Today the Niemann-Pick Spain Foundation came to deliver the check for 71,056.25 euros that will fully finance a pilot study to determine the efficacy and safety of the drug Efavirenz to alleviate the cognitive deterioration of patients with this minority disease of genetic origin and for which there is currently no treatment.
The event was attended by Dr. Gabriel Capellá, director of IDIBELL, Dr. Montserrat Figuerola, manager of the South Metropolitan Territorial Area, Dr. Javier Tapia Martínez, Deputy Medical Director of the Bellvitge University Hospital (HUB), and Dr. Jordi Gascón, principal investigator of the IDIBELL Neurological Diseases and Neurogenetics group and section head of the HUB Neurology Service, who will lead the study. Dr. Gascón has appreciated the effort and commitment of the fundation in promoting research as a tool to improve the patients life quality.
The Niemann-Pick Spain Foundation is made up of patient families and is supported by volunteers, friends, and collaborators. Its work is divided into three main pillars: family orientation, dissemination, and research. For the latter, they created the Juan Girón scholarships that support projects focused on finding new treatments for this disease, such as that of Dr. Gascón.
An antiretroviral against HIV for the treatment of Niemann-Pick type C
The trial seeks to determine the effectiveness of Efavirenz in reducing learning and behavior problems in patients and thus improving their quality of life. This is a drug used as an antiretroviral against HIV that improves the function of an enzyme that regulates the distribution of cholesterol in the synapse, which makes it a great candidate for the Niemann-Pick test.
Until now, the results obtained in mice were encouraging. For this reason, they began translational research at IDIBELL and the HUB in 14 of the 27 patients over 14 years of age diagnosed with Niemann-Pick type C in Spain.
More information: A generic antiviral could improve the life expectancy of patients with a rare disease without a cure
The Bellvitge Biomedical Research Institute (IDIBELL) is a biomedical research center created in 2004. It is participated by the Bellvitge University Hospital and the Viladecans Hospital of the Catalan Institute of Health, the Catalan Institute of Oncology, the University of Barcelona and the City Council of L’Hospitalet de Llobregat.
IDIBELL is a member of the Campus of International Excellence of the University of Barcelona HUBc and is part of the CERCA institution of the Generalitat de Catalunya. In 2009 it became one of the first five Spanish research centers accredited as a health research institute by the Carlos III Health Institute. In addition, it is part of the “HR Excellence in Research” program of the European Union and is a member of EATRIS and REGIC. Since 2018, IDIBELL has been an Accredited Center of the AECC Scientific Foundation (FCAECC).