Drugs trials and research

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Heidi

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We've heard about a trial for an immunosuppressive drug which could cut down the amount of insulin Ross has to inject. Originally this was going to be happening in London but now we're being offered Sheffield or Bristol.

Two questions -

First, has anyone tried any of these trials and how did they get on?

And secondly, does anyone have any experience of research centres in Sheffield or Bristol?


My feeling was that we'd investigate everything and we were particularly keen when there was the trial in London as that's only an hour from us. Now I'm not so sure...but if it could help....and of course finally the choice is Ross's not mine.
 
Personally I wouldn't be too happy about taking immunosuppressive drugs unless they were absolutely necessary as they can cause all sorts of nasty side efects and make you more susceptible to other illnesses.

A friend of mine had to have a kidney transplant just over 10 yrs ago and as a result of taking the immunosuppressive drugs for so long has now developed a severe problem with her gallbladder which is going to have to be removed.

So unless you really need to take them I would advise against it.
 
Heidi

As far as i know, and i'm not important enough to be let loose on clinical trials yet...🙂, you have to be recommended to a trial based by a consultant, maybe the one running the trial. He has to prove that you are a suitable candidate and might benefit from treatment. You can't just volutenteer at this stage, but you might be able to contact the doctor in charge of the trial and ask for Ross to be considered. The doctor will be trying to prove that the treatment he's testing will be better than the alternative.
The group of selected patients will be split into at least two groups, one who will have the active drug, and ones that will have the alternative treatment(s) or a placebo. You won't know what group you are in, and nor will the doctor. The only person who is likely to know is the pharmacist in charge of the trial or maybe the techs dispensing. But the idea is that if nobody knows, nobody can be swayed by money, sympathy or favouritism.
The drug will either be new or an old drug being used on a new illness (which sounds like what this is) but it will have been tested on people to make sure it's safe, and that the side effects aren't dangerous. Drugs are licensed for particular treatments for particular treatments called indications and it sounds like the doctor in charge is looking for a new license to treat diabetes with immunosuppresants.
As Phil has correctly explained, immunosupressants are usually given to people with transplants to stop them being rejected, but are also given to people with eczema, rheumatoid arthitis, leukaemia and non-hodkins lymphoma and some other cancers. Examples include Tacrolimus, Ciclosporin, Methotrexate and azathioprine. Phil's also correct in saying that the side effects can be nasty, they're designed to suppress your immune system so people talking them have to be very watchful for infections. The list of other side effects go on a bit too, but patients taking these sorts of drugs tend to be under careful supervison by medical staff and have blood tests regularly. Saying that, unfortunaltely, i've seen many children and young people take these drugs regularly, and in some cases, i'm sure, they've been incredibly helpful and indeed, have saved lives.
I've got no experience of hospital services in Bristol or Sheffield, and my knowledge of clinical trial is learnt from observation and being nosey🙂. I trained in the pharmacy at Great Ormond Street, but most hospitals are running a range of trials at any given time. Trials involving children and young people are particularly strictly observed and regulated. I think they have to explain any risks that they are aware of before anybody starts treatment, especially after the incidents in Northwick Park a few years ago (don't worry, that would have been at an ealier stage of the trial process to see if the drug was safe for use in healthy volunteers, if the drug has got to the stage of being offered to patients with a disease then it will have passed that and been declared safe, in this case it's probably been used in other patients for some time already).
If you've got any idea of what drug is involved or would like to know more, you can contact me and if i don't know, i'll ask somebody who does.🙂

Rachel
 
Wow, thank you Rachel!

Phil, I had much the same feelings about the whole idea before I began to look further into this.

The drug concerned is Teplizumab and it would appear that it's been trialled lots of times around the world and in the UK - now it seems they're trying to work out the best regime for the longest coverage.
According to the things I've read this drug prolongs the 'honeymoon' period from a few months after diagnosis to anything up to five or more years.

It sounds interesting and could potentially be something of an improvement for Ross, but (and it's a big but) if he doesn't want to do it once we find out more then we won't. At the moment we're looking at anything and everything.

Thanks
H
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Teplizumab is a new drug to me, but it sounds like Monoclonal antibody like Infliximab which is used in Rheumatoid artritis. The Macrogenics website (they manufacture the stuff for Lilly i think) think it will help protect beta cells in the pancreas from more damage if given to patients who have only just been diagnosed with, or more acurately just developed type 1 diabetes. Like you say, in theory it would reduce the "severity" of diabetes if given early enough, could mean that a really lucky person might lose just a few beta cells, and have a form of diabetes which was somewhere between type 1 and type 2, at least for a while.
"In Phase 2 trials of teplizumab, a single brief course of teplizumab delivered within the first six weeks following diagnosis was shown to improve c-peptide responses, reduce HbA1c levels and reduce insulin requirements for at least two years. This translated into better metabolic control for the treated individuals for the two-year duration of the study. These findings are being further studied in the Prot?g? and Prot?g? Encore clinical trials, described below."
This is from Macrogenics' website (www.macrogenics.com), all the trials they mention are in the states, but i could have a word with the pharmacists who cover the appropraite areas and see if they are running Protege in this country.

Rachel
 
They're running a trial in London for adults and there may be one in either Sheffield or Bristol for children if all the paperwork goes through.

We shall see....

Thanks Rachel

H
x
 
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