Patients in cell therapy Carvykti trial in remission after 5 years
1/3 of patients given single infusion free from disease progression, data show
One-third of patients with hard-to-treat myeloma were alive and free from disease progression five years after a single infusion of the cell therapy Carvykti (ciltacabtagene autoleucel), according to data from a clinical trial.
The findings were presented at the 2025 American Society of Clinical Oncology annual meeting, held May 29-June 2 in Chicago and online, and simultaneously published in The Journal of Clinical Oncology in a study titled, “Long-Term (≥5-Year) Remission and Survival After Treatment With Ciltacabtagene Autoleucel in CARTITUDE-1 Patients With Relapsed/Refractory Multiple Myeloma.”
The study was sponsored by Janssen, now Johnson & Johnson Innovative Medicine, which co-developed Carvykti with Legend Biotech.
“Across our multiple myeloma portfolio and pipeline, we are shifting from treating to progression to treating to cure,” Jordan Schecter, MD, vice president of research and development for multiple myeloma at Johnson & Johnson Innovative Medicine, said in a company press release. “Our focus is to extend patient survival, and based on our expertise of the disease biology, develop treatment regimens with curative potential.”
Carvykti is a CAR T-cell therapy that’s approved in the U.S. as a second-line treatment for multiple myeloma. The therapy works by collecting T-cells (a type of immune cell) from a patient, then engineering the cells to equip them with a molecular weapon called a chimeric antigen receptor or CAR that targets BCMA, a protein expressed by myeloma cells. The engineered T-cells are then infused back into the body to go after the cancer cells.
Cell therapy patients in remission don’t require additional treatment
The new data come from a Phase 1/2 clinical trial called CARTITUDE-1 (NCT03548207). In the study, Carvykti treatment was given to 97 people with myeloma that was relapsed or refractory, meaning the cancer had failed to respond to prior treatments or had come back after initially responding to other treatments.
The new data show that the median overall survival time for these patients was slightly more than five years after a single infusion of Carvykti. Historically, the median overall survival time for people with heavily pretreated relapsed or refractory multiple myeloma has been about one year, the researchers noted.
Of the 97 participants, 32 were alive without signs of disease progression, and without requiring additional lines of treatment, at least five years after one infusion of Carvykti. The researchers noted that these patients with long-term progression-free survival were similar to the overall study population in terms of starting characteristics such as the presence of high-risk genetic changes in their cancer cells.
“This new evidence shows how a single infusion of Carvykti can help patients survive without disease progression much longer than previously thought possible in this setting, and without any maintenance or subsequent treatment. In a heavily pre-treated population, a third of patients remained treatment- and progression-free for at least five years,” said Peter M. Voorhees, MD, co-author of the study and a professor at Wake Forest University School of Medicine.
A dozen of the patients who showed long-term progression-free survival underwent detailed testing over the course of the trial, and findings showed that all of them were negative for minimal residual disease, essentially meaning that they had no detectable myeloma cells. These data imply that Carvykti may have “curative potential,” at least in some patients, the researchers wrote.
The long-term safety data from CARTITUDE-1 were consistent with the treatment’s known safety profile. Among the patients who showed long-term progression-free survival, two developed other types of cancer and there were four serious infections reported.
In a separate presentation at the ASCO meeting, researchers discussed data from an ongoing Phase 3 study called CARTITUDE-4 (NCT04181827), which is comparing the effects of Carvykti against standard-of-care myeloma treatments in patients of different subgroups, such as those with high-risk genetic changes or with extramedullary disease (cancer cells outside the bone marrow). Results showed that, across all the subgroups, survival outcomes were consistently better with Carvykti than with standard-of-care treatments. Data from this study, as well as CARTITUDE-1, formed the main basis for the U.S. approval of Carvykti as a second-line myeloma therapy.
“[Carvykti] improved [progression-free survival] and [overall survival] vs [standard of care] in all subgroups … These data continue to support a positive benefit-risk ratio for [Carvykti],” the researchers wrote.
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