A novel immune therapy using patients’ own genetically engineered immune cells has shown promising results in treating multiple myeloma, marking a potential breakthrough in a disease long considered incurable. The therapy, known as CAR T-cell treatment, was administered in a recent clinical trial involving 97 patients who had exhausted other treatment options. Prior to the study, most participants were given a prognosis of just one to two years to live.
The five-year follow-up data, published in the Journal of Clinical Oncology, revealed that 82 of the patients remained alive and healthy, many with no detectable signs of cancer and without needing further treatment. According to Professor Sundar Jagannath, the lead investigator from Mount Sinai Hospital in New York, the findings meet the criteria for declaring these patients cured.
Multiple myeloma is a blood cancer affecting plasma cells within the bone marrow. These cancerous cells multiply uncontrollably, creating lesions in multiple areas of bone marrow, which leads to symptoms such as fatigue, bone pain, repeated infections, fractures, and kidney failure. The disease often goes undiagnosed until complications arise, with about one-third of patients first identified after emergency hospital visits due to fractures or kidney issues.
Traditional treatments include chemotherapy, radiotherapy, targeted drugs, bisphosphonates to protect bones, and stem cell transplants. Although stem cell transplants can extend survival, many patients experience relapse within three years, and treatments typically require ongoing administration with significant side effects such as fatigue, infections, and nerve damage.
CAR T-cell therapy offers a new approach by collecting a patient’s own T-cells, reprogramming them in the lab to recognize myeloma cells, and infusing the engineered cells back into the patient in a single dose. These CAR T-cells then actively seek out and destroy cancer cells throughout the body. While CAR T-cell therapy has been used previously for other blood cancers, this trial represents a significant milestone for multiple myeloma treatment.
However, the treatment is not without risks. Patients undergo chemotherapy prior to infusion to reduce existing immune cells, making them temporarily vulnerable to infections. Nearly all patients experience cytokine release syndrome, an inflammatory response that can cause fever and confusion. Most cases are mild, but approximately 4 percent become severe, involving dangerously low blood pressure and organ stress requiring intensive care. These complications are typically manageable with prompt intervention using medications such as tocilizumab and steroids.
Cost is another challenge, with a single CAR T-cell infusion priced around £350,000. The therapy is not currently available through the National Health Service (NHS) for multiple myeloma, though some private hospitals offer it and clinical trials are ongoing in the UK to evaluate its use earlier in the disease course.
Experts have described the results as unprecedented. Professor Graham Jackson, a haematologist at Newcastle Hospitals NHS Trust not involved in the trial, called it a “game-changer” and said it is the most effective treatment seen for myeloma to date. The therapy’s potential to provide a treatment-free remission marks a significant advancement for patients with a disease that historically has been difficult to cure.