MCRI team create blood stem cells in lab in world-first

Children with leukaemia and other blood diseases could soon find a “perfect match” for a bone marrow transplant thanks to these lab-made stem cells.

Children with blood diseases including leukaemia and bone marrow failure disorders may soon be treated with “perfectly matched” blood stem cells created in a lab.

Melbourne researchers revealed on Tuesday they had created blood stem cells that closely resembled those in the human body.

A world-first, it has the potential to transform bone marrow transplants in children and be a lifesaver for those who do not have a matched donor.

The team, led by Murdoch Children’s Research Institute (MCRI), describe it as a “significant” discovery.

MCRI Professor Andrew Elefanty was part of the research team that created blood stem cells in the lab that closely resemble those in the human body. Picture: MCRI

MCRI Professor Andrew Elefanty says it is important as while a blood stem cell transplant is often a lifesaving treatment for childhood blood disorders, not all children find an ideally matched donor.

“Mismatched donor immune cells from the transplant can attack the recipient’s own tissues, leading to severe illness or death,” he said in a statement.

“Developing personalised, patient-specific blood stem cells will prevent these complications, address donor shortages and, alongside genome editing, help correct underlying causes of blood diseases.”

Professor Elefanty said the next stage would be clinical trials with patients in Melbourne, expected to start in around five years with government funding.

MCRI Associate Professor Elizabeth Ng said the team had made a significant discovery in human blood stem cell development. Picture: MCRI

MCRI’s Elizabeth Ng said the team had been working “for years” to understand how to generate blood cells in the laboratory.

“This study has overcome a major hurdle for producing human blood stem cells, which can create red cells, white blood cells and platelets, that closely match those in the human embryo,” Associate Professor Ng said.

Red blood cells transport oxygen, white blood cells are the body’s immune defence and platelets are necessary as they cause clotting to stop bleeding.

“The ability to take any cell from a patient, reprogram it into a stem cell and then turn these into specifically matched blood cells for transplantation will have a massive impact on these vulnerable patients’ lives,” Associate Professor Ng said.

Riya Mahajan was diagnosed with aplastic anaemia and successfully treated with a bone-marrow transplant. Picture: Supplied

“The greatest benefit is that the blood stem cells we make in the dish will be perfectly matched to the patient, so they will not experience a lot of the side effects.

“We hope the treatment and recovery will be very successful and patients will be able to resume a normal life and won’t need immune suppressants because they are perfectly matched.”

The researchers said in the study immune deficient mice were injected with the lab engineered human blood stem cells.

It found the blood stem cells became functional bone marrow at similar levels to that seen in umbilical cord blood cell transplants, a proven benchmark of success.

Riya Mahajan with her father Gaurav Mahajan who says it is great the MCRI team is helping to create stem cells in the lab. Picture: Supplied

Melbourne schoolgirl Riya Mahajan was diagnosed with aplastic anaemia, where the body stops producing enough new blood cells.

Her father Gaurav said she never really responded to medications and eventually doctors at the Royal Children’s Hospital recommended a bone-marrow transplant.

Mr Mahajan said he and his wife Sonali were both tested and each found to be 50 per cent matches, but it was decided to go ahead with the transplant to save their daughter.

He described a rigorous process where Riya’s immune system was suppressed to prepare for the transplant and a three-month stay in hospital.

“She lost her hair during chemotherapy sessions, she lost 10kg and was isolated and not able to meet anyone but her mum and dad,” he said.

Riya Mahajan with her mother Sonali. Riya was diagnosed with aplastic anaemia and was successfully treated with a bone-marrow transplant. Picture: Supplied

The transplant was successful and today Riya is getting her life back.

“She is out of danger and back at school after two years away and able to eat well and is regaining her physical strength,” Mr Mahajan said.

“We are so grateful for this new research, it is great news that researchers are creating stem cells in the lab.

“My best wishes to the team because I understand as a parent, and we as a family, it is very difficult to see your child going through so much pain and I think this new research will go a long towards helping children in the future.”

Researchers from the University of Melbourne, Peter MacCallum Cancer Centre, University of California Los Angeles, University College London and the University of Birmingham also contributed to the findings that were published in Nature Biotechnology.