Havana, Jan 30.- A phase III clinical trial to evaluate the efficacy and safety of nasal administration of NeuroEPO in adult patients with spinocerebellar ataxias is being successfully developed in different provinces of the country by medical institutions and scientific centers attached to the Biocubafarma group.
Cuba is the country in the world with the highest prevalence of Ataxia type SCA2, at a rate of 36.2 cases per 100 thousand inhabitants, although its frequency in the province of Holguín is 183 cases per 100 thousand individuals. Internationally, the incidence of this type of ataxia is 3 to 5 cases per 100 thousand people.
Doctor of Science Luis Velázquez Pérez, Second Degree specialist in Neurophysiology and Neurology, founder of the Center for Research and Rehabilitation of Hereditary Ataxias (CIRAH) of Holguín and president of the Academy of Sciences of Cuba, reported that this phase III trial It follows a phase I/II carried out between 2015 and 2016.
As he explained, the results of that study showed an improvement in the cerebellar syndrome among the treated patients and an improvement in the cognitive manifestations in them, in addition to a high safety profile of NeuroEPO, a molecule developed by the Center for Molecular Immunology and which is being applied to different neurodegenerative diseases.
"One year after that trial began - added the scientist - the patients who received NeuroEPO showed a stabilization of the disease, while those who received a placebo showed a worsening in accordance with the natural history of the disease."
“NeuroEPO in SCA2 ataxia. From preclinical studies to clinical trials in humans”, was the presentation that focused this week's meeting between scientists and experts on Health issues, with the first secretary of the Central Committee of the Party and president of the Republic, Miguel Díaz-Canel. Bermúdez, and the member of the Political Bureau and prime minister, Manuel Marrero Cruz.
During the usual exchange with representatives of the country's medical and scientific community, held this Tuesday, Díaz-Canel sent congratulations to the workers of the Institute of Neurology and Neurosurgery for the new anniversary of the Neurology Hospital, founded on January 29 of 1962.
With the participation of the vice prime ministers Inés María Chapman Waugh and Jorge Luis Perdomo Di-Lella, and moderated by the Minister of Health, Dr. José Ángel Portal Miranda, in the exchange it was reported that the Phase III clinical trial of NeuroEPO in adult patients with spinocerebellar ataxias, placebo-controlled, randomized and double-blind, has the general objective of evaluating the efficacy and safety of the product in these patients.
As explained by Dr. Velázquez Pérez, ataxia means loss of coordination of movements and defines neurodegenerative diseases in which ataxia is the main clinical sign.
Regarding type SCA2, he explained that it is a serious multisystem hereditary disease, with a progressive course until death and whose age of onset is around 30 years and has a clinical course leading to disability in ten years.
In Cuba, every year, 75 new subjects are diagnosed with the mutation and 25 patients die. In the country, 2,400 people are reported to have the disease and 10,000 asymptomatic relatives, belonging to 200 families.
Regarding the clinical manifestations, the specialist described, among others, the so-called ataxic gait in one hundred percent of the patients, as well as language disorders. Meanwhile, slowing of saccadic eye movements affects 91 percent, muscle atrophy 75 percent, peripheral neuropathy 85 percent, swallowing abnormalities 76 percent, sleep disturbances 90 percent, and cognitive dysfunction in 85 percent of patients.
Despite a group of barriers that limit therapies for ataxias, a disease that does not have a definitive cure at the moment, Cuba is committed to developing them and making them free and universal for all Cubans who suffer from it or have genetic risks of being affected.
The objective is to stimulate the cellular and molecular mechanisms of neuroplasticity, improve damaged motor and cognitive functions, slow the progression of the disease, increase the independence of patients and thus improve the quality of life of all our compatriots who suffer from it, and also collaborate with the world.
Together with NeuroEPO and other experiences, the comprehensive and intensive rehabilitation of these patients in our country has allowed a significant improvement in cerebellar motor symptoms, gait speed, posture and coordination. (Text: Presidency of Cuba) (Photos: Revolución Studies)