Anna Falk

Purpose and goal: There are three main aims of IndiCell: 1)Develop iPS cell-based therapy for two model project, Parkinson’s disease and Macular degeneration 2)Create a development pipeline for iPS cell therapies that includes state-of-the-art workflows addressing generic processes, competence and quality controls 3)Become an important global actor within stem cell therapies Expected results and effects: Development of precision medicine with iPS cell therapy for Parkinson’s disease and Macula degeneration. Increase the speed and number of translations of stem cell therapies from pre-clinical transplantations to patients and identify innovation potential in techniques, processes, devices and knowledge. Put Sweden on the global map of stem cell therapies. Increase the awareness of stem cell therapies among the public. Approach and implementation: IndiCell will be executed by implementation of 6 workpackage where 4 will address the process from donation of cells for reprogramming, enter into a specialized GMP facility via production of iPS cells, therapeutic cells, sorting of cells, quality testing of cellular products and then exit of cells from GMP to bedside and local washing and preparation of cells for transplantation to the patient. 2 work packages which deal with monitoring, defining and creating innovations and one dealing with the project management and actively prepare for the future after the Vinnova funding.

My research group builds cellular models of the human brain using the Nobel Prize-winning reprogramming technique. This technology makes it possible to take a skin cell from a person and then in the lab direct the cell backwards in development to become like an embryonic stem cell. In order to build as similar a model as possible of the human brain, we then use growth factors to direct the stem cells so that they form three-dimensional mini-brains known as organoids. These organoids contain structures and cell types found in the real brain, such as cerebral cortex structure, nerve cells, supporting tissue cells, and neural stem cells. To study how cancer cells build up a brain tumor, it is common for researchers to transplant cancer cells from human tumors into the mouse brain and then follow how the tumor grows and also study how different drugs treat the growth. These are important experiments that in the future may result in new treatments and medicines, the questions I ask in this research project are whether there are alternative methods to these experiments and whether the development of organoids as a model could mimic the animal brain. We plan to create organoids that correspond to the "right" part of the brain to study glioblastoma and medulloblastoma. The goal of this project is to develop and use organoids to replace part of the studies where the researchers transplant tumor cells into animal brains to study tumor growth. We hope to achieve two goals, first to develop these models so that they become useful and thus reduce time, costs and animals. The second goal is to use the organoid models we developed to study medulloblastoma and glioblastoma growth in collaboration with research groups who are experts in cancer research. We expect to answer questions about how important the environment, 3D organization, intrinsic genetic and epigenetic mechanisms are for tumor growth.