New Immunotherapy Treatments for Myeloma Show Positive Results

(Posted on Tuesday, October 7, 2025)

Long-term remission in multiple myeloma—a blood cancer once considered incurable—is finally becoming a reality. A single infusion of immunotherapy has kept patients healthy for more than five years. This is an unprecedented milestone for this tough-to-treat blood cancer. For decades, people with multiple myeloma lived through cycles of treatment, remission, and relapse. Most ran out of options within just a few years. New results suggest that a single round of this cell-based treatment could significantly improve the outlook for many patients.

A recent study in the Journal of Clinical Oncology found that patients receiving cilta-cel not only achieved deep remissions but also survived years longer than expected. These outcomes are especially striking for those with advanced, treatment-resistant disease, where survival has historically been measured in months rather than years.

Why This Matters

Multiple myeloma affects tens of thousands of people worldwide every year. While new drugs over the past two decades have improved life expectancy, the disease has almost always come back. For patients whose cancer progresses despite the mainstay drugs—proteasome inhibitors, immunomodulators, and CD38 antibodies—the outlook has been grim.

Cilta-cel belongs to a new class of cell-based cancer treatments known as CAR T-cell therapies, which are further described in the book CAR T. This therapy uses immune cells to combat cancer. Specifically, this type of therapy reprograms a patient’s own immune cells to recognize and attack myeloma cells. The five-year results reported here provide the clearest evidence yet that CAR T therapy can deliver durable control of this difficult disease.

The Five-Year Outcomes

About half of the participants were still alive five years after receiving cilta-cel, and roughly one in three remained in remission. On average, patients lived more than five years and enjoyed nearly four years without disease progression. By comparison, patients who had exhausted traditional options in earlier studies typically lived less than a year. For some, whose cancer became undetectable after the infusion, remissions have lasted longer than anything seen before.

Importantly, these benefits were widespread. Even older adults, patients with high-risk disease, and those heavily pretreated in the past showed meaningful improvements. This broad efficacy profile indicates that cilta-cel may be applicable to a wide range of patients with relapsed or refractory myeloma. These results suggest cilta-cel may offer lasting disease control and, for some, long remissions.

Safety and Side Effects

As with most powerful treatments, there are risks. For example, the response to cilta-cel varies. Some patients relapse after initial remissions, demonstrating the disease’s adaptability. Ongoing research specifically aims to identify biological markers that can predict which patients benefit most from cilta-cel and to determine the mechanisms behind treatment resistance. Additional studies plan to test combination approaches to improve the durability of response, and to evaluate when administering cilta-cel provides the best patient outcomes.

The most common side effects are cytokine release syndrome—a strong inflammatory reaction—and neurological effects, which can be serious but are usually treatable with medical care. Nearly all side effects occur in the first days after infusion and tend to resolve with appropriate treatment. Over the long run, no new safety issues have emerged.

The trial represents an initial step in evaluating the potential of cilta-cel. Ongoing studies aim to more precisely determine its safety and efficacy in earlier disease stages, test its use in patients who remain responsive to standard therapies, and assess its effectiveness in combination with other targeted agents. These studies also aim to identify which patient groups are likely to experience the greatest benefit. Preliminary data suggest that patients with less heavily pretreated disease may achieve better outcomes. This likely reflects improved immune function.

Looking Ahead

The implications of cilta-cel’s success extend beyond multiple myeloma. The ability to reprogram a patient’s immune cells to achieve durable remissions supports the broader promise of cellular therapies in oncology. Similar approaches have already demonstrated efficacy in other hematologic malignancies, including lymphoma and leukemia, and efforts are underway to adapt these strategies for use in solid tumors such as breast and lung cancer. Significant challenges persist, including the complexity of manufacturing, high treatment costs, and variability in patient response. Nevertheless, the accumulating evidence suggests that immune-based therapies represent a durable and transformative advance in cancer care.

The durability of five-year outcomes with cilta-cel is a source of optimism for both clinicians and patients. In a disease historically characterized by frequent relapse, sustained remission following a single therapy marks a significant milestone. While cilta-cel does not yet represent a universal cure, it highlights the progress made in myeloma treatment and the potential for further advances in the field. The key takeaway is clear: durable remissions and significantly improved survival are now within reach for patients with advanced disease, and continued research will be pivotal in turning these breakthroughs into standard treatment options.