It is significant for research organizations to have results in each phase of the pipeline in order to move towards the day when a treatment will be available. Not only can DSRTF point to promising results in each area, we can boast one of the shortest intervals between funding and outcome. Below are some highlights from our results to date. You can also learn more about our approach and DSRTF's Research and Development Pipeline.
Five Potential Drug Targets
The results from the Discovery research phase are the most significant. Before DSRTF’s founding there were no brain mechanisms identified to be associated with cognitive impairment in Down syndrome. By funding work at Stanford University and Johns Hopkins University DSRTF helped to identify five new potential drug targets.
Three Candidate Drugs
The Target Validation and Drug Discovery phase is where the basic discoveries are more rigorously tested and the molecules and compounds that have shown potential in the target validation stage advance to the drug discovery phase, where important aspects such as dosage, efficacy and initial safety assessments are made. The promising results of the Discovery phase have enabled DSRTF to fund a number of projects in this phase, three of which can now be considered "candidate drugs."
One Important Test Battery
The Arizona Cognitive Test Battery or ACTB is a battery of tests developed and validated with funding from DSRTF. These tests will be essential for testing the efficacy of potential treatments in clinical trials. The tests were designed to measure cognitive function associated with three distinct parts of the brain so that drugs targeting these areas can be measured effectively. The tests are currently being enhanced to more closely correlate with tests given to the mouse models of DS so that there will be better predictability of potential efficacy in humans.