Luciano Costa waited three years to see how patients with an aggressive bone-marrow cancer would fare on an experimental immunotherapy treatment. Reviewing the results gave him goose bumps. “Holy shit,” the oncologist thought.
More than 80 percent of patients who got the therapy were still alive without their cancer spreading after nearly three years, compared with 30 percent of patients in a control group who received a standard treatment, the clinical trial found.
All of the patients in the trial had a cancer called multiple myeloma that had come back after previous treatments, part of a historically grim cycle in which each reprieve gets progressively shorter until they die of the disease. Remarkably, patients on the investigational therapy have remained in remission much longer than has been possible with approved drugs, said Costa, lead author of the study published in December in the New England Journal of Medicine.
The treatment carried risks: 71 percent of the people who got it developed serious reactions, including infections, and 7 percent died. (By contrast, 62 percent of those in the control group had bad reactions, which were fatal for 6 percent of those patients.) Still, the results are raising hopes among physicians and researchers about finding a potential cure for multiple myeloma, something that was unthinkable not long ago.
“We’re entering a new era where dying from this disease is no longer going to be the norm,” said Costa, director of the Multiple Myeloma Program at the University of Alabama at Birmingham and a consultant for Johnson & Johnson, which developed the regimen and sponsored the study.
Officials at the Food and Drug Administration contacted Johnson & Johnson after the study was unveiled in November. Last month, the agency said it had awarded the drugmaker a new, controversial voucher designed to accelerate the approval of promising medicines.
Johnson & Johnson said last month that the therapy “has the potential to change the standard of care” and could be an outpatient option, adding that serious infections subsided by taking steps to fight infection and providing less frequent dosing. The new treatment is a combination of two immunotherapy drugs, Tecvayli and Darzalex Faspro, that were individually approved by the FDA.
The mainstay of advances in treating multiple myeloma is redirecting the body’s immune system to attack the cancer. Even as recent studies have fueled optimism that a cure could be in reach, experts caution that harnessing the immune system is incredibly complex and fraught with risk.
“We do expect that acute and chronic toxicities” with therapies like Tecvayli “will continue to pose a challenge for broad access to the regimen,” Daina Graybosch, an analyst at Leerink Partners, wrote in a note to clients when the paper published in December.
In multiple myeloma, cancer cells multiply in the bone marrow and hijack the body’s ability to fight disease, sowing tumors in bones throughout the body and hollowing them out. The National Cancer Institute estimates that some 36,000 people were diagnosed with the cancer last year and that about 12,000 died of it.
In the study, 587 patients with multiple myeloma who had undergone up to three prior treatments were randomly assigned into two groups: The control group received the typical standard of care consisting of Darzalex plus a steroid and another drug for multiple myeloma, while the rest received the two-drug combination.
Darzalex latches on to myeloma cells and flags them for the body’s immune system to eradicate. Tecvayli grabs on to myeloma cells and the body’s T cells and brings them together to kill the cancer. Combined, the drugs deliver a one-two punch, said Attaya Suvannasankha, a professor at the Indiana University School of Medicine who was not involved in the clinical trial.
With adequate supportive measures, “the infection risk is quite manageable,” said Suvannasankha, who has also been paid as a scientific adviser to a Johnson & Johnson subsidiary and other companies developing cancer drugs. One major uncertainty, she said, is how strong of a dose, and for how long, to give patients to avoid overstimulating immune cells “to the point where they are exhausted and cannot fight.”
For some patients in the clinical trial, the results have been life-changing. Sally Herring, 69, learned that she was at risk of developing multiple myeloma more than a decade ago and that she would be dealing with it for the rest of her life.
“It was crushing, absolutely crushing,” said Herring, a mother of four and an Episcopal priest in Birmingham, Alabama. “Knowing that eventually I was probably going to be in a train wreck and there wasn’t anything I could do about it.”
The cancer struck just as she’d been counseled by her doctors: She was diagnosed in 2017, had a stem cell treatment coupled with chemotherapy that gave her a reprieve, and then the cancer returned in 2022. That’s when she enrolled in the clinical trial for the Tecvayli-Darzalex combination, and three years later, her bloodwork shows no trace of myeloma.
For the most part, Herring has experienced mild side effects from the monthly injections that she likened to a 24-hour flu, plus recurring sinus infections, and she still works full-time. She knows she’s lucky, having watched friends with the same cancer die “excruciating, ridiculously horrific deaths,” she said. Every month, she knows that her bloodwork could show that the cancer has returned. But the disease is “not the big, bad bogeyman anymore that it used to be.”
The Tecvayli-Darzalex results come on the heels of another breakthrough drug — developed by Johnson & Johnson and partner Legend Biotech — that harnesses the body’s immune system. In this approach, a patient’s plasma is filtered to collect their T cells, which are then engineered to recognize myeloma. These modified cells are then multiplied in a lab and infused back into the patient’s bloodstream to attack the cancer. A study published in June showed that a third of the 97 patients in the clinical trial remained alive, with no progression of their cancer, five years after a single infusion of the drug, Carvykti.
“We’re now starting to talk about cure for the first time” in myeloma, Mark Wildgust, a Johnson & Johnson executive, said of Carvykti at an investment conference in November, according to a recording archived by S&P Global Market Intelligence. In evaluating effectiveness, he said, “cure is the benchmark.”
Carvykti, approved in 2022 by the FDA to treat multiple myeloma after patients have undergone four other therapies, has the advantage of being a one-and-done infusion. But it is also available only at select, specialized treatment centers and effectively out of reach for many patients with multiple myeloma. By contrast, the injections of Tecvayli and Darzalex can be given at cancer centers large and small.
Hearn Cho, chief medical officer of the Multiple Myeloma Research Foundation, said the 80 percent of patients whose cancer hadn’t progressed after three years on the Tecvayli-Darzalex combination is unprecedented. “It didn’t just outperform the control arm,” he said. “It smashed it.”
The foundation is researching the optimal dose of Tecvayli with support from Johnson & Johnson. “It’s like conducting an orchestra,” Hearn said. “It’s not like dropping a bomb.”
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