What We Fund

All funding programs support translational research to accelerate the development of therapeutics for neurodegenerative diseases of aging.

As the Institute defines it, these diseases include:

  • Alzheimer’s disease
  • Dementia with Lewy bodies
  • Frontotemporal dementia
  • Multiple system atrophy
  • Parkinson’s disease
  • Progressive supranuclear palsy
  • Vascular contributions to the above diseases (not stroke-mediated vascular disease)
  • Prodromes to the listed diseases, including
    • Mild cognitive impairment as prodromal to Alzheimer’s disease
    • REM sleep behaviour disorder as prodromal to Parkinson’s disease

Proposed projects may relate to any disease(s) but must have impact on the diseases above and will be adjudicated based on their potential impact on these diseases.

 Projects must meet two conditions to be eligible:

  1. Be translational research that helps accelerate the development of therapeutics for neurodegenerative diseases of aging
  2. Be the development of a therapeutic and/or tool

The Institute defines translational research to be applied research towards developing therapeutics for the prevention and/or treatment of human disease. For example, for small molecule drug development, this includes target validation to Phase II clinical trials. Basic/discovery research, including but not limited to understanding disease mechanisms and discovering genes implicated in disease, is not in scope.

Clinical trials definition:

The Institute defines a clinical trial to be research in which one or more human subjects are prospectively assigned to one or more interventions to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.  A clinical trial sub-study is defined by the Institute to be a study investigating a question not addressed by the main trial, and which may involve obtaining additional measurements and data collection from a sub-group of all participants from the main trial.

Therapeutics definition:

The Institute defines a therapeutic to be a pharmacological approach (including small molecules, biologics, cell therapies and vaccines, including drug repositioning and repurposing), medical device, surgical intervention, or magnetic or electrical brain stimulation. Therapeutics can be for symptomatic relief, disease modification, or prevention.

Identification of novel therapeutics is in scope (e.g., high throughput compound screens); however, identification of novel therapeutic targets, including genes implicated in disease, is not in scope.

Notes about complementary approaches – updated 2019

Based on the success of the 2018 pilot allowing interventions of diet, physical activity, sleep and nutritional supplements, other kinds of “complementary” or “lifestyle” interventions are now eligible. This includes but is not limited to speech therapy, cognitive therapy, music therapy, and social interaction. Projects are eligible for funding through the Rapid Response, Transformational Research, and Early Phase Clinical Trials programs. Applications on these topics are eligible if they meet our disease and project scope criteria above and the following criteria:

  • Includes specific supportive evidence/rationale (published literature or unpublished data) to justify further investigation.
  • Similar experimental design is used to test the approach as would be implemented to test therapeutics, including appropriate control groups.
    • Any interventional trials should address, as best as possible, the potential confound of placebo effect.
  • Measures outcomes relevant to neurodegenerative diseases of aging (as defined by the Institute).
  • Interventions are being investigated in relevant human cohorts and/or appropriate disease models (e.g., cell culture, in silico, or animals).

Examples of projects that are considered out of scope for this program call, but could be considered in scope for future programs:

  • An exercise intervention aimed at reducing obesity, blood pressure and resting heart rate in subjects with subjective cognitive impairment.
  • Testing whether a 12-month paleo diet intervention improves cognition in healthy older subjects.

Examples of projects that are considered in scope:

  • In silico screen of a library of nutritional supplements to select those that reduce amyloid plaques.
  • Testing whether a 12-month paleo diet intervention improves MoCA scores or amyloid deposition in subjects with mild to moderate AD.

If you are interested in applying with a project that incorporates complementary approaches and/or lifestyle interventions, you are encouraged to contact Matthew Sacheli (+1-416-967-7828, matthew.sacheli@westonbrain.org) to discuss whether your project is in scope.

Tools definition: – updated 2018

Tool: An item that accelerates development of therapeutics, e.g., imaging techniques or reagents, biomarkers, and diagnostics.

  • Tools must have direct impact on the translational development of therapeutics (as defined by the Institute, i.e., target validation to phase IIa clinical trials) for neurodegenerative diseases of aging and will be valued only on their ability to do this.
    • Any value the tools contribute to basic research will not be taken into consideration. For example, tools will not be valued for their ability to identify new targets or understand disease mechanisms.
  • Projects covering only the discovery/identification of a tool are out of scope.

Notes about biomarkers

  • Biomarkers must be being developed for human disease diagnosis, prognosis, for patient stratification to clinical trials or to predict response to therapies (surrogate for a clinical endpoint).
    • Biomarkers should measure pathology of the disease (e.g., fluid, imaging or tissue biopsy derived biomarkers) and not be based on behavioural phenotypes (e.g., gait or grip strength).
    • Genetic biomarkers including somatic mutations, SNPs, epigenetics and gene products are in scope if they meet the other eligibility criteria.
  • If the project includes biomarker identification:
    • The project must also include experiments to validate the biomarker.
    • All the samples/data necessary for identification and validation must already be available/collected unless there is sufficient justification to collect new samples/data (e.g., samples cannot be stored).
    • Validation of biomarkers must occur in a well-characterized human subjects/samples/data. This validation must be in samples/data from different subjects than those used to identify the biomarker.
    • Post mortem tissue can only be used for validation of biomarkers previously identified in living subjects.

An identified biomarker is defined by the Institute as one that meets the following 4 conditions:

1.  Specific item(s) or signature to be measured can be defined;

  • For e.g.,
    • Presence of a particular bacterium
    • Disease-specific EEG signature
    • Specific brain structure with reduced volume
    • Single protein increased
    • Precisely defined fingerprint
      • If the biomarker is a fingerprint of a family of proteins or a signature of brain volume changes, the precise fingerprint or signature to be replicated must be previously determined. For e.g., omics studies for the purpose of identifying biomarker patterns or signatures are out of scope.
    • Exact identities of multiple individual factors that may be useful individually or as a specific composite
  • It is not in scope to know that a family of proteins is affected or that brain volume is changed overall, if the specific item or signature that is the biomarker cannot yet be specified. For example, a single protein is not considered an identified biomarker if only the family of proteins were previously identified to be affected

2. In what it will be detected (e.g., which tissue/fluid), using what assay, and for what disease, can be clearly stated;

3. Specific item(s) (or signature) to be measured has been shown to be detectable in humans or human-derived samples/data in the tissue/fluid to be tested;

4. Compelling data exists to justify moving to validation (as defined by the Institute).

  • The most compelling data is likely in humans or human-derived samples/data with a relevant disease
  • The most compelling data will likely allow for a power calculation
  • Data from pathophysiologically relevant animal models could be considered if those animal data are compelling

Biomarker validation is defined by the Institute as:

  • Testing a previously identified biomarker in a sufficient number of appropriate, comparable, well-characterized human subjects/samples/data to determine whether it is a sensitive and/or accurate biomarker.
    • If the proposed assay is different than the one used for initial biomarker identification, or if the assay will be used in a different type of specimen (e.g., different tissue/fluid or different species) then preliminary data must be provided to demonstrate that the assay works appropriately. For example, if a biomarker was identified using an assay in CSF and you are proposing to use the same assay to validate a biomarker in blood, there must be preliminary data demonstrating the assay works in blood.
    • Replication studies are not considered to be validation, e.g., using subjects with a different disease stage, or subjects on different drug regime if that regime could affect the biomarker.

For cognitive assessment tools and clinical assessment instruments

  • If developing a cognitive assessment tool or a clinical assessment instrument, the tool must be being tested in patients with a relevant disease.
  • Requires discussion of why the new assessment would be better than existing ones.

Important things to know about the Institute:

  • We do not fund basic research. We only fund translational research.
  • Funds are provided contingent on meeting milestones. If your project is awarded, funds are provided in tranches as experimental milestones are successfully completed.
  • Our application process is interactive. You will likely receive feedback on your application and may be asked to make modifications.
  • Many projects are declined at the Letter of Intent (LOI) stage. Only ~15% of LOIs are invited to the Proposal phase, so that applicants and reviewers spend their time on Proposals that have an excellent chance of being funded. Proposal funding rates have ranged from 30-100%.
  • We provide more than funding. Our grantees may also benefit from things such as expert advice from our scientific advisors, industry exposure, and networking opportunities.