in the bone marrow
Researchers in the UK have developed a new method of producing megakaryocytes from human pluripotent stem cells (hPSCs).
The team said their method generated large quantities of functional megakaryocytes that produced functional platelets.
They believe this work brings us one step closer to manufacturing platelets for transfusion.
The research was published in Nature Communications.
“Making megakaryocytes and platelets from stem cells for transfusion has been a long-standing challenge because of the sheer numbers we need to produce to make a single unit for transfusion,” said study author Cedric Ghevaert, MD, PhD, of the University of Cambridge and NHS Blood and Transplant in Cambridge, UK.
“We have found a way to ‘rewire’ the stem cells to make them become megakaryocytes a lot faster and more efficiently. It is a major step forward towards our goal to one day make blood cells in the laboratory to transfuse to patients.”
Dr Ghevaert and his colleagues employed a “forward programming” strategy in which 3 transcription factors—GATA1, FLI1, and TAL1—were used drive the differentiation of hPSCs into megakaryocytes.
The team said the forward-programmed megakaryocytes matured into platelet-producing cells that could be cryopreserved, maintained, and amplified in vitro for more than 90 days. The average yield was 200,000 megakaryocytes per hPSC.
The researchers generated platelets from the forward-programmed megakaryocytes via culture. And tests suggested these platelets were functionally similar to donor-derived platelets.
“The success of this research team in producing megakaryocytes in the laboratory has paved the way for the ultimate goal—manufacturing platelets for transfusion,” said Edwin Massey, MBChB, associate medical director for diagnostic and therapeutic services at NHS Blood and Transplant, who was not involved in this research.
“It will, however, be many years before a process for the large-scale production of platelets is developed. Donated platelets will still be needed by patients for the foreseeable future, either as part of a blood donation or by dedicated platelet donation using a machine collection process.”
