NIH Cancer Therapy Trial May Raise Hopes for Celgene, Bluebird

  • Researchers use engineered immune cells to target myeloma
  • Companies pursue similar approach to fighting blood cancer

A U.S. National Institutes of Health trial of a genetically engineered T-cell therapy resulted in some remissions in patients with advanced blood cancer, a promising sign for the approach that also is being pursued by drugmakers Celgene Corp. and Bluebird Bio Inc.

Researchers treated 11 advanced multiple myeloma patients with a therapy known as CAR-T, in which a patient’s immune cells are removed, engineered to recognize a protein expressed by cancerous cells, and then returned to the patient. The tailored T-cells in this trial were modified to recognize a protein called BCMA. While other versions of CAR-T have been tried in patients, this is the first time a BCMA-targeting CAR-T was used in humans.

Biotechnology firms Celgene and Bluebird have an agreement to jointly develop CAR-T treatments targeting BCMA and will begin their first trial in 2016. Bluebird’s product is "similar to but not identical" to the NIH’s treatment, according to lead researcher James Kochenderfer, who said in an e-mail that he has a research agreement to develop CAR products with Bluebird.  

The patients in the NIH trial received different dosing levels of the treatment. Of the two who got the highest dose, one experienced complete remission, and signs of the cancer were wiped out in the bone marrow plasma of the second patient, according to a summary of the study published Monday. The full results will be presented at the annual conference of the American Society of Hematology in December.

Variable Results

Of the remaining patients who received lower dosage levels, one experienced partial remission, seven had “stable disease,” meaning their cancer was still detectable but had stopped growing, and one had a “transient” partial remission lasting two weeks, according to the abstract.

“Multiple myeloma is a malignancy that is almost always incurable, so new treatments are clearly needed," Kochenderfer said by e-mail. “One of the most interesting findings of this study was elimination of multiple myeloma from a patient with multiple myeloma that was extremely resistant to chemotherapy."

Side effects also rose when patients got a bigger dose of the therapy. The three patients with the best responses all showed signs of cytokine release syndrome, an immune system response that can be fatal. Kochenderfer said the toxicity was "substantial, but reversible."

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