Disease: Multiple Myeloma; Melanoma; Synovial Sarcoma; Myxoid/Round Cell Liposarcoma, MRCL, (NCT03399448)

Disease info:

Multiple myeloma is a cancer that develops in the bone marrow, the spongy tissue found in the center of most bones. Multiple myeloma is characterized by abnormalities in plasma cells, a type of white blood cell. These abnormal cells multiply out of control, increasing from about one percent of cells in the bone marrow to the majority of bone marrow cells. The abnormal cells form tumors within the bone, causing bone pain and an increased risk of fractures.

Melanoma is a type of skin cancer that begins in pigment-producing cells called melanocytes. Synovial sarcoma is a rare and aggressive soft tissue sarcoma. 
Myxoid liposarcoma is a subtype of liposarcoma, tumors that arise in the body’s fat tissue.

Frequency:
Multiple myeloma occurs in approximately 4 per 100,000 people per year; there are currently about 100,000 affected individuals in the United States.
Official title:
Phase 1 Trial of Autologous T Cells Engineered to Express NY-ESO-1 TCR and CRISPR Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T Cells)
Who:

Principal Investigator: Edward Stadtmauer, MD

Locations:

United States, Pennsylvania

Study start:
Sep. 5, 2018
Enrollment:
3 participants
Gene editing method:
CRISPR-Cas9 guide RNA
Gene:
TCRα, TCRβ and PD-1 genes (NYCE T Cells)
Delivery method:
Transduced with a lentiviral vector to express NY-ESO-1 and electroporated to disrupt expression of endogenous genes.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Terminated

Description

This is a first-in-human trial proposed to test HLA-A*0201 restricted NY-ESO-1 redirected T cells with edited endogenous T cell receptor and PD-1.

Autologous T cells transduced with a lentiviral vector to express NY-ESO-1 and electroporated with CRISPR guide RNA to disrupt expression of endogenous TCRα, TCRβ and PD-1 (NYCE T Cells).

Last updated: Jul. 17, 2020
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