Accelerating new therapies for Alport spectrum disorders
Alport Spectrum Disorders (ASD) are rare inherited kidney diseases that often affect children and young adults, leading to progressive kidney damage and, in many cases, early kidney failure requiring dialysis or transplantation. For patients and families, the disease imposes a lifelong medical, psychological, and social burden, with limited treatment options beyond symptom management.
The ALP-RARE project has been selected for funding under the highly competitive ERDERA call, with national support in Cyprus provided by the Research and Innovation Foundation (RIF). The project aims to accelerate the development of innovative therapies for Alport Spectrum Disorders (ASD), a group of rare inherited kidney diseases that currently have no approved treatments targeting their root cause. Unlike traditional drug discovery approaches, ALP-RARE focuses on optimizing therapies on top of existing standard-of-care treatments, combining drug repurposing with novel protective compounds. This strategy allows faster clinical translation while reducing development risk, an especially critical consideration in rare diseases.
Alport Spectrum Disorders (ASD) are caused by genetic alterations affecting type IV collagen, a key structural component of the glomerular basement membrane, also known as the filtration barrier. Alterations or defects in type IV collagen disrupt the structure and function of this barrier, resulting in progressive kidney damage in patients, which often leads to chronic kidney disease and, in many cases, kidney failure requiring dialysis or kidney transplantation. Current standard-of-care (SOC) treatments, such as ACE inhibitors and angiotensin receptor blockers, can slow disease progression but do not address the underlying molecular defect.
ALP-RARE will establish a robust preclinical validation and therapy development pipeline designed to improve how new treatments are identified, tested, and prepared for clinical translation. A central concept of the project is the combination of standard of care (SOC) with selected small molecules, aiming to enhance therapeutic benefit while building on existing clinical practice. By integrating drug repurposing strategies and novel protective compounds, the project seeks to preserve kidney function and extend kidney lifespan in affected individuals.
The research approach combines advanced laboratory mouse models, artificial intelligence tools, and multicenter preclinical randomized controlled trials — an approach that mirrors human clinical trial standards to enhance reliability and predictability. Cutting-edge technologies such as patient-derived kidney models, organ-on-chip systems, advanced imaging, and multi-omics analyses will be used to better understand disease mechanisms and identify the most effective treatment combinations.
ALP-RARE brings together scientific experts from six European countries and works closely with patient advocacy organizations. Through biobank.cy CoE, Cyprus operates as a central hub for coordinating advanced preclinical research, data integration and translational strategy across the European consortium composed of leading scientists in the field, working closely with patient organizations. Specifically, the Karolinska Institute and Guard Therapeutics Int. (Sweden), the University of Munich and the University Medical Center Göttingen (Germany), Erasmus University Rotterdam (Netherlands), Meyer Children’s Hospital (Italy) and the patient association Alport Selbsthilfegruppe (Germany). The University College London (United Kingdom) and the European Kidney Patients’ Federation, based in Spain, also participate.
Patient representatives actively contribute to defining research priorities, shaping clinically meaningful outcomes, and guiding dissemination activities to ensure that the project addresses real-world needs and challenges faced by patients and families.
By strengthening the bridge between laboratory research and human trials, ALP-RARE seeks to reduce the high failure rate of drug development in rare diseases and accelerate the path toward precision therapies for people living with ASD.
Over its 36-month duration, the project aims to lay the scientific and regulatory groundwork necessary for future clinical testing, ultimately contributing to improved quality of life and delayed kidney failure for patients affected by this rare genetic condition. Beyond its immediate scientific objectives, ALP-RARE will deliver validated disease models, predictive biomarkers, and regulatory-ready data packages that can be used by future clinical trials and development partnerships, creating lasting value beyond the project’s lifetime.
By focusing on delaying disease progression rather than promising a cure, ALP-RARE aims to deliver realistic, meaningful benefits that can extend kidney lifespan and improve daily life for people living with Alport Spectrum Disorders.
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