NIH invites applications to develop clinical trials for co-occurring conditions in individuals with Down Syndrome

[Andy]

So the very thing that isn't on offer for ME research apparently is on offer for Down Syndrome.

This funding opportunity announcement (FOA) encourages Exploratory/Developmental Phased Innovation (R61/R33) grant applications to support development of clinical trials to treat critical and co-occurring health conditions in individuals with Down syndrome. The proposed research aims should be milestone-driven. The total project period for an application submitted in response to this FOA may not exceed five years. This FOA provides support for up to two years (R61 phase) for preliminary/developmental/planning studies, followed by possible transition of up to three years of expanded clinical trial support (R33), although the total duration of the award may not exceed five years. This FOA requires measurable R61 milestones.

https://grants.nih.gov/grants/guide/rfa-files/RFA-OD-19-018.html

The overarching emphasis is on the acquisition of knowledge that can be translated into new, enhanced, or tailored therapies for co-occurring conditions in individuals with Down syndrome. Applications should focus on delivering better healthcare outcomes and should seek solutions that are effective and deliverable. Research is needed to develop or test new and innovative adaptations of current drugs, interventions, and therapies for the treatment and management of co-occurring conditions in Down syndrome. These therapies should be implementable and sustainable in clinical settings beyond the research environment. Research applications may address a particular co-occurring condition at a particular stage of development or across the lifespan in people with Down syndrome.

This new research initiative expands many of the research objectives and opportunities previously highlighted in the 2014 Down Syndrome Directions: NIH Research Plan on Down Syndrome. More recent discoveries have enhanced our understanding of chromosome segregation and chromosome silencing, identified certain proteins and neurotropic factors involved in brain development using mouse models, and uncovered the role of interferons in immune dysregulation, each of which have the potential to lead to development of novel therapies for individuals with Down syndrome, as well as broader applications. People with Down syndrome are often excluded from clinical research, such as trials of potentially beneficial drugs and therapeutics that are used to treat the same condition in the general population.

There is great value in connecting people with Down syndrome to therapies that could improve their overall health and quality of life. And there is great interest in the Down syndrome community in participating in clinical research, based on experience from NICHD’s “DS-Connect®: The Down Syndrome Registry,” (https://DSConnect.nih.gov), an online survey tool that introduces individuals with Down syndrome and their families to research opportunities. A comprehensive clinical cohort study with deep phenotyping and exploration of pan-‘omics' will permit identification of biomarkers and outcomes for the co-occurring conditions in Down syndrome. Coupled with development of a clinical trials readiness program, and informed by basic science discoveries, this combination of resources could have a great impact on addressing health disparities that exist for people with Down syndrome and could also lead to the development of therapies to improve outcomes for those with and without the condition.

The purpose of this FOA is to encourage research and increase infrastructure support to address component 3 of the INCLUDE project, namely to promote clinical trials for co-occurring conditions in individuals with Down syndrome. This includes research on the development of technologies (e.g., wearables to monitor biomarkers of health), screening (e.g., effectiveness in typical primary care settings), access and management through new delivery systems (e.g., telehealth), system barriers (e.g., availability of services) as well as behavioral research at the individual patient level (e.g., patient outcomes). Research is needed to develop or test new and innovative adaptations of current drugs, interventions, and therapies, for the treatment and management of co-occurring conditions in Down syndrome. Solutions should be implementable and sustainable in settings beyond the research environment and should have the potential to address disparities in health care for individuals with Down syndrome.

 

[wigglethemouse]

I noticed that there is only one month to submit the applications. Why have such a short open window to submit applications.......... unless this is posted with a particular project winner in mind..........