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PAPP-A: A Novel Therapeutic Target

Undergraduate #5
Discipline: Biological Sciences
Subcategory: Biochemistry (not Cell and Molecular Biology and Genetics)
Session: 1

Brian W. Basinski - Grand Valley State University
Co-Author(s): Co-Author(s): Ahmed Mady, Ph.D., University of Michigan, MI; Nurul Ansari, Ph.D., University of Michigan, MI; Zaneta Nikolovska-Coleska, M.S., Ph.D., University of Michigan, MI



The metalloprotease pregnancy-associated plasma protein-A (PAPP-A), plays a significant role in the IGF system, a cellular pathway that controls metabolism, growth, and cell differentiation. PAPP-A targets and cleaves the insulin-like growth factor binding protein-2 (IGFBP-2) -4, and -5. The IGFBPs bind to and sequester the insulin growth factor (IGF), an endocrine growth hormone. Once the IGFBPs are cleaved they release IGF. Therefore, PAPP-A functions to increase the bioavailability of IGF. IGF binds to and signals the tyrosine kinase receptor named insulin growth factor receptor (IGFR). PAPP-A has been shown to have a role in modulating aging and age-related changes. Gene knock-out of PAPP-A in mice shows increased lifespan and reduced tumorigenicity of cancer. Therefore, the development of small-molecule inhibitors of PAPP-A can be used to recapitulate these effects. Characterized small molecule inhibitors of PAPP-A will lead to the development of chemical tools that will provide insight into the biology and role of PAPP-A in aging.
We discovered a lead compound from a high-throughput screen. From this lead, we synthesized analogs and validate their activity in inhibiting PAPP-A with two orthogonal assays. We used a Quenched Fluorescent Assay which detects PAPP-A activity using an IGFBP-4-derived fluorescent peptide. To determine a range of PAPP-A activity we established the minimum control which contained no PAPP-A and the maximum control which contained PAPP-A, both controls contained no compounds. Additionally, we designed a western blot which detects PAPP-A activity by identifying IGFBP-4 full-length and IGFBP-4 cleaved segments using a polyclonal antibody. The minimum and maximum IGFBP-4 cleavage were determined as controls, where the minimum cleavage reaction contained IGFBP-4 without PAPP-A and the maximum cleavage reaction contained IGFBP-4 with PAPP-A, both controls contained no compounds. PAPP-A activity in the western blot was validated by the introduction of stanniocalcin-1 (STC1), an endogenous inhibitor of PAPP-A, as a positive control. We conclude that this class of compounds is the first to show inhibition of PAPP-A. Further studies are required to understand how the inhibition of PAPP-A enzymatic function modulates aging.
References: Lawrence, J. B.; Oxvig, C.; Overgaard, M. T.; Sottrup-Jensen, L.; Gleich, G. J.; Hays, L. G.; Yates, J. R.; Conover, C. A. The Insulin-like Growth Factor (IGF)-Dependent IGF Binding Protein-4 Protease Secreted by Human Fibroblasts Is Pregnancy-Associated Plasma Protein-A. Proc. Natl. Acad. Sci. U. S. A. 1999, 96 (6), 3149-3153.
Conover, C. A.; Bale, L. K.; Mader, J. R.; Mason, M. A.; Keenan, K. P.; Marler, R. J. Longevity and Age-Related Pathology of Mice Deficient in Pregnancy-Associated Plasma Protein-A. Journals Gerontol. – Ser. A Biol. Sci. Med. Sci. 2010, 65 A (6), 590-599.
Gallagher, E. J.; LeRoith, D. Minireview: IGF, Insulin, and Cancer. Endocrinology 2011, 152 (7), 2546-2551.

Funder Acknowledgement(s): Funder Acknowledgement(s): This research is supported by Glenn Foundation and Calico LifeScience (Z.N-C.) and by the National Science Foundation through the Interdisciplinary REU Program in the Structure and Function of Proteins at the University of Michigan - College of Pharmacy (B.B.).

Faculty Advisor: Dr. Zaneta Nikolovska-Coleska, M.S., Ph.D., zanetan@med.umich.edu

Role: I performed the validation tests on all compounds of a novel class of inhibitors of PAPP-A (~30 compounds total), in three assays. I used a quenched fluorescent assay, this experiment was performed in dose-response (DR) and each compound was tested independently 3 times. I also helped designed a western blot assay (WBA), characterizing PAPP-A activity and optimal concentrations of protein and substrate. With WBA I independently tested all compounds in DR independently 3 times. Finally, I tested all compounds in a cell viability assay, cell line A673.

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This material is based upon work supported by the National Science Foundation (NSF) under Grant No. DUE-1930047. Any opinions, findings, interpretations, conclusions or recommendations expressed in this material are those of its authors and do not represent the views of the AAAS Board of Directors, the Council of AAAS, AAAS’ membership or the National Science Foundation.

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