California - UK collaborative opportunity in translational stem cell research: disease team call for proposals
This call is now closed for applications and is only available for reference purposes.
The Medical Research Council (MRC) has agreed to partner the Californian Institute of Regenerative Medicine (CIRM) in its Disease Team Request for Applications (RFA), which aims to progress preclinincal stem cell research towards clinical testing. The MRC is therefore calling for proposals from UK groups active in the preclinical domain that can link to Californian research teams to establish high quality, multidisciplinary translational research programmes. Funded programmes will be milestone-driven and have the maturity and critical mass to reach the stage of a phase I clinical trial within four years.
- Background
- Remit
- Funding available for invited proposals
- Assessment process
- Terms and conditions of the MRC award
- Intellectual property
- Consortium agreement
- Monitoring Performance
Background
Stem cell research offers enormous potential for the development of novel therapies for the treatment of acute and chronic disease and serious injury. The application of stem cell research to clinical problems is in its infancy, and though few stem cell-based therapies have progressed into clinical testing to date, recent progress has suggested that there are opportunities in a number of areas for translation towards the clinic. Such research is an inherently multidisciplinary process, requiring the combined strengths of academic and clinical researchers, together with the expertise of the biotechnology and pharmaceutical sector. Collaborative arrangements between industry and academic institutions are likely to be needed in order to assemble teams with the requisite complementary expertise to successfully translate promising basic findings into viable therapeutics.
Applications to this call will seek to establish such partnerships, based on scientific excellence and involving teams in both the UK and California, in order to deliver an interdisciplinary programme of translational stem cell research.
Remit
Aim of call: Californian-UK Disease Team Research Awards (CIRM RFA 09-01)
The purpose of the California Institute of Regenerative Medicine (CIRM) Disease Team Research Awards is to accelerate potential therapies based on stem cell research toward clinical testing. To facilitate this goal, CIRM intends to support actively managed multidisciplinary teams engaged in milestone-driven translational research, in partnership with collaborative funding partners. The MRC has agreed to partner CIRM in this effort, and will fund the participation of a limited number of UK groups within this programme. The mission of the programmes funded under this call will be to conduct the necessary research and regulatory activities to prepare and file an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) (or an equivalent body), before Phase I clinical testing can begin.
In addition to MRC, CIRM has agreed to partner three other agencies under this RFA - the State of Victoria, Australia; the Cancer Stem Cell Consortium of Canada; and the Spanish Ministry of Science and Innovation (MICINN). Through this ‘Collaborative Funding Partner’ programme, California-based Principal Investigators (PIs) can collaborate with researchers eligible for funding by any of these agencies, such that if a collaborative funding proposal is approved, CIRM will fund all project work done within the State of California and the partner will fund all project work within its jurisdiction. Thus MRC will fund the UK component of awarded collaborative projects, subject to the available funds.
Funding available for invited proposals
- Under RFA 09-01, CIRM intends to commit up to $210 million to support up to 12 awards. Projects will be funded for up to four years, with justifiable total project costs of up to $20 million per project for California-based activity.
- MRC has made up to £5m available to support the involvement of UK teams in this RFA. It is anticipated that this will fund the UK-based activity in one or two of the top-ranked fundable proposals involving a UK team.
- UK participation will be funded as an MRC award to the eligible UK organisation under standard arrangements. Funding will be provided at 80 per cent FEC
Assessment process
Submission of an application for the CIRM Disease Team Research RFA involves a two-step process. Any qualified PI may submit one brief preliminary application. Applicants submitting the most promising, competitive and responsive proposals will be invited to submit a detailed, full application. The process will be administered by CIRM.
Key assessment criteria will be:
- Project objective, rationale, and significance
- Scientific maturity
- Feasibility of the preclinical research and development plan
- Qualifications of the PI and Co-PI(s)
- Collaborations, resources, and environment
The review will be undertaken by CIRM in association with the MRC and the other collaborative funding partners under an agreed joint process.
Collaborative preliminary applications will be evaluated under the same review standards as CIRM-only Disease Team preliminary applications, and will be neither favoured nor disfavoured in the review process.
Terms and conditions of the MRC award
Standard MRC terms and conditions will apply to the UK component of a collaborative disease team award. These are specified in the applicants’ handbook, and encompass:
- the core Research Councils UK terms and conditions
- the additional MRC-specific terms and conditions relating to medical research
- the MRC supplementary terms and conditions for research grants involving human stem cells.
Further additional terms will also apply to the CIRM-MRC collaborative disease team awards, such that it will be a condition of MRC funding that:
- The consortium partners (CIRM-funded or otherwise) agree to abide by the Code of Practice for the UK Stem Cell Bank and for the Use of Stem Cell Lines. This includes the requirement to deposit any hESC line derived using MRC funds in the UK Stem Cell Bank, and to seek approval from the Steering Committee for the UK Stem Cell Bank for the import, export or use of any hESC lines in the UK.
- The consortium partners (CIRM-funded or otherwise) agree to provide free access to the UK research community to all publication related biomedical mMaterials generated during the course of the project, to provide consistency with the CIRM requirements for California.
Intellectual property
- Intellectual property generated in the course of the collaborative project through MRC funding will be owned by the host UK institution, who will have the right to manage and exploit the project intellectual property.
- Participating UK groups will be bound by the specified CIRM IP provisions in relation to activities within the State of California. UK partners would only be required to comply with the CIRM IP provisions relating to joint inventions when these were directly linked to CIRM funded research.
- MRC wishes to assure itself that the funded UK institutions are able to manage and exploit effectively the intellectual property generated from the MRC-funded research. In agreement with the CIRM position, MRC will reserve march-in rights to ensure that IP generated during the course of the project using MRC funding can be fully exploited for the national benefit and that of the research organisation involved.
Consortium agreement
- The terms of the collaboration funded under the disease team RFA must be determined early in a proposal’s development and relevant agreements put in place by the start of the project. Collaboration arrangements should ensure transparency in the project design and in the analysis and publication of results (including if these are negative). Consideration should also be given to issues such as: relative responsibilities, governance arrangements, indemnity, intellectual property rights, reporting and access to data and samples.
- MRC funding will not be released until an appropriate consortium agreement has been agreed and signed by all collaborative partners, and approved by MRC and CIRM (and any other collaborative funding partner).
Monitoring Performance
- For collaborative disease team awards involving UK groups, MRC and CIRM will be involved with the project PIs in the active management of the projects. In addition to annual Progress Reports, the PIs will be expected to provide 1) quarterly updates; 2) routine communication by the PI or Project Manager; and 3) participation in Evaluation Meetings. MRC and CIRM will have access to all grantee-generated progress and financial reports across the full scope of the project’s work.
- MRC and CIRM will meet periodically to review the progress of the collaboratively funded projects, and funding may be withdrawn should there be a failure in making satisfactory progress. These Evaluation Meetings will typically occur at the key decision points in development projects, at which time go / no go decisions will be made. These decision points occur:
1. between the preclinical research and development phases, where a significant step-up in funding is required, and the decision is made whether to initiate IND enabling activities; and
2. upon completion of critical IND enabling studies, and the team is moving towards a regulatory filing for first-in-man studies.
