New FDA CAR-T Therapy Approvals Change the Paradigm for Multiple Myeloma Patients

As reported by the international oncology news and information provider OncLive, FDA approvals of two key CAR-T therapy modalities has given new hope for people with relapsed and/or refractory multiple myeloma. A cancer that forms in blood plasma cells, multiple myeloma inhibits the ability of these important white blood cells to produce antibodies that fight infection.

Multiple myeloma presents itself when a preponderance of cancerous plasma cells build up in the bone marrow, crowding out healthy white blood cells and making it impossible for the immune system to effectively identify and attack pathogens. In fact, these cancerous plasma cells produce abnormal proteins that exacerbate the condition by causing a number of harmful complications.

Unfortunately, multiple myeloma has extraordinarily high rates of relapse and refraction due to its unique genomic evolution. A reoccurrence of cancer that had previously gone into remission, relapse can be absolutely heartbreaking for a multiple myeloma patient. Even worse, with each relapse, patients generally face a dramatically increased risk of the disease becoming refractory (irresponsive to treatment).

For this reason, CAR-T therapy experts, such as Massachusetts General Hospital Center for Multiple Myeloma director Noopur S. Raje, MD, are celebrating the recent approval of two key multiple myeloma treatments by the US Food and Drug Administration (FDA). Idecabtagene vicleucel (commonly known as “ide-cel” and marketed under the brand name Abecma) and ciltacabtagene autoleucel (commonly known as “cilta-cel” and marketed under the brand name Carvykti) offer new hope for people who have either relapsed or refractory multiple myeloma.

A champion of increased cancer treatment accessibility, Dr. Raje gave a provocative presentation about CAR-T therapy products for the treatment of multiple myeloma at the 26th Annual International Congress on Hematologic Malignancies in February of 2022. She views drugs such as ide-cel and cilta-cel as critical assets in the fight against multiple myeloma and its serious immune deficiency implications. In addition to severely limiting the human body’s capacity to fight infection, multiple myeloma can lead to serious bone problems, reduced kidney function, and low red blood cell counts.

One of two CAR-T therapy products with FDA approval, ide-cel was officially sanctioned for use in multiple myeloma treatment in March of 2021. Ide-cel has been proven to produce a CR (complete response) rate of nearly 40 percent, which translates into a PFS (progression-free survival) duration of roughly 22 months. While these results are certainly promising, Dr. Raje was eager to answer the question “Can we do better?”.

A year after ide-cel, the FDA gave approval to cilta-cel in March of 2022. This CAR-T therapy product promises a significantly longer duration of response. In fact, data from initial cilta-cel studies is remarkable, suggesting an exceedingly high response rate of 97 percent among the 5-time refractory patient population. Cilta-cel also boasts a CR rate of 55 percent and leaves roughly 72 percent of patients progression-free a full two years after treatment.

Viewing ide-cel and cilta-cel as just the first two forays into the world of multiple myeloma CAR-T therapy, Dr. Raje welcomes their FDA approval with tremendous excitement. She hailed these CAR-T therapy products as world-changers or people who suffer from relapsed and/or refractory multiple myeloma in particular.

Although she is extremely thankful that the approvals of ide-cel and cilta-cel will give multiple myeloma patients access to a much broader treatment landscape, Dr. Raje regrets the fact that these CAR-T therapy products aren’t “off-the-shelf” and as readily available as she would like them to be. The ultimate goal, according to Dr. Raje, is to provide customized, designer-specific CAR-T therapy for each person with multiple myeloma.