# Injectable Biomaterial for Treating Hypoplastic Left Heart Syndrome

> **NIH NIH R01** · EMORY UNIVERSITY · 2020 · $677,417

## Abstract

Much progress has been made in the last 30 years to improve survival of children with CHD, but
what was once a fatal condition is now a manageable long-term disease. Of these, children with
Hypoplastic Left Heart Syndrome face the worst outcomes as they are left with a single ventricle
(the right ventricle) that ultimately fails. These children are either left with long-term morbidity
such as poor exercise tolerance, decreased executive function, and poor social skills. At the
worst, they are placed on the transplant list where short-term survival in the highest need cohort
is 50%, and grafts have a maximal longevity of roughly 12 years before needing a new heart.
The goal of this study is to provide FDA-enabling data for a combination therapy of two Phase 1
FDA approved treatments; cardiac progenitor cells and decellularized porcine cardiac
extracellular matrix. Cell therapy is promising in children, but similar to adult studies the cells
do not persist for long and are transplanted to a diseased microenvironment. In 3 separate aims,
we will examine the efficacy of this combined therapy in both immunocompromised and
immune-competent rats, followed by large animal pig studies. These data will not only provide
critical data for an FDA IND, but will also shed light on potential physiological mechanisms by
which these therapies act.
With congenital heart disease occurring in nearly 1% of all births and representing the most
common birth defect, the potential impact of this study cannot be understated. Information
gleaned from this study will not only improve the lives of nearly 40,000 children per year and
their families, but it would also generate greater societal impact in terms of economic and
educational outcomes. With both individual therapies under early clinical trials in humans, this
proposal is the next logical step toward enhancing pediatric cell therapy.

## Key facts

- **NIH application ID:** 9842365
- **Project number:** 5R01HL146147-02
- **Recipient organization:** EMORY UNIVERSITY
- **Principal Investigator:** Karen L Christman
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $677,417
- **Award type:** 5
- **Project period:** 2019-01-01 → 2022-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9842365

## Citation

> US National Institutes of Health, RePORTER application 9842365, Injectable Biomaterial for Treating Hypoplastic Left Heart Syndrome (5R01HL146147-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9842365. Licensed CC0.

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