I dont know the answer but biologically it seems that there is more than just the down regulating of the lymphocytes going on inside these boys bodies @George1972
Hi George,
The Dr Voit interview is like going to the well. One keeps drawing up new and vital pieces of the jigsaw puzzle that is DMD and the mechanism of action for ATL1102. I do have a theory on what is happening biologically that crystallised from one of the draws out of that well. Lets start with the conundrum that you outlined;
the mechanics of muscle turning into fat and how, to date, that is a one way process!!! [Voit] goes on to say that “if something comes along to prove that is not the case, that would be a real COUP”
So, if its a one way process .. well how did the MRI data for ATL1102 show a decrease in the fat fraction %???
We can understand that stopping the inflammatory cascade could stabilise the fat fraction percentage but it beggars understanding as to how the administration of an anti-CD49d therapy could result in a decrease in the fat fraction or in other words an increase in functional muscle mass. You see that should only be possible if the boys could produce dystrophin.
Here's the thing. Does an increase in functional muscle mass imply that a level of dystrophin production becomes biologically active following the administration of ATL1102? Professor Voit makes the point that boys that are missing exon 44 for instance, are able to produce minute amounts of dystrophin. The dystrophin, however, normally get broken down by the inflammatory action in the muscle. I haven't been able to dig deep into the literature to discover whether all boys are capable of producing some level/type of dystrophin whether its a minute level, as in the case of the missing exon 44, or a truncated version. Its interesting that in the case of eteplirsen for instance, the drug does only produce a minute level of dystrophin and in the case of SRP-9001, a micro-dystrophin that is in fact a smaller version of the dystrophin gene that carries specific functional regions. Did some of the boys in the Phase II trial have the missing exon 44 or did some of the boys have a truncated version of dystrophin based upon what the gene was able to read?
I don't have the answers but do note that, according to the PPMD website, Duchenne occurs because there is not enough dystrophin protein in the muscle cells or the dystrophin protein does not work correctly. At first glance that is helpful to my line of questioning but its not a validation from a scientific publication.
In short I ask the question:
Does the mechanism of action for ATL1102 include the protection of minute levels of dystrophin or a version of micro-dystrophin that become biologically active in exerting a functional outcome?
- Forums
- ASX - By Stock
- PER
- ATL1102 US Patent - very interesting
ATL1102 US Patent - very interesting, page-11
-
-
- There are more pages in this discussion • 34 more messages in this thread...
You’re viewing a single post only. To view the entire thread just sign in or Join Now (FREE)
Featured News
Add PER (ASX) to my watchlist
(20min delay)
|
|||||
Last
13.5¢ |
Change
0.005(3.85%) |
Mkt cap ! $121.7M |
Open | High | Low | Value | Volume |
13.5¢ | 13.5¢ | 13.0¢ | $378.0K | 2.903M |
Buyers (Bids)
No. | Vol. | Price($) |
---|---|---|
1 | 5000 | 13.0¢ |
Sellers (Offers)
Price($) | Vol. | No. |
---|---|---|
13.5¢ | 516956 | 4 |
View Market Depth
No. | Vol. | Price($) |
---|---|---|
1 | 5000 | 0.130 |
4 | 1441013 | 0.125 |
7 | 89909 | 0.120 |
4 | 1061892 | 0.115 |
5 | 322225 | 0.110 |
Price($) | Vol. | No. |
---|---|---|
0.135 | 516956 | 4 |
0.140 | 564703 | 9 |
0.145 | 162235 | 7 |
0.150 | 993514 | 9 |
0.155 | 150261 | 3 |
Last trade - 16.10pm 15/10/2024 (20 minute delay) ? |
Featured News
PER (ASX) Chart |
The Watchlist
JBY
JAMES BAY MINERALS LIMITED
Andrew Dornan, Executive Director
Andrew Dornan
Executive Director
SPONSORED BY The Market Online