by Nick Miller
February 18 2009
source The Age (Melbourne, Australia)
As nine-year-old Mikaela Brundell looks around the labs at the Murdoch Children’s Research Institute, she can see two things that may one day save her life.
One is the lab itself: the home of breakthrough stem-cell research that has just brought a cure for her cystic fibrosis one step closer. The other is her little brother Aiden, who unknowingly donated his umbilical cord blood for storage, with its rich crop of stem cells that could hold the key to a successful treatment.
Last week MCRI researcher Faten Zaibak published the results of a three-year study that has been internationally hailed as a step towards a cure for genetically inherited diseases such as cystic fibrosis or Friedreich ataxia. For the first time, Dr Zaibak has successfully placed a large chunk of DNA - with the faulty gene that causes the disease successfully fixed up - into a cord blood stem cell. That stem cell can then transform into proper, functioning lung cells that could one day help a cystic fibrosis patient breathe easier and live a full life.
“What we have shown is we can use that (cord blood) stem cell to deliver DNA, and potentially correct any genetic defect,” Dr Zaibak said. “It is very difficult to introduce an entire gene into a cell.”
A treatment using this method is still years away, but it could come soon enough for Mikaela, now that other treatments have extended the lifespan of cystic fibrosis patients. “It’s all steps along the way,” said Professor Bob Williamson, research co-author and professor of medical genetics at the University of Melbourne.
Mikaela’s father, Shane Brundell, said Aiden’s cord blood had been collected for the institute’s stem cell research - but also as insurance for the children’s future.
“We were advised of the possibility of storing cord blood, the long term prospect that with continued research there was a slim possibility they might find a cure,” he said.
“We were told it would be a long term project. In just three years to have this development is very exciting. It has given us a lot of hope. One day there might be a cure for Mikaela and other people in her situation.”
Mr Brundell said he was grateful to the researchers, as well as the groups supporting the research such as Cystic Fibrosis Australia for giving his family hope.