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Product Development Programs- Bridging the Gap: Early Translational Research Awards Instructions for Applicants


Thursday, August 07, 2014


CPRIT furthers cancer research in Texas by providing financial support for a wide variety of projects relevant to cancer research. 

This Request for Applications (RFA) solicits applications for research projectsaddressing critically important needs related to the diagnosis, prevention, and/or treatment of cancer. The objective of this award is to "bridge the gap" between promising new discoveries achieved in the research lab oratory and commercial development by funding advancement toward investigational new drug (IND) clearance or investigational device exemption (IDE) approval for thetherapeutic, device, or diagnostic assay through activities up to and including preclinical proof-of-principle data that demonstrate applicability to the planned clinical scenario. 

The work funded under this RFA must be deemed sufficiently robust such that successful completion would result in identification of a "lead" compound, assay, or device that, as a next stage, could be taken into full development in compliance with International Conference on Harmonization (ICH) Guidelines and U.S. regulatory guidance documents and regulations. Applicants must identify a clear path of development consistent with the Target Product Profile outlined in the application.

The goal of awards made in response to this RFA is to fund innovative cancerresearch from target identification to "lead candidate" stage, according to a defined Target Product Profile, that projects a clear path to full commercialdevelopment. This award allow s the opportunity to develop proof-of-principle data necessary to bring promising cancer research projects to lead stage in preparation for full commercial development according to Food and Drug Administration (FDA) regulations. Funding may be provided for intermediate steps according to established milestones (often referred to as "stage gates") consistent with those utilized by pharmaceutical/ biotechnology therapeutic, diagnostic, and/or device companies for "target identification to lead"development (i.e., achievement of planned Target Product Profile [Draft Package Insert]) prior to full development activities. The Target Product Profile should include the parameters below; the questions are intended to guide the thinking proc ess and may include, but are not limited to, the examples provided. 
1. Identification of a target that is applicable to human cancer treatment. Is intervention with this target likely to lead to a therapeutic, diagnostic, or medical device that could be useful in the treatment of cancer? 
2. Selection of a lead compound, assay, or device technology based on thetarget. Is the identification of potential developmental candidates based on a set of in vitro tests followed by selection of a lead candidate based on considerations (as appropriate for the candidate) of pharmacodynamic parameters and the results of preclinical, in vivo, proof-of-principle studies in relevant animal models of disease? 
3. Description of a high-level clinical development plan detailing each of theclinical studies the preclinical work is meant to support. Designing thepreclinical program requires an understanding of the duration of the clinical studies required by regulatory authorities. Consequently, a brief outline of each of the Phase I, Phase II, and Phase III studies necessary to obtain regulatory approval and reimbursement funding must be sketched out prior to deciding which toxicology studies would be required. 

Additionally, for therapeutics the following apply : 
- Intended route of administration and dosing regimen. Is the intended route of administration and dosing regimen consistent with accepted convention and medical need for the therapeutic, or will the use of this new agent require a paradigm shift (more frequent or less frequent dosing, new route of administration), and if so, what impact will it have on current standard of care? Optimization of the lead to ensure desired characteristics, including, but not limited to, the following studies: 
1. Absorption, distribution, metabolism, excretion (ADME), including, but not limited to, relevant studies based on route of administration. 
2. Safety (studies as mandated by ICH Guidelines). 
3. Biomarkers (assays) that potentially target specific patient populations forclinical trials. 
4. Biomarkers (assays) that can serve as potential pharmacodynamic markers of clinical activity during early clinical trials designed to demonstrate proof-of-concept. 
5. Proposed current Good Manufacturing Practice (cGMP) (including estimated costs) that can be scalable from Phase I through Phase III. Include information if there are possible plans for formulation. 

Successful applicants should be working in a research environment capable of supporting potentially high impact studies. Access to a clinical environment and interaction with translational cancer physician-scientists are highly desirable. 

Areas of interest include translational preclinical studies that establish proof-of-concept. A detailed preclinical development plan that demonstrates thetranslation of the preclinical work to the eventual clinical studies will be required. 

The current trend strongly favors programs with a strong proof-of-concept that can be undertaken at an acceptable level of risk. Increasingly, this is taken as a clear preclinical indication of a population subset or biomarker approach allowing selection of an enhanced patient population more likely to respond tothe therapy. 

Examples of fundable projects include those that incorporate the study of potential biomarkers of use for the clinic, such as biomarkers for selection of patients (e.g., tumors with mutations in EGFR, DDR2, BRAF) and/or biomarkers that can be utilized as p harmacodynamic end points (e.g., measurement of bone degradation products in preclinical animal studies andearly clinical studies of treatment of bon e metastases), tissue distribution, preliminary stability or other "drugability" criteria or safety pharmacology studies conducted in compliance with ICH Guidelines and, thus, usable in a formal FDA regulatory submission. Applicants who plan to perform IND-enabling studies should document that they have experience and proficiency in doing such studies.

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