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 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.

Nasal administration of the neuroprotective candidate NeuroEPO to healthy volunteers: a randomized, parallel, open-label safety study.

BACKGROUND: Delivery of therapeutic agents as erythropoietin (EPO) into Central Nervous System through intranasal route could benefit patients with neurological disorders. A new nasal formulation containing a non-hematopoietic recombinant EPO (NeuroEPO) has shown neuroprotective actions in preclinical models. In the current study, the safety of NeuroEPO was evaluated for the first time in humans. METHODS: A phase I, randomized, parallel, open-label study was carried out in healthy volunteers. They received, intranasally, 1 mg of NeuroEPO every 8 h during 4 days (Group A) or 0.5 mg of NeuroEPO (Group B) with the same schedule. The working hypothesis was that intranasal NeuroEPO produce <10% of severe adverse reactions in the evaluated groups. Therefore, a rigorous assessment of possible adverse events was carried out, which included tolerance of the nasal mucosa and the effect on hematopoietic activity. Clinical safety evaluation was daily during treatment and laboratory tests were done before and on days 5 and 14 after starting treatment. RESULTS: Twenty-five volunteers, 56% women, with a mean age of 27 yrs. were included. Twelve of them received the highest NeuroEPO dose. Twenty types of adverse events occurred, with headache (20%) and increase of hepatic enzymes (20%) as the most reported ones. Nasopharyngeal itching was the most common local event but only observed in four patients (16%), all of them from the lowest dose group. About half of the events were very probably or probably caused by the studied product. Most of the events were mild (95.5%), did not require treatment (88.6%) and were completely resolved (81.8%). No severe adverse events were reported. During the study the hematopoietic variables were kept within reference values. CONCLUSIONS: NeuroEPO was a safe product, well tolerated at the nasal mucosa level and did not stimulate erythropoiesis in healthy volunteers. TRIAL REGISTRATION: Cuban Public Registry of Clinical Trials RPCEC00000157 , June 10, 2013.

Caracterización preliminar de los protocolos de investigación de residentes de Ciencias Médicas / Preliminary characterization of research protocols of residents in Medical Sciences

Introducción: El conocimiento científico se caracteriza por ser racional, sistemático y verificable, lo que requiere una correcta estructura del protocolo de investigación. La finalidad de un protocolo es la correcta planificación y ejecución del estudio mediante la organización del trabajo de forma lógica, enfocada y eficiente. Objetivo: Describir los protocolos de tesis de los residentes de ciencias médicas. Métodos: Se realizó una investigación descriptiva de corte retrospectivo para la caracterización de 61 protocolos de tesis de los estudiantes de las disciplinas de Medicina general integral y Estomatología general integral del curso 2019-2020. Se confeccionó una planilla para la recolección de los datos extraídos de los protocolos revisados y se elaboró una base de datos para su almacenamiento. Resultados: Del total de protocolos revisados, 36 (59 por ciento) fueron de Medicina general integral y 25 (41 por ciento) de Estomatología general integral. El 90 por ciento de los protocolos de Medicina general integral son estudios observacionales, de estos el 97 por ciento estudios transversales, y de ellos casi el 90 por ciento estudios descriptivos. Los protocolos de Estomatología general integral presentaron resultados similares: el 96 por ciento son estudios observacionales, de estos 92 por ciento transversales y de ellos el 95 por ciento de tipo descriptivo. Acápites tan importantes como la operacionalización de las variables, la descripción de los métodos para la recolección de datos y las consideraciones éticas para el trabajo con humanos se consideran en menos del 25 por ciento de todos los protocolos revisados. Conclusiones: Se demuestra una marcada insuficiencia en la redacción de los protocolos de investigación por parte de los residentes de Medicina general integral y Estomatología general integral(AU)

Introduction: Scientific knowledge is characterized by being rational, systematic and verifiable, for this an adequate initial planning of the research work is required, which is achieved through the correct structure of the research protocol. The planning of a protocol is the correct planning and execution of the study by organizing the work in a logical, focused and efficient way. Objective: To characterize the thesis protocols of the students of the disciplines of comprehensive general medicine and comprehensive general stomatology of the 2019-2020 academic year. Methods: A retrospective descriptive investigation was carried out for the characterization of 61 thesis protocols of the students of the integral general medicine and integral general stomatology disciplines of the 2019-2020 academic year. A spreadsheet was prepared to collect the data extracted from the revised protocols and a database was prepared for storage. Results: Of the total protocols reviewed: 36 (59 percent) from integral general medicine and 25 (41 percent) from integral general stomatology. 90 percent of integral general medicine protocols are observational studies, of these 97 percent cross-sectional studies, and of them almost 90 percent descriptive studies. Regarding the integral general stomatology protocols, the results are similar: 96% are observational studies, of these 92 percent are cross-sectional and 95 percent of them are descriptive. In all, sections as important as the operationalization of the variables, the description of the methods for data collection, and the ethical considerations for working with humans are considered in less than 25 percent of all the protocols reviewed. Conclusions: A marked insufficiency in the writing of the research protocols by the residents of integral general medicine and integral general stomatology is demonstrated(AU)

Trabajo comunitario integrado de estudiantes de Ciencias Médicas frente a la COVID-19, Policlínico Rampa, 2020 / Integrated Community Work of Medical Sciences Students Facing COVID-19, Rampa Community Clinic, 2020

Introducción: En Cuba se realizan colosales esfuerzos para la prevención y control de la COVID-19, como es el pesquisaje activo realizado por estudiantes de Ciencias Médicas, a modo de trabajo comunitario integrado a la propia comunidad. Objetivo: Caracterizar el trabajo comunitario integrado desarrollado por estudiantes de Ciencias Médicas en el enfrentamiento a la COVID-19 en el área de salud del Policlínico Rampa. Métodos: Se realizó un estudio observacional de tipo descriptivo, retrospectivo, longitudinal, para analizar los datos provenientes del trabajo comunitario integrado. La pesquisa tuvo una primera etapa, entre marzo-julio 2020, al inicio de la pandemia en el país, y una segunda en septiembre-octubre 2020, dada la contingencia sanitaria existente en la capital. Se analizaron esencialmente las viviendas visitadas y las cerradas, personas pesquisadas, cantidad de adultos mayores, así como los sospechosos de contagio diarios. La evaluación del trabajo comunitario integrado se basó en la asistencia y la calidad de la pesquisa. Resultados: Se pesquisó como promedio por día el 21 por ciento del universo poblacional en la primera etapa y el 35 por ciento en la segunda etapa. El número de sospechosos detectados fue bajo. El número de viviendas visitadas y de adultos mayores pesquisados fue también superior durante la segunda etapa. Aunque el trabajo comunitario integrado fue más productivo y mejor evaluado en esa etapa, se consideró meritoria la pesquisa activa desarrollada por los estudiantes en ambos periodos. Conclusiones: El trabajo comunitario integrado realizado por estudiantes de las Ciencias Médicas, además de ser parte de su formación integral, representa un aporte necesario para enfrentar la COVID-19, de gran relevancia en un área con elevada prevalencia de adultos mayores(AU)

Introduction: In Cuba, boundless efforts are being made for the prevention and control of COVID-19, such as the active screening carried out by students of Medical Sciences, as a community work integrated into the community itself. Objective: To describe the integrated community work fulfilled by students of Medical Sciences in the confrontation with COVID-19 in the health area of Rampa community clinic. Methods: An observational, descriptive, retrospective, longitudinal study was carried out to analyze the data from integrated community work. The investigation had a first stage, from March to July 2020, at the beginning of the pandemic in the country, and a second stage from September to October 2020, given the existing health contingency in the capital city. Essentially, the homes visited and closed were analyzed, as well as the people surveyed, the number of older adults, and the daily contagion suspects. The evaluation of integrated community work was based on the assistance and the quality of the research. Results: An average of 21percent of the population universe was surveyed per day in the first stage and 35percent in the second stage. The number of suspects detected was low. The number of homes visited and aged adults surveyed was also higher during the second stage. Although integrated community work was more productive and better evaluated in this stage, the active research carried out by the students in both periods was considered meritorious. Conclusions: The integrated community work carried out by students of Medical Sciences, in addition to being part of their comprehensive training, represents a necessary contribution to face COVID-19, of great relevance in an area with a high prevalence of aged adults(AU)

El discreto encanto de los medicamentos / The discreet charm of drugs

El presente artículo es un resumen de un grupo de conferencias sobre la industria farmacéutica mundial ofrecidas entre 2010 y 2013 en diferentes foros nacionales e internacionales. El comportamiento de la producción y consumo de medicamentos en el mundo, teniendo en cuenta que se trata de un bien social que se utiliza para prevenir enfermedades y restablecer la salud del ser humano, ofrece un panorama extraño en relación con las reales necesidades de la población. Se analiza cuáles son los elementos implicados en ese comportamiento. Comienza con una caracterización de la industria farmacéutica mundial contemporánea y analiza tres aspectos que considera están relacionadas con estos resultados: la investigación médico-farmacéutica, la promoción e información médica y el funcionamiento de las agencias reguladoras nacionales de medicamentos. La industria farmacéutica mundial ha aportado enormes ventajas al tratamiento de las enfermedades. Es un hecho irrefutable, pero la Gran Pharma no es una industria cualquiera y requiere ser rentable. La globalización y el neoliberalismo han creado patrones de motivación y conducta basados fundamentalmente en los intereses del mercado. Debemos estar alertas en el futuro. Este nuevo siglo introduce nuevos retos a los pueblos, sus gobiernos y a la Gran Pharma en relación con la salud y la enfermedad y su enfrentamiento(AU)

This article summarized a group of lectures on the world drug industry delivered from 2010 to 2013 in a number of national and international fora. Taking into account that a pharmaceutical is a social asset used to prevent diseases and recover human health, the behavior of the drug production and consumption worldwide shows a awkward overview in terms of the real needs of the population. An analysis was made on the elements involved in such behavior, beginning with a characterization of the world current drug industry and analyzing three aspects related to the results: medical-drug research, medical promotion and information and operation of the national drug regulatory agencies. The world drug industry has provided huge benefits for the treatment of diseases and this is an undeniable fact; however, the Big Pharma needs to be profitable. Globalization and neoliberalism have created patterns of incentives and behaviors mainly based on the market interests. We should be aware of that in the future. This new century poses new challenges for the peoples, their governments and for the Big Pharma in terms of health, disease and how to face it(AU)