Objective/Rationale:
The development of Parkinson’s disease (PD) is linked to the accumulation of the toxic protein alpha-synuclein. Systems within neurons able to clear alpha-synuclein become overwhelmed in PD. Junaxo is developing therapies aimed at improving the ability of such systems to reduce alpha-synuclein. Trehalose is a safely consumed disaccharide sugar that enhances autophagy (cell clean up), reduces brain alpha-synuclein levels and prevents pathology in a pre-clinical model of PD. Several studies have been designed in this program of research to develop trehalose as a therapeutic candidate ready to enter clinical trials.
Project Description:
We will identify blood and brain levels of trehalose associated with efficacy in an alpha-synuclein model of PD and define dosing strategies to translate these levels to non-human primates. This administration strategy will then be used to assess efficacy of trehalose in an alpha-synuclein-based non-human primate model of PD.
Relevance to Diagnosis/Treatment of Parkinson’s Disease:
Trehalose could be used as orally-administered therapy to slow or reverse the progression of PD.
Anticipated Outcome:
We will define the optimal brain and plasma levels of trehalose in terms of providing efficacy in pre-clinical models. In doing this, we will define target dosing to be used in proof-of-concept clinical trials. As trehalose is already FDA-approved as a safely consumed product, it will be ready to move into the clinic relatively rapidly.