PROCHYMAL Phase 3

Hi All,

Just wondering what if anything people on the board know about this new Drug.  Is anyone involved in the testing on Phase 3 or 2 would like too hear more about it.

Thanks

Jay

Hi Jay,

I took part in the phase 3 trial and can answer most questions you might have. Basically I am convinced I got the real cells the first randomization and I healed 50-60% in 28 days! My CDAI score dropped 120 points in that same time. After I got re-randomized I did not receive any further benefit. But the benefit from the first randomization lasted 3+ months. Personally, I am on the edge of my seat waiting to see how this one pans out.

It's fairly intense, but very worth it. Have you gone through the screening tests yet? Do you know if you're at a colonoscopy site? (Some sites are required to get videos of the colonoscopy at D0 and D28 for comparison. That's what my site did.) So, I had an EKG, CAT scan, colonoscopy, blood work, urine test... I think that's it, along with CDAI, of course.

If you are eligible, you will have 4 infusions in 2 weeks -- D0, D4, D7, D14. Most of those days you will also complete the IDBQ and do some blood work and urine sample. They premedicate with steroids and Benadryl. The infusion bag and tubing are covered in a brownish wrap so that neither you nor the nurses can see the packaging. The infusion itself is only an hour. But, the cells are stored frozen in a local blood bank. they do not call to start the defrost process until you show up at the office. So, what I found is that from the time I stepped into the infusion room until the beginning of the infusion was about 1 hr 45 min. Then the hour for the infusion and then they add an observation at the end. I believe that was 2 hours. (longer if you show reactions) The entire process was about a 5 hour event. (Thank goodness for great nurses and trial coordinators!)

I didn't feel much during the infusion itself. The first couple of times I had a warming sensation in my chest -- sort of like the contrast dye, but just in the chest. That went away by the 3rd infusion.

Other than that, they use DMSO to preserve the cells, so expect to stink of creamed corn/garlic (different opinions) for a couple days after. It doesn't last long, fortunately! I could never smell it on myself, but my daughter would plug her nose when I came near! I could taste it in my mouth though. It's emitted through the pores, mouth, urine, etc.

If you get the real cells, with what I understand, the response rate is quite high. I felt noticably better after a week or so. It was really an amazing med. I cannot wait to see what happens with it! And, I am so excited to hear another person entering the trial. Where are you located? I drove to Seattle from Portland to be a part of it!

Thanks for all the details. I would be going to Kentucky, about 3 1/2 hours away, but it could be well worth the trip.  That is a colonscopy site, not really looking forward to that part.  They are in the process of reviewing my rather large file so I hope to hear soon.  How long did the relief last?  I've never been in remission or even close to it so anything would be great. There is a follow up study.  If you have improvement and then start having problems within six (I think) months, you can basically go through it again, but it is randomized also.  Again, thanks for the great info.

Jumper -- You asked if I had any idea on the timeline. I don't, unfortunately. I suspect it will be years, despite the fast-track/orphan drug designations. Phase 3a was to enroll (you can check you consent form) about 250 people. Last I heard there were not very many people who had been enrolled. (You can ask that too of your trial coordinator. If you find out the current count, I'd love to know!!) It also sounds like they are planning a Phase 3b trial of another ~250 people. I sure wish they'd do an open label follow-on study! ;-) But they said they have no plans. (Of course, one never knows...) So, it's really about how quickly they can enroll the study patients. The bulk of the study data is captured with in a few months. And, fast-track should guarantee quick results once it is submitted for approval.

I never went into full remission, but I did get much of my life back. The benefit lasted about 3 1/2 months. I went into the second portion of the trial, but after being re-randomized, whatever I received didn't give me any additional benefit. I always wonder how long I would have stayed well had I been able to get all three series of good infusions.

It's definitely worth the 3.5 hr drive! That's what I did and I don't regret it one moment. You get to know the local hotel staff pretty well! ;-)

I'd love to stay updated on your progress!

I found this Guys and Gals and I was prety excited after reading about what is going on with this.

http://www.osiristx.com/products_prochymal.php

Hello all- I am new to this forum but thought I would jump in here. I just started the Phase III Prochymal study in Minneapolis, MN. I go in for my second infusion on this Friday the 28th. I havent seen a change yet in my crohn's disease, but I am sure it takes time to "tackle" three years of Crohn's abuse. I know that I could be receiving the placebo but I thought I would just give it a try cuz nothing else worked. I am so far the only one in the trial here, but I was bugging them for the last 6 months to start ,so i am not suprised. They told me that they hope to enroll 10 patients at this site. Anyways, from what I have read and researched for the last year or so this seems to be the best thing coming up, as far as new treatments go. Seems like the research and technology are finally catching up to this disease. I remember hearing about stem cells like 10 years ago and thinking that they were going to be something that happened when I was an old man. I am only 28 now and when I was diagnosed three years ago at the age of 25 there wasnt even any mention that I know of treating this disease with stem cells. It seems like all of this has kind of sprung up in the last couple years or so. I know that they having been doing research for years, but it seems like its just starting to take shape now. Well, Ill stop my rambling about this but I am just really interested and hopeful in the stem cell technology for this disease. I hope that it works for anybody who has this disease and anybody that has any disease!

Hi northernguy... So good to hear of others in the trial! It's a difficult one to enroll for since the inclusion criteria is so restrictive. The site I was at in Seattle has only enrolled 2. I am not sure how many they are slated to enroll, but they've only added me and one other! Slow going, which depresses me. I wish it would go faster because, since it's on fast-track, once it's submitted for approval it should come out relatively quickly. Then again, one never knows with the FDA!

I could tell a difference in my Crohn's by about Day 10. Day 7 I suspected that I was on the real cells but by Day 10, I'd had a definite drop in CDAI -- maybe 50 points or so. By Day 28 I'd dropped about 130 CDAI points! This is the drug I hold on for...

Well- Had my second infusion today. Went just as well as the first one.I have my day 7 next Tuesday, and then I have to wait another week for the day 14. Don't want to jinx anything but it seems like my constant stomach rumblings have settled down a bit. Hopefully things just get better and better from here on out.

Thanks for the good thoughts sr5599! The smell is a rather odd thing. Seems like you can taste it right away in the back of your throat and then after a while you can kind of smell it coming off of your body. My brother in-law picked me up from the University of Minnesota today and he said that he could almost smell me before I got into the vehicle. Oh well, like you said, Ill take the smell if that what makes me feel better, not a big deal .

I agree with you about this Prochymal treatment being relatively unknown. The only thing that I can imagine is that there arent many people that know about it. Maybe it is a publicity thing too, i dunno! Seems to me like it should be getting more attention than it is though. I just hope that it hurries up and gets approved by the FDA, but I would imagine that it wont happen for like another year and a half or longer. I remember hearing on the company's conference call that they didnt think that it would be approved until the end of 2009. It is actually further along in the Phase III trials for Graft Vs. host Disease I guess. I wonder if it was FDA approved for that indication if the GI docs could perscribe it off- label for us Crohn's patients? Kind of like what they do for other drugs like Imuran. I was on Humira before it was approved for Crohn's, so it wouldnt really be any different than that I guess. Only problem was getting the insurance company to cover it.

Its too bad that you cant continue getting the Prochymal if it helped you out. It seems like that is almost unethical to me that they cant give it to you just cuz you didnt improve in the second arm of the trial. Im sure that it was just cuz you didnt get the real thing on the second arm which sucks. How do you feel about taking the Tysabri?

Hey Northernguy, how are you feeling? More improvement? Sure is amazing how fast it happens on that stuff. Boy, even hearing the end of '09 as a hopeful approval time is happy for me. I fear longer than that.

How do I feel about being on Tysabri? Well... Not great, but I don't have a choice right now (besides the permanent ileostomy which is even harder to swallow knowing that Prochymal helps me). I am at day 10 from my first Tysabri infusion and I *think* I am sensing a positive path. It's hard to know. Sometimes I think I am definitely responding. Other times I feel frustrated and that nothing has changed. I fight to feel like a walking PML bomb. But, I cannot worry about that now. I want to stay intact until Prochymal hits the market! And, if Tysabri helps, then I guess it's a risk I am willing to take.

LOL -- any word on your eligibilty? I hope you get all your documentation set quickly! I know it's hard to sit and wait...

Hey Northernguy, How are you feeling? Hope all is well.

northernguy-- I was told that you are only randomized twice. Once for 603 and once for 610. I had my first set of infusions for 610, saw no benefit and have not gone back for the third. (Not eligible I guess since I started Tysbabri.) Anyway, I asked the trial coordinator if I was randomized again for the third round of infusions. She said no and confirmed with Duke. It sounds like you will qualify for 610, though. It's a 100 point drop, right?

I did continue to improve through the 28 days. Then I pretty much stayed there for 3 more months.