Medical Professional Publications

Innovation and Discovery

(From the January 2018 Issue of MedStat)

Science Readers Select Gene Therapy Research as "Breakthrough of the Year"

The readers of the journal Science selected gene therapy research conducted at Nationwide Children’s Hospital as the 2017 “Breakthrough of the Year.” The study was led by Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at The Research Institute and published in November by the New England Journal of Medicine. Dr. Mendell and his team have developed a gene therapy to replace the mutated gene responsible for spinal muscular atrophy type (SMA) 1. Children with SMA type 1, who often do not survive past their second birthdays, have such severe muscle weakness that most never hold up their heads on their own. After receiving this gene therapy, some children were sitting up and even walking.

Read more about the research, under “Gene therapy triumph,” and other the winners and runners-up here.

Spinal Anesthesia Appears to be an Effective Alternative to General Anesthesia

A growing number of research studies indicate general anesthesia may have neurotoxic effects on children. In response, urology surgeons and anesthesiologists at Nationwide Children’s Hospital have sought alternatives and found success using spinal anesthesia. V. Rama Jayanthi, MD, chief of the Section of Urology, and Emmett Whitaker, MD, attending anesthesiologist at Nationwide Children’s, say planning and partnership led to effective use for a wide range of urology procedures. They report a high success rate, less need for systemic sedatives and a more efficient operating process in the Journal of Pediatric Urology.

Read more in this Pediatrics Nationwide article

A Promising Regimen for Transplanting Stem Cells from Unrelated Donors to Children with Sickle Cell Disease

The only cure for sickle cell disease is through hematopoietic stem cell transplantation but many children with the disease can’t take advantage because they lack a matched sibling donor who’s disease-free. Physicians at Nationwide Children’s now report that a new medication regimen holds promise for transplanting the stem cells from an unrelated donor. Hemalatha Geetharani Rangarajan, MD, a physician in the Department of Pediatric Hematology, Oncology and Blood & Marrow Transplant, and colleagues found a combination of medications appears to support engraftment while a single dose of the drug cyclophosphamide following transplantation appears to be key to preventing graft versus host disease. Their study is in the journal Biology of Blood and Marrow Transplantation.

Read more in this Pediatrics Nationwide article

Researchers Urge New Fertility Counseling Guidelines for Growing Population of Childhood Cancer Survivors

A Nationwide Children’s survey found that more than three quarters of adult survivors of childhood cancers want to have children of their own but don’t know if they can. And, most say that learning now that they’re infertile would cause marked distress, a study published in the journal Human Reproduction reports. The research team, anchored by Cynthia Gerhardt, PhD, psychologist and director of the Center for Biobehavioral Health in The Research Institute at Nationwide Children’s, calls for new guidelines for fertility counseling and testing early on. Children who would not have survived decades ago are living longer due to better diagnoses and treatments, but cancers and treatments continue to carry high risks for infertility.

Read more in this Research Now article

myChildren’s mobile app

iPhone and Android.

Download Today! »

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000