Blog

15 March 2019

Developing therapies to make my dream come true.

Gail E. Besner, M.D.
H. William Clatworthy Jr., Professor of Surgery
Chief, Department of Pediatric Surgery
Principal Investigator
Center for Perinatal Research

The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio

Despite six decades of research, the morbidity and mortality of necrotizing enterocolitis remain relatively unchanged and this is completely unacceptable. It is critical to our goal of eradicating this devastating disease that a diverse array of stakeholders including parents, physicians, nurses, therapists, dieticians, scientists, and company CEOs get together to share experiences and to develop strategies to prevent and treat it. The SIGNEC annual meetings are tremendously inspiring, and in 2017 Professor Khashu asked everyone there to write a sentence about the disease. I wrote that my dream is to never have to operate on a baby with NEC again. 

It is remarkable how quickly NEC can destroy parts of the bowel, and having to perform surgery on a baby with the knowledge that survival will be challenging is heartbreaking. So, the focus of the Besner Laboratory is to develop potential novel therapeutic strategies for preventing NEC and to make my dream come true. As I explained at SIGNEC in 2017, we have identified a novel delivery strategy that can improve the ability of probiotics to protect the intestines from NEC. The strategy involves the enteral delivery of the probiotic Lactobacullus reuteri in its biofilm state, which dramatically reduces the incidence of NEC after administration of just one single dose. All of the components of this delivery system are Generally Recognized as Safe (GRAS) by the US Food & Drug Administration, making this an exciting potential therapy for NEC that can be translated to the bedside in the near future.

I am one of the Scientific Founders of Scioto Biosciences, a preclinical stage company devoted to the development of innovative therapies based on the delivery of microbiome therapeutics. The Scioto Platform has the potential to enhance efficacy wherever live bacterial therapeutics are used , and as Dr Ravi Patel has written, aside from breast milk, no other therapies have a stronger effect on decreasing the risk of NEC than probiotics. Scioto Biosciences is working closely with the FDA to begin Phase I human clinical trials in 2019.

Further studies from the Besner Laboratory have investigated the administration of stem cells in the prevention of NEC. Recent findings demonstrate that multiple different types of stem cells derived from either amniotic fluid, bone marrow, or the intestine itself, all protect the intestines from NEC with equivalent efficacy. In addition, exosomes (nano-sized particles) secreted from these stem cells are as effective as the stem cells themselves in protecting the intestines from NEC, thus representing a potential non-immunogenic, non-cell-based therapy for NEC in the future.

If you would like more information, please go to the Center for Perinatal Research at Nationwide Children’s; to follow us on Twitter, look up @NCHPedSurg and @nationwidekids. The buttons below will bring up my SIGNEC presentation (as it is a large file, it has been divided into two parts).

Stem Cell and Probiotic Therapy for NEC

Previous blogs

NEC and the chaotic gut microbiome
Dr Christopher Stewart
Faculty Fellow
Institute of Cellular Medicine
Newcastle University

Probiotics and Evidence-based Medicine

Ravi Mangal Patel, MD, MSc
Associate Professor of Pediatrics
Division of Neonatology
Emory University School of Medicine

Light a candle with us on October 15th

Joanne Ferguson
Jennifer Canvasser
Erin Umberger
Simone Rosito

Our best opportunity to reduce judgement calls

Nigel J Hall MRCPCH FRCS PhD
Associate Professor of Paediatric Surgery
University of Southampton
Consultant Paediatric and Neonatal Surgeon
Southampton Children’s Hospital

Defining NEC: beginning to shed light in a dark room.


Steven McElroy, MD
Associate Professor of Pediatrics – Neonatology
University of Iowa

The Big Little Warriors of Brazil

Simone Rossito
Founder
Instituto Pequenos Grandes Guerreiros

Fighting the phantom menace of necrotizing enterocolitis

David J. Hackam, MD, PhD
Chief of Pediatric Surgery and Surgeon-in-Chief
Johns Hopkins Children’s Center

The NEC Biorepository

Misty Good, MD, MS
Assistant Professor of Pediatrics
St Louis Children’s Hospital

 

Conquering NEC, step by step

Dr Minesh Khashu
Founder
Special Interest Group in Necrotizing Enterocolitis