\u201cThis project aims to promote the development of MBF-118, a novel drug discovered, developed and patented by Medibiofarma, as a therapy for Marfan syndrome and, by extension, for other aortopathies, both genetic and non-genetic, thereby significantly improving patients\u2019 quality of life,\u201d explains Gustavo Egea, Professor in the Department of Biomedicine at the Faculty of Medicine and Health Sciences, who leads UB\u2019s participation in the project.<\/p>\n
A promising mechanism of action<\/h3>\n
Currently, there are no pharmacological therapies capable of effectively stopping or delaying the aortic damage associated with Marfan syndrome. The main therapeutic option is surgical repair of aortic dissection or rupture, which is not without risks. In this scenario, the compound MBF-118 emerges as a promising pharmacological alternative thanks to its ability to selectively and controllably activate the PPAR\u03b3 receptor, whose expression is markedly reduced in the Marfan aorta.<\/p>\n
This receptor is mainly known for its role in fatty acid and glucose metabolism, but several studies have shown that it also plays a relevant role in the vascular system. According to Egea, through this mechanism of action, the drug could \u201chelp restore balance in aortic tissue and exert beneficial effects on the integrity and functionality of the aortic wall, cellular signalling and probably also on blood flow, thus reinforcing its potential as a new therapeutic option.\u201d<\/p>\n
Positive preclinical and clinical results<\/h3>\n
The compound MBF-118 has already passed several stages of pharmaceutical development. Data obtained to date \u2014both in animal models and in a proof-of-concept study in patients with Crohn\u2019s disease\u2014 indicate that the drug is safe and well tolerated in humans. Moreover, it shows adequate absorption and reaches sufficient and sustained blood concentrations over time to induce the desired therapeutic effects.<\/p>\n
In this context, the project will make it possible to evaluate the therapeutic potential of MBF-118 in an animal model that closely represents the clinical progression of Marfan syndrome in patients, as well as to carry out the toxicological studies required to meet the regulatory requirements for future long-term clinical trials in patients. \u201cThis work will help validate the compound\u2019s effect in the prevention\/attenuation of aortic aneurysms and dissections and their pathophysiology,\u201d highlights Egea, whose research group brings extensive experience in studying the pathological mechanisms of aortic diseases.<\/p>\n
In addition, new formulations of the compound will be developed throughout the project. \u201cThis will help patients follow the treatment more easily, with particular consideration for paediatric patients with Marfan syndrome,\u201d adds the researcher.<\/p>\n
Achieving these objectives will facilitate the designation of MBF-118 as an orphan drug for this genetic disorder. Orphan drugs are intended for the treatment of rare diseases and benefit from regulatory and financial incentives that support their development. This designation will accelerate the launch of a phase II clinical trial with MBF-118 in patients with this disease.<\/p>\n
The project, entitled \u201cResearch and development of a new therapy for the treatment of Marfan Syndrome (Marfan-PPARg)\u201d, has a duration of three years and reference number CPP2024-011398. The University of Barcelona has obtained funding of 267,550 euros under the 2024 call for public-private collaboration projects, funded by the Ministry of Science, Innovation and Universities, the State Research Agency and FEDER funds.<\/p>\n
Caption: Members of the Vascular Cell Biology group (UB-IDIBAPS).<\/p>\n
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