You are all aware of the Losartin paper by R. Cohn and colleague and like many other drugs/supplements/nutraceuticals, we continues to discuss this within the community and ask various physicians to prescribe Losartin for our boys. And some parents are giving it to their son, with or without the blessing of the physician. So, the botton line is that there is no evidence other than on study on the mdx mouse. And you have already heard the words, "there is a great deal of difference between the mdx mouse and the boys". And they are right. And from our side of things we ask "well, if there is potential gain with few (or no) side effects, why not?" and TIME matters. We want to EndDuchenne and as fast as possible.

So, where is the happy middle? I'm not sure we will find that tonight, but I did poll a few physicians (7) for some opinions and thought you might be interested in their responses. The responses fell into 3 broad categories.
1. One physician said - the recommended dose of Losartin is 25mg/day. The Marfan trial (and probably the dose range for a dmd trial) is 150-200mg/day. This dose is very high and increases the risk for toxicity. This physician did not thin 25mg or 50mg/day would have any effect.
2. Another physician said there is no evidence and they would not recommend it. When asked about a trial, this physician suggested trials cost millions of dollars and 'why spend money on a bandaid, when more meaningful trials are in development (referring to exon skipping. utrophin upregulation (summit, catalyst compounds), etc
3. Another physician repeated the words above and worried that patients would 'jump ship' if another, potentially more useful trial was announced. This physician also discussed the fact that most trials involve a minimum of 18 mo. to develop, recruit patients, analyze the data and report.
I thought you would like to know. I have some other ideas I'm exploring as well. Stay tuned.
Pat

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Comment by Jacobs Mommom on October 15, 2008 at 3:52pm
What about those boys whose mutations are in the area that affects the heart more? Wouldn't it be better to try Losartan on them now then to not do anything at all?

I was told that the doctors know that some mutations affect certain areas of the body more and there is a chart that explains it but the only one I saw was on another site that was very complicated for those of us not in the health field to understand.

Jacob has a deletion of 42 and 43 and needs 44 skipped (for what it's worth) and if this area affects the heart more than why not put him on Losartan or anything that might keep his heart stronger now instead of waiting till a problem arises?

Pat I understand what you are saying but some of these doctors are just covering their own you know whats and why can't the parents sign some kind of waver if they want to try something that they believe would help?

I just don't believe that these doctors can be 100% involved in each patient. At least that's been our experience.
Comment by Julie Garcia on October 15, 2008 at 2:34pm
Michelle/Ofelia, I agree. Pat, I know you are trying all you can for our families and it is appreciated beyond words.

The time to act is now and not too ponders as our children slip away. I know we must do things properly and safely. I know and I understand this but I believe there are things we can do today.

It seems we can have things in place to provide measurements. As the Duchenne Connect data base is built up why can't we pull from this as one resource or set up another category in terms of patient/parents interest. in using FDA approved drugs or supplements. List the categories under considerations. Have the protocol in place and the families work through the data base/their doctor to gather desperately needed data. Set it up so this information is a clinical trial that is well monitored and controlled. Too many what ifs and we can't. What about the families living every day watching for signs of the progression and then watching as the progression unfolds.

Controls can be set up and established and put in place to make this work so there is a protocol and documentation to help get coverage. We can think out of the box on this and make it work.

As it is now we have a community who loses faith in the process and will move forward on their own placing their child on supplements, FDA drugs and we have no measurement in place. What if the choices we make with this is actually speeding up the progression? But then what else, right now, can we do besides sit back and watch the progression?

Right now, we sit back, watch and hope something available will come up and maybe our child will be one of the lucky ones. Or, are they really the lucky ones because there is no proof or promise really at this time. Those who have an opportunity are taking risks and it is not an easy decision. We are all frightened for our children’s future.

We don't have much in place at this time to help this generation, right now, today. So, families will forge ahead and hope and pray what they are doing will be of benefit to their child and will not harm them. We have no measurement to say, yes it is helping.

So, until we organize around how to better use resources already available to us for clinical trials my final question is else you got?
Comment by Ofelia Marin on October 15, 2008 at 12:48pm
Well said Michelle. The discussion about losartin started early in 2007 (Cohn's paper was published Feb 07) and now, 18+ months later we are still discussing how to measure benefit?

The more meaningful trials mentioned are in development but still long time away, not to mention how many years it will take until one of these drugs (IF any) will be FDA approved.

Someone posted an analogy on another site and with his permission I will post it here since I find it great:

"Your house is on fire. You have called the fire department and they have sent an old hand-pumped machine to put out the blaze (steroids). It's only got a limited amount of water but it is making a difference in slowing down the fire's progress.

You and your family are standing at the back of the house watching and hoping that a modern fire truck will appear soon to put the blaze out before it's too late and there's nothing to save (exon-skipping etc.).

You notice the garden hose in the garden (Losartan). It's connected to the water supply and water does flow from it when you turn it on.

You don't know whether it will make much difference or not...

So...who is going to stand by and NOT try the garden hose on the fire?!"
Comment by Michelle Scaglione on October 15, 2008 at 1:54am
We dont have time to wait for clinical trials for each and every supplement or drug. If there is a way to think ouside the box when will that happen? Our sons dont have a minute to lose. I am in debt due to trying to save my son. My financial burden is so deep I cant find a way out but I am so saddened with what is happening to my son I just cant worry about it now. I agree we cant afford to pay for these compounds but what other choice do we have. I sit here scared to death with what lies ahead for my son.
Michelle

Staff
Comment by Pat Furlong on October 14, 2008 at 9:55pm
I totally agree Michelle, but I think we have to think outside the box and develop perhaps new models or systems to measure benefit in an effort to collect evidence about a certain drug, supplement, etc. Otherwise families are giving their son compounds that are not covered by insurance, thereby causing increased financial pressure on families. I think there are potentially more streamlined ways to collect evidence. Bandaids are better than nothing at all, but it is first identifying which bandaids are really bandaids and then insuring availability to all, rather than adding financial burden.
Comment by Michelle Scaglione on October 14, 2008 at 8:41pm
Dear Pat

I disagree with the the doctors. I feel a "bandaid" is better than nothing at this point.
Michelle Scaglione

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