I will see if I can get access to this newsletter. I am sure the company will post whatever results that they can 'make public' as it becomes available.
Remember, this being the first set of trials since 2003 - when Henry Gelsinger unfortunately passed away during a gene therapy trial - the main purpose of these trials is to ensure safety. Of course, that death was related to the nature of the disease (immune deficiency) and the nature of the treatment required to treat that particular disease - however the whole thing gave gene therapy a very bad press and delayed other gene therapy trials for years.
Genetix Pharmaceuticals seems to have established safety - so far - and some efficacy with their first patient few patients. Let's see how this continues to go. Remember also the inital attempts to cure a form of blindness earlier this year, in which the dose given was minute because they were testing only for safety - and yet there were able to see significant improvement - not the full effect because the dose was very small.
Please see the following thread:
http://www.thalassemiapatientsandfriends.com/index.php?topic=1675.0Gene therapy for a rare type of inherited blindness improved the vision of 4 adult patients!!
Success was reported by two separate teams of scientists treating Leber congenital amaurosis (LCA). As reported by the National Post in Canada.
A common cold virus (RPE65) was used to deliver the normal version of the gene into one eye of each patient (the other eye was used as a control). The patients went from almost complete blindness (only being able to detect hand movement) to actually reading lines on an eye chart with the treated eye.
These patients were not expected to improve much - because they already had significant damage and were given only low doses to test for safety of the procedure. Surprisingly, the gene therapy actually provided benefit for them.
This is great news for patients with LCA, gene therapy and anyone with a genetic disorder. I hope that this increases confidence in gene therapy!!
I think that it is very important for us to understand the purpose of these studies - and watch for the ongoing results before we get either too excited or disappointed. Frankly, I think what was said at the Singapore conference was premature and frustrating. Gene therapy needs support and making these comments - not fully understanding the context can be harmful.
If I come accross the CAF article I will let you all know. Things
seem positive right now form what we can all see, there is reason to hope but no one can say for sure.
Best,
Sharmin