Media Release oct02

[Guest guest]

This is great news from our Hsp. They are looking for US financial

backing as usual. I also have the newspaper print out with the mouse on

it if anyone wants me to scan it and send it to them.

(Australia)

_____

Text Box: 72 King Road

North Adelaide SA 5006

http://www.wch.sa.gov.au

MEDIA RELEASE

Monday October 21st 2002

World-first gene therapy for Cystic Fibrosis targets lung stem cells ?

The genetically - inherited disease cystic fibrosis causes severe,

unrelenting lung disease in children and adults worldwide. Approximately

1 in 2,500 infants are born with this disease and only half survive past

30 years of age.

Now, researchers from the Women's and Children's Hospital, Adelaide have

developed a novel system of gene therapy for lungs affected by cystic

fibrosis, involving a natural compound found in our lungs which

'conditions' lung airways to allow cells to take up the therapeutic

gene.

Our lungs have developed highly effective ways to protect us from

allergens, irritants, dust, viruses and other foreign particles. But

according to principal medical scientist in Pulmonary Medicine, Dr

Parsons these defences also hinder effective gene therapy in our lungs.

" Using a mouse model of cystic fibrosis Limberis, a PhD student in

our lab, has helped us develop a system to briefly overcome these

defences. As the cells lining the mouse nose behave in much the same way

as human lung cells - this enables us to use the nose airways in mice to

easily develop and test out gene therapy treatments.

" By instilling a single dose of a detergent found naturally in low

amounts in our lungs, we are able to 'condition' cells to take up the

gene needed to treat cystic fibrosis.

" Viruses are very good at transferring their genetic material into

cells and we make use of this by getting useful parts of an inactive and

highly-modified human immunodeficiency virus type 1 (HIV 1) to safely

transfer the cystic fibrosis gene into cells. We use this modified HIV

because it is one of the few viruses that can give long-lasting gene

transfer, " Dr Parsons says.

Using this system, the research team has shown, for the first time in a

living animal, that long-lived gene therapy for cystic fibrosis is

possible. Not only do the airway cells take up the correcting gene, but

these cells also show substantial recovery from the cystic fibrosis

defect for, so far, at least 110 days.

" Airway cells are replaced every three months so our findings are

particularly exciting because they imply we are in fact targeting airway

stem cells through this approach - some of the therapeutic gene must

have been passed on from these parent stem cells to their daughter cells

for the effect to persist beyond three months, " Dr Parsons says.

Continued on page 2

Continued from Page 1

'Gene therapy for Cystic Fibrosis targets lung stem cells ?'

Research funding is now being sought to establish the most effective

dose and timing for giving the detergent together with rigorous safety

checks on the highly modified HIV-1 based virus particle used for the

gene transfer.

Another senior member of the team, molecular biologist Dr Don Anson,

explains, " Last year we published a method which vastly increases the

safety with which HIV-1 can be modified and used to transfer genes

without causing disease.

" We are now working to further improve on this method in order that

patients and their families will feel totally confident to eventually

take part in human trials of this gene therapy for cystic fibrosis, " Dr

Anson says.

The work from this research is to be published in Human Gene Therapy,

volume 13 #16 on October 20.

Members of the Research Team are:

Dr Parsons (Principle Medical Scientist, Pulmonary Medicine)

Dr Don Anson (Senior Molecular Biologist, Chemical Pathology)

Dr Fuller (Molecular Biologist, Chemical Pathology)

Ms Limberis (PhD student, Pulmonary Medicine)

To arrange interviews with Dr Parsons please contact:

Dr Edna Bates or Ms

Ostermann

Manager, Research Communications Director of Media and

Community Relations

* ( 618) 8161 7388 * mobile 0401 125 630 (after

hours)

email: batese@... ostermannc@...

Women's and Children's Hospital http://www.wch.sa.gov.au