Gene therapy clinical trials for LINCL

NYP/Weill Cornell gene therapy clinical trial yields promising results for Batten disease

http://www.eurekalert.org/pub_releases/2008-05/nyph-ncg050908.php

The above link is about a paper* just published in the journal Human Gene Therapy which describes a study that was designed to assess the safety of a new treatment for late infantile NCL, also called late infantile Batten disease, which uses a ‘gene therapy’ vector to surgically deliver a healthy gene into several different areas of the brains of affected children. It has shown that in a very small number of children with late infantile Batten disease, the gene therapy procedure is practical and safe. Perhaps more importantly for families, in some children who received this particular form of gene therapy their disease did not progress as quickly as might be expected. This is obviously an important step forwards in research aiming to find a cure for late infantile Batten disease...

The Batten Disease Family Association is a registered charity which aims to support affected families, promote awareness of and research into the disease. We have therefore prepared a statement with our Medical Advisor to the BDFA, Dr Ruth Williams from the Evelina Children’s Hospital, Guy's and St Thomas' NHS Foundation Trust and Dr Jon Cooper, Institute of Psychiatry, King’s College London who is the chair of our BDFA Scientific Advisory Committee. See our BDFA Statement on LINCL Gene Therapy 13.05.08

* Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA [Worgell et al Human Gene Therapy May 2008]

For updates on this and other developments in Batten Disease research, come and join our discussion meeting on what is being done towards finding therapies for Batten Disease and what can we do to provide a better quality of life for our children? King's College London 17^th June 2008

Further information can be obtained from info@bdfa-uk.org.uk.