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VCID: Conduct clinical research informed by basic research findings (Milestone 4.U)

In Progress

Timeline Start - End

2020 - 2027

Research Implementation Area

Translational Tools, Infrastructure, and Capabilities

AD-Related Dementias Focus

Incorporate VCID mechanisms derived from basic science animal/human studies into the design of human trials targeting prevention or treatment of dementia/mild cognitive impairment.

Success Criteria

  • At least one new VCID clinical project or trial informed by basic VCID science research outcomes (e.g. biomarkers that emerge from basic studies) and basic mechanisms.

Summary of Key Accomplishments

A strong body of evidence suggests that individuals who have vascular brain damage (e.g., white matter damage) as well as individuals who have had a stroke are at increased risk of cognitive decline and dementia. In addition, building on a rich body of basic research on blood clotting mechanisms and proteins, researchers developed a drug that protects rat and mouse brains from brain bleeds during ischemic stroke as well as later white matter damage. The drug, known as “3K3A-APC”, was recently shown in an NINDS-supported Phase 2 clinical trial (RHAPSODY) to be safe in humans and protect from brain bleeds due to ischemic stroke. The drug manufacturer is planning to launch a Phase 3 trial shortly focused on ischemic stroke treatment. Future findings from controlled clinical trials on 3K3A-APC could include data on cognitive and dementia-related outcomes, illustrating a promising basic science to bedside story of a potential treatment of human brain vascular disease.

The key accomplishments summary is current as of July 2022.

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