Allison Frances O'Neill, M.D., CLIP Investigator Dana-Farber Cancer Institute

Hepatocellular carcinoma (HCC) is the second most common form of liver cancer occurring in pediatric patients. Many of these patients do not survive long-term, even if they initially respond to chemotherapy, because their disease has already metastasized, or spread to other organs. However, checkpoint inhibition—a type of immunotherapy that can unleash T cells against tumors—has been relatively effective in adults with HCC, and may offer a promising option for pediatric HCC patients, too.

To that end, Dr. O’Neill will soon start enrolling patients to a multi-institutional phase II clinical trial in which pediatric patients with relapsed or refractory HCC will be treated with checkpoint inhibition targeting the PD-1 pathway. In addition to evaluating the benefits of this approach for pediatric patients, Dr. O’Neill also aims to characterize the tumor immune environment for each patient’s tumor.  This will potentially allow pairing of response rates with the immune characteristics of each tumor while identifying factors that might be prognostic and predictive of response, all with the goal of improving care for this patient population.