A recent clinical trial has demonstrated that a single infusion of genetically engineered immune cells can produce durable remissions in patients with multiple myeloma, a form of blood cancer that has traditionally been difficult to cure. The findings, published in June in the Journal of Clinical Oncology, showed that five years after receiving the therapy, 82 out of 97 participants remained alive and cancer-free.
The participants in the trial, led by Professor Sundar Jagannath of Mount Sinai Hospital in New York, had previously exhausted standard treatment options and faced a prognosis of one to two years to live. The new therapy involves extracting a patient’s own T-cells—immune cells responsible for fighting infections—and modifying them in the laboratory to recognize and destroy myeloma cells. This treatment, known as CAR T-cell therapy, is delivered as a single infusion.
Multiple myeloma originates in the bone marrow when plasma cells become malignant and multiply uncontrollably. The disease can cause bone damage, kidney failure, and impairment of the immune system, leading to symptoms such as fatigue, back pain, and recurrent infections. Diagnosis is often delayed due to vague symptoms, with approximately one-third of patients first identified after complications such as fractures or kidney issues.
Current standard treatments include chemotherapy, radiotherapy, targeted drugs, bisphosphonates aimed at protecting bones, and stem cell transplants. Although stem cell transplants help many patients temporarily, a significant proportion experience relapse within three years. Other therapies such as immune-boosting drugs, proteasome inhibitors, and antibodies have improved survival rates but generally require ongoing treatment and do not achieve long-term remission.
Experts have described the CAR T-cell therapy results as unprecedented in the management of multiple myeloma. Professor Graham Jackson, a consultant haematologist at Newcastle Hospitals NHS Trust, highlighted the potential for this therapy to offer true cures, a term seldom associated with this cancer until now.
However, the use of CAR T-cell therapy is not without challenges. Prior to infusion, patients undergo chemotherapy to deplete existing immune cells, increasing their risk of infections. Almost all patients develop cytokine release syndrome, an inflammatory response that can range from mild to severe. Severe cases may involve dangerously low blood pressure and organ dysfunction, requiring intensive care, but fatalities are rare due to effective treatments including tocilizumab and steroids.
Cost is another consideration. The therapy, which typically requires only one infusion, costs approximately £350,000 and is not yet available through the National Health Service for multiple myeloma patients. Some private clinics in the UK offer the treatment, and ongoing trials are evaluating the efficacy of administering CAR T-cell therapy earlier in the disease course.
The trial’s positive outcomes mark a significant advance in multiple myeloma treatment, raising hopes for improved survival and, potentially, cure rates for patients who previously had limited options.