Experimental gene therapy appears to have helped a patient with advanced pancreatic cancer


An experimental treatment appears to have succeeded in halting the progression of a woman’s advanced pancreatic cancer, doctors reported in the New England Journal of Medicine on Wednesday.

The apparent success of the therapy – which involves modifying the genes of immune cells so that they attack tumor cells – could be a major advance in the treatment of not only pancreatic cancer, but other cancers as well.

“I’m really excited about this,” said Dr. Carl June, who more than a decade ago pioneered a different type of immunotherapy for certain blood cancers. June did not participate in the new report.

Kathy Wilkes, 71, of Ormond Beach, Florida, was diagnosed with pancreatic cancer in early 2018. She first underwent at least eight rounds of chemotherapy, along with radiation therapy and an operation called a procedure of Whipple to remove part of his pancreas.

Within a year, however, the cancer had spread to his lungs.

Kathy Wilkes, 71, of Ormond Beach, Florida, received experimental gene therapy in 2021 to fight her advanced pancreatic cancer.Courtesy of Kathy Wilkes

“When I spoke to the oncologist in my hometown and asked him what to do, he only had one answer, and that was chemotherapy. And I said, ‘It’s not my answer,’” Wilkes told NBC News.

She found a 2016 case report, also published in the New England Journal of Medicine, which detailed how a person with advanced colon cancer was helped by an experimental type of gene therapy targeting a cancerous mutation called KRAS G12D. .

“I thought, ‘This is the trial I want.’ I knew this was the ordeal that was going to save me, save my life. I just had that feeling,” Wilkes said.

With that in mind, she reached out to the report’s author, Eric Tran. Tran was at the National Institutes of Health when he treated the colon cancer patient, but had since moved to the Providence Cancer Institute in Portland, Oregon. It was there that Wilkes found him and inquired about undergoing the same type of therapy.

It turned out that Wilkes had the same genetic mutation as the colon cancer patient, despite different forms of cancer. Tran, who participated in her therapy, was also the author of the latest New England Journal report.

The experimental approach involved taking a sample of Wilkes T cells, a type of immune cell that attacks invaders in the body. The scientists then genetically modified these cells, reprogramming them to recognize and attack tumor cells.

The T cells were then multiplied billions of times in a lab, before being returned to Wilkes’ body via a single intravenous infusion.

The approach is reminiscent of CAR-T therapy, the form of treatment developed by June at the University of Pennsylvania.

“This is potentially a unique treatment,” said Dr. Rom Leidner, co-author of the new report and co-director of the Head and Neck Cancer Treatment Program at Providence Cancer Institute, of the new therapy.

Kathy Wilkes with her husband.
Kathy Wilkes with her husband.Courtesy of Kathy Wilkes

Wilkes’ infusion took place on June 14, 2021. Within a month, the tumors in his lungs had shrunk by more than half, according to the report. Six months later, the tumors were reduced to 72% of their original size.

It’s unclear how long the treatment may last, however.

But the new therapy is a “living drug,” said Leidner, who treated Wilkes, meaning the modified T cells should continue to grow and proliferate within the immune system, and they should watch for the return. cancer.

Wilkes says her cancer remains stable, but she will undergo additional tests and scans this month as part of an annual update.

Pancreatic cancer is one of the deadliest forms of the disease. It is rarely found before it spreads and is therefore more difficult to treat. According to the American Cancer Society, only about 11% of patients are expected to survive five years after diagnosis.

Another pancreatic cancer patient who received the same treatment at Providence Cancer Institute did not survive. It is not known why the treatment appears to have worked in one person but failed in another.

“We’re working hard to try to answer that question,” Tran said. “If we understand the mechanism, it could help us develop better therapies.”

“An encouraging result”

Wilkes’ doctors were able to target his tumors thanks to a mutation called KRAS. While the majority of pancreatic cancers have a KRAS mutation, Tran said only about 4% of pancreatic cancer patients have the mutation along with a specific molecule on the cell surface needed to be eligible for this. particular therapy.

But the mutation is not limited to pancreatic cancer, said Dr. Eric Rubin, editor of the New England Journal of Medicine. The therapy, he said, therefore has the potential to be used against a variety of cancers.

“This particular mutation is common in tumors that arise from epithelial cells, such as lung, ovarian and pancreatic cancers,” Rubin said at a press conference Wednesday. “We have for the first time an approach that could allow the treatment of a wide variety of tumors beyond the small number of tumors that CAR-T cells can be used in a very specific type of immunotherapy.

Still, Rubin emphasized caution. “It was an encouraging result, but it’s certainly far from a cure,” he said.

Much more research is certainly needed, experts agree. Tran and Leidner are now recruiting patients for a Phase 1 clinical trial to continue studying the therapy.

A potential factor that could explain Wilkes’ positive results is that his pancreatic cancer had spread to his lungs rather than his liver, said Dr. Ryan Carr, a pancreatic cancer expert at the Mayo Clinic in Rochester, Minnesota. Carr, who was not involved in Wilkes’ treatment, said in his experience patients whose tumors have spread to the lungs tend to have more favorable outcomes than patients whose cancer has spread to the liver. , a more common site of metastasis.

“In most situations, when we have these lung metastases, they don’t really cause symptoms in the patient,” Carr said. “It’s still a poor prognosis, but we know there’s something different about them, and they’re a bit better off than those who spread to the liver.”

June said he had had the same experience.

As these details are worked out, June and Carr argue that the new therapy appears to be a major breakthrough in the treatment of pancreatic cancer.

“I think cancer research is going to pick up and build on that,” said June, director of the Parker Institute for Cancer Immunotherapy at the University of Pennsylvania. “The real question is not if but when this type of therapy becomes a reality to cure patients with now deadly cancers.”

CORRECTION (June 1, 2022, 10:45 p.m. ET) A previous version of this article incorrectly stated which patients would respond to gene therapy. These are patients with the KRAS mutation plus a specific molecule on the cell surface, not patients with a subtype of the KRAS mutation.

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