Losartan

[MitchellsDad]

Very well stated!!!

In addition, losartan is a drug that has a good safety profile and has been studied and approved for use in pediatrics (unlike many other drugs that are currently used in children).

[David Feder]

Researchers always say that in five years there will be a treatment to cure DMD. But these five years will never pass. I do not like to use hard words but some researchers deceive parents for a long time.

[anne]

Hello. I am new to this site. Thank you David for starting it. I have a 10 year old daughter with congenital muscular dystrophy. She has been on prednisone for the last 4 months with a tremendous response. She was ambulatory prior to steroids though losing weight, falling, tripping and getting weaker (unable to climb stairs, get off floor). On steroids, she is walking the dog to the end of the block, less afternoon fatigue, and climbing stairs. There is a subset of congenital muscular dystrophies (the dystroglycanopathies) that appear steroid responsive, but it has not become a best practice treatment. I am trying to decide whether adding losartan would give her longterm additional benefits, both muscle and potentially cardiac (unknown if her subtype will lead to heart involvement). After reading the posts, it appears that the losartan effect may be more subtle than steroids, but upon discontinuation the absence of losartan demonstrates its efficacy. My daughter is mute, uses sign and a talking computer to communicate so I am a little worried about starting an additional medication with her limited ability to communicate how it makes her feel, though we got through steroids which did cause some increases in behavior. I would also like to make people aware of a new site for CMD, www.curecmd.org for those with congenital muscular dystrophy or who know of someone with this diagnosis.

[David Feder]

Anne:
It's good to know that your daughter is responsive to steroids. She has any cardiac dysfuction? As you see previously losartan can prevent cardiac dysfuntion and I think that concomitant use with steroids can be a good option.
My son use losartan almost two years ago. He use several others medications as deflazacort, juven, protandim, coq10, lipoic acid, creatine, carnitine, etc. etc. In DMD, in young adults is commom to worsenig of cardiac and respiratory function. The disease is progressive. But unlike, my son had last year an important improvement in cardiac and respiratory function. I can't attribute this effect to any of the drugs but I believe that losartan may have contributed.
David

[michelle scaglione]

Dear David
How much Losartan does your son use?

Michelle

[David Feder]

My son use 37,5mg (mornig) and 25mg (night). He present cardiac dysfunction and fibrosis and need carvedilol too.
David

[antonis]

This was posted by Pat Furlong in her blog:

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

Dr Feder what is your opinion on the clinicians' comments?

[David Feder]

It is very interesting that Pat Furlong put this issue in debate. I don’t agree with the opinion of doctors heard by Pat. In summary they say: clinical trials with losartan will be expensive, lengthy and is best invest in treatment to cure DMD. In other words: it is not worth investing in this generation of patients. This is already clear that the clinical trials for the treatment of the disease (exon skiping, stem cells, and others) are taking longer than planned and we hardly success for all patients before more than 10 years. The dose of 150mg to 200mg is very high and is not used even in severe hypertension. The possibility of toxicity is high in special in childrens. Treatments with steroids and the use of noninvasive ventilation improved patients survival and further altered should be sought to improve the quality of life and survival, more and more. If the medical community decide not to carry out clinical trials with losartan only left to patients and their families starting treatment with losartan and even if the effect is not good for preventing the progression of muscle weakness are obvious benefits in reducing cardiac dysfunction.
We can not invest all the money in curative treatments. When I started Fight for a Future my son was a teenager and many parents with young children not shared by my pessimism, today, unfortunately, many changed their idea and realized that the progression of research is very slowly.
David