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The rare liver cancer fibrolamellar carcinoma, or FLC, affects only one out of 5 million people, usually children or young adults with otherwise healthy livers.
Because FLC is so rare, it’s difficult to estimate five-year survival rates, which range between 44 and 68% with surgery and 2 to 17% without surgery. There is no curative therapy for FLC.
Its rarity also makes the disease difficult to study in the lab because tumor samples are few and far between.
“You see maybe four or five cases a year, which means every case is precious and important for researchers trying to understand the disease and what drives the tumor,” said rare-cancer expert Taran Gujral, PhD, an associate professor in the Human Biology Division of Fred Hutch Cancer Center.
He recently received a four-year, $1,275,000 grant from the U.S. Department of Defense to study FLC and four other rare liver cancers to make the most of the rare and small samples provided by hospitals.
Working with UW Medicine surgeon Raymond Yeung, MD, founder of the UW Medicine Liver Tumor Clinic, Gujral’s lab will characterize the molecular biology of rare liver tumors, generating fundamental genetic data.
The data will help Gujral’s team form hypotheses about what makes the tumors grow and what might make them shrink.
They will then make models of human tumors to test those hypotheses — both in cells that are cultured and manipulated in the lab, and in genetically engineered mice implanted with tumor cells removed from patients — a crucial step before testing drugs and therapies in patients directly in clinical trials.
The data and preclinical models will be shared publicly to foster collaboration among rare-cancer researchers through a program Gujral directs called TRACER, Fred Hutch’s recently launched Transformative Rare Cancer Initiative.
The power of patient advocates to target funding
The DOD funding represents the power of patient advocates to influence research into specific diseases.
Rare cancers affect fewer than six out of 100,000 individuals, but collectively they account for more than a fifth of all diagnosed cancer cases globally. Because they affect so few individuals, drug companies have little incentive to invest in finding treatments and cures.
The National Institutes of Health provides the largest share of federally funded biomedical research, including the National Institute of Cancer, which is the largest funder of cancer research in the world. NIH grants comprise about 75% of the total sponsored funding at Fred Hutch, which consistently ranks among the top independent research institutes in NIH funding dollars.
But organizations lobbying for more research on specific diseases have also found success by lobbying Congress directly, which channels additional funding through the DOD.
Responding to lobbying from patient advocates, Congress appropriated about $25 million to the DOD for breast cancer research in 1992. The next year, Congress increased the funding to $210 million and put the U.S. Army in charge of the Congressionally Directed Medical Research Programs.
Over the years, the CDMRP increased the range of health care issues it funded, and its budget grew accordingly, receiving about a $1.2 billion appropriation in FY 2020.
That funding included $7.5 million for a newly established rare cancers research program, thanks in part to lobbying from the Rare Cancer Coalition at the National Organization for Rare Disorders, which also successfully advocated for a congressional resolution recognizing Sept. 30 as Rare Cancer Day.
The coalition’s co-founder, John Hopper, is also a founding member of TRACER’s advisory board.
Gujral’s four-year grant from CDMRP specifies three aims:
- Collection, annotation, and functional multi-omics characterization of rare liver tumors.
- Development and dissemination of rare liver tumor preclinical models.
- Development of web-based resources for in-depth exploration of molecular and clinical data.
Collaboration is key
TRACER provides a platform for researchers studying different cancers to come together under one umbrella to share their findings and challenges.
Gujral wants to accelerate rare cancer research with TRACER through collaboration, but also provide new directions for that research by revealing the basic mechanisms driving rare cancers.
Once they understand the biology of the rare liver cancers they are studying with the DOD grant, Gujral’s lab can generate — and share — new data and new pre-clinical models needed to find possible drugs and therapies faster.
“Taran's been an absolutely invaluable investigator,” said Kurt Losert, CEO of the Fibrolamellar Cancer Foundation, which has funded some of Gujral’s research. “We know that his expertise in developing preclinical models and his willingness to freely collaborate with other investigators throughout the world is critical to advancing the understanding of all rare diseases, including fibrolamellar.”
