One Year After Approval: How Effective is China’s First BCMA-Targeted CAR-T Therapy in Treating Multiple Myeloma?

### One Year After Approval: How Effective is China’s First BCMA-Targeted CAR-T Therapy in Treating Multiple Myeloma?

Multiple Myeloma

“In the past 20 years, the treatment of multiple myeloma has advanced rapidly, thanks in large part to the swift progress in drug development. Over the past year, China’s first independently developed and the world’s first fully human BCMA-targeted chimeric antigen receptor T-cell (CAR-T) therapy was approved. In our real-world clinical applications, we have treated over 20 patients with an overall effectiveness rate of nearly 100%.”

On June 30, 2024, exactly one year after the approval of China’s first BCMA-targeted CAR-T therapy (Iquilonsen Injection), and also the world’s first fully human BCMA-targeted CAR-T therapy, how effective has the treatment been for patients? Professor Qiu Lugui, Director of the Lymphoma Treatment Center at the Chinese Academy of Medical Sciences Hematology Hospital, was interviewed by the People’s Daily Health Client.

“Multiple myeloma has a slow onset and early stages often show no obvious symptoms, making it easy to misdiagnose. To date, once diagnosed, the vast majority of patients experience one or more relapses, entering a refractory state, which is an incurable disease,” Professor Qiu Lugui told the People’s Daily Health Client.

Professor Qiu explained that current drugs for treating multiple myeloma fall into three categories: immunomodulators, proteasome inhibitors, and CD38 monoclonal antibodies. The indications for these drugs have gradually moved from refractory cases to frontline treatments, transforming multiple myeloma from a deadly disease with a median survival of around three years to a relatively controllable malignant hematological tumor with a median survival of 10 years or more after systematic multi-drug therapy.

CAR-T cell therapy is a cutting-edge technology for treating malignant hematological tumors. China’s independently developed fully human BCMA-targeted CAR-T drug (Iquilonsen Injection) is designed for multiple myeloma patients who have relapsed or whose disease remains uncontrolled despite traditional treatments including proteasome inhibitors and immunomodulators.

One particularly memorable case for Professor Qiu was a 70-year-old patient. “At that time, the patient was extremely weak and had already undergone all available treatments, including two types of immunomodulators, two types of proteasome inhibitors, CD38 monoclonal antibodies, and intensive chemotherapy, with no other effective options left,” recalled Professor Qiu. “However, the patient had a strong desire to live. Seeing his eager eyes, we couldn’t remain indifferent.”

“After confirming with the patient, we decided to proceed with the fully human BCMA-targeted CAR-T Iquilonsen therapy. One month after the treatment, the first evaluation showed complete remission. To date, the patient remains in complete remission,” said Professor Qiu.

“In real-world applications, we have treated over 20 patients with an overall effectiveness rate approaching 100%. However, due to the high cost of the drug and the fact that it is not covered by medical insurance, making the drug accessible remains a challenge,” Professor Qiu told the People’s Daily Health Client. “Currently, there are two methods to address this issue: one is to meet the needs of economically disadvantaged patients through commercial insurance; the other is to meet the needs of patients who meet the criteria for inclusion in CAR-T clinical research.”

“Additionally, in the year since the approval of China’s first CAR-T therapy for treating multiple myeloma, not only domestic patients but also patients from Europe, Asia-Pacific, Africa, and other regions have come to China for CAR-T treatment. Overall treatment driven by dynamic prognostic stratification will be the future path to cure for multiple myeloma patients both in China and globally,” said Professor Qiu.