Engineered stem cells breakthrough could save lives

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Carrying out stem cell research in the lab (SBS) Source: SBS News

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Australian scientists have made a world-first breakthrough creating lab engineered blood stem cells that closely resemble those found in the human body. They say the discovery could one day put an end to the search for 'perfectly matched' bone marrow donors that are often needed to treat those with leukaemia serious and blood disorders.


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Melbourne-based Gaurav and Sonali Mahajan were in India when they first noticed bruises on their daughter's legs.

Riya, who was 11 at the time, had also been suffering fevers and feeling fatigued.

Tests revealed she had extremely low levels of platelets in her blood. The normal range is between 150,000 to 450,000. Riya's were at 4,000.

The discovery fast-tracked the family's return to Australia.

Gaurav says they boarded a flight to Melbourne once Riya's condition was considered stable enough for her to fly.

"As soon as we got off the flight we went straight to the hospital. It was very stressful and it was a bit of a shock for everyone in the family. [It was] just out of the blue. She wasn't unhealthy or anything, she was very normal."

Riya started therapy at Melbourne's Royal Children's Hospital within days of her return to Australia

"I was just confused an kind of scared, and the treatment was quite hard on your body, you get nauseous, it hurts, you're in pain."

After almost six months of therapy, including blood transfusions every second day, a bone marrow transplant was recommended

Gaurav says finding a perfectly matched donor was a difficult and agonising task.

"We were just waiting to see if we could find a match and so it was obviously a very nerve-wracking and anxious time."

After months of waiting, Riya's mother stepped in becoming her daughter's donor, despite being just a 50 per cent genetic match.

The transplant was successful but without a perfect donor match, Riya suffered complications and had to isolate in hospital for three months

"Besides the physical hardship, there is a mental aspect to it as well. She couldn't see any other kids for six months."

In a world-first, Australian scientists have created lab engineered blood stem cells - similar to those in humans - and successfully transplanted them into mice.

They say future treatments could involve taking a sample of a patient's skin, blood or hair, reprogramming their cells and then transferring them back to the patient.

Professor Ed Stanley from the Murdoch Children's Research Institute was involved in the study.

"This kind of technology could potentially be used to regenerate their blood cells and give them back a working bone marrow." 

Researchers acknowledge animal studies aren't perfect and clinical trials in humans are about five years away.

But their discovery could one day address donor shortages.

Lead-researcher Associate Professor Elizabeth Ng says it could also reduce the side-affects suffered by those unable to find a perfectly matched donor.

"They will be able to recover and rebuild a blood system following treatment without any of the side effects usually associated with blood stem cells that they get from a donor. "

The speed and cost of the technology are hurdles that will need to be overcome, the researchers say but the Mahajan's say it could prove life-changing for families like theirs.




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