Abstract Presenter: Alfred Garfall, MD, director of autologous hematopoietic stem cell transplantation, section chief for myeloma, hematology-oncology, and associate professor of medicine in hematology-oncology at the Hospital of the University of Pennsylvania

Abstract Title: Phase II Multicenter Trial of Idecabtagene Vicleucel (Ide-cel) Followed By Lenalidomide Maintenance for Multiple Myeloma Patients with Sub-Optimal Response after an Upfront Autologous Hematopoietic Cell Transplantation: Top Line Results from the BMT CTN 1902 Clinical Trial

Session: Late Breaking Abstracts, 3:15 – 4:10 p.m. on Feb. 15, Ballroom B

Presentation Time: 3:15 – 3:33 p.m.

What is your presentation/abstract about?

“We are presenting results of a phase 2 clinical trial led by BMT CTN in which anti-B-cell maturation antigen (anti-BCMA) CAR T cells were evaluated in multiple myeloma patients who had failed to achieve a complete response (CR) after standard first-line therapy consisting of induction, high-dose melphalan, autologous stem cell transplantation, and three months of lenalidomide-based maintenance therapy.

“The CAR T-cell therapy we evaluated was idecabtagene vicleucel (ide-cel). We found that 63% of patients achieved CR after ide-cel, 87% improved their overall response to first-line therapy, and 95% achieved minimal residual disease (MRD)-negativity, which is a strong predictor of long-term survival.

“We were impressed by these outcomes since this was a high-risk group of patients. Prior to ide-cel, nearly half our patients had achieved only a partial response to potent first-line therapy. In addition, we showed that CAR T-cell therapy in this new clinical setting was safe and feasible, with only low-grade cytokine release syndrome and no neurologic toxicity, and that patients were able to safely resume standard-of-care lenalidomide maintenance after recovery from the CAR T-cell therapy.” 

How did you first become interested in this topic?

“Our standard first-line therapies for multiple myeloma are quite effective, but patients who fail to achieve CR have poorer outcomes. Since CAR T-cell therapy has shown great promise in relapsed multiple myeloma, we were interested in whether we could integrate CAR T cells into first-line therapy for these patients who had not achieved CR, with the goal of improving their long-term outcomes. We had some basic questions about how well CAR T cells would work against the low-level of myeloma that remains after standard first-line therapy, whether patients early after transplant could tolerate CAR T-cell therapy, and whether patients would be able to resume maintenance therapy with lenalidomide, which is a standard-of-care post-transplant therapy.”

What are you working on next related to this topic?

“These are relatively early results of the trial. We are interested in seeing how these patients do over the longer term. In addition, these patients were very cooperative with an intensive schedule of specimen collection, including multiple bone marrow biopsies, on which we conducted a battery of immunologic and pharmacokinetic correlative studies. We are looking forward to digging into these data and correlating them with patient outcomes. These studies may improve our understanding of the clinical outcomes and yield insights on how to improve CAR T-cell therapy in this clinical setting.” 

How do you hope your work influences the field?

“We think our results provide rationale for a phase 3 study that would definitively evaluate CAR T cells as a post-transplant therapy to improve response. More generally, our results are supportive of CAR T cells as a consolidation therapy, i.e., using CAR T cells to target residual disease that remains after some other prior therapy. Though we tested ide-cel in the post-transplant setting, there are several other clinical settings where this consolidation strategy could be developed, e.g., in the post-induction setting or as a consolidation of second- or later-line therapy. In addition to the very favorable efficacy we observed, the toxicity profile was very favorable, avoiding some of the more concerning toxicities that have been observed with CAR T-cell therapy in patients with relapsed disease.”

What excites you most about transplantation and cellular and gene therapy today?

“What excited me is the amazing dynamism of the field, right at the interface of cutting-edge science and clinical medicine.”

Is there anything else you would like Tandem Meetings attendees to know about your abstract?

“This trial, BMT CTN 1902, was the first CAR T-cell trial to be conducted in BMT CTN. It was a very gratifying collaboration among the BMT CTN sites, the BMT CTN sponsor and administrative team, and the product manufacturer, BMS. In addition, I would like to express my gratitude on behalf of all the investigators to the patients who participated in this trial and their families. All these patients enrolled in this trial after just completing an intensive course of first-line myeloma therapy, including autologous stem cell transplantation, so it was especially generous of them to commit so much time and effort to participate in a trial as complex as this.”