​​NEPTUNE and CureGN Driving Precision Nephrology Forward, With Alessia Fornoni, MD, PhD

Efforts to better understand and treat glomerular diseases are increasingly being driven by large-scale, collaborative research initiatives that move beyond traditional disease labels. Longitudinal cohort studies such as Nephrotic Syndrome Study Network (NEPTUNE) and Cure Glomerulonephropathy (CureGN) are reshaping how clinicians and researchers conceptualize kidney disease, shifting the focus from histologic patterns to underlying molecular mechanisms and patient-specific biology.

In an interview with HCPLive at the World Congress of Nephrology (WCN) Conference in Yokohama, Japan, Alessia Fornoni, MD, PhD, a Professor of Medicine, Molecular and Cellular Pharmacology, and Biochemistry at the University of Miami Miller School of Medicine and Director and Chair of the Peggy and Harold Katz Drug Discovery Center, said these natural history studies provide a critical foundation for linking clinical presentation, demographics, and long-term outcomes. By integrating detailed phenotypic data with genomic and molecular analyses, they enable a more precise understanding of disease heterogeneity.

“Natural history studies are really the best way to understand the correlation between demographic, clinical presentations and outcomes,” she said. “I've been engaged in this study since the very beginning, and I have to say that most of my science has really been developed thanks to those studies that have allowed the de-risking drug development strategies and target identification by bringing clinical data to the bench.”

She notes that this approach has been instrumental in bridging a longstanding gap between nephrology and fields like oncology, where biomarker-driven treatment strategies are already well established.

One of the most significant contributions of these networks, she explains, is their ability to inform target discovery and therapeutic development. Drawing on biopsy-based transcriptomic data, researchers have identified novel molecular pathways involved in disease progression.

Fornoni says these advances are also beginning to reshape clinical trial design. Traditionally, nephrology trials have enrolled broad patient populations based on histologic diagnosis alone, often resulting in mixed outcomes. She emphasizes that molecular stratification offers a more targeted approach. By matching patients to therapies based on molecular phenotype, researchers may improve the likelihood of demonstrating treatment efficacy and accelerate drug development.

Despite this progress, she cautions that these approaches remain largely within the research domain. While the ability to identify activated pathways in individual patients is advancing, it has not yet translated into routine clinical decision-making. Fornoni underscores the importance of transparency and patient engagement as these tools evolve, particularly as clinicians begin to communicate complex molecular data to patients.

Looking ahead, she describes a field on the cusp of transformation. With a growing pipeline of targeted therapies and increasingly sophisticated tools for patient stratification, nephrology is moving toward a more precise, individualized model of care that holds promise for improving outcomes across a range of glomerular diseases.

“It's really an exciting time in nephrology now with so many new drugs. We used to sit there and just prescribe RAAS inhibitors or angiotensin receptor blockers, and having all these disease and soon that the tool to match the right drug to the right patient makes nephrology a very exciting discipline to practice,” she said.

Editors’ note: Fornoni reports relevant disclosures with Travere, Alexion, Novartis, Vera Therapeutics, Bayer, Aion, and others.

References

1. NIDDK. Nephrotic Syndrome Study Network (NEPTUNE). Accessed March 30, 2026. https://repository.niddk.nih.gov/study/115

2. NIDDK. Cure Glomerulonephropathy (CureGN). Accessed March 30, 2026. https://repository.niddk.nih.gov/study/171