Major scientific breakthrough may lead to treatment of leukemia and bone marrow failure

The study, led by the Murdoch Children’s Research Institute (MCRI), revealed that this achievement could soon lead to personalized treatments for children with leukaemia and bone marrow failure disorders.

The research team explained that it has overcome a major hurdle to producing human blood stem cells, which can create red blood cells, white blood cells and platelets, closely matching those found in the human embryo.

“The ability to take any cell from a patient, reprogram it into a stem cell and then turn it into blood cells specifically matched for transplantation would have a huge impact on the lives of at-risk patients,” said Elizabeth Ng, an assistant professor at MCRI.

In the study, immunodeficient mice were injected with human blood stem cells modified in the lab and were shown to develop functional bone marrow at levels similar to those seen in cord blood cell transplants, a proven measure of success.

The study also found that stem cells grown in the lab could be frozen before being successfully transplanted into mice, mimicking the process of preserving donor blood stem cells before transplanting them into patients.

The findings could lead to new treatment options for a range of blood disorders, said Ed Stanley, a professor at MCRI.

“Red blood cells are essential for transporting oxygen, white blood cells provide our immune defense, and platelets cause blood to clot to stop bleeding. Understanding how these cells develop and function is like solving a complex puzzle. We can eventually understand and develop personalized treatments for a range of blood diseases, including leukemia and bone marrow failure,” he added.

Developing patient-specific, personalized blood stem cells would prevent complications from transplants, such as organ rejection, and address the problem of a shortage of donors, said co-author Andrew Elefante, of MCRI.

He explained that the next stage, which is likely to take place within about 5 years, will include conducting a phase I clinical trial to test the safety of using blood cells grown in the laboratory in humans.

The study was published in the journal Nature Biotechnology.

Source: Medical Express