Medical Professional Publications

Innovation and Discovery

(From the September 2016 Issue of MedStat)

Researchers at Nationwide Children’s Hospital Describe New Type of Cancer Therapy

A study conducted at Nationwide Children’s Hospital has found that a new chemotherapy is effective against both pediatric and adult cancers, and that it allows other chemotherapies to more readily reach their targets. Timothy Cripe, MD, PhD, principal investigator in the Center for Childhood Cancer and Blood Diseases in The Research Institute at Nationwide Children’s, is senior author of the study, which was published online in July in the journal Pharmaceutical Research. Researchers found that the novel class of antitumor amphiphilic amines (RCn), and RC16 in particular, is 10 times more effective in harming tumor cells than regular cells.

Read the Pediatrics Nationwide article.

AveXis Receives FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy Type 1

AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, announced in July that the U.S Food and Drug Administration has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough Therapy Designation is based on preliminary clinical results from an ongoing trial conducted in collaboration with The Research Institute at Nationwide Children’s and The Ohio State University. SMA, which causes motor neuron loss and weakness, is the most common genetic cause of infant death, and SMA Type 1 is the most severe form, accounting for approximately 60 percent of cases at birth.

Read the News Room article.

Researchers Use Sequencing Technique to Identify Cause of Congenital Heart Disease in At-Risk Patients

Whole exome sequencing (WES) has the ability to identify disease-causing mutations, contributing to the development of personalized medicine and bridging a crucial gap between scientific knowledge and clinical application. A recent study from , >Vidu Garg, MD, Kim L. McBride, MD, MS, and Peter White, PhD, at Nationwide Children’s Hospital is the first to demonstrate that WES can be applied to clinical testing of congenital heart disease, the most common type of birth defect, affecting approximately 40,000 births per year in the United States. The study was published in Circulation: Cardiovascular Genetics and the types of congenital heart disease affecting patients in the analysis included atrial septal defects (ASD), patent ductus arteriosus (PDA), tetralogy of Fallot (TOF) and pulmonary valve dysplasia.

Read the Pediatrics Nationwide article.

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