Research Approach

The LuMind Research Down Syndrome Foundation’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 clinical work associated with four of the targets.

The Research Development Pipeline

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

In 2017, as part of the newly defined vision, the LuMind RDS Foundation Board of Directors established Four Scientific Research Categories to guide the expansion of the research agenda beginning in 2018: 1) Prevent Alzheimer onset; 2) Improve cognition; 3) Develop gene therapies; and 4) Advance understanding.

 MRI Brain scan

 Prevent Alzheimer onset
  • Encourage industry-led clinical trials of therapeutic approaches
  • Develop necessary resources: assessment scales, registry, other

 Head symbol with internal mechanisms represented as cogs

 Improve cognition
  • Translate Down syndrome-specific approaches to clinical trials
  • Validate Down syndrome-specific cognition targets

 DNA sketch with section being erased by pencil

 Develop gene therapies
  • Silence extra chromosome
  • Restore the right level of specific gene(s)

 Sign pole with When, How, What visible signs

 Advance understanding
  • Discover novel targets (stem cells, sequencing/phenotyping)
  • Improve mouse models to better test novel targets

These Four Scientific Research Categories articulate the future focus of the organization as it builds on the successes of the current research portfolio that has been funded by the foundation since 2004.

The Research Plan

Since the beginning, the Foundation 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, the Foundation 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 four potential therapeutic compounds currently in clinical trials.

An example of the Foundation’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 LuMind RDS’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

LuMind Research Down Syndrome 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.