Department of Defense Grant: Bone Marrow Failure Investigator-Initiated Research Award

The FY22 BMFRP IIRA will offer two funding levels with different intent:

Funding Level 1 (FL1): To support studies that further develop ideas, expand upon key discoveries, and have the potential to make significant advances in research, patient care, and/or quality of life in the FY22 BMFRP IIRA Focus Areas. IIRA applications may involve basic, translational, and clinically oriented research, including studies in animal models, research with human anatomical substances, and research with human subjects, as well as correlative studies associated with an existing clinical trial; however, FL1 awards may not be used to support a clinical trial. Multidisciplinary collaborations are encouraged.

Funding Level 2 (FL2): To support Investigational New Drug (IND)-enabling efforts. The BMFRP recognizes the scientific and financial challenges associated with advancing promising, potentially life-changing, therapeutic agents from the laboratory to clinical evaluation. Data related to lead compound characterization; formulation and stability; absorption, distribution, metabolism and excretion; dose/response; and toxicology are required before clinical trials can commence. The proposed studies under the FL2 IND-enabling efforts are expected to be empirical in nature, product-driven, and focused on the accumulation of data for a lead therapeutic candidate(s). At least one, and no more than three, lead therapeutic candidates must be named at the time of application submission to meet the intent of the FL2 mechanism. Library screening or drug optimization studies do not meet the intent of FL2. At the end of the period of performance, the cumulative data should be sufficient to submit an IND to the Food and Drug Administration (FDA). The intent of FL2 awards is to perform the necessary evaluation of promising therapies that will lead to clinical trials; however, clinical trials themselves are not supported by this mechanism. FL2 applications must address the FY22 BMFRP Focus Area “Find effective BMF treatments and cures”.

The following are significant features of this award mechanism:

• Impact: Proposed research projects should address a central critical issue or question in BMF disease research or clinical care. High-impact research, if successful, will significantly advance current methods and concepts for the prevention, detection, diagnosis, and/or treatment of BMF diseases.

• Translational Potential: The translational potential of the project should be considered and described. Applications should address how the research will translate findings into prevention strategies and/or a cure for BMF diseases.

• Preliminary Data: Observations that drive a research idea may be derived from laboratory discovery, population-based studies, a clinician’s first-hand knowledge of patients, or anecdotal data. Applications must include preliminary and/or published data that are relevant to the mission of the BMFRP and support the proposed research project. Any unpublished preliminary data provided should originate from the laboratory of the Principal Investigator (PI) or a member(s) of the research team.

• Multidisciplinary Collaborations: Applicants are encouraged, but not required, to form multidisciplinary teams of investigators who bring specific skills that contribute to the successful completion of the project. This can include both intellectual input and research resources (e.g., supplies, reagents, equipment, personnel, services, tissue samples, access to patients or populations).

• Correlative Studies: Applications to FL1 are encouraged to propose correlative studies of open/ongoing or completed clinical trials to better characterize treatment response and provide deeper insights that can be used to develop future clinical trial endpoints or support personalized medicine approaches.

Partnering PI Option: The IIRA encourages applications that include meaningful and productive collaborations between investigators and includes an option for more than one PI. Electing to submit to the partnering PI option does not influence the total direct cost limit as outlined in Section II.D.5, Funding Restrictions. One PI will be identified as the Initiating PI and will be responsible for the majority of the administrative tasks associated with application submission. The other PI will be identified as a Partnering PI. Both PIs should contribute significantly to the development of the proposed research project, including the Project Narrative, Statement of Work (SOW), and other required components. If recommended for funding, each PI will be named to an individual award within the recipient organization. For individual submission requirements for the Initiating and Partnering PI, refer to Section II.D.2, Content and Form of the Application Submission.

The FY22 BMFRP IIRA will offer two funding levels with different intent:

Funding Level 1 (FL1): To support studies that further develop ideas, expand upon key discoveries, and have the potential to make significant advances in research, patient care, and/or quality of life in the FY22 BMFRP IIRA Focus Areas. IIRA applications may involve basic, translational, and clinically oriented research, including studies in animal models, research with human anatomical substances, and research with human subjects, as well as correlative studies associated with an existing clinical trial; however, FL1 awards may not be used to support a clinical trial. Multidisciplinary collaborations are encouraged.

Funding Level 2 (FL2): To support Investigational New Drug (IND)-enabling efforts. The BMFRP recognizes the scientific and financial challenges associated with advancing promising, potentially life-changing, therapeutic agents from the laboratory to clinical evaluation. Data related to lead compound characterization; formulation and stability; absorption, distribution, metabolism and excretion; dose/response; and toxicology are required before clinical trials can commence. The proposed studies under the FL2 IND-enabling efforts are expected to be empirical in nature, product-driven, and focused on the accumulation of data for a lead therapeutic candidate(s). At least one, and no more than three, lead therapeutic candidates must be named at the time of application submission to meet the intent of the FL2 mechanism. Library screening or drug optimization studies do not meet the intent of FL2. At the end of the period of performance, the cumulative data should be sufficient to submit an IND to the Food and Drug Administration (FDA). The intent of FL2 awards is to perform the necessary evaluation of promising therapies that will lead to clinical trials; however, clinical trials themselves are not supported by this mechanism. FL2 applications must address the FY22 BMFRP Focus Area “Find effective BMF treatments and cures”.

The following are significant features of this award mechanism:

• Impact: Proposed research projects should address a central critical issue or question in BMF disease research or clinical care. High-impact research, if successful, will significantly advance current methods and concepts for the prevention, detection, diagnosis, and/or treatment of BMF diseases.

• Translational Potential: The translational potential of the project should be considered and described. Applications should address how the research will translate findings into prevention strategies and/or a cure for BMF diseases.

• Preliminary Data: Observations that drive a research idea may be derived from laboratory discovery, population-based studies, a clinician’s first-hand knowledge of patients, or anecdotal data. Applications must include preliminary and/or published data that are relevant to the mission of the BMFRP and support the proposed research project. Any unpublished preliminary data provided should originate from the laboratory of the Principal Investigator (PI) or a member(s) of the research team.

• Multidisciplinary Collaborations: Applicants are encouraged, but not required, to form multidisciplinary teams of investigators who bring specific skills that contribute to the successful completion of the project. This can include both intellectual input and research resources (e.g., supplies, reagents, equipment, personnel, services, tissue samples, access to patients or populations).

• Correlative Studies: Applications to FL1 are encouraged to propose correlative studies of open/ongoing or completed clinical trials to better characterize treatment response and provide deeper insights that can be used to develop future clinical trial endpoints or support personalized medicine approaches.

Partnering PI Option: The IIRA encourages applications that include meaningful and productive collaborations between investigators and includes an option for more than one PI. Electing to submit to the partnering PI option does not influence the total direct cost limit as outlined in Section II.D.5, Funding Restrictions. One PI will be identified as the Initiating PI and will be responsible for the majority of the administrative tasks associated with application submission. The other PI will be identified as a Partnering PI. Both PIs should contribute significantly to the development of the proposed research project, including the Project Narrative, Statement of Work (SOW), and other required components. If recommended for funding, each PI will be named to an individual award within the recipient organization. For individual submission requirements for the Initiating and Partnering PI, refer to Section II.D.2, Content and Form of the Application Submission.