Promise in Personalized Liver Cancer Treatment

An illustration of DNA with a syringe in the middle.

Illustration by Antonio Giovanni Pinna

Adding a personalized anti-tumor vaccine to standard immunotherapy is safe and about twice as likely to shrink cancer as standard immunotherapy alone for patients with hepatocellular carcinoma, the most common type of liver cancer, according to a clinical trial led by researchers at the Johns Hopkins Kimmel Cancer Center and its Convergence Institute.

Hepatocellular carcinoma (HCC) is one of the leading causes of cancer-related deaths worldwide. Fewer than one in 10 patients survive five years post-diagnosis. Existing immune therapies such as PD-1 immune checkpoint inhibitors, aimed at releasing restraints cancer cells place on the immune system, have limited effects.

A preliminary clinical trial led by Kimmel Cancer Center investigators, published in Nature Medicine, shows that adding a personalized anti-tumor vaccine to PD-1 inhibitor therapy may improve patient outcomes.

The study enrolled 36 patients with HCC. All patients received the PD-1 inhibitor pembrolizumab in combination with a personalized anti-tumor vaccine. The most common adverse effect associated with the vaccine was mild injection site reactions. There were no serious adverse events. Nearly one-third of the patients treated with the combination therapy saw their tumors shrink — about twice as many patients as seen in studies of anti-PD-1 therapy alone in HCC. About 8% had a complete response with no evidence of tumor left after the combination treatment.

“We are at an exciting time in new therapy development. Personalized vaccines are the next generation of vaccines that are showing promise in treating difficult cancers when given with immune checkpoint therapy.”

Elizabeth Jaffee, deputy director of the Kimmel Cancer Center and director of the Convergence Institute 

“The study provides evidence that a personalized cancer vaccine can enhance clinical responses to anti-PD-1 therapy,” says Johns Hopkins oncologist Mark Yarchoan, the study’s lead author. “A larger randomized clinical trial will be needed to confirm this finding, but the results are incredibly exciting.”

Decades of experience and research with cancer vaccines from study co-author Elizabeth Jaffee, deputy director of the Kimmel Cancer Center, and other visionary Johns Hopkins scientists have made the successful trial possible. Jaffee and her colleagues saw the potential of cancer vaccines early on and worked to overcome challenges to their development.