Abstract
To date, there is no curable treatment option for non-hereditary degenerative cerebellar ataxia. Here we report the case of a patient with sporadic adult-onset ataxia (SAOA) who underwent allogeneic bone marrow-derived mesenchymal stem cell (MSC) therapy via the intrathecal route. A 60-year-old male patient visited our clinic complaining of progressive gait disturbance that commenced two years ago. Upon neurologic examination, the patient exhibited limb dysmetria and gait ataxia. Brain magnetic resonance imaging (MRI) revealed cerebellar atrophy whereas the autonomic function test was normal. The patient was diagnosed with SAOA. The medications that were initially prescribed had no significant effects on the course of this disease and the symptoms deteriorated progressively. At the age of 64, the patient was treated with allogeneic bone marrow-derived MSC therapy. The subsequent K-SARA (Korean version of the Scale for the Assessment and Rating of Ataxia) scores demonstrated a distinct improvement up until 10 months post-administration. No adverse events were reported. The improved post-treatment K-SARA scores may suggest that the MSC therapy can have a neuroprotective effect and that stem cell therapy may serve as a potential therapeutic option for degenerative cerebellar ataxia.
Highlights
Cerebellar ataxia is an impairment of motor coordination and balance associated with the dysfunction of the cerebellum and its afferent and efferent pathways [1]
The K-SARA scores at the beginning of the stem cell administration had gradually deteriorated since the initial diagnosis
A recent study has found that the growth rate and expression of the neuroprotective factors of mesenchymal stem cell (MSC) in patients w4it.hCocnecrleubseiolnlasr ataxia were decreased as opposed to MSCs in healthy people [10]
Summary
Cerebellar ataxia is an impairment of motor coordination and balance associated with the dysfunction of the cerebellum and its afferent and efferent pathways [1]. We report a case of a sporadic adult-onset ataxia (SAOA) patient that was treated with allogeneic bone marrow-derived MSC therapy via the intrathecal route. The MSC (CS20BR08) used in this study was developed at Corestem’s Good Manufacturing Practice (GMP) verified facility and originated from haplotype matched bone marrow from a donor. In the final process of the CS20BR08 production, cell surface markers CD29, CD44, CD73, CD90 and CD 105 (BD Pharmingen, Heidelberg, Germany) and negative markers CD34 and CD45 (BD Pharmingen, Heidelberg, Germany) were confirmed by flow-cytometry for the subsequent characterization of the MSCs. Under the qualification control protocol, all sterility tests including safety from bacteria, fungi, viruses and mycoplasma were confirmed. The final products, CS20BR08, were suspended in cerebrospinal fluid at 1 × 106 cells/kg and were administered to patients through an intrathecal injection twice four weeks apart. The study was approved by the Institutional Review Board of Kyungpook National University Chilgok Hospital (2017-12-002)
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