Myeloma Cure?

While this was a small Phase I study, the results were considered so impressive that Celgene plans to begin enrolling patients this month for a larger, potentially pivotal trial that could position bb2121 to become the third approved CAR-T.
While this was a small Phase I study, the results were considered so impressive that Celgene plans to begin enrolling patients this month for a larger, potentially pivotal trial that could position bb2121 to become the third approved CAR-T.

(Reuters) – More than half of patients with advanced multiple myeloma who had run out of therapeutic options remained in complete remission after receiving bluebird bio Inc’s experimental gene-modifying immunotherapy in a small, early stage study, according to updated data released on Sunday.

Of 18 patients who received a therapeutic dose of bb2121, all but one responded to the treatment, a 94 percent response rate, while 56 percent remained in remission with a median follow-up of 40 weeks after treatment.

Researchers, who reported the data at the American Society of Hematology meeting in Atlanta, said the initial response to the treatment was very quick and that many of the patients continued to improve over time.

Patients in the Phase I dose-escalation study had received seven prior treatment regimens, including regimens with the newest multiple myeloma drugs, such as Johnson & Johnson’s Darzalex, and had undergone at least one stem cell transplant before receiving bb2121, which is being co-developed with Celgene Corp.

“Some of these patients were going to hospice until they got this,” said Dr. Jesus Berdeja, the study’s lead investigator.

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