A press release from U.C.-San Diego possibly shed new light on a potential treatment for Kennedy's Disease. An excerpt of the article and a link to the actual article can be found on my Living with Kennedy's Disease blog:

Mutant-protein-in-muscle

I have the feeling we are getting closer and closer. (My fingers are crossed)

Thanks for posting this link, Bruce. The research certainly looks very promising, although given the need for diligent labwork, clinical trials on humans are probably some years away, yet. Nonetheless - My fingers are crossed, too.

A little digression - the reference to mice reminded me of a tongue-in-cheek article that appeared many years ago in Scientific American. To stop research spoiling his pleasures in life, the author appealed for the breeding of tougher lab rats that would be totally unaffected by smoking a daily Havana accompanied by a Big Mac and several shots of Bourbon!

good morning Bruce,
I'm 31 year old suffering from muscle pain and weakness for nearly 3 years and lastly diagnosed as SBMA.
I'd like to ask you 2 questions,
1st why i hear no one talking about leuprorelin .i saw the abstracts of the 2 trails
1st published in
February 2009
( The patients treated with leuprorelin acetate for 144 weeks exhibited significantly greater functional scores and better swallowing parameters than those who received placebo)

and newer one in
2010 Sep
(48 weeks of treatment with leuprorelin did not show significant effects on swallowing function in patients with spinal and bulbar muscular atrophy, although it was well tolerated)

however
leuprorelin treatment was associated with a greater reduction in barium residue after initial swallowing than was placebo in patients with a disease duration less than 10 years .
--------------------------------

2nd question is,
i read that you take dutasteride ,can i start taking it now?
my testosterone level is slight high or at upper border.
i did prostate specific androgen as preparatory step to avodart.
thanks

Hi Ahmed,

I will try to give answers for you. I think that it is safe to say that neither the trial with leuprorelin or the trial with dutasteride were as successful as was hoped. If I remember correctly, the leuprorelin had some effects on swallowing and that was about it. For dutasteride, there were possible effects on muscle strength but they were not statistically significant. Even if effective, neither drug would reverse the effects of KD but only slow the loss of strength. I believe that Dutasteride has fewer side effects and that may be why it is more popular. I do take dutasteride and to be honest, I cannot really tell if it has had an effect - I am still getting weaker although I do not know if the rate of this is slower as I do not know what would have happened if I did not take it. In my opinion, neither drug is what we are looking for in a treatment. I would strongly suggest that you share your research with your physician and decide together what is the best approach.

Ed provides a good synopsis and his recommendation is right on. I know of several men that take dutasteride. None see it as a cure or an end to the muscle wasting. I also know of several who took it during the trial and did not see any improvement.

I know of two men who stopped taking dutasteride and within weeks saw a decline in strength, so they went back on it.

The way it was explained to me is that some of the trial participants saw a slight improvement in strength (<3%) and some showed no improvement or a very slight decline. The third group continued to decline. One recommendation that came out of the study was that it should have been longer to see the long-term impact (both positive and negative).

Personally, I feel it has helped slow the progression process. And, I have seen no significant side effects. Because of this, I continue to take it. I am substantionally older than you, so I have no clue how age enters into the factor.

Whether it will help you, know one knows. Print out the studies, bring them to your doctor and discuss them.

Let's all hope that the current pre-trial work and pending trial for IGF-1 shows significant positive results.

Thanks Ed,
leuprorelin was tested on swallowing muscle (cricopharyngeal muscle in one test)(and esophygeal musle=the residual of barium swallowed, in the 2n test)these muscles were selected from other body muscles for accurate monitoring i think.
at least there was slight effect as you said(leuprorelin had some effects on swallowing )why not used?
i don't have the full article of the trials.

thanks Bruce,
what about dutasteride and cancer prostate ??can you help me about this??
why not leuprorelin??

You need to discuss your concerns as well as any other health issues with your doctor. He is best prepared to answer your questions. Since neither treatment is a cure or an effective treatment. You and your doctor have to way potential benefits against potential side effects and then come up with the best course of action.

In my opinion, Leuprorelin, after reading the trial results, seemed to extreme for me for the potential of little gain.

Thanks for this, Bruce. What is the drug treatment that they mention?

This is too far over my head to explain it, so I went to Wikipedia for help and it is still over my head.

"Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off". This is because mRNA has to be single stranded for it to be translated. Alternatively, the strand might be targeted to bind a splicing site on pre-mRNA and modify the exon content of an mRNA.[1]

This synthesized nucleic acid is termed an "anti-sense" oligonucleotide because its base sequence is complementary to the gene's messenger RNA (mRNA), which is called the "sense" sequence (so that a sense segment of mRNA " 5'-AAGGUC-3' " would be blocked by the anti-sense mRNA segment " 3'-UUCCAG-5' ")."