Immunotherapy for children: an option for childhood liver cancer?

(Image: Adobe Stock. Illustration: Patrick Bibbins/Boston Children’s Hospital)

You may have seen advertisements for immunotherapy drugs, or even know someone whose cancer has been treated with these drugs. These drugs, which use the patient’s own immune system to help fight cancer, are becoming increasingly popular for treating solid tumors in adults. However, immunotherapy has been less successful in treating cancer in children.

The reason is that tumors in children differ from those in adults, explains Dr. Allison O’Neilldirector of the Liver Tumor Center at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “Most solid tumors in children are less complex than those in adults,” she says. “This provides doctors with fewer targets for the immune system to recognize, making immunotherapy drugs less effective in childhood cancers.”

But hepatocellular carcinoma (HCC) could change that. This rare and aggressive form of childhood liver cancer shares enough similarities with its adult counterpart to make it a good disease to study immunotherapy treatments for, says Dr. O’Neill.

Understanding Immunotherapy

When your child has a cold, his immune system is mobilized to fight it: immune cells called T cells recognize and destroy viruses, bacteria and other foreign substances that they perceive as harmful. The immune system also tries to fight cancer. It can be tricky, though. Some cancer cells are able to evade immune cells or even prevent them from functioning properly.

Immunotherapy attempts to help the immune system work better to identify and kill cancer cells. Different immunotherapy drugs achieve this in different ways. Some block certain immune system checkpoints, allowing immune cells to react more strongly to cancer. Others stimulate the ability of T cells to fight cancer. And still others strengthen the immune system through various mechanisms.

Two studies could help find answers

The United States Food and Drug Administration (FDA) has approved immunotherapy drugs to treat a number of cancers in adults. Now, Dr. O’Neill hopes two ongoing studies will open doors for the use of immunotherapy in childhood liver cancer.

The first, a trial in pediatric patients with HCC, aims to learn more about how the drug pembrolizumab — already FDA-approved for adults — might allow immune cells to better recognize liver tumors in patients. pediatrics. This study will also enable in-depth exploration of the immune environment of pediatric HCC tumors and an analysis of how circulating and tumor-associated immune cells change in response to treatment with this drug. This trial is open and recruits eligible patients aged 1 to 30 years.

The second study, conducted with Eureka Therapeutics, will seek to investigate the safety of a modified T-cell receptor capable of recognizing a protein (called alfa-fetoprotein) produced by both HCC and hepatoblastoma tumors. This new T cell therapy is currently being studied in adults and is currently being studied for the first time in pediatric patients. This trial is open and is currently enrolling eligible patients ages 12 and older, but will eventually enroll children ages 1 to 21.

“These studies will provide significant insight into the immune landscape of these difficult-to-treat tumours,” says Dr. O’Neill. “They will undoubtedly help us learn more about the role of immunotherapy in pediatric liver cancer.”

To find out more, contact the Liver Tumor Center.

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