Markey home to two breakthrough therapeutic cancer vaccine trials

LEXINGTON, Ky. (Jan. 29, 2024) — The same mRNA technology behind COVID-19 vaccines is now being used in the fight against cancer, and the University of Kentucky Markey Cancer Center is among an elite group of institutions offering these promising new treatments through clinical trials.

The UK Markey Cancer Center is home to two groundbreaking trials using mRNA vaccines to target pancreatic cancer and nonsmall cell lung cancer — cancer types that are among the most aggressive and challenging to treat.

Unlike preventive cancer vaccines like the HPV vaccine, therapeutic vaccines are designed to reduce tumor growth in patients with cancer or prevent recurrence. The vaccines work by “training” the immune system to recognize specific molecular markers, called antigens, that distinguish cancer cells from healthy cells. The approach is part of immunotherapy, which has emerged as a fourth pillar of cancer treatment alongside surgery, chemotherapy and radiation therapy.

“These trials represent a remarkable evolution in how we approach cancer treatment,” said Zhonglin Hao, M.D., Ph.D., medical oncologist and medical director of Markey Cancer Center’s Clinical Research Office. “By using mRNA technology, we can now create treatments that are precisely targeted to each patient’s specific type of cancer.”

Creating custom vaccines for each patient

The phase II clinical trial for pancreatic cancer evaluates an mRNA vaccine called “autogene cevumeran” among patients whose tumors have been removed by surgery. Each vaccine is completely individualized: After surgical removal, each patient’s tumor is sent to BioNTech in Europe, where scientists sequence its DNA and create an mRNA vaccine targeting that patient's specific cancer markers. The vaccine is given in combination with checkpoint inhibitors, drugs that allow the immune system to mount a stronger attack against cancer.

The treatment offers hope for patients with pancreatic cancer, which has a five-year overall survival rate of only 10-12% and recurrence rates as high as 80% after surgery.

“When it comes to treatment, pancreatic cancer is one of those diseases where we’ve made little, if any, progress over the last couple of decades,” said Joseph Kim, M.D., Markey Cancer Center chief of surgical oncology and principal investigator for the pancreatic cancer trial. “This represents a potential major breakthrough, especially considering that even after successful surgery, many patients experience recurrence due to microscopic disease that isn’t visible through imaging.”

Early results from the phase I trial for the pancreatic cancer vaccine have been encouraging, showing that the vaccine induced immune responses associated with reduced risk of recurrence.

Smart vaccine targets lung cancer’s vulnerabilities

The mRNA vaccine in the nonsmall cell lung cancer trial takes a different approach by teaching the immune system to recognize six different tumor markers associated with nonsmall cell lung cancer, which represents about 85% of all lung cancer cases. Nonsmall cell lung cancer is well-suited for immunotherapies including mRNA vaccines because tumors carry a high number of mutations that can be targeted, says Hao, who is leading the trial at Markey.

“What makes this approach exciting is that it’s targeting markers that are common across many lung cancer patients,” said Hao. “This treatment could potentially help a broader population of patients while still maintaining the precision of mRNA technology.”

Developed by BioNTech, the vaccine called BNT116, is being studied in the phase I trial to assess its safety and efficacy on a small group of patients, with a target enrollment size of 130 across seven countries. Researchers are studying how the vaccine works alone and in combination with other treatments, including chemotherapy and immunotherapy in preventing relapse after surgery or in stage 4 cancer.

Local access to the latest treatments

These trials bring promising new treatments closer to home for patients in Kentucky, which has higher incidence rates of pancreatic cancer and nonsmall cell lung cancer, with outcomes notably worse in Appalachian regions.

Markey’s selection as one of only 17 U.S. sites for the pancreatic cancer trial and one of five for the lung cancer trial stems from its status as a National Cancer Institute-designated Comprehensive Cancer Center — one of just 57 in the nation. This designation places Markey among centers leading the development of new cancer treatments.

“Clinical trials represent the future of cancer care,” said Hao. “When patients participate in trials at Markey, they gain access to new treatments years before they become widely available. For those with limited options, this can make a real difference.”