Scientists have discovered a new treatment option for the aggressive type of childhood cancer that forms in muscle tissue.
In a breakthrough, the scientists have successfully brought about rhabdomyosarcoma cells to change into normal, healthy muscle cells.
“Every successful medicine has its origin story. And research like this is the soil from which new drugs are born,” said Cold Spring Harbor Laboratory Professor Christopher Vakoc.
“The cells literally turn into muscle,” Vakoc said.
“The tumour loses all cancer attributes. They’re switching from a cell that just wants to make more of itself to cells devoted to contraction. Because all its energy and resources are now devoted to contraction, it can’t go back to this multiplying state,” he added.
Cancer is a disease that develops when cells divide uncontrollably and begin to destroy body tissues.
Rhabdomyosarcoma, a type of cancer that is usually seen in children and adolescents, normally originates in the skeletal muscle when cells therein mutate and start procreating and spreading all over the body.
One treatment that has shown positive results for the lethal pediatric cancer is differentiation therapy which arose when scientists detected that leukaemia cells are not completely mature, equivalent to undifferentiated stem cells that have not yet comprehensively evolved into a specific cell type.
This therapy compels those cells to continue their development and discern into specific mature cell types.
Vakoc and his team created a new genetic screening technique. By deploying genome-editing technology, the researchers tracked the genes that, when disrupted, would compel RMS cells to become muscle cells.
Then a protein called NF-Y came into the picture. With NF-Y harmed, the scientists detected an incredible mutation.
The new relationship discovered between NF-Y and RMS may set forth the chain reaction required to bring differentiation therapy to patients. The technology could also be practical for other cancer types. If so, scientists may someday work out how to turn other tumours into healthy cells.
“This technology can allow you to take any cancer and go hunting for how to cause it to differentiate,” Vakoc explains. “This might be a key step toward making differentiation therapy more accessible.”
Previously, Vakoc along with his team successfully transformed Ewing sarcoma cells into healthy tissue cells.
The Ewing sarcoma and RMS discoveries were supported by local families who had lost their family members to these cancers. “They came together and funded us to try to find, with some desperation, a new therapeutic strategy,” said Vakoc.
The study has been published in the Proceedings of the National Academy of Sciences.