DEFEND-1 reports as a negative trial


Terribly disappointing.

Just got this news from our study doctor. So sad for you & Samantha. We will keep searching for anything to make life better for people with T1.

Don’t lose hope…I’m sure other researches will come…

I’m not sad about this at all. I am long past the honeymoon stage, so even if I got the drug it didn’t benefit me. I always wondered about the single dosing thing. Seems to me that it would be more effective with a maintenance scheduled of infusions like in the adjuvant cancer setting.

However, from the phase 2 data, there were a subset of people who did truly benefit. So for other people, especially younger people (eg sam) there could be a marked benefit.

I think you’re right, there may still be benefits. Tolerx says Defend-1 didn’t meet the primary endpoint, which was preserving beta cell function. A secondary endpoint, I guess, would be improving control and reducing insulin need. That may still happen. Plus the benefit of being proactive and realizing that there may be something in the pipeline that will have an even better outcome. (Still a little hard to hear the news.)

yeah, but that secondary endpoint will not get FDA approval. Not for a drug that is pry going to cost, i’m guessing here based on chemo and this being an orphan drug, $10,000-25,000 for an 8 day infusion.