How the PICALM protein affects the blood–brain barrier, nerve cells, and amyloid/tau buildup in Alzheimer’s

PICALM: Role in the pathogenesis and treatment of Alzheimer vascular blood-brain barrier clearance dysfunction, neuronal dysfunction, and amyloid-beta, tau and neurodegenerative disorders

NIH-funded research University of Southern California · NIH-11297571

Researchers are trying to boost a brain protein called PICALM to help the blood–brain barrier remove amyloid and tau and protect people with Alzheimer’s disease.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Southern California NIH-funded
Lab location1 site (Los Angeles, UNITED STATES)
Project IDNIH-11297571 on NIH RePORTER

What this research studies

This project uses new mouse models that lack PICALM in brain blood vessels or in neurons to see how that loss leads to amyloid and tau buildup and neuron damage. The team will track how PICALM controls protein trafficking across the blood–brain barrier and inside neurons, and how its loss increases ER stress and vulnerability to toxic proteins. They will test approaches to raise PICALM levels to restore clearance of harmful proteins and to protect nerve cells. The goal is to identify strategies that could be developed into treatments for people with Alzheimer’s in the future.

Who could benefit from this research

Good fit: People with late‑onset Alzheimer’s disease or those who carry genetic variants that reduce PICALM function would be the most relevant candidates for future PICALM‑targeted treatments.

Not a fit: People without Alzheimer’s disease or whose dementia is driven by non‑amyloid/non‑tau causes are less likely to benefit from PICALM‑focused approaches.

Why it matters

Potential benefit: If successful, this work could lead to therapies that improve removal of amyloid and tau, protect neurons, and slow progression of Alzheimer’s disease.

How similar studies have performed: Genetic and laboratory studies have linked PICALM to Alzheimer’s risk, but therapies that boost PICALM are largely experimental and have not yet been proven in people.

Where this research is happening

Los Angeles, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Alzheimer disease dementiaAlzheimer syndromeAlzheimer's DiseaseAlzheimer's disease pathology
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.