Research Progress Announcement – January 2013
Please find a short announcement which has been written by THE CATS FOUNDATION and the research team based in the UK. If you have any questions regarding the below, then please get in touch using the contact us form here.
Research progress announcement
“We would like to calm some rumors regarding the UK study into Tay-Sachs. As many of you may be aware, the study taking place in the US has run into difficulties recently and this will unfortunately delay the programme there (please refer to the NTSAD or Cure Tay-Sachs websites for more information).
However, although gene therapy studies in the UK and US seek to achieve the same goal of a treatment for Tay-Sachs (and also Sandhoff in the UK), the clinical trials themselves will be slightly different at each location. This is due to differences in the planned study design and separate regulations in both regions.
We would like to reassure our supporters that the study in the UK which The CATS Foundation supports is not expected to be affected by the delays in the US. The team is awaiting the results of a funding application to UK government sources (the MRC). The outcome should be known within a few weeks. If granted, the further work on the complex European regulatory documentation will resume so that the differently staged UK clinical trial should be able to start (as planned from the outset) in about two years from initiation of the work.
We would like to thank everyone who has, and continues to support The CATS Foundation. The work we continue to undertake with the research team is taking us a step closer to finding a treatment for Tay-Sachs and Sandhoff disease.”
Daniel Lewi (Charity Director), The CATS Foundation and the research team