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Permalink Reply by Jan on May 16, 2009 at 9:42pm

Check out joshualevy.com "Cures for Diabetes". He lists all the trials, what phase they are in, and has links to the research papers. You will be able to go to one web site and get tons of information. Good luck. We did try to get my niece into Cd3 study with Dr. Herald when she was diagnosed at 8, but the criteria at that time was she had to weigh 80 pounds -- an impossibility, as she was more like 50 pounds at the time. If you think it's safe, I say go for it!

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Permalink Reply by Wendi on May 17, 2009 at 7:39am

Thank You. I tried to go to that website, but it has nothing about diabetes on it?
My daughter is 11, she weighs 90 lbs. I sent them an email (Herold) he sent one back, we will see, it's a recent onset, not new onset, so she might qualify.
have you ever looked into faustman? faustmanlab.org. It looks hugely promising and I want to get involved in this. (on the cure front.)

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Permalink Reply by ger on May 17, 2009 at 6:31pm

Here's the website:

http://cureresearch4type1diabetes.blogspot.com/

Faustman is very promising but it's a bit behind other ongoing trials that have pretty solid results.

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Permalink Reply by Wendi on May 18, 2009 at 6:24am

Thank you for the website. It's all very interesting. some of it seems so promising. Our center has the GAD trial but my daughter was too young. By the time it opens up to her age, she will be past eligibility for new onset. but we will try the recent onset at Herold maybe.

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Permalink Reply by Marnie on May 18, 2009 at 8:34am

I also tried to get in and missed the window by weeks. I truly wish I could have been apart of it

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Permalink Reply by Wendi on May 18, 2009 at 9:09am

where do you live and how long since diagnosis? maybe you can try a recent onset trial.
I think though that you could be one of the 1/3 of the patients that receive a placebo....

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Permalink Reply by Sara Noble on May 26, 2009 at 9:38pm

I was diagnosed last December at age 27 and made the decision to participate in the Protege study (http://www.protegediabetes.org/), a clinical trial of the monoclonal antibody teplizumab. I am not familiar with abate, but based on your question and description I would surmise it is similar to teplizumab. Macrogenics, backed/funded at least in part by Eli Lilly, is sponsoring this research. The trial is fairly involved, but I would be glad to give you more insight into my experience. You must make the decision to participate and begin dosing within 90 days of T1 diagnosis, so it's a very short amount of time to make a big decision and take action. I screened for the study in February and completed the first of two total 2-week dosing cycles in March (intravenous drug administered daily at research facility; I am participating at a site in San Antonio, TX, about 75 miles from my home in Austin, TX). I am very confident that I was one of the treatment groups (there is a placebo group in this phase 2 study) given the side effects I experienced, all consistent with other participants. I will go for my second dosing cycle during the first two weeks in September, and will continue blood work and monitoring through March 2011. My research coordinator actually contacted me a few weeks ago because they are trying to gauge participants' willingness/interest in extending the observation period from 2 years to 5 years. I take this as a good sign that the drug has shown some positive results, as the timing of this extension has come along right as the first study participants are nearing the end of their original 2 year commitment. I hope this means many of the first to participate are still experiencing good results/benefits from the drug and the researchers would like to continue to monitor them to see how long those results will go on. I also this link with more information about the study extension: http://clinicaltrials.gov/ct2/show/NCT00870818. I would gladly extend my participation, in the hopes that this will give researchers more data to work with. I will say making the decision to participate is something I really mulled over and struggled with, but in the end I have no regrets and I am glad I decided to participate. As I was doing my own research, looking for others who had participated in this sort of clinical trial, I found one family's account of their experience (thus far) in a teplizumab drug trial very helpful. I encourage you to check out their blog: http://teplizumabdallas.blogspot.com/. I also recommend looking at clinicaltrials.gov for other similar studies. Please feel free to contact me if you would like more information on my experience, I would be glad to share. You can e-mail me at sara.noble@gmail.com.

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Permalink Reply by Wendi on May 27, 2009 at 3:58am

Hi Sara,
I am now looking at the DELAY study at Yale. It is Teplizumab for recent onset, 4-12 months. it is 50/50 that you get the placebo. It is at Yale, 1 hour away. It is for my 11 year old daughter. so the scheduling is somewhat more complicated. same course, I believe, 14 days straight, 30 minute infusion. stay there 1/2 day. I think she will not want to spend her off time during the summer doing this. I am worried about any side effects, and I am worried to take her there every day for 14 days figure out who will take care of my son and then get the placebo.
I think I will email you. I really appreciate your info, I spent an hour on the phone with them yesterday.

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Permalink Reply by Sara Noble on May 29, 2009 at 10:06pm

Hi Wendi,

I would love for you to e-mail me and be glad to answer any questions you might have about my experience. As for the side effects, here's a brief description of what I experienced. Note I did not begin experiencing any side effects until the 3rd or 4th day of dosing, and the doctors and nurses at the research facility warned that everyone will react differently to dosing so even some people in the treatment group might not exhibit side effects. I will add that I will never know for certain if I was in a treatment or placebo group, as the study is double-blind and, unless some odd medical circumstance comes up in the future that requires me to know, I will never find out whether or not I received drug. I can only suspect that I was in a treatment group due to the side effects I experienced, known side effects common to other people who have received teplizumab treatment...

First side effect: Low white blood cell count. In my study, they were doing safety labs (blood draws) every morning to make sure that it would be okay to proceed with that day's dosing. There is a certain type of white blood cell count that goes very low during dosing (around the 4th-6th day of dosing), and then picks back up around the 10th day, and finally returns back to normal after the dosing is over. My count dropped very low (the doctor supervising at my site said it was the lowest count she had seen of the 9 participants they have had up to that point). They advised me to be sure to wash my hands, stay out of crowds, etc., so I would not get sick. I heeded their advise and did not experience any illness. However, I was extremely tired starting on about the 4th day of dosing and did not regain my energy until after dosing had completed. I am not sure if this was related to my blood cell count, or some other factor, but I definitely slept a lot, especially during days 4-9.

Second side effect: Extreme itching, redness, and bumpy rashes, followed by weeks of dead skin peeling. Beginning on about the 3rd or 4th day of dosing, I began experiencing moderate itching on the palms of my hands. I actually did not think much of it and could not see any rash at first. Then, over the next few days, the itching got more severe and tiny bumpy rashes began to appear on my palms, eventually spreading to the tops of my fingers and wrists, armpits, and genitals. Rashes are another known/common side effect so part of the dosing regimen includes daily intravenous doses of Benadryl, but I developed the rashes nevertheless. The itching and rashes progressed throughout the first dosing period, but subsided once I was finished dosing. All of the affected skin did peel, however, and it took weeks for it to all shed off and my skin to return to normal. I will note that I am not generally an allergy-prone person. I have no known food, drug, or environmental allergies and have never developed a rash of any other sort in my memory, so this was a bit of an anomaly for me. The nurse at the site said she had commonly seen the same sort of rash develop on people's torsos, but mine was limited to the areas I mentioned above.

I did not have any other noteworthy side effects. In the disclosure forms, I remember nausea was another common side effect. There was one day I had an upset stomach and total loss of appetite, but it was limited to one afternoon/evening and not really painful or anything. Just not very hungry, more interested in sleeping.

I did have some complications with the PICC line, unrelated to dosing. I do plan to get another PICC line put in for the second dosing cycle, but that was a huge pain. I got the PICC put it the day before my treatment was to begin, and everything was smooth as could be. The local anesthetic worked fine, and I found the whole process kind of interesting. I can describe in more detail if you want, but not sure if that's part of the study you are looking at or not. It's basically a long-term IV with an in and an out port to facilitate blood draws and intravenous dosing. I had mine inserted at a radiologist's office in what was basically a clean room or OR, but you stay awake during the procedure. They had an MRI xray thing hooked up to a live feed so they could see on screen where the PICC line was inside me. I could actually see my heart beating and lungs breathing! Took about 30 minutes to prep everything with me on the table, and only about 3-4 minutes for the doctor to do the actual insertion. Anyway, no problems at all for the first 8 or so days. Then I went in and they could not get blood to draw or saline to push in, and so I had to go back to the radiologist's office where I had the PICC inserted to get it looked at. They determined that I had formed something called a "fibron sheath," (probably spelling it wrong), which the doctor described as a sort of scab that had developed around the tip of the end of the PICC line as my body's way of trying to protect itself from the "foreign object" that had intruded (the line). We discussed a few options, and elected to leave the port/hole in my arm in place, pull out the tube, and replace with a new one. However, they did not give me local anesthetic for the swap and it was very uncomfortable. If you have to get it replaced, I strongly advise that you insist on getting the local anesthetic, even if they tell you it won't be too bad since the hole is already there. Trust me, it IS that bad... They inserted the replacement line and it was too long, so they had to pull it out, cut it, and insert again (double ouch!!). Went back to the research site and everything was in working order again. Day 9 complete. Then I went in for day 10, and again the same issue. Could not draw blood or push saline, so I had to go back to the radiologist again. This was definitely the most discouraged I felt throughout the course of the study. The radiologist had a revised theory--that the fibron sheath had been knocked loose, only to re-attach to the new tube we inserted very quickly. Discussed options once again. We decided to move to the other arm and insert a new PICC line, which would end up in a slightly different spot hopefully safe from the evil little fibron sheath I had come to despise! I was not at all excited to be getting yet another PICC line, but I sucked it up and I guess the 3rd time's a charm because that one worked through the remaining days of dosing. Despite the PICC line complications, I will do it again because it beats getting sticked over and over for blood draws and dosing (at least 2 sticks per day in the research regimen/protocol for my study, more if they can't find a good vein on the first try). Everyone who is participating at the site I'm in (9 total people to date) has chosen to get the PICC line, and I was only the 2nd person to have any complications. The girl with complications before me was able to get everything resolved with the first solution they tried for me.

It sounds like your dosing regimen would be very similar or identical to mine. It was a logisitcal challenge for me as well. I work for a publishing company where we are used to working cross-location and much of my job can be handled via conference call meeting and e-mail/computer, so I chose not to take medical leave from work. My boss was very supportive and allowed me to work remotely, and I just did the best I could to call in for meetings and work during the hours I was stuck at the research site each day. Commuting was not a good option for me, given the schedules and horrendous rush-hour traffic I would have to endure between Austin and San Antonio. Luckily, my parents live in San Antonio so I stayed with them during the dosing. My husband came down to stay with me on the weekends, but I wasn't much fun as I spent all my time sleeping. I do hope you can find someone to help care for your son if you guys choose to participate. The chance of getting placebo in my study was 20%, and then the other 80% of people fell into 3 different dosing groups, each receiving different amounts of the drug. The whole taking off work and sitting in the research facility for hours a day only to get a placebo was definitely something I had to think through, but I ultimately made peace with myself that, even if I got the placebo, I would still feel like I was doing something--taking some sort of action--to help someone somewhere by participating. It is a big commitment, and the prospect of not getting any sort of personal benefit from such a huge investment is hard to swallow, definitely something you will need to come to terms with as I did. I spent a lot of time thinking that through before deciding to participate. I basically decided that, should I not participate because of that risk/fear, I was letting diabetes win and that was even harder for me to swallow. Adding to my doubts, I have always been skeptical of all drugs, and prior to this whole diabetes thing, had always looked to alternative medicine and treatments for any minor ailments I had (have almost never been sick in my life until the last 6 months). If you had told me a year ago that I would choose to participate in an clinical drug trial, I never would have believed it. As I mentioned in my first post, choosing to participate in this research was a very difficult decision but I have no regrets whatsoever.

Hope you are finding success in working through all the adjustments necessary for a happy, healthy life with diabetes. Best wishes to you and your family as you think everything through and figure out whether or not this is right for your daughter. Again, you are more than welcome to e-mail me. I can send you my phone number via e-mail as well if you'd rather chat over the phone.

-Sara
sara.noble@gmail.com.

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Permalink Reply by Scott Strumello on September 24, 2009 at 4:55pm

I have attached the initial clinical trial results for the drug teplizumab, which is the subject of the Protege study. Be aware that while it sounds very promising, it is not without adverse events. For example, in the attached journal entry, the authors conclude that increased doses of teplizumab were associated with greater adverse events without improved efficacy. They also posit that drug may marginate rather than deplete T cells. At the Children With Diabetes Friends for Life Conference, Dr. Harlan from the NIJH wisely pointed out that the anti-cd3 drugs such as teplizumab can also cause recurrent mononucleosis which can potentially increase one's propensity to develop lyphoma. Just realize that like all phase 1 clinical trials, this was done on only a handul of patients. However, on the bright side, the authors in the attached journal submission also believe that c–peptide levels may remain detectable up to 5 years after treatment, although that is conjecture at this point in time.

Attachments:

• teplizumab.pdf, 449 KB

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Permalink Reply by Wendi on September 25, 2009 at 4:07am

Thanks Scott,
I read it. I think for my daughter I am relieved we didn't do it. There is so much in the pipeline and there is so much we don't know about longer term adverse effects....

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