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:
- Be translational research that helps accelerate the development of therapeutics for neurodegenerative diseases of aging
- Be the development of a therapeutic and/or tool and/or complementary approach
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.
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.
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.
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, firstname.lastname@example.org) to discuss whether your project is in scope.
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 under development for human disease diagnosis, prognosis (including rate of progression), stratification to clinical trials, measuring disease progression, and/or to predict/measure response to therapy (e.g., 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 cognitive, neuropsychological or 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, it must meet the following criteria to be in scope:
- 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).
Institute definitions for biomarkers
1. Description of the biomarker:
A. Specific item(s) or signature to be measured can be defined, for example,
- Disease-specific EEG signature
- Specific brain structure with reduced volume (e.g., MRI analysis)
- Single protein changed (e.g., measured in fluid or by neuroimaging approaches)
- 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 must be previously determined. For example, -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
B. Method: Type of assay or technique that will be used for the detection of the biomarker can be stated clearly.
C. Human population: Disease population or clinically relevant subgroup the biomarker is being developed for use in, OR that you wish to study the biomarker in, can be stated clearly.
D. Type of specimen: The type of specimen (e.g., which fluid or tissue) that will be used for the detection of the biomarker can be stated clearly.
2. Detection: Specific item(s) or signature to be measured has been shown to be detectable in humans, or in the kind of human-derived samples/data that will be tested in the proposed study.
The first time measuring the chosen biomarker in human samples in the population of interest versus controls using the proposed assay.
Carrying out the same work as the initial experiments, except using a different, independent set of well-characterized human samples/data and/or cohort to determine whether it is a sensitive and/or accurate biomarker. Can be done at the same or different research site(s).
- 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.
- Compelling data must exist to justify moving to validation:
- 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.
- Replication studies are not considered to be validation.
- Post mortem tissue can only be used for validation of biomarkers previously identified in living subjects.
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.