Early Anito-cel Data Shows Promise in Multiple Myeloma
- Early clinical trial results show the drug, anito‑cel, may offer powerful multiple myeloma control with a gentler safety profile than other CAR T-cell therapy.
- Anito-cel uses a new CAR T design meant to reduce inflammation while still attacking cancer cells effectively.
- CAR T-cell therapy is sometimes called a “living drug” because it uses your own immune cells, which are reprogrammed to attack a unique aspect of your cancer.
- In a small phase 1 study of heavily pretreated patients, nearly everyone responded to treatment, and many had long‑lasting remissions with high rates of deep responses.
- The most common side effect was cytokine release syndrome (CRS), which can cause fever, chills, low blood pressure, or low oxygen levels.
Anito-cel is a type of BCMA-directed CAR T-cell therapy. BCMA is a protein found on myeloma cells and is an important target for several newer myeloma treatments.
Read More“The recently published phase 1 NEJM study of anito-cel in 38 heavily pretreated patients showed impressive depth and durability of response with an encouraging safety profile,” Dr. Gurbakhash Kaur, an assistant professor of medicine in the Division of Hematology and Medical Oncology at the Icahn School of Medicine at Mount Sinai, tells SurvivorNet.
Dr. Kaur adds that the phase 2 iMMagine-1 data presented at ASH 2025 reinforces these findings in a larger cohort.
“If approved later this year, I believe anito-cel will become an important addition to our treatment armamentarium for patients with relapsed or refractory multiple myeloma,” Dr. Kaur said.
Anito-cel: Breaking Down the Early Data
The first clinical study of anito-cel included patients whose myeloma had returned or stopped responding after several previous treatments.
These were a particularly heavily treated group of patients, with all of them having received multiple classes of myeloma therapy.
All 38 patients had a measurable response to treatment, and 79% achieved a complete response or better. A complete response means that standard testing could no longer detect evidence of myeloma.
Overall, 92% of patients achieved at least a very good partial response.
Many patients also achieved measurable residual disease negativity, meaning that even highly sensitive testing could not detect remaining myeloma cells. Responses appeared to be durable, with a median duration of response of approximately 29 months.
Potential Side Effects
Like other CAR T-cell therapies, anito-cel can cause side effects related to activation of the immune system. The most common was cytokine release syndrome (CRS), which can cause fever, chills, low blood pressure, or low oxygen levels.
WATCH: What is Cytokine Release Syndrome (CRS) and How is It Managed?
In this study, most CRS was mild to moderate, and there were no cases of severe CRS at the recommended dose. Some patients also developed immune effector cell-associated neurotoxicity syndrome (ICANS), a temporary neurologic side effect that can cause confusion, difficulty speaking, sleepiness, or other changes in brain function.
Most cases were mild, and severe ICANS was uncommon. Notably, no delayed or non-ICANS neurologic complications were observed in this study, although the study was too small to determine whether anito-cel has a lower risk of these complications than other CAR-T therapies.
What’s Next?
One of the most interesting aspects of anito-cel is its CAR design.
Rather than simply developing another treatment against the same target, researchers have changed the way the CAR T-cells recognize BCMA. The goal is to maintain strong anti-myeloma activity while potentially reducing unnecessary immune activation.
The early clinical results suggest that this approach shows promise, but additional studies are needed to understand whether the CAR design translates into meaningful advantages for patients.
Expert Resources on CAR T-Cell Therapy
- CAR T-Cell Therapy And The Hope For A Cure In Multiple Myeloma
- CAR T-Cell Therapy Explained: What Diffuse Large B-Cell Lymphoma Patients Should Know
- CAR T-Cell Therapy Side Effects Can Be Serious, But Many Are Short-Lived
- CAR T-Cell Therapy: A Promising New Approach to Relapsed Multiple Myeloma
- Bridging Treatments as a Springboard for Successful CAR T-Cell Therapy
- Can You Afford the High Cost of CAR T-Cell Therapy?
It is important to remember that anito-cel is still an investigational therapy and is not FDA-approved. The initial study was relatively small and did not directly compare anito-cel with currently available CAR-T treatments.
In addition, the study did not include patients who had previously received BCMA-targeted bispecific antibodies, an increasingly common treatment approach for myeloma. It is therefore not yet known how well anito-cel will work in patients whose myeloma has already been treated with another BCMA-directed therapy.
Overall, the early results with anito-cel are encouraging. The therapy produced very high response rates, deep responses, and durable disease control in patients with heavily treated multiple myeloma, while the early safety findings were also favorable.
Larger clinical trials and longer follow-up will help determine where anito-cel fits among the growing number of cellular and immune-based treatments available for multiple myeloma.
Questions To Ask Your Doctor
- Is CAR T-cell therapy an option in my case?
- At what point should I consider treatment with CAR T-cell therapy?
- What risks and side effects should I be aware of?
- Are there clinical trials testing anito-cel that I should consider?
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