Rare Disease Report

Blastic Plasmacytoid Dendritic Neoplasm Study Begins for CAR T-Cell Candidate

AUGUST 18, 2017
Mathew Shanley
Cellectis announced today that the Phase 1 clinical study testing their CAR T-cell therapy, UCART123, to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) at the MD Anderson Cancer Center has begun, and the first patient was given the drug.

The interleukin-3 receptor, or CD123 antigen, is often expressed across acute myeloid leukemia (AML) and BPDCN cells. Before UCART123, the first allogeneic, “off-the-shelf” gene edited CAR T-cell product candidate to target CD123, no similar treatments had been investigated.

BPDCN is a rare and aggressive hematological malignancy in the bone marrow, that often also affects the skin and lymph nodes. Previously referred to as “natural killer cell” lymphoma or leukemia, it is categorized as a subtype of AML. Men are more likely than women to receive a BPDCN diagnosis, and confirmation of the diagnosis typically comes between age 60 and 70.

“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 statement. “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.”

The initial administering of UCART123 is part of a Phase I clinical study being conducted by 3 professors at the MD Anderson Cancer Center, and will investigate the safety and efficacy of the therapy in relapsed and refractory BPDCN patients.

There is currently no standardized therapeutic approach to BPDCN, and because of its rarity, no ideal treatments have been defined. Some patients, however, do respond to specific chemotherapy regiments, but relapse is common with current treatments, and overall survival is 8-12 months.

In June, UCART123 was administered in Cellectis’ AML Phase I clinical trial at Weill Cornell Medicine, New York-Presbyterian Hospital.
“We are excited to be enrolling our first patient with UCART123 and are hopeful that this novel immunotherapy modality will prove to be a significant and effective weapon against AML,” said Principal Investigator Dr. Gail J. Roboz, Professor of Medicine at Weill Cornell Medicine and Director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

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