The Annual Review issue of Pediatric Research contains important reviews and original reports focusing on stem cell therapy for the treatment of neonatal lung disease (1), pediatric brain injury (2), congenital anomalies (3), and congenital heart disease (4). The authors describe the potential of these approaches to advance the care of children with life-threatening conditions and those with significant long-term chronic morbidities. The authors also describe the promise, ethical challenges, and limitations of these evolving therapeutic approaches. In considering these important advances, several themes are worthy of consideration, especially as related to potential cell-based therapies in neonates. These themes can be broadly categorized into study design, recruitment, funding and sponsorship, and ethical and regulatory complexities.

A clear understanding of the unique characteristics, cell source, and potential of the cellular therapies under development in each study is important in evaluating these emerging therapies. It is inevitable that clinical trials in children will result in conflicting results due to differences in study populations, cellular therapy administration regimens, and outcome measures. As such, proper description and evaluation of the characteristics of individual clinical trials are critical, especially for neonates, for whom developmental and physiological immaturities impart additional challenges.

Log in or create a free account to read this content

Gain free access to this article, as well as selected content from this journal and more on nature.com

Access through your institution

or

Sign in or create an account
Continue with Google
Continue with ORCiD