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A surge in clinical trials investigating potential idiopathic pulmonary fibrosis therapies offers patients new hope, according to a recently published review.

Although effective treatments for idiopathic pulmonary fibrosis (IPF) remain elusive, the number of clinical trials currently investigating potential IPF therapies is “unprecedented,” according to a review published online in Pulmonary Therapy.

“This report on the many ongoing trials and investigational drugs highlights the efforts made by pharmaceutical companies, researchers, and doctors to prolong survival and improve the quality of life for patients suffering from this terrible disease,” wrote corresponding author Giacomo Giulianelli, MD, University of Padua, and colleagues.

Pathogenesis Insights & Trial Surge

IPF is marked by progressive fibrosis and destruction of lung architecture; without intervention, median survival is only three years, according to the authors. However, over the past decade, advances in understanding IPF pathogenesis—driven by genetic predisposition, aging processes, and environmental pollutant exposure—have fueled a dramatic uptick in drug development, offering hope for patients.

Unmet Needs

Research shows that pirfenidone and nintedanib, the antifibrotic agents constituting the current standard of care, slow forced vital capacity decline, reduce hospitalizations, and extend median survival. Nevertheless, neither therapy alleviates symptoms nor fully arrests disease progression. The authors emphasize that this therapeutic gap underscores the urgency for more potent and symptom-relieving interventions.

Emerging Targets

Investigational agents target key pathogenic players—alveolar macrophages, fibroblasts, and epithelial cells, the authors explained. REGEND001, an autologous P63+ basal progenitor cell product, demonstrated safety and tolerability in phase 1 trials; a phase 2, placebo-controlled study is slated for completion in July 2025.

“Inhaled molecules and others aimed at treating symptoms related to IPF, such as cough, are also gaining attention,” the review noted. Targeted inhalation could minimize systemic adverse events while delivering higher drug concentrations directly to alveoli, a “fascinating” idea, the authors noted. Clinical-stage inhaled candidates include treprostinil—an inhaled phosphodiesterase-5 inhibitor—and ARO-MMP7, an RNA interference agent designed to downregulate matrix metalloproteinase 7, thereby mitigating fibrosis and inflammation.

Transforming IPF

The authors suggested that continued elucidation of disease mechanisms will not only guide future drug development but also equip clinicians and researchers to transform IPF from a fatal diagnosis into a manageable condition, concluding, “There is certainly still much to understand about the pathogenesis and mechanisms underlying the development of IPF.”