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.

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Comment by Michelle Scaglione on October 16, 2008 at 10:34pm
Pat this is directed to the FDA not you: How dare the FDA call DMD chronic and not terminal. I am livid to say the least. Just because they changed the age to 27 that makes it less severe? Who has the right to call it chronic and not terminal? With what evidence did all of it suddenly change? Just age? And the last time I checked 27 and quite young to die. My son is 16 and yes hes alive but is he living? Living the life of a normal 16 year old. By the time he makes it 27( I hope he does )will he want to given the fact all he will have left to move is his fingers? Why dont they come join me in my house with my son who is suffering from DMD and see if they think its chronic. Why dont they talk to the parents who have lost their sons recently to DMD at 16 and 17. See if they think its not terminal. Personally I think they are using this new age change just so they dont have to put the money into research. Because thats what it all comes down to, money! With any luck maybe their kids or grandkids will get DMD see if they still think it chronic and not terminal.
Comment by Ofelia Marin on October 16, 2008 at 4:32pm
I do not think we are talking about the same Protandim study are we? The one I mentioned was done in Europe and contains results form 16 parents (very low sample size), all completed the study, 1 parent did not see any improvement; all results, negative or positive were reported.

If we would be able to collect data from a higher number of parents, we would certainly be able to learn more. Do you plan to involve statisticians, or only to collect the data and look at trends etc.? I expect disagreement with everything that does not involve a rigorous statistical analysis and a good experimental design. But even a guideline for parents (about supplements etc.) is more than we have today, correct? For example, if I knew that 75% of parents observed positive results and no side effects with supplement/drug X, I would feel better in paying the high price. It is only a guideline as long as we don't have a placebo controlled clinical trial. Is this the best we could do at this point? What do we have today (talking about the ones where there is agreement among physicians), steroids (lots of side effects, disagreement about the starting age etc.), CoQ10, VitD and Calcium? Any additional information about other drugs/supplements would be helpful.

Comment by Pat Furlong on October 16, 2008 at 3:44pm
i agree. well, there is a good deal of disagreement about the protandim information bec. there were parents who did not see anything and stopped, so it only includes those who reported benefit.
DMD does come with a huge burden. NIH conveined a workshop on burden of care, though it was preliminary. Some years ago the australians conducted a burden of care study, which suggested a cost of3M over a lifetime. High unmet need - it is the basis for our position with advocacy and in other areas.
Comment by Ofelia Marin on October 16, 2008 at 3:36pm
Collecting data and looking at trends sounds good to me. In fact I did read an observational study about Protandim that was created using parent's reported data. The results were quite significant.

No matter how we think about it, DMD comes with such a high cost for care (500K / non-ambulatory year ?). It would be great to use part of the money for a treatment improving quality of life.

Comment by Pat Furlong on October 16, 2008 at 3:07pm
i'm not comparing a migraine with dmd, simply suggesting that one needs to make decisions based on available information and if we were able to collect information in a way that is statistically relevant, it would be useful and could do that (actually are already working on the tool via duchenneConnect). Losartin is inexpensive, BUT the dose required is 6x normal which gets into potential toxicity. So docs are reluctant (or simply refuse) to order it outside of a trial. And trials are very expensive and take time and pull from a small pool of patients. I am simply trying to develop opportunities that may help us understand the value of, for instance, supplements, nutraceuticals because we do not have sufficient time or patients to do trials on every approved drug that has potential impact. Another issue is that there are trials in development that may have substantial impact on the disease process and families would absolutely want/need to participate and might consider jumping trials.
BY the way, FDA no longer categorizes dmd as terminal, rather a chronic disease. I understand it is ultimately lethal, but mean age of death is now >27 .
I'm just thinking about loud about how we might work together, identify "bandaids' if you will, in a systematic way and take advantage of the more significant opportunities. We are developing the tool, parents would need to find a doc willing to participate and then willing to be actively engaged in collecting information.
Comment by Ofelia Marin on October 16, 2008 at 2:58pm
Pat, I understand what you are saying, I really do! I am a statistician and I know that having some data, even for as small a sample as 100 would be helpful.

Problem is that we cannot compare a migraine with DMD. If I would have a migraine about which I knew that it is going to kill me then I would try the $5000 drug, but that is just me. We have to keep in mind that DMD is terminal and we do not have anything except the steroids coming with so many side effects!

Then, as far as I know Losartan, it is not very expensive (am I wrong here?), it is very easy to take -- comes in a pill form, it is FDA approved to be used in children and comes with less side effects than steroids. The results in mdx looked good and several parents reported positive results. So I say, let’s start collecting the data.

Comment by Pat Furlong on October 16, 2008 at 11:21am
Thanks for your example about the fire, but here is another example you might think about and probably the answer is somewhere in between. If you have a violent migrane, throwing up and in immense pain and someone says - take these 5 things, every hour, costing $5000 and it MAYreduce the headache by 5%. All decisions are complex and very individual. No one wants to stand by and do nothing. It is not acceptable. On the other hand, all decisions have to be thought through very carefully - what is expected, what does it mean in terms of expense, impact on family members and potential toxicity. If we could develop a tool to captuer data in a systematic way, getting the community engaged to report what they see, problems involved, and report also if they stop and for what reason. This would help a great deal.
Imagine if 300 people are taking (losartin, haelan, protandim, juven, you name it) and of those 270 report that they see something, that the compound is easy to take, not expensive and this is over 1 year -we have some hard data, anecdotal for sure, but something.
On the other hand if 100 families are giving their son something, at first reporting something very good but after 1 year,only 20 familes continue and 80 tell us the reason why they discontinued - it will tell us something as well. I'm only suggesting that if we work together, actively report in a systematic way, we could learn a great deal more and might (just might) have a better opportunity to convince physicians to write the script or insurance to cover something or heck, sometimes to convince others in the family with a negative attitude.
p .

Comment by Pat Furlong on October 16, 2008 at 11:06am
Hi, I'm just the messenger. I'm working to develop tools that will allow us to actively participate in data collection and hopefully identify trends at least which might help convince physicians aobut a certain compound. I agree we have no time to waste, but we need to do what we can (safely) within the community and work to develop efficient, meaningful trials. We don't have time to be spinning wheels.
Comment by Julie Garcia on October 15, 2008 at 4:37pm
Pat, you did mention at the conference about using Duchenne Connect to gather information as I re-call. We just need something established and in place so we can meet all our objects, providing evidence to insurance companies and most importantly helping our children now.

Comment by Pat Furlong on October 15, 2008 at 3:59pm
I totally agree with all of you. There is no time and clinical trials take time and money. No question. I actually think we need to develop another model and to the extent possible, we are trying to do that on DuchenneConnect. It is a tool we can use to follow individuals on supplements and/or approved drugs if we do it carefully. While we would not have evidence in the rigorous way a clinical trial might provide, we could perhaps understand 'trend'. It would be essential (for instance) to enter information about the start of a certain compound and progress (impression of benefit or not) AND important to include information related to stopping a certain compound and reason why the decision was made. I know how important it is to try in an effort to identify something that provides benefit, no matter how small, it is equally important to understand the other side as well... if you feel something is not working or if your son won't take it for some reason or if you decide to stop based on your assessment of benefit/expense/etc. We absolutely need to work together on this in a systematic way. Only a few people saying something about a supplement is not sufficient, as a community we need to actively engage in understanding the value of collecting data.
This is one of the reasons we set up DuchenneConnect. Companies are more and more interested in patient entered data. For example, Santhera was interested in specific questions as they develop their trial protocol. DuchenneConnect now has 1200 patients registered and was able to provide Santhera specific answers to their questions.
I agree families should forge ahead, but if we are smart about how we do it, we are likely to identify trends over time that will help us make better decisions .

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