Australian scientists have made a world-first stem cell breakthrough that could put an end to agonising searches for bone marrow donors needed to treat critical conditions.
The findings, published in Nature Biotechnology, could eventually benefit patients like Riya Mahajan, who was diagnosed with life-threatening blood disorder aplastic anaemia at the age of 11.
She had blood transfusions every couple of days during a global search for a matching bone marrow donor, but none could be found so her mother, who was only a partial match, stepped in instead — but the lifesaving choice came with side effects.
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However Murdoch Children’s Research Institute scientists have now successfully created lab-grown blood stem cells that are similar to those in humans and can survive beyond a Petri dish.
Stem cells can become any type of cell in the body and, until now, no lab-grown cells have successfully been transplanted into mice.
Clinical trials in humans are still about five years away, but the discovery has sparked hopes of improving treatment options for people with leukaemia, serious blood disorders and children with tumours who need rigorous chemotherapy.
Riya suffered complications after her surgery and had to be isolated in hospital for three months while on immune-suppressing medication.
“Chemotherapy was probably the hardest period for us as a family because she lost her hair during the chemo sessions,” Riya’s father Gaurav said.
“She was getting fed through a nasal gastric tube, lost a lot of weight, I think nearly 10kg and the isolation can also have a mental impact.”
The transplant was ultimately successful, but her father hopes the discovery will prevent other children from going through a similar ordeal.
“The hardest thing (is) to see your child going through so much pain,” he said.
“This new research will go a long way in helping kids who are suffering from similar conditions.”
Associate Professor Elizabeth Ng said, although animal studies are “not perfect”, 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.
“The blood stem cells that we make in the dish will be perfectly matched to the patient, so the patient will not experience a lot of the side effects that Riya, for instance, has experienced,” she said.
“We hope in fact the treatment will be successful, recovery will be very successful and patients will be able to resume a normal life very, very quickly.”
Professor Ed Stanley stopped short of calling it a cure but said it would be an important part of a patients’ treatment, and it would remove the need to suppress their immune system.
“In many ways for patients with bone marrow failure who fail medical therapies, the transplant is itself a curative process, so it depends on what the underlying illness is,” he said.
The next step will involve ensuring the technology is safe and effective, with the team already shifting their focus to cell manufacturing and clinical use.
