to offer screening so couples have a choice of prenatal testing if they wish to take this route.  The United States CF Foundation has been particularly good at testing better antibiotics, drugs to loosen the mucus in the lungs, and medicines to aid digestion.  The British CF Trust has put a lot of emphasis on new methods of gene therapy, to deliver healthy copies of the CFTR gene.  There are groups in London, Paris and in Australia studying whether embryonic stem cells and bone marrow cells can be made to form lung cells.  However, to the best of our knowledge, our project is the only one that is studying whether cord blood stem cells, which are universally agreed to be ethical and safe, could be used for CF therapy without rejection.

What is innovative about the approach you are taking?

To date, cord blood stem cells have been used to treat children (and adults) with leukaemia and similar blood diseases.  Because over 6000 cord blood transplants have been carried out, it is known that they are safe, and also seem to suppress immune rejection when used to replace bone marrow.  We have organised free collection of cord blood from siblings of every child with CF in New Zealand and Australia.  We will use these cells in a new and different way, to form lung cells rather than blood cells.  There are also many thousands of cord bloods in public banks that could be available for transfer, if we can show that cell therapy is safe and effective.  This grant will support research to determine whether cord blood is free from problems of rejection if used for lung cell therapy for cystic fibrosis, an essential step towards treatment.

Our objective is to show that cord blood stem cell therapy for cystic fibrosis is ethical, safe and will be effective in restoring lung function.  If the therapy is useful for CF, it may also help others, both children and adults, with lung diseases such as asthma or COPD.

If you achieve your objectives what will that mean to those suffering from the disease or to the knowledge advancement of this disease?

The importance of this research for CF is that it represents a different approach to either pharmacological treatment or gene therapy.  We know from mouse models that stem cells, if they are not rejected, can correct the ion transport defect in the lung.  Even a small improvement in lung function could make a big difference to children and young adults with CF.

Is there national or international collaboration on your research project?

Our group is based in Melbourne, but we have collaborations with many groups around the world, including Professors Liz Simpson in London, Henry Erlich in San Francisco, David Cook in Sydney and Gesine Kogler in Germany.  We are in frequent contact with Professor Keith Grimwood in Wellington and Professor Peter Gluckman in Auckland, as we are very aware of the need to work as closely as possible with interested colleagues in New Zealand.  We have already established cord blood collection for families with a child living with CF across New Zealand and Australia, and the first stem cell collection for a CF family in fact took place in New Zealand.

Radio Interview with Professor Bob Williamson

‘Tune Your Engine’

What science is still learning about umbilical cord blood indicates its potential could be enormous...

Click here for full interview. Cure Kids is funding his research into Cystic Fibrosis.