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Bellicum CAR-T therapy shows promise in phase 1/2 pancreatic cancer study

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Bellicum Pharmaceuticals Inc. said its cell therapy BPX-601 showed promise in an early-stage study in patients with advanced pancreatic cancer.

The 12-patient phase 1/2 trial enrolled subjects with advanced pancreatic cancer which has spread across the body and whose disease express a protein called prostate stem cell antigen.

BPX-601 is a chimeric antigen receptor T cell, or CAR-T, therapy, which involves taking a person's own immune cells and modifying them to fight cancer cells when infused back into the body. The effects of Bellicum's therapy are designed to be boosted by administering a drug called rimiducid, which acts as an activation switch for the treatment.

Patients treated with BPX-601 but were not given rimiducid showed limited expansion in the CAR-T cells and the cells did not persist.

Meanwhile, four patients given a single dose of rimiducid seven days after being treated with BPX-601 showed a significant expansion of CAR-T cells, with the expansion ranging from 3x to 20x. The CAR-T cells also persisted by over three weeks in three patients who were also given rimiducid.

Further, four out of six evaluable patients treated with BPX-601 and given a single dose of rimiducid achieved stable disease — no new tumors appear and known cancers neither shrink or grow — while two patients saw their tumors shrink by over 20%.

Side effects of the therapy were consistent with those commonly observed in advanced cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies.

Houston-based Bellicum plans to study BPX-601 further in patients with prostate and gastric cancers, with additional doses of rimiducid and along with a more complete conditioning regimen. CEO Rick Fair said the company plans to report updated results for the therapy in 2019.