Celgene Corp. and Bluebird Bio Inc.'s chimeric antigen receptor T-cell, or CAR-T cell, therapy showed disease reduction in certain late-stage blood cancer patients and extended their life while keeping the disease at bay.
CAR-T cell therapies work by manipulating a patient's own disease-fighting T cells and then infusing them back into the body to fight blood cancers.
The phase 1 trial, named CRB-401, evaluated the companies' bb2121 investigational anti-B-cell maturation antigen CAR-T cell therapy in 43 patients with late-stage multiple myeloma whose disease had returned or did not respond to treatment. Patients in the study were heavily pre-treated with prior myeloma treatment regimens.
Multiple myeloma is an incurable blood cancer that forms in plasma cells — a type of white blood cell — that are responsible for producing antibodies. It is characterized by a recurring pattern of remission and relapse.
Celgene and Bluebird's CAR-T cell therapy is designed to target a protein called B-cell maturation antigen, or BCMA, that is present on the surface of multiple myeloma cells. When these modified CAR-T cells are administered back into patients, they attach to BCMA on the surface of the cancer cells and kill the cells in the process.
In the trial, 22 patients received a dose of more than 150 million CAR-T cells. Out of these patients, 95.5% saw a reduction in their disease and 50% saw the disappearance of all signs of cancer — known as a complete remission. Patients in the trial who received 150 million CAR-T cells or more lived for a median of 11.8 months without progression in their disease.
The therapy reduced the disease in 57.1% of the 14 patients who received a dose of 150 million CAR-T cells, while 42.9% of these patients achieved complete remission. Out of the 3 patients who received a dose of 50 million CAR-T cells, 33.3% saw a reduction in their disease and lived for a median of 2.7 months without the disease progressing.
Bb2121 has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration and priority medicines eligibility from the European Medicines Agency.
Bluebird and Celgene have entered into an agreement to co-develop and co-promote bb2121 for the potential treatment of patients with relapsed or refractory multiple myeloma in the U.S. Celgene has also sponsored a phase 2 study, named KarMMa, which is recruiting in North America and Europe, to evaluate bb2121 further in such patients.
The 2018 American Society of Clinical Oncology meeting is expected to bring together more than 32,000 professionals from all over the world, with more than 2,500 study abstracts to be presented on-site and an additional 3,350 abstracts to be published online.
