Duchenne muscular dystrophy was found to respond well to breast cancer drug Tamoxifen - some of the features of the disease were reversed in an animal experiment.
This is very promising news as there is currently no treatment available for alleviating the long term symptoms of Duchenne muscular dystrophy (DMD).
DMD is a disorder that affects around 1 in 3,600 boys and is very rarely found among girls, it weakens the musculoskeletal system due to a mutation in the dystrophin gene - which is responsible for coding vital muscular proteins. It is characterized by respiratory and cardiovascular problems, fatigue, muscle weakness and progressive difficulty walking.
The researchers observed the effects of giving tamoxifen to a mouse model of DMD for a year. They were surprised at the end results, noting remarkable improvements in muscle force and cardiac structure. In the heart, fibrosis was reduced by nearly 50% and they noticed an increase in the thickness of the muscle fibers in the diaphragm, improving overall respiration.
People suffering from DMD have muscle fibers that are extremely susceptible to stress and experience decreased overall muscle function. Tamoxifen was found to greatly improve this and restore: muscle function, the structure of leg muscles and muscle contraction. The whole body strength of mice treated with tamoxifen doubled and even tripled in some instances.
The authors add:
"Our findings of a slower rate of contraction and an enhanced resistance to fatigue in muscles from tamoxifen-treated dystrophic mice are of significance for the pathophysiology of muscular dystrophy."
Tamoxifen restored muscular ability
The study involved a wire test to assess the strength of mdx5Cv mice treated with tamoxifen versus mice receiving no treatment at all. They compared their ability to pull themselves up on a wire - an exercise that demands quite a bit of strength and energy - and observed how long they hung onto the wire with their forelimbs before falling.
The authors found that mice on the tamoxifen treatment were able to hold onto the wire for a few seconds longer compared to the mice not undergoing treatment. This reveals that tamoxifen treatment could indeed be very effective at restoring muscle strength among those with DMD.
Tamoxifen works by restoring plasma creatine kinase (CK) activity. Mice without DMD had levels of activity about three times greater than those with the condition - tamoxifen was able to normalize their activity. The authors believe that this effect is achieved because of an estrogen receptor mechanism, especially since the drug is able to significantly raise levels of estrogen receptor β2.
The authors note that tamoxifen has been shown to be effective at treating muscle tissue levels a lot lower than reported in previous studies - which were carried out on normal rodents. In conclusion, this finding suggests that tamoxifen way prove to be a very good treatment option for people suffering from DMD.
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for know about tamoxifan treatment in detail
posted by MEENA GARG on 23 Mar 2013 at 4:10 am
sir, i am diagnoised as a pt. of myotonic muscular dystrophy type1 tested by genome test from CCMB hydrabad(india) now i am on treatment of tab. phenytoin,nucal-d,lanoxin0.25mg . Please tell me about effectiveness of tamoxifan in my case.
I am in age 22 with the disease LGMD. Whomever is affected by MD I want to say that there is no treatment. Physiotherapy is the only way to keep sustain. I want to say that please don't get fooled by any kind of treatment whichever is shown in you tube and all...
I also have a son (now 33yrs old ) with Muscular Dystrophy. We found out when he was 3. As he progess we put him in a wheel chair, he also has a cough machine to help him with colds ( very important to have) also he has a bi-pap to help him sleep better at night. You can contact Muscular Dyrophy asociation and they can refer you on where to go. If you are on your own you can go thur the state and get assitance for income,caregiver, go down and talk to them.If your child is young you can go to shrinner hospital and they can help with your child. There are resourses though them. hope that I helped a little.
'Muscular Dystrophy Responds To Tamoxifen Treatment'
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