OrganoTherapeutics in collaboration with University of Luxemburg has published interestingin vitro and in vivo results on the effects of HPBCD treatment of Parkinson disease .
The etiology of Parkinson’s disease (PD) is only partially understood despite the fact that environmental causes, risk factors, and specific gene mutations are contributors to the disease. Biallelic mutations in the phosphatase and tensin homolog (PTEN)-induced putative kinase 1 (PINK1) gene involved in mitochondrial homeostasis, vesicle trafficking, and autophagy are sufficient to cause PD.
PD patient-derived cells presented differences in their energetic profile, imbalanced proliferation, apoptosis, mitophagy, and a reduced differentiation efficiency to tyrosine hydroxylase positive (TH+) neurons compared to controls’ cells. Correction of a patient’s point mutation ameliorated the metabolic properties and neuronal firing rates as well as reversing the differentiation phenotype, and reducing the increased astrocytic levels. Treatment with 2-hydroxypropyl-β-cyclodextrin increased the autophagy and mitophagy capacity of neurons concomitant with an improved dopaminergic differentiation of patient-specific neurons in midbrain organoids and ameliorated neurotoxicity in a mouse model.
Javier Jarazo, Kyriaki Barmpa, Jennifer Modamio, Cláudia Saraiva, Sònia Sabaté-Soler, Isabel Rosety, Anne Griesbeck et al. (2021) Parkinson’s Disease Phenotypes in Patient Neuronal Cultures and Brain Organoids Improved by 2-Hydroxypropyl-β-Cyclodextrin Treatment. Movement Disorders https://doi.org/10.1002/mds.28810