World’s 1st lab-grown blood stem cells could aid in bone marrow transplants

A bone marrow transplant can save the lives of children suffering from leukemia or bone marrow failure. However, finding a perfect match can be a daunting challenge.

Researchers have successfully developed human blood stem cells that closely mimic natural human cells. They call it a “world-first breakthrough.”

Interestingly, these cells can be reprogrammed from any patient’s cells, offering the potential for personalized treatments.

“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,” said Elizabeth Ng, Associate Professor from Murdoch Children’s Research Institute (MCRI).

She added: “Prior to this study, developing human blood stem cells in the lab that were capable of being transplanted into an animal model of bone marrow failure to make healthy blood cells had not been achievable. We have developed a workflow that has created transplantable blood stem cells that closely mirror those in the human embryo.”

Shows promise in mice

In laboratory tests, the engineered blood stem cells effectively developed into “functional bone marrow” in immune-deficient mice. Interestingly, the cells matched the performance of umbilical cord blood transplants.

Moreover, the lab-grown cells were successfully frozen and thawed without compromising their ability to function as bone marrow. This ability to freeze the lab-grown cells offers practical advantages for storage and transportation, making them more accessible for patients.

For years, finding a suitable donor for a blood stem cell transplant has been a major challenge for children with blood diseases.

“Mismatched donor immune cells from the transplant can attack the recipient’s own tissues, leading to severe illness or death,” said Andrew Elefanty, MCRI Professor.

“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,” he added.

Clinical trials likely in next five years

Three types of blood cells work together to maintain our health: red blood cells transport oxygen, white blood cells protect against disease, and platelets prevent bleeding. This study could help in understanding their complex interactions for human health.

“By perfecting stem cell methods that mimic the development of the normal blood stem cells found in our bodies we can understand and develop personalized treatments for a range of blood diseases, including leukemias and bone marrow failure,” said Ed Stanley, MCRI Professor.

The researchers expect to initiate clinical trials within the next five years to assess the safety and performance of the lab-grown blood cells.

The press release highlights the experience of Riya, a young girl from India. She was diagnosed with aplastic anemia, a rare blood disorder characterized by the body’s inability to produce enough blood cells. Her family faced a daunting journey as they sought treatment for her condition.

After initial misdiagnosis, Riya was found to have bone marrow failure and required frequent blood transfusions. The family relocated to Australia for specialized care, where Riya underwent a bone marrow transplant from her mother, despite being a half-match.

Riya’s recovery was challenging, with complications and a prolonged recovery period. Her family is hopeful that the recent breakthroughs in blood stem cell research will offer hope and personalized treatments for future patients facing similar challenges.

“This research will come as a blessing to so many families. The fact that one day there could be targeted treatments for children with leukemia and bone marrow failure disorders is life-changing,” said Riya’s mother.

The findings have been published in the journal Nature Biotechnology.