# Novel Therapeutic Approaches for NPC Disease

> **NIH NIH R01** · UNIVERSITY OF ILLINOIS AT CHICAGO · 2022 · $582,175

## Abstract

PROJECT SUMMARY/ABSTRACT
Niemann-Pick disease, type C1 (NPC1), is an autosomal recessive, neurovisceral disorder, and patients
typically succumb to complications of the disease in the early adulthood years. The clinical phenotype of NPC1
is broad including both central nervous system and peripheral dysfunction and currently there is no FDA-
approved therapy. The enclosed proposal seeks to develop and understand the mechanism of action of a new
class of peptides to ameliorate cholesterol storage and associated phenotypes of NPC1. Our central approach
is to address the biochemical deficits upstream of the NPC1 protein. First, we will understand how defined
protein and lipid biomarkers respond to peptide treatment. Second, we will perform a DMPK study and
investigate lifespan extension with treatment. Third, we will investigate the mechanism of action by which
these peptides reduce cholesterol storage. To carry out the proposed project, we will leverage our expertise in
mass spectrometry, biochemistry and molecular biology techniques.

## Key facts

- **NIH application ID:** 10520232
- **Project number:** 1R01NS124784-01A1
- **Recipient organization:** UNIVERSITY OF ILLINOIS AT CHICAGO
- **Principal Investigator:** Vinay Aakalu
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $582,175
- **Award type:** 1
- **Project period:** 2022-07-01 → 2027-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10520232

## Citation

> US National Institutes of Health, RePORTER application 10520232, Novel Therapeutic Approaches for NPC Disease (1R01NS124784-01A1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10520232. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*