How mRNA vaccines could help fight cancer

Although vaccines against infectious diseases are used as a preventive measure, cancer vaccines are primarily used as treatments. Cancer vaccines target antigens on tumor cells so the immune system will recognize and destroy them.

Currently, there are two types of mRNA cancer vaccines in development: "bespoke" or personalized vaccines that are tailored to a specific target or "off-the-shelf" vaccines that target antigens shared across all patients.

"The appeal of cancer vaccines is that you can make it much more specific – you can basically give the immune system written instructions," said David Pinato, a consultant medical oncologist at Imperial College Healthcare NHS Trust. "It's almost like an identikit of the tumour cells, which is more precise."

According to Heather Shaw, a consultant medical oncologist at University College London Hospital, personalized mRNA cancer vaccines are "one of the most exciting things we've seen in a really long time."

"This is very much an individualised therapy and it's far cleverer in some senses than a vaccine," Shaw said. "It is absolutely custom-built for the patient – you couldn't give this to the next patient in the line because you wouldn't expect it to work."

Shaw also noted that the ultimate goal of cancer vaccines is to permanently cure patients of the disease. "I think there is a real hope that these will be the gamechangers in immunotherapy," she said.

Current progress with mRNA cancer vaccines

Although research into cancer vaccines is still preliminary, several companies have reported promising results from clinical trials. According to MIT Technology Review, there are currently around 400 ongoing clinical trials for cancer vaccines, and roughly 50 are testing personalized vaccines.

Moderna and Merck

Moderna and Merck have partnered on an experimental cancer vaccine that targets melanoma, a deadly form of skin cancer. In a mid-stage clinical trial, the vaccine, along with Merck's immunotherapy Keytruda, reduced the risk of death or cancer recurrence after three years by 49% compared to Keytruda alone. The companies are also testing the treatment combination for lung cancer in a phase 3 trial.

Moderna also recently launched three clinical trials for an experimental mRNA vaccine therapy for patients with bladder cancer, kidney cancer, and a type of skin cancer called cutaneous squamous cell carcinoma.

BioNTech and Genentech

BioNTech and Genentech have partnered to create a personalized mRNA vaccine targeting pancreatic cancer. Pancreatic cancer is one of the deadliest cancers, and many treatments, including chemotherapy and radiation, are largely ineffective against it.

In a small trial, 16 patients with pancreatic cancers received immunotherapy, a cancer vaccine, and a standard chemotherapy regimen following surgeries to remove their cancer. Of the patients, half responded to the vaccine. After three years, six of the eight patients have not seen their cancer recur. The companies are currently testing the efficacy and safety of the vaccine in a larger patient group in a phase 2 clinical trial.

BioNTech is also currently testing mRNA vaccines for melanoma and head and neck cancers in phase 2 trials.

University of Florida and iOncologi

In a recent study published in Cell, researchers at the University of Florida and iOncologi reported the results of a personalized mRNA cancer vaccine for glioblastoma, a type of aggressive brain cancer.

In a phase 1 trial, researchers tested the vaccine in four human patients and 10 pet dogs that had spontaneously developed brain cancer. According to a press release, the dogs lived for a median of 139 days, much longer than the usual 30- to 60-day survival time, and the human patients survived or lived without disease for "longer than expected."

Currently, the researchers are working on an expanded phase 1 trial that will include up to 24 pediatric and adult patients. After they establish dosing, the researchers plan to enroll 25 pediatric patients in a phase 2 trial.

NYU Langone Health

Researchers at NYU Langone Health's Perlmutter Cancer Center are currently testing the immunotherapy drug pembrolizumab and an experimental mRNA vaccine V940 in patients with metastatic melanoma.

In a phase 2b trial, 22.4% of patients who received the immunotherapy treatment and the vaccine saw their cancer return within three years of follow-up, compared to 40% of patients who received the immunotherapy treatment alone.

Based on these results, FDA gave the combination treatment a Breakthrough Therapy Designation. Currently, the researchers are working on a randomized, double-blind phase 3 study for the combination treatment and are aiming to enroll over 1,000 patients across the United States and Australia. (Reed, Axios, 5/7; Fleck, Medscape, 5/2; Willyard, MIT Technology Review, 5/3; Kirby, The Independent, 2/4; Constantino, CNBC, 12/14/23; Floersh, Fierce Biotech, 5/7; Stallard, Memorial Sloan Kettering Cancer Center, 4/7; Gregory, The Guardian, 4/26; NYU Langone Health news release, 3/26)

Breakthroughs in treatment and diagnostics offer the promise of personalized, tailored care for every patient. However, healthcare leaders face four key challenges in delivering this level of "bespoke care" at scale. Learn how to navigate these obstacles to improve healthcare access for all patient