UB identifies allopurinol as a potential new therapeutic approach to prevent aortic aneurysms in Marfan syndrome
A research team from the University of Barcelona has identified a new therapeutic application for allopurinol, a widely used clinical drug, in the treatment of aortic aneurysms associated with Marfan syndrome. This rare genetic disorder affects connective tissue and can cause progressive enlargement of the aorta, increasing the risk of dissection, rupture and premature death.
The research was led by Dr Gustavo Egea, Professor at the Department of Biomedicine of the Faculty of Medicine and Health Sciences of the University of Barcelona and researcher at IDIBAPS (August Pi i Sunyer Biomedical Research Institute), in collaboration with researcher Isaac Rodríguez-Rovira and researcher Victoria Campuzano from CIBERER (Networked Biomedical Research Centre for Rare Diseases).
Results obtained in preclinical models have shown that allopurinol can prevent the formation of aortic aneurysms and slow their progression. The beneficial effect of the drug does not appear to be related to its well-known action on uric acid levels, but rather to its antioxidant properties, which help reduce the oxidative stress involved in the deterioration of the aortic wall.
Current pharmacological options for Marfan syndrome have limited effectiveness in preventing or slowing aneurysm progression. As a result, many patients eventually require preventive aortic surgery, a complex intervention that may involve significant risks and complications. In this context, allopurinol could represent a safe, accessible and non-invasive alternative to delay or prevent disease progression.
One of the technology’s main strengths is its clinical translation potential. Allopurinol is a well-known, low-cost drug with a widely documented safety profile, which could facilitate its therapeutic repositioning. In addition, the European Medicines Agency has granted allopurinol orphan drug designation for the treatment of Marfan syndrome.
“This research shows how knowledge generated within the university can create new therapeutic opportunities from already approved drugs. In the case of Marfan syndrome, allopurinol could offer a safe, affordable and potentially transformative option to reduce the risks associated with aortic aneurysms,” says Dr Gustavo Egea.
The Bosch i Gimpera Foundation, the University of Barcelona’s knowledge transfer office, is promoting this technology with the aim of identifying industrial partners interested in establishing licensing or collaboration agreements to advance its clinical development and commercial exploitation.
The technology, identified under reference UBTT0509, is currently undergoing in vivo preclinical validation and represents a significant opportunity to accelerate the development of new therapeutic solutions for patients affected by a rare disease with major clinical and social impact.