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  • The role of IGF pathway in polycystic kidney disease (MCF-CDB) (MCF-CDB) Jacksonville, Fla.

    The purpose of this study is to

    evaluate the potential of PAPP-A as a therapeutic target in ADPKD, we collaborated with Calico and performed the following studies under a sponsor research agreement (SRA) using an anti-PAPP-A monoclonal antibody (anti-PAPP-A) developed by Calico. These studies demonstrated an improvement in total kidney volume (TKV), reduction in cystic and tumor burden, and beneficial effects in survival, frailty index, and renal function.

    Thus, our published studies and the preclinical studies performed under the Calico SRA confirm that the IGF/PAPP-A pathway is a therapeutic target in ADPKD, and that anti-PAPP-A treatment is beneficial in this mouse model of cystic disease. However, the specific cell types that express PAPP-A in ADPKD kidneys is unknown and their identification may provide valuable insights into the mechanism of action of the antibody.

    We hypothesize that PAPP-A is highly induced in ADPKD kidneys and drives the cystic growth mainly through IGF dependent manner. We further propose that PAPP-A expression will correlate with the cystic disease in progressively growing ADPKD kidneys.  The study will help to understand role of PAPP-A in cystic progression in ADPKD and to identify the cell type/pathway relevant to the IGF/PAPP-A axis and validate the human relevance of this pathway in ADPKD; which will further help us to clinically validate the relevance of PAPP-A/IGF pathway in the ADPKD patients.

Closed for Enrollment

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