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Roneparstat: Development, Preclinical and Clinical Studies

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Heparanase

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1221))

Abstract

A growing interest around heparanase and its role in cancer, inflammation and other diseases prompted the identification of specific inhibitors of this enzyme and the exploration of their potential therapeutic role. Roneparstat, a 15–25 kDa N-acetylated and glycol split heparin, is one of the most potent and widely studied heparanase inhibitors. These studies generated a large body of data, which allowed to characterize Roneparstat properties and to endorse its potential therapeutic role. Multiple Myeloma represents the indication that most of the studies, including the phase I clinical trial, addressed. However, Roneparstat antitumor activity activity has been documented in other cancers, and in non-oncological conditions.

In addition, assessing Roneparstat activity in different experimental models contributed to understanding heparanase role and the biological factors that may be affected by heparanase inhibition in more detail. Finally, some studies elucidated the molecular mechanisms regulating the enzyme-inhibitor kinetics, thus providing important data for the identification and design of new inhibitors.

The objective of this chapter is to provide a comprehensive overview of the most significant studies involving Roneparstat and discuss its potential role in therapy.

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Acknowledgments

The authors are thankful to Israel Vlodavsky and Ralph Sanderson for the invitation to prepare this chapter and for having enjoyed a very stimulating scientific interaction with them during the whole development process of Roneparstat. Their pivotal research has been and still is inspiring to the whole field. The authors want also to thank Giuseppe Giannini for revising the manuscript and all the pre-clinical and clinical investigators that have been involved in studies with Roneparstat for the important contribution provided, without forgetting the phase I patients and their families, whose consent and participation have been fundamental.

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Authors and Affiliations

  1. Leadiant Biosciences S.p.A, Rome, Italy

    Alessandro Noseda

  2. Freelance, Medical Consultant (formerly Leadiant Biosciences SA employee), Riva San Vitale, Switzerland

    Paola Barbieri

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  1. Alessandro Noseda
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  2. Paola Barbieri
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Correspondence to Alessandro Noseda .

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Editors and Affiliations

  1. Technion Integrated Cancer Center (TICC) Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Haifa, Israel

    Israel Vlodavsky

  2. Department of Pathology, University of Alabama at Birmingham, Birmingham, AL, USA

    Ralph D. Sanderson

  3. Technion Integrated Cancer Center (TICC) Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Haifa, Israel

    Neta Ilan

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Noseda, A., Barbieri, P. (2020). Roneparstat: Development, Preclinical and Clinical Studies. In: Vlodavsky, I., Sanderson, R., Ilan, N. (eds) Heparanase. Advances in Experimental Medicine and Biology, vol 1221. Springer, Cham. https://doi.org/10.1007/978-3-030-34521-1_21

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