Erythropoietin in Spinocerebellar Ataxia Type 2: Feasibility and Proof‐of‐Principle Issues from a Randomized Controlled Study

Background Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is...

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Published in:Movement disorders 2022-07, Vol.37 (7), p.1516-1525
Main Authors: Rodriguez‐Labrada, Roberto, Ortega‐Sanchez, Ricardo, Hernández Casaña, Patricia, Santos Morales, Orestes, Padrón‐Estupiñan, Maria del Carmen, Batista‐Nuñez, Maricela, Jiménez Rodríguez, Daise, Canales‐Ochoa, Nalia, Peña Acosta, Arnoy, Medrano Montero, Jacqueline, Labrada Aguilera, Pedro Enrique, Estupiñán Rodriguez, Annelie, Vazquez‐Mojena, Yaimee, Almaguer Gotay, Dennis, Aymed‐García, Judey, García‐García, Idrian, Torres Vega, Reydenis, Viada González, Carmen, Valenzuela Silva, Carmen M., Silva Ricardo, Yanelis, Columbié Ximelis, Jorge, Tribin Rivero, Kenia, Valle Cabrera, Roselin, García‐Rodriguez, Julio Cesar, Crombet Ramos, Tania, Amaro‐González, Daniel, Rodriguez‐Obaya, Teresita, Velázquez‐Pérez, Luis
Format: Article
Language:English
Subjects:
Ataxia
clinical trial
Clinical trials
Cognitive ability
Erythropoietin
human‐recombinant erythropoietin
Intranasal administration
Latency
Movement disorders
Neurological diseases
Patients
Placebos
Saccadic eye movements
Spinocerebellar ataxia
spinocerebellar ataxia type 2
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Summary:Background Several pieces of evidence have shown the neurotrophic effect of erythropoietin (EPO) and its introduction in the therapeutic practice of neurological diseases. However, its usefulness in the treatment of spinocerebellar ataxia type 2 (SCA2) has not been proven despite the fact that it is endogenously reduced in these patients. Objective The study aims to investigate the safety, tolerability, and clinical effects of a nasally administered recombinant EPO in SCA2 patients. Methods Thirty‐four patients were enrolled in this double‐blind, randomized, placebo‐controlled, phase I–II clinical trial of the nasally administered human‐recombinant EPO (NeuroEPO) for 6 months. The primary outcome was the change in the spinocerebellar ataxia functional index (SCAFI), while other motor, neuropsychological, and oculomotor measures were assessed. Results The 6‐month changes in SCAFI score were slightly higher in the patients allocated to NeuroEPO treatment than placebo in spite of the important placebo effect observed for this parameter. However, saccade latency was significantly decreased in the NeuroEPO group but not in placebo. The frequency and severity of adverse events were similar between both groups, without evidences of hematopoietic activity of the drug. Conclusions This study demonstrated the safety and tolerability of NeuroEPO in SCA2 patients after 6 months of treatments and suggested a small clinical effect of this drug on motor and cognitive abnormalities, but confirmatory studies are warranted. © 2022 International Parkinson and Movement Disorder Society.
ISSN:0885-3185
1531-8257
DOI:10.1002/mds.29045