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

Disease info:

Multiple Myeloma  is a form of cancer that occurs due to abnormal and uncontrolled growth of plasma cells in the bone marrow.
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 to disrupt expression of endogenous genes
Gene:
TCRα, TCRβ and PD-1 genes (NYCE T Cells)
Vector:
Transduced with a lentiviral vector to express NY-ESO-1 and electroporated
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

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).