Scientists Target Cure for Blood Disorders and Immune Diseases

Researchers identify mechanisms that trigger hematopoietic stem cell production.

Scientists working to identify the process of stem cell generation may soon be able to target a cure for a range of blood disorders and immune diseases.

Published August 13, 2014 in Nature, researchers from the Australian Regenerative Medicine Institute at Monash University and the Garvan Institute of Medical Research, identified mechanisms in the body that trigger hematopoietic stem cell (HSC) production for the first time. HSCs, which are located in the bone marrow and umbilical cord blood, are significantly important due to their ability to replenish the supply of blood cells in the body.

Although leukemia patients who received HSC transplants have achieved success, experts believe the potential of blood stem cells will allow them to be used more widely.

"HSCs are one of the best therapeutic tools at our disposal because they can make any blood cell in the body,” said lead researcher Professor Peter Currie in a press release. “Potentially we could use these cells in many more ways than current transplantation strategies to treat serious blood disorders and diseases, but only if we can figure out how they are generated in the first place. Our study brings this possibility a step closer.”

An inability to produce HSCs in the laboratory setting have thus far inhibited their wider use, due to a molecular switch that may be necessary for formation, the study noted. The mechanism that is responsible for the switch was a mystery, until the current study.

The researchers examined cells in developing zebra fish, which are known for regenerative abilities and optically clear embryos. The study compiled new information on the signaling process that is responsible for HSC generation.

Utilizing high-resolution microscopy, the scientists recorded how stem cells form inside the embryo and captured in detail the process of formation. The researchers discovered that HSCs require endotome cells, which have stem cell-inducing properties, to help their formation.

"Endotome cells act like a comfy sofa for pre HSCs to snuggle into, helping them progress to become fully fledged stem cells. Not only did we identify some of the cells and signals required for HSC formation, we also pinpointed the genes required for endotome formation in the first place," Currie said. "The really exciting thing about these results is that if we can find the signals present in the endotome cells responsible for embryonic HSC formation then we can use them in vitro to make different blood cells on demand for all sorts of blood related disorder.

"Potentially it's imaginable that you could even correct genetic defects in cells and then transplant them back into the body.”