CAR T-Cell Therapy Produces ‘Remarkable’ Results in Multiple Myeloma, but It’s Still Not a Cure

Researchers Have Questions About the Ideal Timing, Target, and Setting

• A few investigational CAR T-cell therapies for multiple myeloma have shown positive response rates and progression-free survival
• However, this treatment is still not curative
• Researchers have many remaining questions — including when in the disease course is the best time to administer this therapy

Chimeric antigen receptor (CAR) T-cell therapy is showing great promise in multiple myeloma, as evidenced by a trio of investigational therapies presented at this year’s ASCO conference. “CAR-Ts are one of the most exciting technologies that we have in trials, and hopefully in approvals soon,” says Mount Sinai hematologist-oncologist, Dr. Joshua Richter during SurvivorNet Connect’s virtual conference on multiple myeloma developments.

Janssen’s investigational CAR T-cell therapy, JNJ-4528, had a 100% overall response rate, with 86% of patients still alive and progression-free at 9 months. “What was really remarkable, I think, was the depth of response,” says UCSF Health hematologist, Dr. Nina Shah. Another promising candidate, BB2121, had an 83% response rate, and added nearly one year of progression-free survival (PFS) in heavily pre-treated patients. “Never before this had we seen such remarkable data in people with six prior lines of therapy,” she says.

Given the patient population receiving this therapy, eight to 12 months of PFS is “extremely good,” says Mayo Clinic medical oncologist, Dr. Vincent Rajkumar. However, Dr. Shah adds, “it’s still not a cure and we have a lot of work to do.”

Researchers have several open questions about the treatment, including the best target, the best time to administer it, and the best setting. “I think we have more questions than answers at the moment,” Dr. Richter says.

Cost is currently one barrier standing in the way of CAR-T as a curative therapy. New platforms like Prodigy are enabling research centers to make their own CAR T-cells more efficiently and inexpensively. Once this therapy becomes more cost-effective, researchers will have the option of trying it earlier in the course of the disease.

“If we’ve got an immunological platform that’s transformative, and if we bring it earlier, I’m really hopeful that we’ll see incredibly enhanced outcomes that look toward a functional cure for a large number of our patients,” says Dana-Farber Cancer Institute medical oncologist, Dr. Paul Richardson.