Rare Disease Report

Allogeneic CAR-T Therapy to Begin Clinical Trials for 2 Rare Blood Cancers

FEBRUARY 06, 2017
James Radke
The FDA has given the green light for Cellectis to begin its phase 1 study with UCART123, a allogeneic CAR-T cell product to treat 2 rare cancers – acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). 
 
Cellectis plans to begin Phase 1 trials in the first half of 2017.

UCART123 is a gene-edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in AML, tumoral cells in BPDCN.
 
The clinical research for AML will be led, at Weill Cornell, by principal investigator Gail J. Roboz, MD, Director of the Clinical and Translational Leukemia Programs and Professor of Medicine.
 
The clinical research for BPDCN will be led, at the MD Anderson Cancer Center, by  Naveen Pemmaraju, MD, Assistant Professor, and Professor Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine.

About Acute Myeloid Leukemia (AML)

AML is a type of cancer in which the bone marrow makes abnormal myeloblasts. Symptoms include fatigue, recurrent infections and easy bruising. Acute myeloid leukemia is generally a disease of older people and is uncommon before the age of 45. The average age of a patient with AML is about 67 years. The number of new cases of acute myeloid leukemia was 4.0 per 100,000 men and women per year.

About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

BPDCN is a very rare and aggressive hematological malignancy that is derived from plasmacytoid dendritic cell precursors. Similar to leukemia, BPDCN is a disease of bone marrow and blood cells but unlike AML, it can also affect skin and lymph nodes.
 


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