Is Using CAR T-cell Therapy as a Frontline Treatment Option Effective?  

This week's Ask Dr. Durie comes from a patient who has heard that CAR T-cell therapy—the very exciting new immune therapy that has been evaluated in patients with relapsed/refractory myeloma and two CAR T products have been approved by the FDA (Food and Drug Administration). So, the question is: have CAR T cells been used earlier in the disease? 

The answer to that question is yes. At the recent ASH meeting in December 2022—held in New Orleans—there was a presentation by Dr. Juan Du from Shanghai, China. She presented the results using a new CAR-T product in patients with newly diagnosed myeloma—so really a unique new study. She also used a new product with dual targeting, BCMA and CD19, so a double targeting of the myeloma cell and using a new manufacturing technique which makes the CAR T cells available within 48 hours. So really it is a remarkable new development.  

And so, in this treatment protocol, newly diagnosed patients—and she started with patients who have high-risk myeloma—so these are patients with active myeloma, but with the high-risk features such as abnormal chromosomes, plasma cell, leukemia, things like that. So, the CAR-T treatment was given to those patients after initial disease control using Velcade®, Revlimid®, and dex for two cycles or initial two cycles of therapy, followed by the CAR T-cell infusion. 

And so, the initial results were remarkably good. All the patients have had an overall response rate and not only that, but all the patients have also achieved an MRD-negative status. All the patients have had deep, deep responses at this point. 

So, a remarkable, strong benefit using these CAR T cells as an early treatment in patients with newly diagnosed myeloma. 

The CAR T-cell therapy has been well tolerated with only very low cytokine release syndrome, what we call CRS. So that is very, very promising.  

And so, the BOTTOM LINE is that this first study, looking at the use of CAR T cells earlier in the disease course, in this case, for newly diagnosed patients is extremely promising. And so, I think everyone is quite excited to see the results with this rapid production of the cells using a new lab technique and with the new dual targeting CAR T cells, and it looks like this protocol can lead to many more evaluating what looks like can be a very successful approach to early disease control.