COLD SPRING HARBOR, Nueva York, 28 de agosto de 2023 /PRNewswire/ — «Every successful medication has its origin story. And research like this is the groundwork from which new medications are born,» says Cold Spring Harbor Laboratory professor Christopher Vakoc.
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The cartoon model above illustrates the transformation of RMS cells into healthy muscle cells. When NF-Y is depleted from the cells, cancer stops multiplying and begins to acquire typical muscle characteristics and functions. The microscopy images in the bottom row capture real cells before and after this transformation. Cold Spring Harbor Laboratory professor Christopher Vakoc and graduate from the CSHL Biological Sciences Department, Martyna Sroka, have found a way to transform rhabdomyosarcoma cells into muscle cells. As the cells transform, they acquire typical muscle characteristics, including the fusiform shape seen here.
During six years, Vakoc’s laboratory has had the mission to transform sarcoma cells into functioning tissue cells. Sarcomas are cancers that form in connective tissues like muscles. Treatment often includes chemotherapy, surgery, and radiation, procedures that are especially challenging for children. If doctors could transform cancer cells into healthy cells, they would offer patients a completely new treatment option, one that could save them and their families a great deal of pain and suffering.
Rhabdomyosarcoma (RMS), a type of devastating and aggressive pediatric cancer, resembles muscle cells in children. No one knew if this proposed treatment method, called differentiation therapy, could ever work in RMS. It could still take decades. But now, thanks to Vakoc’s laboratory, it seems like a real possibility.
To carry out their mission, Vakoc and his team developed a new genetic screening technique. Using genome editing technology, they searched for genes that, when altered, would force RMS cells to become muscle cells. That’s when a protein called NF-Y emerged. With NF-Y deteriorated, scientists witnessed an astonishing transformation. Vakoc explains, «The cells literally turn into muscle. The tumor loses all the attributes of cancer. They are changing from a cell that only wants to produce more of itself to cells dedicated to contraction. Because all their energy and resources are now dedicated to contraction, they may not return to this proliferative state.»
This new relationship between NF-Y and RMS may trigger the cascading reaction necessary to bring differentiation therapy to patients. And the mission doesn’t stop at RMS. The technology could be applicable to other types of cancer. If so, scientists may someday discover how to turn other tumors into healthy cells. «This technology can allow you to take any cancer and look for how to differentiate it,» explains Vakoc. «This could be a key step in making differentiation therapy more accessible.»
Previously, Vakoc and his team succeeded in transforming Ewing sarcoma cells into healthy tissue cells. The discoveries in Ewing sarcoma and RMS were supported by local families who had lost their loved ones to these cancers. «They came together and funded us to try and find, somewhat desperately, a new therapeutic strategy,» says Vakoc.
Those families and Vakoc’s laboratory may now be the heroes of a new origin story: a scientific breakthrough that could someday help save children’s lives and revolutionize cancer treatment as we know it.
About Cold Spring Harbor Laboratory
Founded in 1890, Cold Spring Harbor Laboratory has shaped contemporary biomedical research and education with programs in cancer, neuroscience, plant biology, and quantitative biology. Home to eight Nobel Prize winners, the nonprofit private laboratory employs 1,000 people, including 600 scientists, students, and technicians. For more information visit www.cshl.edu.
SOURCE Cold Spring Harbor Laboratory
