First Patient with BPDCN Receives UCART123 Dose in Phase 1 Clinical Trial

First Patient with BPDCN Receives UCART123 Dose in Phase 1 Clinical Trial

The first patient with Blastic Plasmacytoid Dendritic Cell Neoplasms (BPDCN) has received a dose of Cellectis’ UCART123 in a Phase 1 clinical trial. UCART123 is a gene edited T-cell drug that targets CD123, which a cell-surface antigen that is present on many types of tumor cells including BPDCN.

The trial will evaluate the safety and efficacy of UCART123 in BPDCN patients in relapsed, refractory, and front-line settings. BPDCN is a rare bone marrow disease which arises from a type of cell called a plasmacytoid dendritic cell precursor. Until recently, it was not recognized as a separate disease and was grouped in with acute leukemia. Due to the rare incidence of BPDCN, there are no standardized approaches to treatment. While current treatments are effective in the short-term, patients almost always relapse with drug-resistant disease.

UCART123 was developed based on the gene-edited Chimeric Antigen Receptors (CAR) T-cells.  CARs are specific receptors that can be expressed on the cell surface of T-cells by a gene editing process. These CARs can then function to target T-cells to a specific antigen that is expressed on cancer cells, which can direct T-cells to its intended target.

Cellectis received approval by the U.S. Food and Drug Administration (FDA) to conduct Phase 1 clinical trials in patients with acute myeloid leukemia (AML) and BPDCN using UCART123 in February 2017. The UCART123 clinical trial (NCT03203369) for BPDCN is being held at the MD Anderson Cancer Center and led by Naveen Pemmaraju, MD; Marina Konopleva MD, PhD; and Hagop Kantarjian, MD.

“We are eager to progress through clinical trials with UCART123, Cellectis’ wholly controlled gene-edited product candidate, next with the treatment of BPDCN, rare but aggressive entity,” said Dr. Loan Hoang-Sayag, Cellectis’ chief medical officer in a press release. “With this innovative treatment, the hope is that our ‘off-the-shelf’ approach will transform the way we think about cancer care and serve as the next step in curing this disease through the power of gene editing.”