Basic Study
Copyright ©The Author(s) 2018. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Nephrol. Sep 7, 2018; 7(5): 96-107
Published online Sep 7, 2018. doi: 10.5527/wjn.v7.i5.96
A small molecule fibrokinase inhibitor in a model of fibropolycystic hepatorenal disease
Prani Paka, Brian Huang, Bin Duan, Jing-Song Li, Ping Zhou, Latha Paka, Michael A Yamin, Scott L Friedman, Itzhak D Goldberg, Prakash Narayan
Prani Paka, Brian Huang, Bin Duan, Jing-Song Li, Ping Zhou, Latha Paka, Michael A Yamin, Itzhak D Goldberg, Prakash Narayan, Department of Research and Development, Angion Biomedica Corp., Uniondale, NY 11553, United States
Scott L Friedman, Icahn School of Medicine, 1 Gustave L. Levy Place, New York, NY 10029, United States
Author contributions: Narayan P, Paka P, Yamin MA, Paka L and Friedman SL substantially contributed to the conception and design of the study, acquisition, analysis and interpretation of data; Goldberg ID has contributed for funding acquisition and scientific advice; Narayan P drafted the article related to the intellectual content of the manuscript; Paka L made revisions for the reviewer’s comments for the final version of the article to be published; Paka P, Huang B and Zhou P also performed in vitro work; Duan B and Li JS performed animal care, experimental dosing, pre and post-surgical procedures in animal models; all authors reviewed and approved the final version of the article to be published.
Institutional animal care and use committee statement: Angion’s Animal Welfare Assurance # A4532-01. All the study protocols were designed to minimize pain and distress to the animals and were reviewed approved by our Angion’s animal care and use committee.
Conflict-of-interest statement: Dr. Narayan reports in addition, Dr. Narayan has a patent null issued.
Data sharing statement: Angion Biomedica is a for-profit small business engaged in developing therapeutics for unmet medical needs. In keeping with corporate policy, data and research resources generated by the company are proprietary. Once all intellectual property that results from the generation of the data and research resources is protected via filing of patents, data and research resources generated will be made available.
ARRIVE guidelines statement: The authors have read the ARRIVE guidelines, and the manuscript was prepared and revised according to the ARRIVE guidelines.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Prakash Narayan, PhD, Research Scientist, Department of Research and Development, Angion Biomedica Corp., 51 Charles Lindbergh Blvd., Uniondale, NY 11553, United States. pnarayan@angion.com
Telephone: +1-516-3261200
Received: March 25, 2018
Peer-review started: March 25, 2018
First decision: April 10, 2018
Revised: July 11, 2018
Accepted: August 11, 2018
Article in press: August 11, 2018
Published online: September 7, 2018
Processing time: 167 Days and 10.2 Hours
Core Tip

Core tip: In autosomal recessive polycystic kidney disease (ARPKD)-congenital hepatic fibrosis (CHF), a genetically acquired and congenital disease, approximately 20-30% of affected patients succumb within the first 1-2 mo of life, with pulmonary insufficiency secondary to renal enlargement as the primary cause of death. For children, nephrectomy and dialysis or kidney liver transplant is often warranted by approximately ten years of age. Other than transplantation, there is no cure for ARPKD-CHF. We report that platelet-derived growth factor and vascular endothelial growth factor are the intermediaries between the cystic and fibrotic components of progressive fibropolycystic disease and ANG3070, a novel dual kinase inhibitor therapy that may serve as an interesting bridge toward hepato-renal transplantation in patients with ARPKD-CHF.