# Patient-Oriented Research in Arginine Deficiency Syndromes

> **NIH NIH K24** · EMORY UNIVERSITY · 2023 · $144,573

## Abstract

This K24 renewal application entitled “Patient-Oriented Research (POR) in Arginine (ARG) Deficiency
Syndromes” seeks 5 years of additional support for protected time to maintain the principle investigator’s (PI)
mentoring activities, promote her career development & provide ongoing momentum to advance the applicant’s
research evaluating ARG therapy in sickle cell disease (SCD) and beyond. ARG is a nutritional supplement &
obligate substrate for the production of nitric oxide, a potent vasodilator that becomes depleted during episodes
of SCD-related pain. The PI & her mentees have completed several randomized, double-blinded, placebo-
controlled trials (RCT) of ARG therapy in children with SCD & pain requiring hospitalization in both the US &
Nigeria. These studies combined found that ARG replacement therapy (ART) has opioid-sparing effects,
improved pain scores & cardiopulmonary function, decreased pain medication use & time-to-crisis-resolution
and was associated with a shorter hospital stay compared to placebo. The PI and colleagues also observed a
dose-dependent impact of ART that improved mitochondrial function & decreased oxidative stress in children
with SCD-pain. With recent NHLBI funding for a multicenter Phase 3 RCT utilizing the Pediatric Emergency Care
Applied Research Network (PECARN), the PI plans to perform the definitive study - Sickle Cell Disease
Treatment with Arginine Therapy (STArT) to determine the efficacy & safety of ART. Pain in SCD is the leading
cause of emergency room (ED) visits & hospitalizations, associated with an increased mortality rate. There are
no current therapies to treat the underlying mechanisms of acute pain, with interventions limited to hydration &
analgesia. The PI hypothesizes that ARG is a safe & inexpensive nutritional supplement with opioid-sparing
effects in children with SCD & pain, with potential value in low-resource environments. The STArT trial will
provide essential data for product development & will ultimately help gain FDA product approval for SCD if
benefits of ART are confirmed. The results of this proposal may ultimately lead to a change in clinical practice
for patients with SCD and targets an NCCIH strategic priority of non-pharmacologic management of pain. The
PI is highly committed to performing POR focused on nutrition science & in growing her mentorship reach to
include a diverse group of undergraduates, medical students, post-doctoral fellows & junior faculty, particularly
women & underrepresented physicians in clinical nutrition research, having mentored over 40 trainees
throughout her career. During the 5-year grant renewal, the candidate will also participate in a number of
leadership & mentorship courses, choosing curricula that will help advance the candidate as an educational
leader & mentor within academic medicine. The environment at Emory University is ideal to foster these goals
and will enhance the career development of the applicant as a mentor champion in integrative...

## Key facts

- **NIH application ID:** 10737718
- **Project number:** 2K24AT009893-06
- **Recipient organization:** EMORY UNIVERSITY
- **Principal Investigator:** Claudia R Morris
- **Activity code:** K24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $144,573
- **Award type:** 2
- **Project period:** 2018-09-24 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10737718

## Citation

> US National Institutes of Health, RePORTER application 10737718, Patient-Oriented Research in Arginine Deficiency Syndromes (2K24AT009893-06). Retrieved via AI Analytics 2026-06-14 from https://api.ai-analytics.org/grant/nih/10737718. Licensed CC0.

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