Original Investigation

Virus-Specific T Cells and Response to Checkpoint Inhibitors in Progressive Multifocal Leukoencephalopathy

JAMA Neurology 10.1001/jamaneurol.2025.5318

January 20, 2026 at 11:00 AM EST

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Are pretreatment JC virus- and/or BK virus-specific T cells in the blood associated with the efficacy of immune checkpoint inhibitors (ICIs) in progressive multifocal leukoencephalopathy (PML)?In this cohort study of 111 patients with PML treated with ICIs, those with detectable virus-specific T cells (n = 21) had significantly higher response rates and longer survival than both T cell–negative patients (n = 22) and those with unknown status (n = 68).These findings suggest that functional virus-specific T cells, as markers of preexisting antiviral immunity, may be associated with better clinical outcomes and reduced toxicity in patients with PML who are treated with ICIs.

Corresponding Author: Thomas Skripuletz, MD, Department of Neurology, Hannover Medical School, Carl-Neuberg-Str 1, 30625 Hannover, Germany (skripuletz.thomas@mh-hannover.de).

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