AUSTRALIAN scientists could be one step closer to curing leukaemia and other blood disorders, after a major breakthrough in stem cell treatment.
Researchers from the Murdoch Childrens Research Institute were able to successfully create blood stem cells in the lab almost identical to those that grow inside a developing embryo.
The groundbreaking discovery could eventually replace bone marrow transplants.
A trio of biology researchers — Dr Elizabeth Ng, Professor Andrew Elefanty and Professor Ed Stanley — led the research and the study was published today in the science journal Nature Biotechnology.
“If we can make normal blood stem cells in the laboratory, we can also use the same technology to be able to study blood diseases like leukaemia or other diseases that affect children and adults,†Prof Elefanty told news.com.au.
“By modelling or recreating those diseases in the dish in the laboratory, that helps us to study what’s gone wrong in those cases and how we can treat them better.â€
The researchers used human pluripotent stem cells, which can differentiate and turn into any type of cell in the body, to create blood cells that very closely resemble those that develop inside an embryo. These can be used as an alternative to bone marrow transplants.
“That’s the stuff we’re born with, that’s the stuff that contains the blood stem cells that will look after us for the rest of our lives,†Dr Ng told news.com.au.
She explained that the blood cells that are found inside bone marrow actually originate from the developing aorta, the main blood vessel of the heart, during embryonic development. These cells travel to the foetal liver to grow for a few months before they migrate to the bone marrow, where they will be required to produce circulating blood cells after birth.
The process of cell development took around two and a half weeks, which is close to the time taken for the blood cells to develop during embryonic development, and the lab-grown blood cells have the right markers on the cell surface.
This breakthrough is important because it gives researchers the ability to model and reproduce various blood disorders, such as Thalassemia and leukaemia, to understand how to direct genetic expression and look at improving current treatment therapies and developing new ones.
It also means that instead of solely relying on bone marrow transplants, where there are often problems with finding suitable donors, scientists can tailor blood stem cells to recipients to reduce the risk of adverse immunologic reactions.
“The reason that we want to make blood cells is that by understanding the way that human blood cells develop, we hope that we can make blood stem cells that can be used to transplant into patients with leukaemia who don’t have a matching donor they need,†says Prof Elefanty.
According to the Australian Institute of Health and Welfare, over 3,600 people will be diagnosed with leukaemia this year, and the five-year survival rate was around 58 per cent.
Aileen Boyd-Squires, CEO of the Children’s Cancer Foundation, says the research is exciting because it allows for a much faster avenue for developing new treatments.
“To this date, it’s been difficult to get a human cell model where they can take the leukaemia-causing genes and put them into a blood cell and then compare them with normal blood cells,†Ms Boyd-Squires told news.com.au.
“It’s really important that they can look at what’s going on in a live cell model rather than just taking a genetic profile.â€
The ability to recreate disease states in cell models means that researchers can test the effects of new drugs and treatment combinations, develop predictive screening systems and, ultimately, discover ways to control gene expression.
“Now that the costs are lower and that there’s better collaboration both within Australia and overseas, I think there’s going to be a proliferation of new techniques and new targets,†says Ms Boyd-Squires.
According to Prof Elefanty, the focus of their research will be to observe blood stem cell maturation in the lab to see how they would cope with transplantation.
“The ultimate test will be when we can get the cells to undergo an additional level of maturation so that we can transplant them,†he said.