# Project-004

> **NIH NIH P01** · INDIANA UNIVERSITY INDIANAPOLIS · 2020 · $2,050,023

## Abstract

Our overall goal is to translate new, small molecule drugs into personalized therapies for severe
asthma and cystic fibrosis (CF). Severe asthma and CF are disabling obstructive lung diseases that have
been a particular focus of our therapeutic development efforts. In our pediatric and internal medicine research
programs, we have developed potentially effective small molecule therapies for both. In many cases, these
therapies are similar or identical in both diseases. For example, S-nitrosoglutathione (GSNO), GSNO-mimetic
compounds and GSNO reductase inhibitors are potentially active in both conditions. However, it is now clear
from our work and that of others that there is substantial clinical heterogeneity in both severe asthma and CF,
and that there are specific patients who will and will not respond to each treatment. As with most drugs,
patients who are non-responders could actually be harmed by unnecessary treatment. We have now
developed noninvasive or minimally invasive biomarker approaches to identify responders and non-responders
to specific treatments. Therefore, we propose in the first phase of this program to use these novel techniques
to identify patients who will and will not likely respond to our proposed therapies. We will study targets and
mechanisms of action for these therapies, including biochemical and epigenetic effects. We will also use these
data to inform studies of interactions between therapies in vitro and in vivo. In the second cycle, we will begin
personalized clinical trials using specific clinical testing and selection criteria developed in the first cycle. Many
severe asthma and CF patients are benefitting already from personalized biological and small molecule
therapies that have recently been developed; our proposed program is meant to complement these novel
therapies in order to optimize the ease and efficacy – and minimize the cost - of treatment for each patient.
Our multidisciplinary program is based strengths in drug development, clinical drug trials, airway redox
chemistry, cell culture, cell physiology and genetics as they relate both to severe asthma and to CF.

## Key facts

- **NIH application ID:** 10011852
- **Project number:** 5P01HL128192-05
- **Recipient organization:** INDIANA UNIVERSITY INDIANAPOLIS
- **Principal Investigator:** Benjamin Gaston
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $2,050,023
- **Award type:** 5
- **Project period:** — → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10011852

## Citation

> US National Institutes of Health, RePORTER application 10011852, Project-004 (5P01HL128192-05). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10011852. Licensed CC0.

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