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TARGETING TAU—OUR HOPE TO SOLVING THE DEMENTIA CRISIS. In our systems-based review of the biomedical landscape supporting research of a cellular protein called tau—a common culprit in a number of neurodegenerative diseases—we make the case that focused strategic investment in tau research and the category of neurodegenerative diseases called tauopathies, could have a transformative impact on the landscape of neurodegenerative diseases writ large.

We have identified specific areas where carefully targeted funding, particularly philanthropic capital, could have an outsized impact on the field.

 

Overview

Tauopathies are a class of neurodegenerative diseases associated with the aggregation of tau protein. These diseases include Alzheimer’s disease, Chronic Traumatic Encephalopathy (CTE), Progressive Supranuclear Palsy (PSP), and more than 20 others including several forms of fronto-temporal dementia. Although the individual tauopathies are typically studied in terms of each unique disease, this report discusses the diseases and their underlying science as a whole because the tau protein appears to play an integral role across all of them.

Tauopathies appear to share common mechanisms that lead to cell death; however, the symptoms experienced by patients vary and have led to a range of distinct diagnoses. Primary symptoms are classified according to the physiological system affected and include motor, cognitive, behavioral, and language.

Diagnosis

Across the tauopathies, no single test can determine the diagnosis or the specific type of tauopathy driving symptoms. Instead, clinicians use a variety of assessment tools such as a physical examination, laboratory tests, and medical history to make a diagnosis.

Symptomatic Interventions

At present, no disease-modifying therapies for the pure tauopathies exist. Researchers are working to develop effective therapeutic agents, which include approaches such as decreasing the amount of tau within the brain.

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Key Unmet Needs Impeding Tauopathy Research Progress

Tauopathies are an extremely complex class of brain disorders that are difficult to study, diagnose, and treat. In collaboration with the Rainwater Charitable Foundation, the Milken Institute Center for Strategic Philanthropy convened world-renowned tauopathy and neurodegeneration experts to discuss the state of the science and the greatest hurdles to research and therapeutic development. The goal of this retreat was to identify impactful and actionable solutions where philanthropic investment could accelerate progress in tauopathy research.

In total, five key barriers were identified and then used to frame the most impactful solutions.

  1. Support basic research. It is imperative to invest in basic research that will fill in knowledge gaps as to how tau mediates neurodegeneration, which could lead to the identification of new therapeutic targets.

  2. Focus on diagnostic tools. Although there are no cures or disease modifying treatments for tauopathies, there are interventions that help to slow or mitigate symptoms. Furthermore, early and accurate diagnosis would improve stratification of patients into clinical trials such that there is a higher certainty that the patient has the specific tauopathy under investigation in the study.

  3. Increase access to patient samples. In order to understand the range and variation of disease, researchers need access to both postmortem patient brain tissue, as well as biospecimens from living patients.

  4. Facilitate data sharing. As neurodegenerative disease research continues to move toward big data approaches and multicenter collaborations, data sharing will be necessary and will lead to reduced duplication of efforts.

  5. Form a streamlined collaborative. The development of a collaborative initiative in oncology between pharmaceutical companies, the U.S. Food and Drug Administration (FDA), the National Institutes of Health (NIH), and relevant nonprofits to standardize and centralize clinical trials led to streamlining and reduced cost. A similar effort could provide transformative change for neurodegenerative diseases and could be facilitated through philanthropic support.

This Giving Smarter Guide was developed with the express purpose of empowering patients, supporters, and stakeholders to make strategic, informed decisions when directing their energy and philanthropic investments into research and development efforts aligned with their interests.