APDA-funding fuels new research in understanding alpha-synuclein aggregation

APDA-funding fuels new research in understanding alpha-synuclein aggregation

In a recently published new study, researchers at Johns Hopkins University School of Medicine, led by Dr. Gabsang Lee, developed a new model for testing whether compounds can interfere in the aggregation of alpha-synuclein, thought to be the primary driver of the pathology of Parkinson’s disease (PD). Dr. Lee received an APDA research grant in September 2022, funding which was crucial in developing this work.

In this paper, Dr. Lee and his team created induced pluripotent stem cells (iPSCs) from people with PD and differentiated them into dopaminergic neurons. They next developed a novel system, named OASIS (Opto-genetic assisted Alpha-Synuclein aggregation Induction System) in which alpha-synuclein production and aggregation within the dopaminergic neurons is regulated by shining blue light on the cells. The system allows for accurate control of alpha-synuclein aggregation within the dopamine cells. They next tested 1,280 drugs on these cells to see which of the compounds were able to reduce aggregation.

Of the long list of potential compounds, BAG956 was identified as having the most potential at reducing aggregation. They then further tested it in both cell systems and in a mouse model of PD. BAG956 not only reduced alpha-synuclein aggregation, but also reduced nerve cell death in dopaminergic cells. In a mouse model of PD, the introduction of BAG956 improved many tests of motor and behavior function. The next step for Dr. Lee’s research team is to continue to study BAB956 to determine if it can be tried in humans as a potential treatment for PD.

This work is particularly significant because not only did it identify a potential compound to help PD, but it developed a system for easy screening of many drugs, which could lead to even more potential treatments.

Dr. Lee expressed his gratitude to APDA for believing in his work, “Thank you for your support! I very much appreciate APDA’s support of our project. I tried very hard to secure research funding for this particular study over three years! Again, thank you so much”.

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