For six years, Professor Christopher Vakoc's lab at Cold Spring Harbor Laboratory has been on a mission to transform sarcoma cells into regularly functioning tissue cells. Sarcomas are cancers that form in connective tissues such as muscle. Treatment often involves chemotherapy, surgery, and radiation—procedures that are especially tough on kids. If doctors could transform cancer cells into healthy cells, it would offer patients a whole new treatment option—one that could spare them and their families a great deal of pain and suffering.
A devastating and aggressive type of pediatric cancer, rhabdomyosarcoma resembles children's muscle cells. No one knew whether this proposed treatment method, called differentiation therapy, might ever work in RMS. It could still be decades out. But now, thanks to Vackoc's lab, it seems like a real possibility.
"Every successful medicine has its origin story. And research like this is the soil from which new drugs are born," says Vakoc. To carry out their mission, Vakoc and his team created a new genetic screening technique. Using genome-editing technology, they hunted down genes that, when disrupted, would force RMS cells to become muscle cells. That's when a protein called NF-Y emerged. With NF-Y impaired, the scientists witnessed an astonishing transformation.
The cartoon model above illustrates RMS cells’ transformation to healthy muscle cells. When NF-Y is depleted from the cells, the cancer stops multiplying and starts to take on typical muscle features and functions. The microscopy images on the bottom row capture real cells before and after this transformation. Credit: Vakoc lab/Cold Spring Harbor Laboratory
"The cells literally turn into muscle," Vakoc says."The tumor 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." The research is published in the journal
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