# Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis - A prospective evaluation

> **NIH NIH R01** · UNIVERSITY OF CALIFORNIA-IRVINE · 2023 · $960,345

## Abstract

PROJECT SUMMARY
Sporadic inclusion body myositis (sIBM) is a rare disorder of aging Americans, causing asymmetric muscle
weakness and severe disability and morbidity. It is currently untreatable, and poorly understood. The
prevalence of sIBM is likely to increase as the proportion of the United States population above the age of 65
years continues to grow. A major barrier to clinical trials in sIBM has been the lack of full understanding of the
natural history of the disease. It remains to be determined whether the rates of disease progression is uniform
and whether the various biomarkers associated with sIBM (anti-NT5c1A antibodies, variant T-cell populations)
influence the natural history and disease behavior. Given the slow rate of disease progression, such
observations cannot be made in the context of a routine clinical trial, and such studies need to be done as a
separate stand-alone observational study. To address these unmet needs, we propose a prospective study
with four specific aims. Aim 1: To determine for the first time whether c1A antibodies mediate disease
progression over a two year interval in patients with sIBM. Aim 2: To perform a detailed morphological,
histochemical, and immunohistochemical analysis of fresh muscle biopsy specimens obtained from a subset of
patients with sIBM. Aim 3: To characterize the distribution of “immunosenescent” lymphocytes in circulating
blood from patients with sIBM. Aim 4: To quantify the decline in the respiratory function of sIBM patients. The
significance of our proposed study is 1) to allow for a detailed characterization of the disease progression in
sIBM over a two-year period, and 2) to explore the relationship of a number of biomarkers associated with
sIBM, and their influence on disease behavior and disease progression. Upon completion of these aims, we
will 1) understand the disease phenotype, including pattern of respiratory involvement, and disease
progression in sIBM better and understand the influence of serum antibodies to NT5c1A antibodies on the
natural history and disease behavior; 2) define differences in serum variant T-cells and cytokine signatures in
sIBM patients and their influence on disease progression and behavior; and 3) understand muscle pathology
and immune cell distribution in sIBM patients and its relationship to NT5c1A antibodies. These findings may
influence future trial design in sIBM. Finally, we will have created a thirteen-site consortium of myositis
treatment centers that will be ready to adopt quickly any future clinical trials aimed at changing the course of
sIBM.

## Key facts

- **NIH application ID:** 10596154
- **Project number:** 5R01AR078340-03
- **Recipient organization:** UNIVERSITY OF CALIFORNIA-IRVINE
- **Principal Investigator:** TAHSEEN MOZAFFAR
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $960,345
- **Award type:** 5
- **Project period:** 2021-04-01 → 2026-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10596154

## Citation

> US National Institutes of Health, RePORTER application 10596154, Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis - A prospective evaluation (5R01AR078340-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10596154. Licensed CC0.

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