WASHINGTON: A groundbreaking treatment that involves genetically modifying the body’s own immune cells has shown remarkable success in reducing the risk of disease progression by 74 percent in individuals with a rare form of blood cancer, according to study results presented on Monday.
The treatment, known as ciltacabtagene autoleucel or Carvykti, was tested in a clinical trial involving 419 patients diagnosed with multiple myeloma. These patients had previously shown no response to the standard frontline drug lenalidomide, a chemotherapy medicine.
“While lenalidomide has been an important treatment for myeloma patients, there is a growing number of individuals whose disease becomes resistant to it,” explained oncologist Oreofe Odejide at the American Society of Clinical Oncology’s annual meeting, where the results were unveiled.
Ciltacabtagene autoleucel was found to deliver remarkably effective outcomes when compared to current treatment options, offering a safe alternative that can be used earlier in the treatment phase, added Odejide, who was not involved in the research.
Multiple myeloma affects plasma cells, a type of white blood cells, and can lead to various complications affecting the bones, kidneys, and immune system. The disease affects approximately seven individuals per 100,000 annually, with 100,000 people impacted in the United States alone. While there is currently no cure, disease progression can be halted for extended periods.
Treatment of Blood Cancer Patients
The risk of multiple myeloma increases with age, with men more susceptible than women, and Black individuals facing a higher risk compared to other races. Immediate treatment is not always necessary, and in cases of slow growth, the disease can be monitored.
During the clinical trial, half of the participants were randomly assigned to receive ciltacabtagene autoleucel, while the other half received the current standard-of-care treatment, consisting of a combination of chemotherapy, steroids, and other drugs.
“After a median follow-up of 16 months, the researchers discovered that ciltacabtagene autoleucel reduced the risk of disease progression by 74 percent compared to standard-of-care treatments,” stated a press release.
Ciltacabtagene autoleucel belongs to a category of treatments known as chimeric antigen receptor (CAR) T-cell therapy, a relatively new approach to combating cancer.
CAR T-cell therapy involves extracting the patient’s own T cells responsible for fighting disease and genetically modifying them in a laboratory. These modified cells are then reintroduced into the patient’s body, where they possess receptors that specifically target and destroy cancer cells.
Both groups of patients in the trial experienced severe to life-threatening adverse events, including infections and low blood cell counts. Seventy-five percent of those treated with ciltacabtagene autoleucel developed Cytokine release syndrome, a condition in which the immune system goes into overdrive, potentially affecting multiple organs and leading to fatalities.
Around five percent of patients treated with ciltacabtagene autoleucel experienced immune effector cell-associated neurotoxicity syndrome (ICANS), which affects the nervous system.
The researchers will continue to monitor the study participants to evaluate long-term effects and assess impacts on their quality of life. The clinical trial was funded by Janssen Research & Development and Legend Biotech USA.
