So many drug trials but what will be the first

[Andy]

Just wondered what everyones thoughts were about all the trials that are going on world wide at the moment and any opinions on what will be the first to actually be a treatment and if so when ?

Exon skipping
Myostatin inhibbitor
Calpain inhibbitor
Albuterol
Stem cell

etc etc

[bhopeful]

I have had high hopes for some kind of Calpain inhibitor like Myodur for the past few years. I think this would be a good intrerim therapy which could possibly replace steroids; however CepTor, the company that is taking Myodur to trial is in financial trouble and may not be able to begin trials as soon as planned (if ever).

[David Feder]

Hi Andy: I am not optimist now. I don't know if the researchers will conclude this trials in few time to use for this generation of patients. Albuterol, myostatin inhibitor, calpain inhibitor and exxon skipping - palliative therapy. I saw only advance to mdx mice.

[Uli]

What is with exon-skipping? They are quite far advanced in the Netherlands, even double-skipping seems possible....

Uli

[David Feder]

Uli:
Exon-skipping can delay the evolution of disease; change DMD in BMD. But the research don't demonstrate that it's possible to delivery oligonucleotides to all muscles and fix all muscle cells.
My disappointment is the slowness of research. For example I send an e-mail to company that discovery pirfenidone (news 14/June) 2 or 3 years ago asking about the possibility of study this drug in DMD. They don't send any answer and only now there is a first study with this drug. To complete the human study of this drug they need more 5 or 10 years ahead. They don't had interesting of more studies of this drug? NO! They problem is that need protect and control all studies because they want receive money....much money when the drug is approved. Probably there are others drugs that could help but few studies now.
David

[bhopeful]

Hi David: I tend to agree with you. I too have contacted drug companies with questions about a drug they were working on for DMD and never received any answers. They will tell you at the conferences that they really want to help the boys with DMD and BMD, but in reality, it's money that drives the train.
Researchers around the world have become very good at treating and sometimes curing mice with DMD, but none of this gets translated into humans.
There is certainly no sense of urgency in the research community or various government agencies in speeding up the process of getting some kind of therapy to our boys.
We keep getting promises but no results.
I pray that somewhere in the world, there is someone who might have the answer and who doesn't care about making money off of us and our boys.
I truly believe that we could have a treatment/cure within one year if all the researchers sat down together and worked on this collectively without any government interference and without any greed in their minds as to who will be the first to discover a cure.
We can only hope and pray that this will happen.

[Uli]

Hi David,

that doesn't sound good. I just hope that there is a major breakthrough smewhere....

Uli

[jog999]

i agree with you all but believe that the mdex consortium in the uk is the way foward because they are scientiest working and trying to prove a therapy in people this could come in time for this generarion

[Andy]

Things are happening slower than perhaps we would like but i believe the safety process is so important. As one specialist said in a speach i saw "its the wanting to do no wrong"

I was recently involved in the myostatin trial and found the results encouraging for the future. There were some problems with dosing amounts on the trial and side effects , if these things were not discovered during trials the patients could be in real trouble ( as if things weren't bad enough just having MD ).

I don't know when the next phase of the myostatin trial will begin but i think its the most advanced new treatment being near the end of the last cohort of the phase I / II

[Luis]

Andy

Could you explain a litle more about your experience with myostatin trial, I am very interesting with this alternative as a treatment for my son BMD 19 years old.

best regards

Luis

[David Feder]

Hi all:
Hurry it's not synonymous of bad quality or risk for patients. The safety it's very important in all trials. Recently several patients had side effects in a clinical trial of a new drug to cancer in UK (one patient died). The pharmaceutical companies have several drugs to test (more than 200) and make few studies with only one drug. Why not test several drugs in several countries in several centers? Why they hide some informations for other researchers? They want parents resigned and optimists.
David

[Andy]

i only had a few doses of the myostatin drug but i felt stronger and more confident on my feet. It could still be years before this drug is licensed however and i know the level of the trial i was on won't finish for a few more months , it just ended in the UK prematurly in part due to slow response from the drug authorities over here

[hb]

As david said, "Why not test several drugs in several countries in several centers? Why they hide some informations for other researchers? "... atleast they can SPEED the process, SOMEHOW. Every country and its people (MD) are willing to take on clinical trial. (atleast, satisfaction of participating is more important to them rather just living on hopes)

Secondly, integration of knowledge on a “common platform” is necessary... integration of researcher, fund raising organisation, pharmaceutical company and patients...

And lastly but honestly, the safety factor has been cooked so much that "fear" has enveloped it... one patient might had died with "one kind" of treatment programme... but why stop others from trying... several are dying everyday around the globe wanting a treatment… These generation patients can contribute/try-finding cures (with themselves getting a fair chance) otherwise the next generation will have to face the same situation ahead, while the cure would still have to wait for the generation next!!!

Right information, knowledge, expertise and infrastructure are all there except for one important missing factor… ‘Sincerity’ … I just wish that atleast someone would make these beautiful hopes/dreams come true with a faithful trial… hb

[bhopeful]

Hi hb: I agree with you 100%, but how do you get the researchers and appropriate pharmaceutical companies as well as the leading organizations like MDA and Parent Project MD to take the lead and change the way the research has been done in the past. Sometimes I think we are still in the dark ages when it comes to the way we do things in medical research.
As I said in an earlier post, I really believe we could have a cure for this generation if the researchers would start working together as an International team and be more efficient and timely in the doing their research.
In the last 5 years that I have been affected by DMD, I have not seen any real urgency in the way research is being approached.
Let's hope that will change.

[David Feder]

Source: www.clinicaltrial.gov/
Muscular dystrophy trials: 17 (included epidemiologic studies and studies about other neuromuscular diseases)
Aids: 720
Lung cancer: 604
Multiple sclerosis: 106.
It`s impossible to advance with so few studies. Money is one problem but it`s not the only problem. Organization must choice the studies that can give results as early as possible.
David