Vishal Patel, MD, on Unmet Needs in Polycystic Kidney Disease


Patel discussed the current standard of care for PKD and the potential advantages of a new gene therapy approach.

“We've gone after the root cause for polycystic kidney disease... People with polycystic kidney disease have mutations in one copy of PKD1 or PKD2, but the other copy is still around in many cells in the kidney. We figured out a way of harnessing that remaining copy and activating it such that it produces more protein. And that, at least in mouse models and human cell lines, seems to slow cyst growth.”

Patients with autosomal dominant polycystic kidney disease (ADPKD) have limited options. The only FDA-approved treatment for ADPKD, tolvaptan, does not address the root cause of the disease and therefore cannot halt its progression.

CGTLive spoke with Vishal Patel, MD, associate professor, Department of Internal Medicine, Division of Nephrology, UT Southwestern Medical Center, about his team’s preclinical research into a potential gene therapy approach to treating ADPKD, which was recently published in Nature Communications. He discussed the unmet needs that currently exist for patients with ADPKD and the limitations of treatment with tolvaptan. He also discussed the genetic basis of ADPKD, highlighting that many patients still have at least 1 functional copy of the disease-targeted genes.

Patel then described the key findings from his team’s research and explained the basic mechanism behind their approach. He also briefly spoke on the potential advantages of the gene therapy approach, which they tested in human cell lines and a mouse model of PKD, if it were to be developed into a new treatment for patient use.

Lakhia R, Ramalingam H, Chang CM, et al. PKD1 and PKD2 mRNA cis-inhibition drives polycystic kidney disease progression. Nat Commun. 2022;13(1):4765. Doi: 10.1038/s41467-022-32543-2
Recent Videos
Manali Kamdar, MD, on Liso-Cel's Ongoing Benefit in the Treatment Lanscape for LBCL
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases
Manali Kamdar, MD, on The Importance of Bringing Liso-Cel to Earlier Lines of Lymphoma Treatment
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart Failure
Steve Kanner, PhD, the chief scientific officer of Caribou Biosciences
Paul Y. Song, MD, the chairman and chief executive officer of NKGen
Lisa Nieland on Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
Manali Kamdar, MD, on Acclimating to Routine CAR T Practice in the Field
© 2024 MJH Life Sciences

All rights reserved.