In a nutshell
This study investigated the safety and effectiveness of a new chimeric antigen receptor (CAR) T cell therapy for relapsed or refractory (does not respond to treatment) multiple myeloma (MM). This study found that this cell therapy was effective, but it had significant side effects.
Some background
MM is a type of cancer of the bone marrow that can lead to abnormal immune cells. After initial treatment, most patients with MM eventually relapse or develop refractory disease. New therapies are needed for these patients. A new CAR-T cell therapy called LCAR-B38M is under investigation for these patients.
CAR-T cell therapy involves removing T-cells (immune cells that help fight infections) from the patient's blood. Then, the T-cells are engineered to make a special protein called CAR. This protein helps the T-cells attack cancer cells. These CAR T-cells are then reintroduced into the patient. The safety and effectiveness of LCAR-B38M therapy for patients with MM are under investigation.
Methods & findings
This study included 57 patients with relapsed or refractory MM. Patients first received cyclophosphamide (Cytoxan). Five days later, patients received LCAR-B38M. All patients received a total of 3 rounds of treatment. Patients were followed-up for an average of 8 months.
Overall, 88% of patients responded to treatment. 68% of patients had a complete disappearance of all signs of cancer.
By the end of the study, 20% of patients had tumor growth or spread (progression). For all patients, the average progression-free survival (time from treatment until disease progression) was 15 months.
Many patients reported side effects. The most common side effect was fever (91%). 90% of patients experienced a severe immune response called cytokine release syndrome (CRS). This syndrome is often accompanied by fever, nausea, and muscle and joint pain.
65% of patients reported severe side effects. The most common of these included low white blood cell count (30%) and low platelet count (cells involved in blood clotting; 23%).
The bottom line
This study found that LCAR-B38M was effective for patients with relapsed or refractory MM. However, most patients experienced severe side effects as a result of treatment.
The fine print
This study did not have a control group. All patients received the same treatment. Therefore, the effects of LCAR-B38M compared to other therapies have not been evaluated. Also, this study involved Chinese patients, so these results may not be applicable to all patients.
Published By :
Journal of hematology & oncology
Date :
Dec 20, 2018