One Third of Near-Death Multiple Myeloma Patients Are Cured by a New CAR-T Immunotherapy

Many consider immunotherapy to be the most promising current approach to curing cancer. One way to implement immunotherapy is to develop CAR-T cells. But there apparently are many ways to develop a CAR-T cell and which, if any, will work is a matter of trial-and-error.

It seems overly-cautious for regulators to require that the most innovative and promising therapies must first be tried on the patients nearest to death, and so least likely to respond. Why not allow patients at earlier stages to volunteer to try the new therapies earlier? They would be taking a bigger risk, but also would have the possibility of a bigger benefit. They would avoid the suffering from current treatments that are known to have major side-effects, and also are known to only extend life for short periods of time; and they would gain a shot at a real long-term cure.

(p. A18) A group of 97 patients had longstanding multiple myeloma, a common blood cancer that doctors consider incurable, and faced a certain, and extremely painful, death within about a year.

They had gone through a series of treatments, each of which controlled their disease for a while. But then it came back, as it always does. They reached the stage where they had no more options and were facing hospice.

They all got immunotherapy, in a study that was a last-ditch effort.

A third responded so well that they got what seems to be an astonishing reprieve. The immunotherapy developed by Legend Biotech, a company founded in China, seems to have made their cancer disappear. And after five years, it still has not returned in those patients — a result never before seen in this disease.

These results, in patients whose situation had seemed hopeless, has led some battle-worn American oncologists to dare to say the words “potential cure.”

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The new study, reported Tuesday [June 3, 2025] at the annual conference of the American Society of Clinical Oncology and published in The Journal of Clinical Oncology, was funded by Johnson & Johnson, which has an exclusive licensing agreement with Legend Biotech.

. . .

The Legend immunotherapy is a type known as CAR-T. It is delivered as an infusion of the patient’s own white blood cells that have been removed and engineered to attack the cancer. The treatment has revolutionized prospects for patients with other types of blood cancer, like leukemia.

Making CAR-T cells, though, is an art, with so many possible variables that it can be hard to hit on one that works.

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The . . . study took on a . . . challenge — helping patients at the end of the line after years of treatments. Their immune systems were worn down. They were, as oncologists said, “heavily pretreated.” So even though CAR-T is designed to spur their immune systems to fight their cancer, it was not clear their immune systems were up to it.

Oncologists say that even though most patients did not clear their cancer, having a third who did was remarkable.

To see what the expected life span would be for these patients without the immunotherapy, Johnson & Johnson looked at data from patients in a registry who were like the ones in its study — they had failed every treatment. They lived about a year.

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. . ., the hope is that perhaps by giving it earlier in the course of the disease, it could cure patients early on.

Johnson & Johnson is now testing that idea.

Dr. Kenneth Anderson, a myeloma expert at Dana-Farber Cancer Institute who was not involved with the study, said that if the treatment is used as a first-line treatment, “cure is now our realistic expectation.”