Short-term Tolerance of Nasally-administered NeuroEPO in Patients with Parkinson Disease

Marité García-Llano, Ivonne Pedroso-Ibáñez, Lilia Morales-Chacón, Teresita Rodríguez-Obaya, Leslie Pérez-Ruiz, Iliana Sosa-Testé, Daniel Amaro-González, María Luisa Bringas-Vega, Marité García-Llano, Ivonne Pedroso-Ibáñez, Lilia Morales-Chacón, Teresita Rodríguez-Obaya, Leslie Pérez-Ruiz, Iliana Sosa-Testé, Daniel Amaro-González, María Luisa Bringas-Vega

Abstract

Introduction: No neuroprotective treatment has been able to successfully halt the progression of Parkinson disease or prevent development of associated complications. Recombinant erythropoetin (EPO), an erythropoiesis-stimulating agent originally indicated in anemia, produced and manufactured in Cuba (iorEPOCIM, CIMAB S.A, Havana, Cuba) has neuroprotective properties. NeuroEPO is a new nasal formulation of recombinant EPO with a low content of sialic acid and without hematopoietic effects. It has neuroprotective effects in animal models.

Objective: Evaluate short-term tolerance of intranasal NeuroEPO in patients with Parkinson disease.

Methods: As part of a monocentric randomized placebo-controlled double-blind study (registered at www.clinicaltrials.gov number NCT04110678), 26 patients with Parkinson disease (stages 1 and 2 on Hoehn & Yahr Scale), were randomly divided into two groups: NeuroEPO (n = 15) and placebo (n = 11), both treated intranasally either with the drug (1 mL, at a concentration of 1 mg/mL of NeuroEPO) or placebo once a week for 5 weeks. At each application, we recorded any adverse events and blood pressure. To assess potential hematopoietic effects of the drug, hematological and biochemical variables were evaluated one week before and one week after the intervention.

Results: There were no significant differences (p = 0.22) between the two groups in terms of frequency of adverse events (20.0% in NeuroEPO and 9.1% in placebo groups). Three patients in NeuroEPO presented nausea, and one vomited (possibly due to the patient's positioning during drug application). One patient in placebo group reported polyuria and nasal irritation. In both groups, the adverse events were mild, brief, required no treatment and did not present sequelae.

Conclusions: Nasally administered NeuroEPO for five weeks in patients with Parkinson disease stages 1 and 2 on Hoehn & Yahr Scale is well tolerated.

Keywords: Cuba; biopharmaceutics; erythropoietin; intranasal drug; neurodegenerative diseases; parkinson disease.

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