Despite advances in medical science—including the development of effective mRNA vaccines amid today’s pandemic—treatment for certain cancers remains lacking. For liver cancer, in particular, the efficiency of treatment options leaves much to be desired, and surgical options are typically available only for children and young adults with no prior liver problems. This is especially true for fibrolamellar carcinoma. While recent genetic studies have made progress in identifying the specific genes that drive the growth of fibrolamellar tumors, available drugs used to treat other types of liver cancer simply don’t work on this condition.

To address this, Dr. Sanford M. Simon, Ph.D and his team at The Rockefeller University took on the challenge to find a potential treatment. They did so by testing over 5,000 compounds — either already approved for other clinical uses, or used in clinical trials — to see whether any of the compounds could be repurposed to treat fibrolamellar carcinoma. The researchers discovered a few classes of therapeutics which destroy fibrolamellar tumor cells growing in mice.

“We decided to be completely agnostic about what we thought would work—we tried everything,” asserted Rockefeller’s Dr. Simon, in a press release. “To our surprise, we found a few compounds that work really well.”

Identifying the perfect therapeutic target for a disease and testing drugs to determine their efficiency can take years. As such, Simon and his team took an expedited approach and found few new classes of therapeutics that appear to effectively kill fibrolamellar tumor cells. Their study, published in Cancer Discovery, also provides molecular explanations for why these drugs can be effective against this type of liver cancer that has remained untreated for years.

Simon’s work is an example of precision medicine, which continues to change the face of cancer treatment, with its tailored approach that targets a patient’s specific condition, genetic composition, lifestyle, and environment.

“You don’t want to give everyone with a limp the same treatment—you want it ‘precisely targeted’ based on whether they have twisted their ankle, broken a bone, or just have a splinter,” explained Simon.

The key to applying precision medicine to cancer treatment, Simon explained, is not blindly testing drugs against cancer mutations, but conducting functional screenings that ask which drugs actually have an impact on the tumor in question.

Another key finding from using their precision medicine approach is that it will not be necessary to screen new cancer drug candidates in cells grown in mice before testing them on human cells. That shortcut saves an extra step that would prolong the cancer research for months; precious time which many liver cancer patients may not have.

Simon and his team tested the compounds on human cells taken directly from patients’ tumors. The success of this approach means physicians may soon be able to biopsy cells from a patient’s tumor and subject those cells to being tested by various drug candidates, in order to find the most effective treatment for a specific patient, in a matter of days.

“Up to this point, I’ve had to tell patients that we don’t have any medicines that are proven to work,” said Dr. Michael V. Ortiz — a pediatric oncologist at Memorial Sloan Kettering Cancer Center – who is also a collaborator on the study. “It’s really thrilling that we finally have some promising drugs to go after, in clinical trials. And, since each individual responds differently, it’s particularly exciting that we had multiple hits, which we can now test in combination with one another.”

With additional work underway, this team’s novel approach shows promise in producing the first therapy for fibrolamellar carcinoma. And while we wait for updates from this exciting new research, let’s heed ADRLF’s critical message: Screen. Vaccinate. Don’t hesitate.

To learn more about their work, visit the Simon Lab’s site here

To learn more about liver inflammation, read more here

To explore lifestyle tips for a healthy liver, visit our blog