New Hope

Hi everyone, the following was published in The Age newspaper on
February 11th 2010. There is also video grab on this subject.

http://www.abc.net.au/catalyst/stories/2608076.htm

to read this article in context, go to
http://www.theage.com.au/national/new-hope-in-hunt-for-ataxia-cure-20100210-nsgm.html

MELBOURNE scientists have made a breakthrough in the hunt for a cure for
Friedreich's ataxia, a genetic disease with no known treatment that
causes slow nerve degeneration and early death.

Teams from the University of Melbourne and the Monash Institute of
Medical Research have made pluripotent stem cells from the skin of FA
sufferers, which could provide a shortcut to a drug-based cure or even
be used directly as a treatment.

The work has been hailed as being at the forefront of worldwide research
into a disease that affects hundreds of Australians.

Friedreich's ataxia shows up in teenage years, as the faulty gene causes
low levels of a protein vital for delivering iron to nerve and heart
cells. Sufferers gradually lose their balance and co-ordination, develop
heart problems and often diabetes.

Melbourne University's Mirella Dottori said the key to the new approach
were Monash's ''induced pluripotent'' stem cells, which had been
manufactured from the skin of the FA patients.

Dr Paul Verma, MIMR's stem cell research program leader, created
Australia's first iPS cells early last year.

Dr Dottori and her colleague Dr Alice Pebay have ''pushed'' these cells
to turn into nerve and heart cells, which can be studied to better
understand the disease.

They can also be used to test existing drugs to see if they reverse the
effect of the faulty gene. And they may even be used in ''regenerative
therapy'', by correcting the faulty gene then re-implanting the cells in
an FA patient.

''It is very exciting - new doors are opened,'' Dr Dottori said.

The research was welcomed by Carrie Beetham, 32, of Hawthorn, who found
she had FA in her teens.

''I knew I would be in a wheelchair at some stage but I didn't know it
was going to move so fast. It is about keeping yourself strong and
keeping your mind strong. The main part is trying to be positive, trying
to enjoy the life that we can enjoy, trying to remain hopeful.''