Doctors: cancer patients cured a decade after gene therapy

In 2010, doctors treated Doug Olson’s leukemia with experimental gene therapy who turned some of his blood cells into cancer killers. More than a decade later, there are no signs of cancer in his body.

The treatment cured Olson and a second patient, according to doctors at the University of Pennsylvania, who said it was the first time the therapy had been studied for so long.

“I’m doing very well at the moment. I am still very active. I was running half marathons until 2018,” said Olson, 75, who lives in Pleasanton, Calif. “It’s a cure. And they don’t use the word lightly.

His doctors describe both cases in a study published Wednesday in the journal Nature. They say the two examples show the treatment, called CAR-T cell therapy, can attack the cancer immediately, then stay inside the body for years and evolve there to keep the disease at bay. These “living drugs” are now used by thousands of people around the world to treat certain blood cancers.

Based on the 10-year results, “we can now conclude that CAR-T cells can actually cure leukemia patients,” said Dr. Carl June, one of the study’s authors.

the single treatment involves collecting the patient’s T cells, the white blood cells essential to the immune system, and genetically modifying them in the laboratory so that they find and attack cancer cells. The modified cells are returned to the patient by IV.

By the time Olson received the treatment, he had been battling cancer for years. When doctors diagnosed him with chronic lymphocytic leukemia in 1996, he said: “I thought I had months to live.

He eventually underwent chemotherapy, and at one point his doctor, Dr. David Porter, suggested he might need a bone marrow transplant. Porter also raised the idea of ​​joining a CAR-T therapy study. Olson, chief executive of a New Hampshire lab products company, said he was excited about science and eager to avoid the transplant.

A few weeks after receiving the treatment, he felt sick for about a week and was hospitalized for three days.

“It was the next week, he sat me down and he said, ‘We can’t find a single cancer cell in your body,'” Olson recalled.

The other patient, retired corrections officer Bill Ludwig, had similar results.

Over time, the researchers say, the modified cells evolved, many of them becoming “helper” cells that work with cancer killer cells. Helper cells eventually became dominant in both patients.

Study author J. Joseph Melenhorst said they were able to isolate and analyze the cells using new technologies, which gave them “very good insight” into the how they persisted in the patient’s body.

Dr. Armin Ghobadi of Washington University in St. Louis, an expert in gene and cell immunotherapy against cancer, called the findings “incredible”. Although the word “cure” is rarely used in the case of cancer, he said it seems that these patients are “very likely” cured.

He was intrigued by the persistence of CAR-T cells and how the living drug evolves.

“It’s really beautiful to see,” said Ghobadi, who was not involved in the study.

At this point, June said, tens of thousands of patients are being treated with CAR-T cell therapies, which have been approved for certain blood cancers by health authorities around the world, including the US Food and Drug Administration. -United. The agency first approved a CAR-T therapy treatment in 2017 developed by Penn and drugmaker Novartis for childhood leukemia.

The Nature study was funded in part by the Novartis Institute for Biomedical Research and in part by grants from the National Institutes of Health.

Scientists hope to see wider use of CAR-T therapies in the future to other cancers. Last year, a CAR-T cell therapy was approved for multiple myeloma, the most common malignancy of the bone marrow in adults. According to the Leukemia & Lymphoma Society, leukemia, lymphoma and myeloma were expected to account for just under 10% of the 1.9 million new cancer cases in the United States last year.

“But the big scientific challenge — and it’s a big challenge — is how to make this work in solid cancers,” like lung, colon and other places, June said.

Even in blood cancers, there are challenges. The therapies are expensive, running into the hundreds of thousands of dollars for the drugs alone. And there is a risk of significant side effects, including an overreaction of the immune system called ‘cytokine release syndrome’ and nervous system problems such as brain swelling.

Both of Penn’s patients did extremely well after treatment. Ludwig traveled the country with his wife in an RV and celebrated family milestones before he died early last year of complications from COVID-19.

Olson said he was extremely grateful for the decade of life since doctors used cutting-edge science to save him.

“What has changed is the dimension of hope. The pace of discovery takes your breath away,” he said. “It’s a whole new world.”