One year ago, a consortium of just over 20 international clinician-scientists came together to understand, and ultimately treat and cure, tau-related disorders (tauopathies) including progressive supranuclear palsy (PSP), frontotemporal dementia with parkinsonism (FTD-P) and corticobasal degeneration (CBD). The consortium fosters a community of leading scientists to share their work openly and quickly in order to speed insights and discoveries. Individuals still follow the established tradition of publishing peer-reviewed results in journals, but frequent, informal group meetings allow collaborators to share ideas and results earlier in the process and incorporate these new perspectives into their own work.
The Tau Consortium is studying the tau (MAPT) gene to understand how various changes in the gene lead to neurodegeneration. Investigators have developed cellular and animal models, and have obtained skin cells from patients with tauopathies to create induced pluripotent stem cells (iPS) and postmitotic neurons. These cell and animal models are being tested with existing pharmaceutical compounds and drug libraries. By understanding the biochemical and clinical significance of each step of tau metabolism, the investigators hope to better understand how the protein functions normally and its role in disease. Is abnormal tau a cause of disease or a product of disease? Approaches to slowing or preventing disease may include altering tau levels, preventing the hyperphosphorylation or misfolding of tau, correcting the misfolding once it happens, preventing or clearing out the sticky aggregations, or many other possibilities. One unique aspect of this consortium is how tightly the basic scientists work with clinicians to develop better ways of understanding and treating these illnesses.