Research Approach

DSRTF’s strategic and robust approach, outlined in our Research and Development Pipeline below, has generated results that are recognized as unprecedented in this field, including the identification of the first five potential drug targets and the validation and pre-clinical work associated with three of the targets.

the Research and Development PipelinE

When DSRTF was started it was imperative that funds be invested in the Discovery phase of the Research and Development Pipeline because at the time of DSRTF’s founding there were no biological mechanisms proven to be associated with impaired cognition in Down syndrome. DSRTF’s initial funding investments paid off well, as today there are five such mechanisms identified.

The Research Plan

Since then, DSRTF has employed a strategic approach of identifying and supporting research projects across the entire pipeline that have the greatest promise for accelerating translation to effective new therapies. For example, DSRTF proactively pushed our initial research results down the R&D pipeline by funding investments in projects to validate the initial discoveries and evaluate their potential as near term drug targets. Simultaneously, we initiated discussions with small biotech firms and large pharmaceutical companies to identify whether proprietary drug development candidates already exist. Once again, the results speak for themselves with three potential therapeutic compounds currently in the pre-clinical stage.

An example of DSRTF’s interest in anticipating and addressing obstacles before they impede research progress is the recent development of a battery of tests to evaluate cognitive abilities in individuals with Down syndrome. Without these tests, there would be no way to measure the efficacy of potential treatments when they are ready for clinical trials.

Scientific Advisory Board

The robust nature of DSRTF’s approach to research is exemplified by our world-class Scientific Advisory Board (SAB), which not only evaluates and suggests direction relative to the current research projects, but proactively identifies the areas with the best potential for future investigation. Another objective of the SAB is that it encourages and facilitates collaboration and information exchange among the research institutions.

Our Research Grants Program

DSRTF has developed our Research Grants Policies and Procedures to underscore characteristics that we’d like to see in the research we fund. These characteristics include collaboration, use of interdisciplinary approaches, open and prompt sharing of project information, results and reagents with each other and with other qualified researchers.