Clinical trial demonstrates that CART therapy improves survival of early relapsed multiple myeloma patients

The results have been published in the New England Journal of Medicine, one of the highest impact journals in medicine. The Clínica Universidad de Navarra is the hospital that has contributed the most patients in a study carried out in 13 countries.

Professionals of the Clinical Trials Unit of the Clínica Universidad de Navarra, the private center that promotes the most clinical trials in Spain.

February 10, 2023

CART lymphocyte therapy has been shown for the first time in a phase III clinical trial to significantly prolong survival in patients with multiple myeloma who had relapsed after having received between 2 and 4 previous lines of treatment. This is the result of a clinical trial in which the Clínica Universidad de Navarra participated, carried out in hospitals around the world and published in the New England Journal of Medicine, one of the most important journals in Medicine.

"The treatment of this disease, the third most frequent hematological cancer, has improved significantly in recent years thanks to the incorporation of new therapies", indicates Dr. Paula Rodríguez Otero, specialist in the Hematological Cancer Area of the Cancer Center Clínica Universidad de Navarra and first signatory of the trial, who explains that "however, patients whose disease relapses after two lines of previous treatment and who have already received the main drugs have an unfavorable prognosis and need new pharmacological strategies".  

"Although CART therapy with the drug ide-cel is approved in patients with advanced stage myeloma, this study is the first to demonstrate its efficacy in a phase III trial, comparing this therapy with standard treatment in patients in early relapse who have already been exposed to the main anti-myeloma treatments," details Dr. Rodriguez Otero. 

The project, promoted by Bristol Myers Squibb, has involved the participation of almost 20 national and international centers from 13 different countries, with the Clínica Universidad de Navarra recruiting the most patients. "We have randomly assigned patients to CART therapy or standard treatment, taking into account that patients assigned to the control arm could receive ide-cel therapy at the time of progression," explains the specialist from the Hematological Cancer Area, concluding that "standard treatment is insufficient, since the median progression-free survival observed with CART therapy exceeds thirteen months, while that observed with conventional treatment is less than four months".

These results support the role of CART therapy in patients with relapsed myeloma who have received at least two prior unsuccessful therapies. At that stage of the disease, the therapeutic options are limited and suboptimal, therefore, according to Dr. Rodriguez Otero, "in light of the results of this work, CART therapy could considerably improve the situation of these patients". 

Treating myeloma with the most advanced therapies

Multiple myeloma is the third most common hematological tumor. It is a cancer of the plasma cells, located in the bone marrow, whose role is crucial in the immune system. "Despite significant advances in treatment, most patients relapse and end up being resistant to the most important drugs available to date," says Dr. Rodríguez Otero.

In this situation, new treatments with different mechanisms of action are needed, such as CART cell therapy. "This therapy consists of the genetic modification in the laboratory of T lymphocytes (one of the main cells of the immune system), in order to reinforce their capacity to recognize and destroy tumor cells," explains Dr. Felipe Prósper, director of the Cell Therapy Department at the Clínica Universidad de Navarra, "it is a therapy that is proving very beneficial for treating hematological tumors in our patients".