دورية أكاديمية

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

التفاصيل البيبلوغرافية
العنوان: Treating amyotrophic lateral sclerosis with allogeneic Schwann cell–derived exosomal vesicles: a case report
المؤلفون: Goldschmidt-Clermont, Pascal J., Khan, Aisha, Jimsheleishvili, George, Graham, Patricia, Brooks, Adriana, Silvera, Risset, Goldschmidt, Alexander J.P., Pearse, Damien D., Dietrich, W. Dalton, Levi, Allan D., Guest, James D.
المصدر: Neural Regeneration Research ; volume 20, issue 4, page 1207-1216 ; ISSN 1673-5374 1876-7958
بيانات النشر: Medknow
سنة النشر: 2024
الوصف: Schwann cells are essential for the maintenance and function of motor neurons, axonal networks, and the neuromuscular junction. In amyotrophic lateral sclerosis, where motor neuron function is progressively lost, Schwann cell function may also be impaired. Recently, important signaling and potential trophic activities of Schwann cell-derived exosomal vesicles have been reported. This case report describes the treatment of a patient with advanced amyotrophic lateral sclerosis using serial intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles, marking, to our knowledge, the first instance of such treatment. An 81-year-old male patient presented with a 1.5-year history of rapidly progressive amyotrophic lateral sclerosis. After initial diagnosis, the patient underwent a combination of generic riluzole, sodium phenylbutyrate for the treatment of amyotrophic lateral sclerosis, and taurursodiol. The patient volunteered to participate in an FDA-approved single-patient expanded access treatment and received weekly intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles to potentially restore impaired Schwann cell and motor neuron function. We confirmed that cultured Schwann cells obtained from the amyotrophic lateral sclerosis patient via sural nerve biopsy appeared impaired (senescent) and that exposure of the patient’s Schwann cells to allogeneic Schwann cell-derived exosomal vesicles, cultured expanded from a cadaver donor improved their growth capacity in vitro. After a period of observation lasting 10 weeks, during which amyotrophic lateral sclerosis Functional Rating Scale-Revised and pulmonary function were regularly monitored, the patient received weekly consecutive infusions of 1.54 × 10 12 (×2), and then consecutive infusions of 7.5 × 10 12 (×6) allogeneic Schwann cell-derived exosomal vesicles diluted in 40 mL of Dulbecco’s phosphate-buffered saline. None of the infusions were associated with adverse events such as infusion reactions (allergic or otherwise) or changes ...
نوع الوثيقة: article in journal/newspaper
اللغة: English
DOI: 10.4103/nrr.nrr-d-23-01815
DOI: 10.4103/NRR.NRR-D-23-01815
الإتاحة: https://doi.org/10.4103/nrr.nrr-d-23-01815Test
رقم الانضمام: edsbas.D27D7FDF
قاعدة البيانات: BASE