Abstract: SA-PO0272
Therapeutic Repurposing of Arimoclomol for AKI
Session Information
- AKI: Mechanisms - Cell Signaling
October 24, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Acute Kidney Injury
- 103 AKI: Mechanisms
Authors
- Sultana, Taposhi Nahid, The Ohio State University, Columbus, Ohio, United States
- Madhavan, Sethu M., The Ohio State University, Columbus, Ohio, United States
- Pabla, Navjot, The Ohio State University, Columbus, Ohio, United States
Background
Acute kidney injury (AKI) is a major cause of morbidity and mortality with no approved pharmacologic therapy. To identify translatable therapeutic candidates, we performed a drug repurposing screen using FDA-approved and investigational compounds in a cell culture model of AKI, which identified arimoclomol as a top hit. Arimoclomol enhances heat shock factor 1 (HSF1)-dependent stress responses and promotes expression of cytoprotective chaperones including HSP70.
Methods
Arimoclomol was evaluated in bilateral ischemia–reperfusion injury (IRI) mouse models of AKI. Kidney function and histologic injury were assessed using blood urea nitrogen (BUN), serum creatinine, and tissue injury scoring. To investigate mechanism, proximal tubule-specific HSF1 conditional knockout (cKO) mice were generated and subjected to ischemic AKI.
Results
Arimoclomol-treated mice demonstrated significantly lower BUN, serum creatinine, and histologic injury compared with vehicle-treated controls, indicating marked protection against ischemic AKI. HSF1 deficiency increased susceptibility to kidney injury, supporting a protective role for heat shock signaling during AKI. Ongoing studies are examining whether the renoprotective effects of arimoclomol require HSF1 activation.
Conclusion
These findings identify arimoclomol as a promising repurposed therapeutic candidate for AKI and support activation of HSF1-mediated stress responses as a protective pathway in ischemic kidney injury.
Funding
- NIDDK Support