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Orphan Drug Designation has been given by the FDA for chimeric antigen receptor engineered T cells directed against the target protein CD4 (CD4CAR) for the treatment of peripheral T-cell lymphoma (PTCL).

FDA. FDA... - Click to enlarge in new windowFDA. FDA

Although there are clinical development programs ongoing with CAR T cells for CD19+ cell hematological malignancies, CD4+ peripheral T-cell lymphomas (PTCLs) have not been targeted by a CAR therapy in a human trial. PTCLs account for 10-15 percent of all non-Hodgkin lymphomas (NHLs) and are more difficult to treat in comparison to B-cell NHLs.


Furthermore, and with few exceptions, T-cell NHLs have poorer outcomes, lower response rates, shorter times to progression, and shorter median survival in comparison to B-cell NHLs. As a result, the standard of care for PTCLs is not well-established, and the only potential curative regimen is bone marrow transplant (BMT).


However, not only is BMT poorly tolerated, but it is not an option for a significant subset of patients with resistant disease. This leaves many patients with no curative options.


CD4CAR is in development for CD4+ T-cell malignancies. The novel CD4-specific chimeric antigen receptor engineered T cells are properly-matched allogeneic human T cells engineered to express an anti-CD4 scFV antibody domain. An initial phase I clinical study is being planned through collaboration between iCell Gene Therapeutics, the NIH, Indiana Clinical and Translational Sciences Institute, Stony Brook University Hospital, and the Division of Blood and Bone Marrow Transplantation and the Clinical Trial Research Unit at James Graham Brown Cancer Center at University of Louisville.