Allopurinol as a New Treatment against Aortic Aneurysm in Marfan Syndrome
Advantages
- Safe, affordable, and repurposed option: Allopurinol is a long‑established drug with an excellent safety profile and wide clinical availability.
- Innovative mechanism: Acts as a primary antioxidant, independently of uric‑acid lowering, with multiple protective effects (XOR inhibition, ROS reduction, endothelial normalization).
- Disease‑modifying potential: Prevent or slow the development and progression of aortic aneurysm in Marfan syndrome mouse models.
- Clinically actionable: Clear roadmap toward clinical translation, including dose optimization and planned clinical trials.
- Accessible long‑term therapy: Potential to transform current MFS treatment strategies thanks to its safety, low cost, and cardiovascular benefits
Goal
Looking for industrial partners interested in a license and/or collaboration agreement to further develop and exploit this asset.
Intellectual Property
Orphan Drug Designation in 2025 (EU/3/25/3041).
Reference
UBTT0509
Contact
Isabel Durán
Email: iduran@fbg.ub.edu
Allopurinol as a New Treatment against Aortic Aneurysm in Marfan Syndrome
Executive summary
A research team has demonstrated that allopurinol -a safe, affordable, and widely used medication- can offer a breakthrough solution for Marfan syndrome. This rare disorder causes dangerous aortic aneurysms that existing pharmacological therapies fail to stop. The team uncovered that excessive oxidative stress is a key factor behind aortic damage. By blocking this oxidative stress, allopurinol protects the aorta and slows the weakening of the vessel wall. These results reveal a powerful opportunity to repurpose a well established drug for a high impact, unmet medical need. Allopurinol stands out as a promising non invasive therapy with strong potential for rapid clinical translation. In recognition of these observation EMA has recently approved allopurinol as orphan drug for treatment of Marfan syndrome.
Introduction
Marfan syndrome is a rare genetic connective tissue disorder that primarily affects the aorta, leading to the formation of aneurysms that may progress to dissection and premature death. Excessive production of reactive oxygen species and the resulting oxidative stress further contribute to the degeneration of the aortic wall. Current pharmacological treatments, including beta blockers and angiotensin II receptor blockers (losartan), have shown limited efficacy in preventing or slowing aneurysm progression. Consequently, many patients eventually require preventive aortic surgery, a high risk procedure associated with complications such as stroke. There remains an urgent unmet need for a safe, effective, and non invasive therapy capable of preventing or significantly delaying aortic aneurysm development in individuals with Marfan syndrome.
Description
Allopurinol (ALO) is a well-known drug originally approved for reducing elevated uric acid levels. In a preclinical study using two different mouse models of aortic disease of Marfan syndrome, ALO demonstrated a remarkable capacity to block aneurysm formation and stop its progression. The drug was effective in both preventive and palliative treatment regimens and significantly increase the survival rate both in males and females. Interestingly, its beneficial effect was not linked to changes in uric acid levels but rather to its strong antioxidant properties, which supports its potential repurposing for Marfan syndrome.
Current stage of development
- Validated efficacy in vivo in a well‑established mouse models.
- Robust experimental evidence demonstrates consistent therapeutic benefits in Marfan syndrome.
- Strong potential for rapid clinical repurposing of allopurinol for Marfan patients, leveraging its known safety profile.