Human Umbilical Cord Mesenchymal Stem Cells for Severe Neurological Sequelae due to Anti-N-Methyl-d-Aspartate Receptor Encephalitis: First Case Report

Abstract

Background: Anti-N-methyl-d-aspartate (NMDA) receptor encephalitis results from altered immune responses and can lead to severe neurological sequelae. This study presents the first case of a patient with significant neurologic impairment following NMDA receptor encephalitis, treated with allogeneic umbilical cord-derived mesenchymal stem/stromal cells (UC-MSCs).

Case Presentation: A 5-year-old girl diagnosed with NMDA receptor encephalitis underwent treatment with immunosuppressive medications and intravenous immunoglobulin (IVIG). Despite intensive therapy, her condition deteriorated, leading to the consideration of allogenic UC-MSC therapy. The patient received three intrathecal infusions of xeno- and serum-free cultured UC-MSCs at a dosage of 10^6 cells/kg.

Assessment and Results: The patient's condition was evaluated using the German Coma Recovery Scale (CRS), Gross Motor Function Classification System (GMFCS), Gross Motor Function Measure–88 (GMFM-88), Manual Ability Classification System (MACS), Modified Ashworth Scale, and Denver II test at baseline and after each UC-MSC administration. Initially, she was in a permanent vegetative state with diffuse cerebral atrophy. Following the three UC-MSC infusions, there was progressive improvement in cognition and motor functions. At the last follow-up, the patient regained the ability to walk, write, and count, with complete recovery of urinary and bowel control. Brain magnetic resonance imaging (MRI) showed a reduction in cerebral atrophy.

Conclusion: The outcomes of this case suggest that UC-MSC therapy may be a promising treatment for autoimmune encephalitis and its associated neurological consequences.