16-patient pancreatic cancer trial: vaccine worked on eight, seven of those alive

NCI works on private-company partnership to fast-track more mRNA cancer vaccines

A small clinical trial of a personalized mRNA vaccine for pancreatic cancer produced the results NPR health correspondent Yuki Noguchi described as eye-popping. According to NPR, the vaccine worked on 8 of the 16 patients in the trial, 7 of whom are still alive. Surgeon and scientist Vinod Balachandran ran the trial at Memorial Sloan Kettering Cancer Center, and Noguchi reported on the work on Nov. 2.

Donna Gustafson, a 72-year-old retired accountant, became the world’s first recipient of an mRNA vaccine designed to treat pancreatic cancer seven years ago. She shows zero evidence of the pancreatic cancer that was supposed to kill her years ago, and NPR described her as living a cancer patient’s fantasy. Gustafson received nine doses of the vaccine in all, along with surgery and chemotherapy.

“I have had 100% quality of life. I am back to where I was prior,” Gustafson said. Her husband, Ed, whom she married 50 years ago, said, “She’s better than that.” When Noguchi asked whether Gustafson was sassier, Ed said, “She’s sassier, and she is in the best shape of her life. There’s no question about that.”

The personalized vaccine was designed using samples of Gustafson’s tumor. The aim was to reprogram her immune cells, training them essentially to recognize distinctive mutant proteins on cancerous cells and kill them. A combination of mRNA, genomics and AI helped create these vaccines, which are also being tested on lung, kidney, bladder and brain cancers.

Gustafson said she was not afraid to be the first person to test the concept against pancreatic cancer. “It was a no-brainer. I knew that statistically, the odds of my survival were low, and I was very happy to be offered some type of hope,” she said.

Nor did the shots feel like chemotherapy. “I had no symptoms, no side effects to the point where I was actually worried that maybe it was not helping,” Gustafson said. It was helping, very swiftly, NPR reported.

In the lab, Balachandran pointed Noguchi to a computer screen of green lines tracking the reproduction of immune cells. “This is - Donna, this is your response,” Balachandran said. The lines for Gustafson shot up and then remained high and flat, reflecting how her immune system mass produced cancer-attacking cells that sustained years beyond her last shot. “Ten thousand fold expansion,” Balachandran said of the spike. “This tells us that that response lasts.”

It was that strength and durability of response, Noguchi said, that for Balachandran blew open the possibility of mRNA vaccines. “Well, if you can generate such a strong immune response in the most toughest of cancers, could this similarly develop cancer vaccines for a broad array of human cancers?” he asked.

NPR host Ailsa Chang set the scene: “It’s been a big year for cancer vaccines using mRNA.” Chang said drug makers Merck and Moderna had just released results from the first major successful trial of mRNA vaccines for skin cancer, which showed big reductions in recurrence and death. NPR health correspondent Yuki Noguchi visited another mRNA vaccine lab to report on why interest is running high.

Noguchi reported that researchers do not yet understand why some trials have failed while others have shown eye-popping results in the form of people like Gustafson. The field’s attention has grown alongside its successes.

The National Cancer Institute is working on a partnership with private companies to fast-track research and development of more mRNA cancer vaccines. Balachandran and Memorial Sloan Kettering are part of that effort, with plans to expand manufacture of personalized vaccines for patients on site next year.

Noguchi described the state of the technology this way: “The technology is now at an inflection point with patients, doctors, researchers and investors.”

For Gustafson, the gains have been measured in family time. “I saw two more grandchildren be born and grow, many birthdays, holidays and Christmases with my family,” she said. Donna and Ed Gustafson said they still feel overwhelmingly lucky. Ed said: “We’ve done so many things. We’ve actually done much more than we probably would have done, you know…” Gustafson added, “If we did not have this experience.” Ed put the family’s outlook more plainly: “Like, honestly, we just live every day for the day now.”