Marie Curie Chair-Planning grant application

Syfte och mål

This proposal will establish Dr. Becanovic as an independent researcher, and assemble a collaborative team of world-class investigators at the Karolinska Institutet (KI), the Berzelii Center Stockholm Brain Institute (SBI), and the Centre for Molecular Medicine and Therapeutics (CMMT) at the University of British Columbia (UBC), to perform translational science with the aim of developing novel therapeutic strategies for Huntington disease (HD). This work is imperative given the current lack of effective treatments or biomarkers for HD.

Resultat och förväntade effekter

This team will focus on dopaminergic stabilizers, a class of pharmacological agents that stabilize dysregulated psychomotor functions through their primary action on dopamine receptors. ACR16 (Huntexil®), which belongs to this class of compounds, is currently in clinical development as the first compound ever to demonstrate efficacy for the motor symptoms in HD patients. This project aims to: 1) test the disease-modifying and neuroprotective effects of ACR16; 2) elucidate the mechanism of action of the dopaminergic stabilizer ACR16.

Upplägg och genomförande

Dr. Becanovic will conduct pre-clinical studies in HD transgenic mice in collaboration with Dr. Leavitt at CMMT (University of British Columbia). These experiments will generate the first data on the effects of the ACR16 in an animal model of HD. This project aims to increase our understanding of the beneficial actions of ACR16 in HD by employment of HD mouse models. This proposal further aims to identify efficacy biomarkers that can be implemented in pre-clinical experimental testing in HD mouse models, and potentially as translational biomarkers in human clinical trials.