Stem-Cell Transplants to Treat Batten Disease
Related News: Spinal / Nervous / BrainOregon Health & Science University's (OHSU) Doernbecher Children's Hospital is getting ready to start a phase I trial to test the safety of stem cell transplants to treat Batten disease, and not necessarily whether it works.
In this trial, six children with symptoms of the disease will be given transplants of stem cells at two different doses and followed for a year.
The stem cells will be surgically implanted into three areas of the brain.
In addition to the stem cells the children will be placed on drugs to help prevent rejection of the cells.
"Batten disease, known as neuronal ceroid lipofuscinosis (NCL), is a rare, inherited neurodegenerative disorder. Children with the condition suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate."
Children eligible for the study are the ones that
- have clinical symptoms of one of two types of NCL: infantile or late infantile NCL;
- and loss-of-function mutations in either the CLN1 or CLN2 gene.
The children will be monitored with standardized measures of development, cognition, behavior and language for 1 year following transplantation.
Dr. Robert D. Steiner, vice chairman of pediatric research and head of the Division of Metabolism at Doernbecher Children's Hospital, said that the hope behind the new research is that the stem cells will evolve into cells that start producing the missing or defective lysosomal enzyme that causes the disease, as was the case in animal studies.
Steiner is also a professor of pediatrics and molecular and medical genetics at Oregon Health and Science University School of Medicine.
Posted on October 17, 2006 11:18 PM