$1,000,000 Translational Research Award
FightMND’s research grant schemes seek to fill the critical translational gap between basic, discovery phase research and later-stage drug development by funding promising preclinical drug development projects, and by funding early phase clinical trials.
The grants are open to researchers at all stages in their career, and seek to encourage collaboration and cooperation between MND research labs, biotechnology companies, pharmaceutical companies, and other industry partners around the world in coordinated, goal driven projects aimed directly at the advancement of new therapies for MND.
One of the core values of FightMND is urgency in our pursuit of a cure, and as a consequence the grants are particularly aimed towards applications demonstrating a bench-to-bedside applicability of between 24 to 36 months.
Our Translational Research grant will support post-discovery, pre-clinical development of therapeutics for MND through the support of a wide range of development activities ranging from validation of therapeutic leads through to the submission of investigational new drug and clinical trial applications to regulatory bodies.
Translational research grant - guidelines
Applications are now open
Researchers across Australia and abroad working in translational research relevant to progressing the development of potential therapies for Motor Neurone Disease / Amyotrophic Lateral Sclerosis (MND/ALS) are invited to apply for funding up to AUD $1,000,000 to support their research projects commencing in 2018.
To be eligible to submit a grant application, applicants must be registered to attend the 2018 Australasian MND Symposium
Applications Open: August 1st 2017
Applications Close: December 18th 2017 6.00pm
Applications Review and Scoring: December 19th to March 2018
Grant Awarded and announced: 2018 Australasian MND Symposium
PLEASE NOTE: These grants are listed on the Australian Competitive Grants Register (ACGR) and eligible institutions are therefore eligibe for additional Government funding through the Research Infrastructure Block Grants (RIBG) and Sustainable Research Excellence (SRE) schemes.
- The proposed projects are expected to be product-driven and focused clearly on therapeutics, and it is anticipated that the agents and/or data generated from these projects will lead directly to the advancement of new therapies for MND to roll out into human clinical trials.
- There is a preference for development projects aimed at sporadic MND with the use of appropriate models of disease to justify such application.
- Standards for Preclinical Study Design: All projects should adhere to a core set of standards for rigorous study design and reporting to maximise the reproducibility and translational potential of the preclinical research. An example of such standards is described in Landis, S.C., et al. (2012), A Call for transparent reporting to optimize the predictive value of preclinical research, Nature 490:187-191
- To improve reproducibility and potential translation of animal data into new treatments for patients;
- Pre-clinical animal studies should follow standardised protocols such as those outlined in the Ludolph et al. (2010), Guidelines for preclinical animal research in ALS/MND: A consensus meeting, Amyotrophic Lateral Sclerosis, 11:1-2,38-45
- Projects using multiple preclinical disease models/strains (eg. SOD1, cytoplasmic mis-localised TDP43, C9orf72 etc) to help demonstrate possible replication of effect of treatment and/or improved application of results will be looked upon favourably
- Applications incorporating additional studies in human tissues and/or iPS lines to strengthen animal data are encouraged and will be looked upon favourably.
- Where relevant, applications for projects must include a realistic timeline of future development to phase 1 trial, with projects demonstrating a true bench–to–bedside applicability between 24 – 36 months considered favourably.
- New Therapeutic Development Grants: Supporting post-discovery, pre-clinical development of therapeutics for MND through support of a wide range of development activities ranging from validation of therapeutic leads to the submission of investigational new drug applications to regulatory bodies.
- Applications supported by these grants must begin with identified lead compounds in hand.
- Applications must include any supporting preliminary data relevant to the phases(s) of the preclinical development process covered by the proposed research.
- Priorities for funding consideration include projects incorporating the following:
- Secondary validation of lead compounds obtained from screening or by other means to demonstrate target selectivity and mechanism of action
- Optimisation of potency and pharmacological properties
- Studies on formulation and stability
- In vitro and in vivo efficacy studies, ADME, toxicology, pharmacokinetics and pharmacodynamics studies for the development of pharmacologic agents to the US Investigational New Drug (IND) and Australian TGA CTN application stage.
- Where possible, applications should include details for the design and implementation of full-scale current Good Manufacturing Practice (cGMP) production of the lead compound and the delivery systems for use in advanced preclinical and initial clinical trials.
- Re-purposing : Supporting hypothesis-driven drug repurposing efforts focused on new therapeutics for MND:
- Testing of compounds currently approved for other indications in established animal models of MND.
- Applications incorporating additional studies in human tissues and/or iPS lines to strengthen animal data are encouraged.
- Proposals should be hypothesis driven and drugs chosen for repurposing testing should either
- target a mechanism of action(s) common to both diseases,
- target a new or novel mechanism of action (supported by appropriate preliminary data or evidence)
- The preclinical drug development process may require resources beyond those available at a single organisation. Therefore, these grants are open to investigators participating in synergistic collaborations focused on testing and developing lead agents for the treatment of MND. Collaborations should be dedicated to a single, synergistic preclinical development project or study rather than an additive set of sub-projects (i.e., the combined efforts of the collaboration must provide greater benefit than the sum of individual research initiatives).
- If a collaboration is proposed, letters confirming/supporting the collaboration are required. If the collaboration is multi-organisational, participating organisations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organisational barriers that might interfere with achieving high levels of cooperation. Details on these processes should be included in the application.
- Biotechnology or pharmaceutical companies are encouraged to apply. Whether a biotechnology or pharmaceutical company applies for this mechanism as an individual applicant or as part of a collaboration, the company is expected to leverage its own resources to complement the funding provided by this contract.
- Applicants with limited MND experience are strongly encouraged to collaborate with those having more substantial expertise in MND research and/or MND model systems.
- A full detailed budget for the proposed project is to be included with year-to-year breakdown must be included. The contributions (if applicable) from other funding sources should also be included.
Acronyms and Definitions for applicants
For the purposes of these grants and project funding, the definition of Motor Neurone Disease (MND) includes the following progressive neurological disorders that destroy motor neurones;
- Amyotrophic Lateral Sclerosis (ALS)
- Primary Lateral Sclerosis (PLS)
- Progressive Muscular Atrophy (PMA)
- Progressive Bulbar Palsy
- Pseudobulbar Palsy
Translational Research is defined as research facilitating the transfer or translation of basic knowledge of disease mechanisms gained in the laboratory into the development of new methods for the treatment and/or prevention of MND in humans. Within this scope, research projects may include:
- The pre-clinical development of new treatments and interventions for MND
- Testing the effectiveness of treatments
Administering Organisation: Organisation that will be responsible for administration of the research project, and the receipt and distribution of grant funds. There can be only one Administering Organisation per grant.
Administering Office/Grants Officer: Person responsible for receiving and administering funds from the Administering Organisation.
Applicant: Researcher who is leading the research – the Principal Investigator (PI). Responsible for the overall direction of the project, and leading and supporting Associate/Co-investigators (CI’s). Responsible for completion and lodgement of the application, and responsible for progress and reporting on the project. This person must be an Australian citizen or permanent resident.
Where the project involves multi-site research, the Applicant must obtain written commitment from all Heads of Departments of collaborative partners not within the Administering Organisation, and must assume responsibility for undertaking and completing the activities outlined in the application.
Associate/Co-investigator(s): responsible for carrying out some aspects of the research under the guidance and leadership of the Applicant/Principle Investigator.
Collaboration and Collaborator/Collaborative Partner(s): All people and organisations involved in the research project are considered to be collaborators. Collaboration may be between a combination of disciplines, departments and/or organisations. Includes organisations or individuals that provide specific resources that contribute to the research. Associate/Co-Investigators represent a specific sub-group of collaborators who are directly involved in the conduct of the project, but are not responsible for the direction and progress of the project.
By encouraging collaborative agreements, the Cure for MND Foundation is asking researchers to consider looking beyond their discrete departments and organisations, and to seek out people who may be doing similar research. This may allow for stronger and higher quality research proposals, and reduce research duplication.
Goods & Services Tax: Goods and Services Tax imposed in accordance with the A New Tax System (Goods and Services Tax) Act 1999, and related Acts and Regulations. GST will be paid on top of grant amounts where appropriate. This will be determined by the administering organisations GST status.
- The maximum period of grant funding and performance is 3 years
- The maximum allowable direct cost for the entire period of performance is $1,000,000 AUD. FightMND does not provide indirect costs.
- More cost-effective studies that do not request the full available funding amount are encouraged.
- The applicant may request the entire maximum funding amount for a project that may have a period of performance less than the maximum of 3 years with appropriate justifications.
- Time-lines must be clearly described (Gannt chart format) with clear go/no go milestones.
- Payment structure will be based on achievement of milestones which will be finalised jointly with the investigators and the scientific advisory board if the study is approved for funding. A minimum of 6 monthly progress reports will be required (see table below).
- Continuity of funding year to year will be dependent on a satisfactory comprehensive progress report from the PI and an itemised financial report at the end of each year.
- Regardless of the period of funding proposed, the applicant may not exceed the maximum allowable costs
- The research project can commence immediately as it is awarded and must commence within 6 months of the time of the notification of the award.
- Travel costs of up to $5,000 AUD per year to attend and/or present at scientific/technical meetings are allowed
- Investigators at all academic levels are eligible to submit applications
- The applicant must be the Primary Investigator (PI) and have the lead role in directing the project, as well as:
- The level of contribution and role of the PI and other co-investigators (CIs) must be clearly defined on the application sheet
- The PI must be an Australian citizen or have permanent Australian residential status
- The PI and majority of the undertaking of the activities contained within the grant application must be based in Australia
- A CV must be provided for all investigators.
- Salary for PI or co-investigators will not be supported.
- Capital equipment, depreciation, or maintenance of equipment will not be funded by the foundations grants
- Cash and in-kind co-contributions from applicants will be viewed favourably
- Any other actual or proposed sources of funding to support the project must be disclosed
- FightMND reserves the right of refusal of any project applications that it deems fall outside these criteria
- Successful applicants will be required to present the planned research project/progress to Cure for MND Foundation donors and supporters at a research symposium to be conducted in Melbourne yearly following funding announcements (date to be confirmed).
- It is an expectation of successful applicants that project findings are to be published in appropriate peer-reviewed academic and professional journals with details sent to the foundation.
Funding recipients will be required to submit reports on a regular basis. The reporting schedule is outlined in the following table:
|Progress against pre-determined milestones and/or targets||6-monthly||Every 6 months from receipt of funds|
|Financial Reports (to be included in progress report)||Annually||Every 12 months from the receipt of funds|
|Final Report||Once Only||At project completion|
|Ad hoc reports||As requested by FightMND||On request with a negotiable time frame not greater than six weeks|
Research Symposium Presentation
|Annually (oral presentation)||At commencement of funding and every 12 months from receipt of funds for the performance of the grant.|
Final decisions will be made by the Clinical Trials and Translational Research Advisory Committee (CTAC) and FightMND.
Please ensure that each of the following sections have been completed and labelled in your application:
Cover sheet: available to download by clicking here
Lay description/Summary (1 page max) – for publication on the FightMND website and newsletter if the application is successful.
- Provide background information necessary for readers without scientific or medical training to readily understand the rationale and feasibility of the proposed research project. It should also clearly describe the scientific objective the project is designed to achieve
- Describe the ultimate applicability of the research (in lay terms):
- What type of MND patients (eg. Sporadic vs familial) will it help and how it will help them (eg. symptom control vs. disease progression)
- What are the potential clinical applications, benefits, and risks?
- What is the project time it may take to achieve a patient-related outcome?
- What are the likely contributions of this study in advancing the development of a therapy for MND?
Research proposal for project: (maximum 8 pages, font size 12). Provide a well-developed, well-integrated, and detailed research plan that supports the translational feasibility and promise of the project including:
- Aims of the project – Stating clearly and concisely which hypotheses are being tested, and the applicability of results to the further development of a potential treatment of MND
- Preliminary and supporting data relevant to the phase(s) of the preclinical development as required, such as
- Research plan and timeline:
- Provide a detailed outline of the project for the full period of performance including clear go/no-go milestones and justifications for such
- Please provide an accompanying gantt diagram outlining this proposed timeline
For Animal Studies:
- Explain how and why the animal species, strain, and model(s) being used can address the scientific objectives and, where appropriate, the study’s relevance to sporadic human MND biology.
- Summarise the procedures to be conducted and describe how the study will be controlled.
- To further support the advancement of a particular therapeutic candidate, studies should aim to demonstrate beneficial effects across a range of outcomes including motor or cognitive function, neurophysiology, histopathology, and survival when using in vivo models of MND.
- Describe the randomisation and blinding procedures for the study, and any measures to be taken to minimise the effects of subjective bias during animal treatment and assessment of results. Provide justification if randomisation and/or blinding will not be utilised.
- Provide a sample size estimate for each arm and the method by which it was derived, including power analysis calculations.
- Describe how data will be handled – including rules for stopping data collection, how outliers will be defined and handled, statistical methods for data analysis, and identification of the primary endpoint(s).
- Attach proof of ethics approval for all proposed animal studies.
In studies utilising iPS lines, investigators should:
- Incorporate uniform differentiation protocols to improve reproducibility
- Use cell lines from a sufficient number of participants per group for studies comparing disease and control
- Use genetically matched (isogenic) mutation-corrected lines when applicable (with rigorous quality control including karyotyping) to control for variability due to intrinsic genetic background of subjects
- address the limitations of and issues related to the immature/foetal nature of the derived experimental tissue and how/if these will be addressed.
Applications should include a description of potential pitfalls and problems areas within the scope of the proposed project and present alternative methods and approaches.
References: (maximum 2 pages)
Impact Statement (maximum 1 page):
- Describe how the project will make an important contribution to MND therapeutic development.
- Describe in general terms how the outcomes of the project, if successful, will be translated to the clinic and made available to MND patients.
Transition Plan (3 pages maximum):
- The applicant must demonstrate that they have access to all intellectual property rights necessary for development and commercialisation
- Where possible, the application should describe/discuss the methods and strategies proposed to move the lead compound(s) into the next phase of development (e.g. clinical trials, commercialisation, and/or delivery to the patient population) after successful completion of this project. The transition plan should include components listed below (where relevant)
- The development and/or commercialisation strategy
- Details of the funding strategy to transition to the next level of development and/or commercialisation (e.g. partners, pharma, internal/external funding opportunities to be applied for)
- A schedule and milestones for transitioning to the next phase of development. Please include a gantt chart.
- A risk analysis for cost, schedule, manufacturability, and sustainability moving forward
Budget Justification including a year by year breakdown (maximum 1 page). Budget items can include:
- Salary for team members
- Direct research costs (regents and consumables)
- Travel costs for one team member to attend the ALS/MND International Symposium to present the findings of the project each year for the duration of the grant (maximum $5,000 per year)
Budget items cannot include:
- Salary for chief investigators
- Large equipment items
- Indirect or overhead costs
- Other sources of funding to support the project should be disclosed.
Curriculum vitae of all investigators (maximum 3 pages each). Please include:
- Academic background
- Present and past employment positions
- Awards and Prizes
- Research grants support (past 5 years)
- Peer reviewed publications (do not include publications “in preparation” or “submitted”)
Collaboration Plan (maximum 2 pages)
- Name(s) of the institution(s) where the work will be carried out
- State in which department and which institution this project will primarily be carried out. A letter from the sponsoring institution(s) confirming laboratory space will be provided for the duration of the project is required.
- Describe the specific role(s) of each collaborator in the proposed project.
- If the project involves a multi-organisational collaboration, participating organisations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organisational barriers that might interfere with achieving high levels of cooperation. Details on these processes should be included in the application including all details of intellectual property ownership, and a description of any appropriate intellectual and material property plans amongst collaborating organisations
HOW TO SUBMIT
All applicants are asked to submit their applications electronically (file size not to exceed 2MB) in Word doc or PDF form (size 12 font). The research project grant application should be forwarded via email to Rebecca Sheean, Translational Research Coordinator, at [email protected]
Each grant application will be independently assessed and scored by the Clinical Trials and Translational Research Advisory Committee:
Peer Review: To determine merit, all applications will be evaluated according to the following scored criteria:
Research Strategy and Feasibility 50%
- How strongly the background data presented supports the applicant’s reasoning that the proposed therapeutic approach / lead compound is feasible for advanced preclinical development and the extent to which the study is product-driven.
- How well the preliminary data support use of a previously identified bioactive compound or group of lead compounds (where relevant)
- How well the experimental design, methods, and analyses including statistical analyses support the proposed study outcomes.
- How well the application identifies potential problems and addresses alternative approaches.
For studies involving animal research:
- How well the animal study (or studies) considers the guidelines for working with MND/ALS animal models and how well it is designed to achieve the objectives, including the relevance of the model and endpoints/outcome measures to be used.
- The extent to which the study (or studies) is designed to achieve the following:
- It’s objectives, including the choice of model and endpoints/outcome measures to be used
- reproducible and rigorous results, including controls, sample size estimation, blinding, randomszation, and data handling
- The extent if which the study attempts to improve translation and applicability of animal data into the wider human MND population through the use of multiple animal models/strains (eg cytoplasmic mislocalised TDP43 models, C9orf72, SOD1), human tissue, or iPS lines.
Studies utilising iPS lines to complement and expand on animal data should:
- Address issues related to the immature/foetal nature of the derived experimental tissue and how/if these will be addressed.
- The investigators should provide details of the differentiation protocols used, the number of participant cell lines per group used for studies comparing disease and control, and processes used to control for variability due to intrinsic genetic background of subjects.
Impact and Transition Potential 25%:
Potential therapeutic impact as well as steps towards clinical translation of the projected study outcomes should be clearly outlined in the Impact/Transition statements within the application.
- The extent to which this study might be expected to impact the development of therapeutics for MND. The application should assess both short-term and long-term impact.
- How the proposed research, if successful, will contribute to a pathway toward making a clinical impact for individuals with, or at risk for, MND, even if clinical impact is not an immediate outcome.
- Whether the identified next level of development and/or commercialisation is realistic.
- Whether the schedule and milestones for bringing the therapeutic compound to the next level of development (next-phase clinical trials, transition to industry, delivery to the patient market, incorporation into clinical practice, or approval by the TGA) are achievable.
- Whether the funding strategy described to bring the compound to the next level of development (e.g., specific potential industry partners, specific funding opportunities to be applied for) is reasonable and realistic.
- Whether the potential risk analysis for cost, schedule, manufacturability, and sustainability is realistic and reasonable.
Personnel and Budget 25%
- How appropriate the research team members’ backgrounds and expertise are for development of the proposed product and conduct of the proposed research;
- How the levels of effort are appropriate for successful conduct of the proposed work.
- Whether the proposed collaborations are established and/or achievable (supporting letters provided)
- How well each collaborating individual’s contribution is described.
- The degree to which the proposed working relationship described in the Collaboration Plan contributes to successful attainment of project objectives.
- How well the application identifies intellectual property ownership, and describes any appropriate intellectual and material property plan among participating organisations (if applicable).
- Whether the applicant has demonstrated that they have access to all intellectual property rights necessary for development and commercialisation.
- Whether the budget is appropriate for the proposed research and within the limitations of this funding opportunity.
In addition, the following criteria will also contribute to the overall evaluation of the application:
- The appropriateness of the scientific environment for the proposed research;
- Whether the research requirements are supported adequately by the availability of and accessibility to facilities and resources (including collaborative arrangements).
- The quality and extent of organisational support.
- To what extent the writing, clarity, and presentation of the application components influence the review.
All applicants are asked to submit their applications electronically (file size not to exceed 5MB) in Word doc or PDF form (size 12 font). The research project grant application should be forwarded via email to Dr Rebecca Sheean, Translational Research Coordinator, at [email protected]
Privacy, Justice and Support
PRIVACY AND CONFIDENTIALITY
All information contained in applications forwarded to FightMND will be regarded as confidential. Documents containing personal information will be handled and protected in accordance with the provisions of the Privacy and Data Protection Act 2014 (Vic). Personal information will only be disclosed with the permission of the individual to whom it relates, or where the Act allows.
Applicants consent to the information supplied as part of their application being disclosed for the purposes of the evaluation and administration of the grant. Such disclosure includes but is not limited to members of the Clinical Trials and Translational Research Advisory Committee (CTAC), independent readers/assessors requested by the CTAC to provide advice on the applications, the FightMND Board, and relevant employees of FightMND involved in the research grant process.
Applicants acknowledge that announcement of the funded grants will involve a dissemination of information to the public about the general nature of the funded grants.
CONFLICT OF INTEREST – CTAC MEMBERS
FightMND requires its evaluation committee (CTAC) members to act in an ethical manner, declare conflicts of interest, and withdraw from considering applications where such conflict does or may exist.
ACKNOWLEDGEMENT OF SUPPORT
Successful applicants are required to acknowledge FightMND in any publications, public announcements, media, and scientific meeting presentations or discussion forums pertaining to research conducted. FightMND logos, materials, and images can be supplied for this purpose if required.
Intellectual Property Policy
As a charity, FightMND is obliged to ensure that the outcomes of its funded research are applied for the public benefit. In some circumstances, this obligation may be best achieved through the protection of intellectual property resulting from the research and the facilitation of commercial exploitation of this intellectual property.
The term “intellectual property” (IP) describes any work or invention that results from original creative thought. IP falls into different categories:
- Copyright – protects written, dramatic and artistic work, software, films, sound recordings and broadcasts
- Patents – protects technical inventions, novel products or processes
- Trademarks – distinguish the goods and services of one organisation from another
- Design rights – protects the visual appearance of products
Some of these protections need to be registered (trademarks, patents) and some do not (copyright, design rights). If the IP is not protected, another individual or organisation may copy the design or commercialise and sell the invention without consent or payment.
Therefore, for grants where FightMND funding may lead to the generation of new intellectual property, the following additional conditions shall apply:
- Any intellectual property developed during the course of conducting research supported by FightMND translational project grant awards under this agreement (Project IP) shall be owned by the Administering Institution.
- The Administering Institution must comply with the National Principles of Intellectual Property Management for Publicly Funded Research (http://www.arc.gov.au/national-principles-intellectual-property-management-publicly-funded-research) by having in place strategies, policies, and procedures for the identification, protection, management, and exploitation of intellectual property, including that resulting from funding by charities.
- The institution should ensure that all persons in receipt of funding from FightMND, or working on funded activity (including employees, students, visiting staff and sub-contractors), are employed or retained on terms that vest in the institution all intellectual property arising from funding by the Foundation.
- The institution and the grant holders are bound to notify FightMND promptly in writing when new Project IP arises from the grant.
- Within 21 days of receiving the notification from the Administering Institution, and prior to the Administering Institution applying for registration of any Commercial IP, FightMND will advise the Administering Institution in writing which one of the following financial arrangements will apply in relation to commercialisation of the Commercial IP:
- All of the costs associated with commercialising of the Commercial IP (including patent and legal costs) will be paid by the Administering Institution. Out of any net proceeds received by the Administering Institution from commercialising the Commercial IP (after all of the Administering Institution’s costs associated with commercialising the Commercial IP have first been deducted), the Administering Institution will pay 10% of all net commercialisation proceeds to FightMND until such time as FightMND has received an amount equal to the amount of the Project Grant funding provided under this agreement multiplied by five (5).
- Ten per cent of the costs associated with commercialising the Commercial IP (including patent and legal costs) will be paid by FightMND as and when the costs fall due, and the remaining 90% of the commercialisation costs will be paid by the Administering Institution. Out of any net proceeds received by the Administering Institution from commercialising the Commercial IP (after all the Administering Institution’s costs associated with commercialising the Commercial IP have first been deducted and FightMND’s costs have been reimbursed), the Administering Institution will pay 10% of all net commercialisation proceeds to FightMND in perpetuity.
- FightMND will not seek any payment from the net commercialisation proceeds arising from commercialisation of the Commercial IP.
- If the Administering Institution does not wish to protect, manage or exploit the IP, or fails to comply with the agreed strategy, FightMND may direct the Administering Institution to take steps to protect the IP at the Administering Institution’s expense or to transfer the IP to FightMND.
- If the Administering Institution wishes to use any third party (other than its recognised technology transfer company) to carry out its obligations with respect to IP, it must provide details to, and obtain prior written approval from, FightMND.