Stem cell therapy for preventing neonatal diseases in the 21st century: Current understanding and challenges
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Updated: Jan 25, 2026

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Published on: April 29, 2007
Christopher R Nitkin1, Johnson Rajasingh2, Courtney Pisano3
1Division of Neonatology, Department of Pediatrics, Children's Mercy Kansas City, Kansas City, MO, USA.
Insights
Mesenchymal stromal cell therapy shows promise for preterm infant diseases like bronchopulmonary dysplasia. Further research is needed to understand mechanisms and optimize treatment for better neonatal outcomes.
Area of Science:
- Neonatal Medicine
- Regenerative Medicine
- Cellular Therapy
Background:
- Preterm newborn diseases, including bronchopulmonary dysplasia and necrotizing enterocolitis, cause significant infant mortality and morbidity.
- Current therapies offer limited benefits, necessitating novel treatment strategies.
- Stem cell therapy presents a promising avenue for neonatal diseases, with encouraging preclinical data.
Purpose of the Study:
- To review the potential of mesenchymal stromal cells (MSCs) in treating neonatal diseases.
- To highlight the promises and challenges associated with the clinical translation of MSC therapy.
- To identify key areas for future research in neonatal stem cell therapeutics.
Main Methods:
- Review of current literature on stem cell therapy for neonatal diseases, focusing on mesenchymal stromal cells.
- Analysis of preclinical findings and ongoing clinical trials.
- Discussion of mechanistic insights, therapeutic optimization, and translational challenges.
Main Results:
- Mesenchymal stromal cells demonstrate potential therapeutic effects through anti-inflammatory and pro-angiogenic mechanisms.
- Significant knowledge gaps exist regarding optimal therapy type (cellular vs. secretomes), timing, and patient selection.
- The interaction between stem cell therapy and standard care is largely unexplored.
Conclusions:
- Mesenchymal stromal cell therapy holds considerable promise for preventing and treating major neonatal diseases.
- Elucidating mechanisms, optimizing therapeutic parameters, and understanding interactions with standard care are critical for successful clinical translation.
- Further research is essential to harness the full potential of stem cell therapy for improving neonatal health outcomes.
Abstract:
Diseases of the preterm newborn such as bronchopulmonary dysplasia, necrotizing enterocolitis, cerebral palsy, and hypoxic-ischemic encephalopathy continue to be major causes of infant mortality and long-term morbidity. Effective therapies for the prevention or treatment for these conditions are still lacking as recent clinical trials have shown modest or no benefit. Stem cell therapy is rapidly emerging as a novel therapeutic tool for several neonatal diseases with encouraging pre-clinical results that hold promise for clinical translation. However, there are a number of unanswered questions and facets to the development of stem cell therapy as a clinical intervention. There is much work to be done to fully elucidate the mechanisms by which stem cell therapy is effective (e.g., anti-inflammatory versus pro-angiogenic), identifying important paracrine mediators, and determining the timing and type of therapy (e.g., cellular versus secretomes), as well as patient characteristics that are ideal. Importantly, the interaction between stem cell therapy and current, standard-of-care interventions is nearly completely unknown. In this review, we will focus predominantly on the use of mesenchymal stromal cells for neonatal diseases, highlighting the promises and challenges in clinical translation towards preventing neonatal diseases in the 21st century.
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