# Exploring autophagy as a target for Alzheimer's Disease

> **NIH NIH R03** · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · 2021 · $169,375

## Abstract

Project Abstract
 Alzheimer’s disease (AD) is a degenerative brain disease that affects more than 5 million Americans and
is the 6th leading cause of death in the United States. There are no known drugs that slow its progression and
given its increasing prevalence, the development of new therapeutic options represents an enormous unmet
clinical need. The discovery of new therapeutic targets and new drugs that can prevent the neurodegeneration
and buildup of neurotoxic protein aggregates, which are the hallmark of AD, are desperately needed. One
emerging pathway that has recently garnered attention is autophagy, a highly conserved catabolic pathway that
is responsible for the degradation and recycling of cellular components ranging from proteins to whole
organelles. There is emerging evidence that autophagic dysfunction is involved in the pathogenesis of AD.
Indeed, buildup of autophagic vesicular structures in neurons is also a hallmark in the progression of the disease.
It is thought that autophagy is responsible for clearing misfolded protein aggregates, and when it is dysfunctional
or can no longer clear the aggregates efficiently enough, neurotoxicity can occur. However, no good drugs exist
that increase autophagy to test this hypothesis and serve as potential new drugs. We are proposing to develop
small molecules to selectively increase the autophagic rate as a novel therapeutic strategy for slowing the
progression of AD. Because protein aggregation is the key phenomenon at the center of AD, we believe that
activating the pathway that clears large protein aggregates could provide a novel therapeutic strategy for this
disease.

## Key facts

- **NIH application ID:** 10194214
- **Project number:** 1R03AG072020-01
- **Recipient organization:** ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- **Principal Investigator:** Michael Block Lazarus
- **Activity code:** R03 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $169,375
- **Award type:** 1
- **Project period:** 2021-04-01 → 2023-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10194214

## Citation

> US National Institutes of Health, RePORTER application 10194214, Exploring autophagy as a target for Alzheimer's Disease (1R03AG072020-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10194214. Licensed CC0.

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