GED-0301, an oral antisense DNA oligonucleotide targeting Smad7 mRNA for the treatment of moderate-to-severe Crohn's disease

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Reported today.
http://news.investors.com/technology/102114-722719-celg-stock-nears-high-on-mongersen-data.htm
ISI Group analyst was especially interested in the four-week data, since that gave him a point of comparison with drugs already on the market. At the 160-milligram dose, the remission rate for this period was 72%; for 40 mg it was 70%, while at 10 mg it was 29%. The remission rate for the placebo group was 14%, meaning that unlike in the two-week data, even the lowest dose of Mongersen produced a noticeable improvement.
Click to expand...
 
rollinstone said:
Would definitely like to hear about scope results. Cdai is so unreliable
Click to expand...

The end of this article also talks about CDAI reliability in determining drug effectiveness. Perhaps the upcoming phase III trials will take that into account if Celgene wants this drug to have any reputable traction.

The drug “looks promising,” says Charles Elson, a mucosal immunologist and gastroenterologist at the University of Alabama, Birmingham, who was not involved in the work. He notes, however, that antisense therapy has been “promising for 20 years.” And as often happens, the result “raises more questions than it answers,” he says.

In particular, it's not clear that how people reported feeling matches up with what’s happening inside their intestines—for example, whether ulcers were healing—Elson says. One particular puzzle is that blood levels of C-reactive protein, which often tracks with inflammation, didn’t reliably drift downward in patients who reported feeling better. Monteleone agrees that focusing on a patient’s symptoms isn’t enough: He wants to take biopsies of affected tissue to “understand what is going on” after treatment.
 
From the press release:
" This phase II trial enrolled 166 adult patients with moderate-to-severe Crohn's disease, defined as Crohn's Disease Activity Index (CDAI) ranging from 220 to 400 at least one week prior to enrollment, with documented inflammatory lesions in the terminal ileum and/or right colon. "

" This phase II trial enrolled 166 adult patients with moderate-to-severe Crohn's disease with documented inflammatory lesions in the terminal ileum and/or right colon. Patients with known lesions in the stomach, proximal small intestine, transverse colon, and/or left colon, strictures, fistulae, perianal disease, extraintestinal manifestations, active or recent infections or a history of malignancy were excluded. `

I would like to know the reason behind the exlucison. I have crohn's involving the left side of the colon. Since the drug acts locally, maybe they can do versions of it to reach to the end of the colon.

Edit: Maybe this is their reason: "The disease may affect any part of the GI tract, from the mouth to the anus, but most commonly affects the end of the small bowel (the ileum) and the beginning of the colon."
 
I asked myself the same question regarding drug release location. From the original article posted in the other recent thread: ''the active compound of mongersen is released in the terminal ileum and right (proximal) colon''

unfortunaletly, I think for those of us with left-sided disease, we wont have much chance with this new drug.

im thinking this drug may be good for flare control in ileo-caecum area, but as a long-term maintenance therapy, I am not so sure since it acts only in this very limited location. CD can move around and since the whole colon is not protected with this drug, Im thinking disease could start in another spot, just like what can happen after a surgery?
 
I wouldn't dismiss it just yet for those of us with transverse or left colon involvement. It is often the coating that determines where the drug will dissolve, perhaps they can adjust this with a different coating or blend.
 
Actually, when reading further in the other thread it clearly states that the coating is released upon reaching a certain ph-level that is typical for the ileum and cecum. I'm sure this can be adjusted to reach the whole colon. There's also the possibility of creating an enema to reach the distal colon.
 

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