Cells in our brains die over time, which is a natural part of aging. However, several neurodegenerative diseases, such as Alzheimer’s and Parkinson’s disease, accelerate this process, thereby wreaking havoc on patients by taking away their memory, dramatically altering their personality, and diminishing bodily control. For many of these diseases, no treatments exist that can reverse or even halt disease progression. A cellular protein called tau is a key culprit in many neurodegenerative diseases.
In collaboration with the Rainwater Charitable Foundation, Association for Frontotemporal Degeneration, Alzheimer’s Association, and CurePSP, the Milken Institute Center for Strategic Philanthropy brought together 30 experts in tauopathies and other neurodegenerative diseases to identify priority areas where philanthropic investment could have a tremendous impact on the field.
Support Basic Research
Insufficient understanding of the molecular underpinnings of the tauopathies is a critical barrier to developing novel therapeutic approaches for Alzheimer’s, PSP, CTE, and others. 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.
Focus on Diagnostic Tools
Investing in enhanced tools that will enable early and distinct diagnosis amongst the tauopathies would be game-changing. 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. Minimizing patient heterogeneity in trials will help to illuminate the distinct biological underpinnings of each disease, as well as differences in disease progression and treatment responses to new therapies. Currently most efforts are focused on developing Alzheimer’s diagnostics using imaging modalities. Philanthropic support should build on these efforts to diversify into other diseases and new modalities to provide clinicians with a robust toolkit.
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. Age-matched control (normal) tissue is also necessary to understand the differences between diseased and normal tissue. These samples are used to identify biomarkers, understand progression, and test novel therapeutics. Philanthropists can support local infrastructure within institutions to gather and maintain patient tissues. Additionally, there is great value in supporting researchers to run large-scale analyses on these tissues, which could be used by many labs through openly sharing the resulting data. This approach minimizes the likelihood of researchers running duplicative studies on these limited resources of tissue samples.
Facilitate Data Sharing
Big data often brings big insights in complex diseases. However, sharing and collaboratively using large data sets requires careful data management, curation, and infrastructure. 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. Philanthropists can contribute to this change by including public data-sharing requirements in the funding agreements and, in some cases, providing funding for curation and infrastructure efforts.
Form a Streamlined Collaborative
Neurodegenerative disease clinical trials have shown very low success rates. Pure tauopathies provide a promising avenue to test tau-based therapeutic approaches because they are specifically characterized by tau aggregation rather than a mix of protein aggregates present in other diseases. However, unique challenges are likely because the pure tauopathies are rare diseases. The development of a collaborative initiative in oncology between pharmaceutical companies, the U.S. Food and Drug Administration, the National Institutes of Health, 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.
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During the past decade, hundreds of clinical trials for neurodegenerative disease have been conducted. However, these trials are costly and have not led to disease-modifying therapeutics. Rethinking the standard approach could be extremely impactful.
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