Case Study

Midwest's First Liver Cell Transplant for Baby with Life-Threatening Urea Cycle Disorder Performed at Children's Memorial




Introduction

Considering the severity of the disease, accumulation of neurotoxic ammonia leads to severe nerve and brain damage. Untreated children experience abnormal physical and mental development. Liver transplantation seems to be the sole cure, but is an exceptionally tedious procedure for neonatal and very young patients.

A clinical trial, which was approved by the U.S Food and Drug Administration in 2010, was held to evaluate the efficacy and safety of liver cell transplantation in patients 5 years old and below. The trial involves enrolling 20 patients across 14 centers in United States of America and Canada, following an analysis of interim results based on an ongoing trial in Germany, which involves newborns with UCD.

Midwest's Baby

The baby who was diagnosed with Citrullinemia received an investigational liver cell therapy sponsored by International Biotechnology firm Cytonet. The transplantation was done at the Children's Memorial Hospital in Chicago.

The baby is now undergoing follow-up care at Rady Children's Hospital, San Diego.

Brief steps in liver transplantation

1. Collection of healthy cells from donated livers not suitable for transplantation
2. Gentle isolation of liver cells, complex processing of cells
3. Infusion of cells into portal veins over 6 days

*Risk of portal thrombosis and systemic shunting of liver cells

Potal thrombosis (PVT)

• Portal vein is a large vein in the abdomen which carries blood from the bowel and other abdominal organs to the liver. PVT occurs when the vein gets blocked with blood clot, causing blood to back up in the vein and leading to high pressures in the vein below it.

Systemic shunting

•  Systemic shunting occurs when blood from the abdominal organs bypasses the liver and is instead sent to the systemic circulation. Therefore, toxins and nutrients are not filtered/absorbed.  

Thoughts of the wise...

"A challenge is enrolling patients since UCD only affects about 400 babies born in the United States each year and diagnosis poses several challenges." Dr. Wolfgang Rudinger, CEO and CSO of Cytonet.

"Unfortunately, because of the rarity of this disease, it's non-specific symptoms and because newborn screening panels do not catch all cases, many babies with severe forms of the disease are not diagnosed or the diagnosis comes too late." Bruce Barshop, M.D., Ph.D., professor of pediatrics and co-director of the Biochemical Genetics Laboratory at the University of California.

"We are pleased to be participating in this trial because it has the potential to provide a treatment option for children with a rare and complicated diagnosis for which there are few options now," said Barbara K. Burton, MD, a pediatrician and director of the PKU program at Children's Memorial.

Reference: Midwest's First Liver Cell Transplant for Baby with Life-Threatening Urea Cycle Disorder Performed at Children's Memorial, WEINHEIM, Germany, Dec. 13, 2011 /PRNewswire/

Link:http://www.prnewswire.com/news-releases/midwests-first-liver-cell-transplant-for-baby-with-life-threatening-urea-cycle-disorder-performed-at-childrens-memorial-135509483.html