Subscribe to the BioNews newsletter for free

Login
Advanced Search

Search for
BioNews

Like the Progress Educational Trust on Facebook


 


 

Stem cell trial for fatal childhood brain disorder is set to start

02 October 2006

By Laura Goodall

Appeared in BioNews 378

The first trial for a proposed stem cell treatment for Batten's disease is about to begin. Researchers at the Oregon Health and Science University's (OHSU) Doernbecher Children's Hospital, US, plan to treat six children with the rare neurodegenerative disorder by using fetal stem cell transplants.

Children with Batten's disease cannot produce the normal amount of a particular 'lysosomal' enzyme. This enzyme is needed to break down the fat and protein particles in the brain, known as lipopigments, which are toxic when they build up in the body's tissues. 'These materials accumulate and interfere with normal cell and tissue function and ultimately cause cells to die', explains Dr. Robert Steiner, the lead researcher and vice chair of paediatric research at Doernbecher Children's Hospital.

The team plans to take neural stem cells developed from fetal tissue by StemCells Inc of Palo Alto, California, and implant them into the children's brains. The researchers hope that these transplanted cells will develop into specialised cells that will produce the correct amount of the lysosomal enzyme needed by those with Batten's disease. If the clinical safety trial is successful, it may mean that stem cell transplants could one day eliminate the debilitating conditions associated with Batten's, such as blindness, communication problems, worsening seizures, and the progressive loss of body movement.

Batten's disease is a juvenile form of Neuronal Ceroid Lipofuscinosis (NCL), which first appears between the ages of 5-10. There are three other types of NCL: two that begin at four years old or younger, and a very rare form that affects adults. According to the National Institute of Neurological Disorders and Stroke (NINDS), Batten's disease is rare and occurs in just two to four of every 100,000 live births in the US, but it is more commonly found in families that have an inherited gene for the disorder. Death from Batten's disease can occur as young as eight years old, although the majority of patients survive until their late teens or early twenties.

The scientists expect to have treated the first child before the end of 2006 and, if everything goes smoothly, will then start treating the remaining five. 'We will begin with one patient, but we are not going to continue on with other patients until all of the safety protocols evaluated in the first patient have been met', says Dr. Nathan Selden, head of the Division of Pediatric Neurological Surgery at Doernbecher and OHSU's School of Medicine.

The team also plan on following up all six children for a year after they are treated to check for any long-term side effects. Steiner tells us that although he hopes that this trial will provide an insight into a potential treatment option for Batten's disease, he cautiously points out that it is early days yet. 'This trial is just the beginning in a lengthy, ongoing effort to determine a safe and effective means to improve the quality of life of those suffering from NCL', he says.

SOURCES & REFERENCES
New Scientist | 25 September 2006
 
Stem-Cell Trial for Neurodegenerative Pediatric Disease to Get Under Way
Forbes.com | 22 September 2006
 

RELATED ARTICLES FROM THE BIONEWS ARCHIVE

06 February 2012 - by Ruth Retassie 
US company StemCells Inc have received Food and Drug Administration (FDA) authorisation to carry out clinical trials of their treatment for one of the leading causes of blindness in over 55-year-olds...
05 December 2011 - by George Frodsham 
Two separate studies have successfully transplanted neurons into the brains of mice. The transplanted neurons are able to send and receive electrical impulses, and can be used to compensate for faulty brain cells, restoring normal function. Both studies sourced the transplanted neurons from embryos – mouse embryos in one case, human embryonic stem cells were used in the other...
18 July 2011 - by Dr Lux Fatimathas 
US researchers have successfully converted human skin cells directly into brain nerve cells, skipping an intermediate stem cell stage. The new technique has the potential to aid research into neurodegenerative disorders of the brain, such as Parkinson's and Alzheimer's....

29 August 2006 - by Dr Kirsty Horsey 
A group of British scientists, medical researchers and funding organisations has written an open letter to the Times newspaper, saying that patients should be warned that some stem cell therapies being offered by clinics are not 'wonder cures'. Many of the therapies available - at home and...
10 March 2006 - by BioNews 
US researchers are set to test a fetal nerve stem cell treatment for Batten disease, a rare and currently incurable genetic brain disorder. Oregon Health and Science University (OHSU) says the trial aims to test the safety of the approach, since transplants of purified nerve stem cells have never been...

HAVE YOUR SAY
Be the first to have your say.

You need to or  to add comments.

By posting a comment you agree to abide by the BioNews terms and conditions


- click here to enquire about using this story.

Published by the Progress Educational Trust

CROSSING FRONTIERS

Public Conference
London
8 December 2017

Speakers include

Professor Azim Surani

Professor Magdalena Zernicka-Goetz

Professor Robin Lovell-Badge

Sally Cheshire

Professor Guido Pennings

Katherine Littler

Professor Allan Pacey

Dr Sue Avery

Professor Richard Anderson

Dr Elizabeth Garner

Dr Andy Greenfield

Dr Anna Smajdor

Dr Henry Malter

Vivienne Parry

Dr Helen O'Neill

Dr César Palacios-González

Philippa Taylor

Fiona Fox

Sarah Norcross

Sandy Starr


BOOK HERE

Good Fundraising Code

Become a Friend of PET HERE and give the Progress Educational Trust a regular donation