Efficacy of CAR T-Cell Therapy in Glioblastoma

New data may support the potential of achieving clinical benefits by using CAR T cells in patients with glioblastoma.

Researchers have identified a molecular target that could allow chimeric antigen receptor (CAR) T-cell therapy to be used in treating patients with glioblastoma. Although the heterogeneous expression of tumor-associated antigens limits the efficacy for CAR-redirected T cells for the treatment of glioblastoma, chondroitin sulfate proteoglycan 4 (CSPG4), a cell surface type 1 transmembrane protein, is highly expressed in a majority of glioblastoma specimens with limited heterogeneity.

“We identified a target that is highly expressed in glioblastoma," said one of the study’s authors, Gianpietro Dotti, MD, in a statement. "While it didn't cure all of the tumors, we think it is very promising as a single agent, and also could be studied in combination with other investigational approaches."

In a research article published this week in Science Translational Medicine, the investigators report that, in a cohort of 46 glioblastoma specimens derived from patients who had received conventional treatment for glioblastoma, CSPG4 was highly expressed in 67% of samples and in lower amounts in 33% of the samples. In 1 specimen, CSPG4 was undetectable. Furthermore, CSPG4 expression was associated with nestin, a marker defining more aggressive glioblastoma, and patients with a high percentage of tumor cells expressing CSPG4 had poorer overall survival (P = .002).

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