Treating amyotrophic lateral sclerosis with allogeneic Schwann cell–derived exosomal vesicles: a case report

• DOI: 10.4103/nrr.nrr-d-23-01815
• Publication Date: 2024-04-05T08:01:16Z
• Authors: Pascal J. Goldsch...; Aisha Khan; George Jimsheleis...; Patricia Graham; Adriana Brooks; Risset Silvera; Alexander J.P. Go...; Damien D. Pearse; W. Dalton Dietrich; Allan D. Levi; James D. Guest
• Keywords: Riluzole; Schwann cell

Schwann cells are essential for the maintenance and function of motor neurons, axonal networks, neuromuscular junction. In amyotrophic lateral sclerosis, where neuron is progressively lost, cell may also be impaired. Recently, important signaling potential trophic activities cell-derived exosomal vesicles have been reported. This case report describes treatment a patient with advanced sclerosis using serial intravenous infusions allogeneic vesicles, marking, to our knowledge, first instance such treatment. An 81-year-old male presented 1.5-year history rapidly progressive sclerosis. After initial diagnosis, underwent combination generic riluzole, sodium phenylbutyrate taurursodiol. The volunteered participate in an FDA-approved single-patient expanded access received weekly potentially restore impaired function. We confirmed that cultured obtained from via sural nerve biopsy appeared (senescent) exposure patient’s cadaver donor improved their growth capacity vitro. period observation lasting 10 weeks, during which Functional Rating Scale-Revised pulmonary were regularly monitored, consecutive 1.54 × 12 (×2), then 7.5 (×6) diluted 40 mL Dulbecco’s phosphate-buffered saline. None associated adverse events as infusion reactions (allergic or otherwise) changes vital signs. Clinical lab serum neurofilament cytokine levels measured prior each varied somewhat without clear trend. A more sensitive in-house assay suggested possible inflammasome activation disease course. trend clinical stabilization was observed period. Our study provides novel approach address possibly patients vesicles. Initial findings suggest this safe.