At The Jackson Laboratory in Bar Harbor, researchers are learning how to treat cancer, one patient at a time, with the help of furry little mice.
Scientists harvest sections of tumors from sick human patients, implanting the cells into potentially dozens of mice and testing various medications and drug cocktails to find out which will best thwart the cancer’s progress. Each “mouse avatar” grows a human tumor that ultimately helps doctors determine how that particular patient will respond to treatment.
A patient with breast cancer, for example, can be treated for her genetically unique form of the disease, rather than risk experimenting with ineffective drugs that carry damaging side effects, said Charles Hewett, executive vice president and chief operating officer at JAX.
“If you’re lucky and you have a great oncologist and a wonderful medical team, your chances of being prescribed a therapy that will work the first time are roughly one in three,” he said.
Now, the Bar Harbor nonprofit research institution is teaming up with a Boston hospital to advance the research. JAX announced this week a partnership with Beth Israel Deaconess Medical Center, which has developed its own mouse for cancer research.
The hospital’s scientists study the disease in thousands of mice they’ve genetically engineered to be susceptible to cancer, much like some people. By better understanding what causes cancer in the mice, researchers hope to better understand what causes the disease in humans.
Beth Israel Deaconess’ “Mouse Hospital,” developed by Dr. Pier Paolo Pandolfi, director of the hospital’s cancer center, allows investigators to test drugs in both mice and humans at the same time, leading to faster results.
Combining the two approaches will allow JAX and Beth Israel Deaconess to learn more about how cancer spreads and develop better treatments to stop it, the institutions’ researchers believe.
“Our two institutions have developed highly complementary mouse modeling platforms that are providing us with tremendous insights into cancer’s mechanisms,” Pandolfi said in a news release announcing the partnership. “By joining forces, we will be able to speed the pace of future discoveries and bring personalized cancer treatments to patients much more quickly.”
The partnership will also include development of a medical curriculum to help cancer clinicians understand how to use genomic tests. The goal is to spread the information’s reach beyond big academic hospitals like Beth Israel Deaconess, a research affiliate of Harvard Medical School, to small community health centers.
“Most of the oncologists working in Maine are aware, they’re quite well-informed, but they don’t have the day-to-day access to some of this that colleagues in the large research and education centers … would have,” Hewett said. “Many of them are hungry for that.”
Mice have long been prized as test subjects in medical research because of the many correlations between their genetic makeup and that of humans. JAX is one of the world’s largest providers of mice for such research, distributing about 3 million each year.
The mouse avatars represent just a portion of the lab’s work, which also takes place at research centers in Farmington, Connecticut, and Sacramento, California.
They’re part of a broader effort to better diagnose and treat cancer based on patients’ DNA. JAX has developed a test that identifies genetic variations in individual tumors through DNA sequencing, allowing researchers to identify which drugs work — or don’t — against those specific variations, Hewett said.
Oncologists can use the information to develop a treatment plan and determine any clinical drug trials patients might benefit from, he said.
“Knowing what those variations are and what drugs are most likely to work can dramatically reduce the trial and error,” Hewett said. “The last thing you want to do is take a cancer drug that’s not going to work.”
The sequencing test is available to a growing number of patients, but it’s not yet covered by insurance, he said. The test has primarily been used for patients who have recurring cancer, a tumor of unknown origin, or cancer that has metastasized — which describes about 700 patients a year in Maine, Hewett said.
While the expense to develop and test avatar mice varies, a “full-blown test” incorporating 10 different drug and medication combinations costs about $50,000, Hewett said.
Additionally, only a certain kind of patient can benefit from the mouse stand-ins. Often that’s individuals for whom frontline therapies failed and their cancer has returned, Hewett said. Researchers must race against the clock to develop and test the tumors quickly enough to identify treatments that will work for patients who may not have long to live.
Some studies have shown mouse avatars to be successful, though some experts question whether the treatments tested on the mice actually help patients live longer. Animal rights activists have also opposed the research.
But JAX and Beth Israel Deaconess see great promise in their work.
“It’s an opportunity to work together and hopefully find some better cures,” Hewett said.


