Progressive multifocal leukoencephalopathy (PML) is a demyelinating disease of the central nervous system caused by JC polyomavirus (JCPyV) occurring in immunocompromised patients who face a disease that is usually progressive and often fatal (Padgett 1971; Tan 2010). Annually, it is estimated that 4,000 people develop PML in the United States and Europe combined (NORD 2015).~This is a randomized, double- blinded, Phase 2 trial in patients with PML due to JCPyV. Patients will receive treatment with a matched virus-specific T-cell product (CE VST01-JC) or placebo, and will then be monitored for response to therapy. The study is designed to evaluate whether CE-VST01-JC infusions will slow and ultimately halt neurological progression in patients with PML compared with placebo as evaluated by modified Rankin Score (mRS).
This is a randomized, double-blinded, Phase 2 trial in patients with PML due to JCPyV. Patients will receive treatment with a matched virus-specific T-cell product (CE VST01-JC) or placebo, and will then be monitored for response to therapy.