Biologically defined neuronal synuclein disease as a tool to advance drug development

Gennaro Pagano; Tien Dam; Geoffrey A Kerchner; Wendy R Galpern; Milton Biagioni; Rajesh Karan; Danna Jennings; M Judith Peterschmitt; Tania Nikolcheva; Patrik Brundin

doi:10.1038/s41531-024-00845-5

Biologically defined neuronal synuclein disease as a tool to advance drug development

NPJ Parkinsons Dis. 2024 Dec 20;10(1):235. doi: 10.1038/s41531-024-00845-5.

Authors

Gennaro Pagano^{1

2}, Tien Dam³, Geoffrey A Kerchner⁴, Wendy R Galpern⁵, Milton Biagioni⁶, Rajesh Karan⁷, Danna Jennings⁸, M Judith Peterschmitt⁹, Tania Nikolcheva¹⁰, Patrik Brundin⁴

Affiliations

¹ Roche Pharma Research and Early Development (pRED), Roche Innovation Center, F. Hoffmann-La Roche Ltd., Basel, Switzerland. [email protected].
² University of Exeter Medical School, London, UK. [email protected].
³ Neumora Therapeutics, Watertown, MA, USA.
⁴ Roche Pharma Research and Early Development (pRED), Roche Innovation Center, F. Hoffmann-La Roche Ltd., Basel, Switzerland.
⁵ Janssen Research & Development, LLC, Cambridge, MA, USA.
⁶ UCB Pharma, Brussels, Belgium.
⁷ Global Drug Development, Novartis Pharma AG, Basel, Switzerland.
⁸ Denali Therapeutics Inc., South San Francisco, CA, USA.
⁹ Sanofi, Clinical Development Neurology, Cambridge, MA, USA.
¹⁰ Product Development Neurology, F. Hoffmann-La Roche Ltd., Basel, Switzerland.

Abstract

In a recent Viewpoint article (JAMA Neurol. 2024;81:789‒90), Okubadejo et al. raised concerns regarding two recent proposals for biological definitions and staging systems for synucleinopathies (the Neuronal Synuclein Disease Integrated Staging System and SynNeurGe system). While acknowledging these concerns, we provide an alternative perspective—that such frameworks represent important steps forward by allowing biologically defined populations to be targeted with precision treatments that can be accurately evaluated using stage-specific outcomes.

Publication types

Letter