Despite novel treatment advances, hepatocellular carcinoma (HCC) remains difficult to treat. This aggressive cancer is often not found until later stages and not all people with HCC can utilize available treatments, like surgery or a liver transplant. Pharmaceutical company Medivir AB is currently working to address this unmet need through developing fostroxacitabine bralpamide (fostrox), which the company describes as an orally administered nucleotide DNA polymerase inhibitor that delivers treatment directly to the tumor.

In a news release from mid-January 2024, Medivir shared that data from an ongoing Phase 1b/2a clinical trial was presented during the American Society of Clinical Oncology’s Gastrointestinal Cancer Symposium on January 19. The trial is evaluating fostrox in conjunction with Lenvima (lenvatinib) as a potential therapy for hepatocellular carcinoma in individuals for whom their first-line or second-line treatments did not work or aren’t tolerable.

Trial data thus far shows that fostrox and Lenvima are safe and well-tolerated. Though a small number of participants discontinued treatment due to adverse reactions, a majority of patients show a solid and sustained response, with one individual having a sustained partial response for nearly 1.5 years. The combination treatment has an overall response rate of 25%, which means that 25% of people have either partially or fully responded to treatment. In this case, it means evidence of cancer has decreased. 61% of patients had stable and controlled disease by 18 weeks (slightly over four months), and the combination treatment seems to stave off disease progression. The median time from treatment to progression is around 5.1 months.

Moving forward, Medivir hopes to advance fostrox into additional clinical studies and hopes that drug approval is on the horizon.

Understanding Hepatocellular Carcinoma (HCC)

Hepatocellular carcinoma is a primary liver cancer, which means that it originates in the liver. This cancer is considered rare but is also the most common form of primary liver cancer. HCC occurs most often in people with chronic liver diseases like liver cirrhosis, iron storage disease, or hepatitis B or C. Additional risk factors for developing HCC include being obese, having diabetes, or drinking lots of alcohol. In Medscape, Dr. Luca Cicalese, MD, FACS notes, however, that 25% of people with HCC have no pre-existing liver disease or risk factors. Dr. Cicalese also notes that the global incidence of hepatocellular carcinoma has increased over the years and is predicted to continue to increase in countries like the United States until 2030.

People with hepatocellular carcinoma may not show symptoms in early stages of their cancer. As the cancer progresses, symptoms may appear. If you experience any of the following symptoms for a period of two weeks or more, consider speaking to a physician:

• Upper abdominal pain or a feeling of heaviness
• A lump in your upper abdomen
• Weakness, fatigue, and/or general malaise
• Nausea and vomiting
• Abdominal distention
• Appetite loss
• Weight loss without trying
• Pale, chalky bowel movements
• Dark urine
• Jaundice (yellowing of the skin, eyes, and mucous membranes)
• Gastrointestinal bleeding
• Extremely itchy skin