Reversing Brain Iron Overload in BPAN by a Natural Small Molecule
Awardee: Young-Ah Seo
Institution: University of Michigan
Grant Amount: $66,366
Funding Period: February 1, 2022 - January 31, 2023
Summary:
The overall goal of this project is to develop new therapeutic strategies that can reduce brain iron overload and iron-induced neurodegeneration in BPAN patients. We have identified that a natural small molecule is exceptionally effective at promoting iron transport. We have now found that iron accumulates in the BPAN cell model and that the resulting iron overload can be mitigated by this small molecule. Building on these preliminary results, this proposal will extensively characterize the capacity for a small molecule to mobilize excess iron from inside cells and will test the overarching hypothesis that small molecule-mediated iron mobilization can mitigate neuronal cell death in BPAN cell models and patient-derived primary fibroblast cells. Completion of the proposed research will advance our fundamental understanding of the mechanistic underpinnings of brain iron overload in BPAN and will build a foundation for the potential therapeutic use of small molecule mobilizers of intracellular iron.