On June 3, 2025, the Journal of Clinical Oncology published a study funded by Johnson & Johnson, which showed that one-third of patients with relapsed/refractory multiple myeloma who were treated with CARVYKTI® (ciltacabtagene autoleucel) had no signs of cancer more than five years post-treatment.[1]
Multiple myeloma is a cancer that forms in plasma cells, a type of white blood cell.[2] These plasma cells build up in bone marrow and crowd out healthy blood cells, leading to complications such as severe bone pain and bone problems, infections, kidney problems, and anemia.[3]
Relapsed multiple myeloma, which nearly all myeloma patients experience after some time, occurs when the cancer returns after an initially successful treatment.[4] Ultimately, for some patients, the relapsed multiple myeloma no longer responds to treatment.[5] This results in relapsed refractory multiple myeloma, which occurs when the disease is non-responsive or progressive on therapy or within sixty days of the last treatment in patients who had achieved a minimal response from the prior therapy.[6] Survival rates for patients with heavily pretreated relapsed refractory multiple myeloma are extremely low, with a median overall survival of about one year.[7]
In 2017, a Chinese study showed that 94% of patients with relapsed or refractory multiple myeloma experienced clinical remission after treatment with chimeric antigen receptor T (“CAR-T”) cells created by Legend Biotech[8], which target B-cell maturation antigen (BCMA), a protein expressed on the surface of multiple myeloma cancer cells.[9] Johnson & Johnson, impressed with the results of the study, partnered with Legend Biotech to test and seek regulatory approval for the immunotherapy, which is now sold under the brand name CARVYKTI®.[10]
In 2018, as part of the regulatory process, Johnson & Johnson and Legend Biotech conducted a two-year study (CARTITUDE-1) to test the safety and efficacy of the Legend Biotech CAR-T cells in adults with multiple myeloma.[11] During this study, 97 patients with heavily pretreated relapsed/refractory multiple myeloma were infused with CARVYKTI®.[12] The vast majority of patients in the study were triple-class refractory, meaning they were resistant to treatment with three of the main classes of treatment for multiple myeloma, namely immunomodulatory imide drugs (IMiDs), proteasome inhibitors (PIs), and anti-CD38 monoclonal antibodies (MAbs).[13] After two years, 55% of the CARTITUDE-1 patients were progression-free (i.e., the multiple myeloma had not returned or had not progressed). Now, more than five years after the patients first received a single infusion of the CAR-T cells, Johnson & Johnson and Legend Biotech have released a promising study following up with the patients from the CARTITUDE-1 study.[14]
Of the initial 97 patients infused with the CAR-T cells, 45 individuals are alive and in long-term follow-up.[15] Amazingly, one-third of the initial 97 patients continue to have no sign of cancer, leading some experts to hypothesize that there may be hope for a cure to multiple myeloma in the future if CARVYKTI® is administered in the earlier stages of the disease.[16]
While the results of the study may mark a significant step forward in the treatment of patients with multiple myeloma, CARVYKTI® is not without cost. CARVYKTI®’s list price is $555,310 for a one-time treatment.[17] And treatment with CARVYKTI® can be grueling, potentially resulting in weeks-long hospital stays and severe side effects.[18] However, despite the costs, given the positive results of the Johnson & Johnson-funded study, CARVYKTI® shows promise for the treatment and potential cure of multiple myeloma.
Editor: Brenden S. Gingrich, Ph.D.
[1] Jagannath et al., Long-Term (≥5-Year) Remission and Survival After Treatment with Ciltacabtagene Autoleucel in CARTITUDE-1 Patients With Relapsed/Refractory Multiple Myeloma, J. Clin. Oncology (2025), available at https://ascopubs.org/doi/pdf/10.1200/JCO-25-00760 (“Jagannath”).
[2]The Mayo Clinic, Multiple Myeloma, https://www.mayoclinic.org/diseases-conditions/multiple-myeloma/symptoms-causes/syc-20353378, last visited June 16, 2025.
[3] Id.
[4] Leukemia & Lymphoma Society, Refractory and Relapsed Myeloma, https://www.lls.org/myeloma/treatment/refractory-and-relapsed, last visited June 16, 2025.
[5] Id.
[6] Alice Hyde, Multiple Myeloma Hub, Defining Relapsed and Refractory MM: The Challenges and Controversies, https://multiplemyelomahub.com/medical-information/defining-relapsed-and-refractory-mm-the-challenges-and-controversies, last visited June 16, 2025.
[7] Jagannath at 1.
[8] HealthTree Foundation ASCO 2017: CAR T Cell Therapy Shows Incredible Results in Myeloma, https://healthtree.org/myeloma/community/articles/asco-2017-car-t-cell-therapy-shows-incredible-results-in-myeloma, last visited, June 16, 2025; Gina Kolata, From No Hope to a Potential Cure for a Deadly Blood Cancer, N.Y. Times, June 3, 2025 (“Kolata”).
[9] Legend Biotech Unveils Groundbreaking 5-Year Survival Data for CARVYKTI® in Multiple Myeloma at 2025 ASCO Annual Meeting, at About CARVYKTI®, available at https://investors.legendbiotech.com/node/9081/pdf, last visited June 16, 2025; Christina Sumners, Your Questions about BCMA and Multiple Myeloma, Answered, The University of Texas MD Anderson Cancer Center, available at https://www.mdanderson.org/cancerwise/your-questions-about-bcma-and-multiple-myeloma–answered.h00-159619434.html, last visited June 16, 2025.
[10] Kolata.
[11] A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma (CARTITUDE-1), NCT03548207, https://www.clinicaltrials.gov/study/NCT03548207?tab=table, last visited June 16, 2025.
[12] Jagannath at 1, 2.
[13] Id.; Joseph Mikhael, Treatment Options for Triple-class Refractory Multiple Myeloma, 20 Clin. Lymphoma Myeloma and Leukemia 1 (2020), available at https://www.sciencedirect.com/science/article/pii/S2152265019320087.
[14] Jagannath at 2.
[15] Id.
[16] Id.; Kolata.
[17] Kolata.
[18] Kolata.
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