But Largaespada believes the real fairy-tale ending will come later, when cancer is considered a largely solved problem.
The Sleeping Beauty tool is essentially a piece of DNA that researchers can make “jump” from one section to another in the chromosomes of cells. This helps the scientists discover which genes and gene pathways are related to cancer development.
Working with genetically modified mice that model human cancers, Largaespada and his team have identified dozens of new cancer genes with Sleeping Beauty’s aid. His lab has reported finding 77 genes tied to colorectal cancer—only seven of which were known previously—and 17 genes tied to liver cancer.
“Now we have a lot of unpublished data on new cancer genes for the [gastrointestinal] tract and liver [and] also for brain tumors, sarcomas, and several other types of cancer,” Largaespada says. “It has really exploded in the lab.”
Funding from the Margaret Harvey Schering Trust for Cancer Research is behind many of Largaespada’s successes. The Schering Trust has been a source of support for his lab since 2006, and most recently it committed another $500,000 to his work. The trust also has funded an endowed chair in cancer genetics that Largaespada has held since the chair was created.
When Largaespada and his colleagues first developed this Sleeping Beauty method for finding cancer genes, they earned a five-year, $1.25 million research project grant from the National Institutes of Health (NIH).
But in Largaespada’s mind, that was just the beginning.
“I wanted to expand the technology so I could show that it would work for other types of cancer besides our initial report, which was on sarcoma and T cell leukemia,” he says. “And I also knew that I wanted to do it fast. The Schering Trust allowed us to get some pilot data showing that the Sleeping Beauty method worked for other types of cancer.”
In fact, that pilot data has earned his lab two additional research project grants from the NIH to study gastrointestinal and liver cancers as well as a new grant from the National Brain Tumor Society.
The Margaret Harvey Schering Trust for Cancer Research was created by the late philanthropists Friedrich Schering and Margaret Harvey Schering, who believed in supporting basic science cancer research to help answer questions about why cancer occurs and how to cure it, according to trustee Mark Gaasedelen.
Largaespada says the trust’s ongoing support is critical to taking his work to the next level. Now he’s connecting the genetics of tumors with clinically relevant traits such as whether a cancer spreads, how it responds to treatment, and whether it recurs.
“These are the things that the clinicians really care about, and all of those things are probably controlled by the genetics of the tumor cell,” he says.
Gaasedelen believes that funding Largaespada’s team’s research is a “perfect match” with the Scherings’ intentions.
“It’s got to be very, very difficult and very, very frustrating at times, but these guys are driven to find answers,” he says. “I feel confident that the money is being well spent.”