SBIR Phase I: Generation of Dopaminergic Neurons from Fat

The broader impact/commercial potential of this Small Business Innovation Research (SBIR) project will be functional neuronal cells derived from human adult adipocytes that will have applications in regenerative medicine. The goal is to develop dopaminergic (DA) neural progenitor cells (NPCs) from transdifferentiated human adult adipocytes using a DA cell induction cocktail. This will have application in cellular therapeutics and research tools for Parkinson's Disease (PD), and other neuronal diseases. In addition, these studies will impact the field of stem cell research and regenerative medicine, since this will be the first demonstration that functional neuronal cells, the main building blocks of brain, spinal cord, and peripheral nervous systems, can be produced from mature fat cells that can be used as cellular therapeutics for several neurological disorders. This SBIR Phase I project proposes to develop new technology for generation of midbrain dopaminergic (DA) neural progenitor cells (NPCs) from adult adipocytes (fat cells), which will used as a platform to develop cellular therapeutics for Parkinson's Disease (PD), and PD research tools. Recently, using a chemical genetics approach (chemical approach or small molecule approach), engraftable midbrain DA neuronal progenitor cells (DA NPCs) from human bone marrow derived mesenchymal stem cells (BM-hMSCs) have been generated. Additionally, DA neuronal progenitor-like cells also had been produced from de-differentiated fat cells (DFAT cells) that have several advantages over BM-hMSCs such as homogeneity of DFAT cell cultures, ease of isolation and low immunogenicity. The goal of Phase I project is to validate and optimize the DA induction protocol for generation of midbrain DA NPC from DAFT cells. Phase II will focus on clinical grade manufacturing of these DA cells and testing their therapeutic effect in several preclinical animal models of PD. Commercial products emerging from Phase I/II work include cellular therapeutics for PD and research tools for PD. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.