Disease & Illness

Scientists Have Discovered A New Gene-Editing Technique That Could Mean Major Advances In Cancer Treatment

This latest research could have big implications.

Scientists are always working to improve cancer treatment so that it’s quicker, safer and more effective for patients. And it seems researchers at the University of California-San Francisco have made a breakthrough discovery in cell therapy that could achieve all three objectives.

In a new study published Wednesday in the journal Nature, USCF scientists detailed how they used cutting-edge gene-editing tools to reprogram T cells, a type of white blood cell. These tools allowed them to transform the cells from your average immune cell — which targets bacterial or fungal infections — into one that attacks cancer. To achieve this, the researchers inserted new genes into T cells taken from patients. According to the study, when these altered cells were returned to the body, they showed the potential to boost immune systems so they could fight cancer on their own.


Researchers tested the promising technology in two ways: by transferring engineered cells into mice implanted with human melanoma tumors, and by repairing a disease-causing genetic mutation in T cells from three children with a rare autoimmune disease.

When they returned the engineered T cells to the mice, the cells went straight to the tumor site and showed anti-cancer activity, according to the study.

“This strategy of replacing the T cell receptor can be generalized to any T cell receptor,” said senior study author Dr. Alex Marson, associate professor of microbiology and immunology at UCSF, in a news release. “With this new technique, we can cut and paste into a specified place, rewriting a specific page in the genome sequence.”


The scientists behind the study are working with UCSF’s Parker Institute for Cancer Immunotherapy to develop altered cells that can treat a variety of cancers, such as non-Hodgkin lymphoma, melanoma and childhood leukemia, according to the New York Times.

Fred Ramsdell, the Parker Institute’s vice president of research, told the Washington Post that the new technique “really opens up the ability for us, as a community, to think about very creative and potentially unique ways to activate a T cell.”


What The Future Holds

But when this new knowledge could turn into real treatments for patients is hard to predict, researchers told the Washington Post. That’s because, as revolutionary as this method may be, there’s still a lot that scientists don’t know. Mainly — and perhaps most critically — is it safe to put the repaired T cells back into people?

“There will have to be discussions with regulatory agencies,” study co-author Kevan Herold, an endocrinologist and immunologist at Yale University, told the Washington Post. “All of us are aware of the potential pitfalls here.”

Human clinical trials testing the new technology should happen within the next one to three years, according to the Washington Post. In the meantime, scientists are seeking approval from the U.S. Food and Drug Administration to use the repaired T cells to treat the three children.


It may take a while before this gene-editing technology becomes a standard therapy. But the research is promising, nonetheless, and could revolutionize treatment for cancer and autoimmune diseases, including HIV and lupus.

“What takes months or even a year may now take a couple weeks using this new technology,” Ramsdell, who was not part of the team behind the study, told the New York Times. “If you are a cancer patient, weeks versus months could make a huge difference.”

“I think it’s going to be a huge breakthrough,” he added.