Patrick McDonough, a young man with a rare cancer of the liver, is working hard to reclaim his life as a college student and music DJ. He’s being helped by doctors and researchers at the Massachusetts General Hospital Cancer Center and friends at the Fibrolamellar Cancer Foundation (FCF).
“One of the key strategies is to partner with the best-in-class thinking to accelerate the path to a cure.”
Touring the lab of Mass General cancer researcher Nabeel Bardeesy, PhD, recently, Patrick placed his hand on his abdomen and described how his own liver tumor has stubbornly resisted treatment since his diagnosis three years ago at age 31. “It’s like Rocky. You can get him in a corner but he keeps coming back at you,” says Patrick who is now seeing some improvement in his condition while undergoing treatment at the Mass General Cancer Center.
Rare Cancer in Young Adults
“Fibrolamellar is a young person’s cancer,” says John Hopper, executive director of FCF. The foundation was founded in 2009 and is now leading the charge to build awareness and find a cure for this extremely rare and deadly disease occurring in only 200 to 300 newly diagnosed teens and young adults worldwide each year.
FCF supports a community of patients and their families. It also raises funds and supports cutting-edge research, such as the work going on in the laboratory of Dr. Bardeesy, an expert in gastrointestinal cancer biology who holds an endowed chair at the Mass General Cancer Center.
“One of the key strategies for the foundation is to partner with the best-in-class thinking we can find to accelerate the path to a cure,” Mr. Hopper said while touring Dr. Bardeesy’s large and busy lab recently. “Dr. Bardeesy brings the knowledge of different types of cancers and connects the dots.”
Alisha Sternenberger, one of FCF’s founders, agrees. She watched her boyfriend, Tucker Davis, succumb to this rare cancer six years ago when he was just 28 years old. During Tucker’s illness he, Alisha, and his family and friends spent many hours searching the internet to locate other patients with the disease. They gradually built a community of people dedicated to supporting patients and their families and raising funds for research and patient care. Today, FCF stands as the only foundation dedicated to granting funds for fibrolamellar cancer support and research, with nearly $6 million invested since 2009.
Following Pathways to a Cure
With a grant from FCF, Dr. Bardeesy is conducting a two-pronged effort to better understand and treat fibrolamellar cancer.
Patients like Patrick are donating tissue samples from their tumors to generate cancer cell lines to be screened against thousands of other cancer cell lines.
First, he is studying alterations in the function of a protein known as PKA, common both to fibrolamellar cancer and to the other cancers he has studied in the liver, bile ducts and pancreas.
This altered pathway seems to divert the normal energy metabolism of cells away from converting nutrients from food into energy toward growing cancer cells instead. Because most patients with fibrolamellar cancer have the PKA mutation, targeting that pathway will help narrow the focus for a cure.
“Verifying that PKA is required for tumor growth and understanding its precise role in the cancer cells are important steps in finding effective drugs,” Dr. Bardeesy says.
Second, and equally important given the ultra-rare nature of this disease, is the collaboration between Dr. Bardeesy and researchers at University of California San Francisco (UCSF). Patients like Patrick are donating tissue samples from their tumors to generate cancer cell lines to be screened against thousands of other cancer cell lines collected at Mass General and UCSF. When a match is found, researchers will look to see which drugs have been used successfully against that cancer in the hope of using the same drug to treat patients with fibrolamellar cancer.
The samples are also being used to generate mouse models, known as patient-derived xenografts, which can be used to further test new treatment strategies for this rare cancer.
A Personal Goal
Meanwhile, Patrick is making regular visits to his Mass General oncologist, Andrew Zhu, MD, PhD, director of liver cancer research at Mass General Cancer Center. There he receives the chemotherapy that is gradually shrinking his aggressive tumor. Because Patrick’s tumor is inoperable, chemotherapy is his best option.
But Patrick and others like him could benefit if Dr. Bardeesy’s research points the way to a more targeted, potent drug to eradicate the tumor completely.
“I was a college student. I’m interested in lots of things and music has always been my passion,” Patrick says. “I would like to get my life back.”
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