PET PET
  • My Account
  • Subscribe
Become a Friend Donate
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • My Account
  • Subscribe
  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements
PETBioNewsNewsStem cell trial for fatal childhood brain disorder is set to start

BioNews

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

Published 9 June 2009 posted in News and appears in BioNews 378

Author

Laura Goodall

Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false colour).
CC BY 4.0
Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false-coloured cryogenic scanning electron micrograph).

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...

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.

Related Articles

Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false colour).
CC BY 4.0
Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false-coloured cryogenic scanning electron micrograph).
News
30 January 2013 • 1 minute read

US stem cell company given green light for blindness trials

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...

Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false colour).
CC BY 4.0
Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false-coloured cryogenic scanning electron micrograph).
News
17 January 2013 • 3 minutes read

Brain cells from embryos: two successful mouse implant studies

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...

Image by K Hardy via the Wellcome Collection. Depicts a human embryo at the blastocyst stage (about six days after fertilisation) 'hatching' out of the zona pellucida.
CC BY 4.0
Image by K Hardy via the Wellcome Collection. Depicts a human embryo at the blastocyst stage (about six days after fertilisation) 'hatching' out of the zona pellucida.
News
15 November 2012 • 2 minutes read

Brain cells made from skin

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....

Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false colour).
CC BY 4.0
Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false-coloured cryogenic scanning electron micrograph).
News
9 June 2009 • 2 minutes read

Warning on untested stem cell therapies

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...

Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false colour).
CC BY 4.0
Image by Sílvia Ferreira, Cristina Lopo and Eileen Gentleman via the Wellcome Collection. Depicts a single human stem cell embedded within a porous hydrogel matrix (false-coloured cryogenic scanning electron micrograph).
News
9 June 2009 • 2 minutes read

Nerve stem cell trial for brain disorder planned

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...

Leave a Reply Cancel reply

You must be logged in to post a comment.

« Sperm donor 'crisis' in UK

Data-Label The UK's Leading Supplier Of Medical Labels & Asset Labels

RetiringDentist.co.uk The UK's Leading M&A Company.
easyfundraising
amazon

This month in BioNews

  • Popular
  • Recent
13 June 2022 • 2 minutes read

Drop in diversity of blood stem cells leads to old-age health issues

6 July 2022 • 1 minute read

Frozen embryo transfers linked to high blood pressure in pregnancy

5 July 2022 • 1 minute read

Anorexia in pregnancy linked to increased risk of complications

5 July 2022 • 2 minutes read

Pregnancy after breast cancer treatment does not increase risk of recurrence

5 July 2022 • 1 minute read

No difference between fresh and frozen sperm for IUI

4 July 2022 • 2 minutes read

Shorter IVF protocol reduces risk of OHSS

Subscribe to BioNews and other PET updates for free.

Subscribe
  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856

Subscribe to BioNews and other PET updates for free.

Subscribe
PET PET

PET is an independent charity that improves choices for people affected by infertility and genetic conditions.

  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Navigation

  • About Us
  • Get Involved
  • Donate
  • BioNews
  • Events
  • Engagement
  • Jobs & Opportunities
  • Contact Us

BioNews

  • News
  • Comment
  • Reviews
  • Elsewhere
  • Topics
  • Glossary
  • Newsletters

Other

  • My Account
  • Subscribe

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856