Statin Drugs Could Help Patients With Duchenne Muscular Dystrophy | Chemical & Engineering News
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Web Date: September 28, 2015

Statin Drugs Could Help Patients With Duchenne Muscular Dystrophy

Drug Discovery: Cholesterol-lowering compounds improve muscle function in mice with the disease
Department: Science & Technology
News Channels: Biological SCENE
Keywords: Duchenne muscular dystrophy, simvastatin, statins, oxidative stress

Chances are you know someone taking a statin: About one in five adults between the ages of 40 and 75 are on one of the cholesterol-lowering drugs.

A new study suggests that the popular drugs could help another set of patients: children with Duchenne muscular dystrophy (DMD), a rare but lethal muscle degeneration disorder. Researchers at the University of Washington report that simvastatin (Zocor) significantly improves muscle function in a mouse model of the disease (Proc. Natl. Acad. Sci. USA 2015, DOI: 10.1073/pnas.1509536112).

The compound’s possible therapeutic effects for muscular dystrophy aren’t due to its knack at reducing cholesterol levels. Instead, the researchers think statins could slow the progression of the disease through their known ability to reduce inflammation, oxidative stress, and fibrosis, a form of scarring.

These three processes contribute to the progressive muscle degeneration that leaves those affected by DMD, who are mostly boys, in wheelchairs by the time they reach their teens. The average life expectancy for patients is 25.

DMD “is a very complicated disease, and that’s why, to date, there hasn’t been an effective therapy,” says Nicholas P. Whitehead, who led the study. The disease is caused by mutations in the gene for a muscle protein called dystrophin. This has led some researchers to investigate treatment strategies such as gene therapy to replace the defective dystrophin gene.

These strategies will have to overcome significant technical hurdles and face regulatory scrutiny before they can reach the clinic, so Whitehead and his colleagues have been looking for possible pharmaceutical treatments for the disease that could be approved more quickly. They turned to statins because the drugs are known to reduce oxidative stress, a destructive player in the disease. Also, some of the drugs, such as simvastatin, are approved for use in children.

In a series of experiments, the researchers gave the drug to mice with mutations in their dystrophin gene, starting the treatment at three points along the disease’s progression—before symptoms started, at an early stage, and at a late stage. The drug showed therapeutic effects at each point.

Compared with untreated mice, simvastatin-treated animals had an 85% lower level of creatine kinase, a marker for muscle damage; exerted 40% more force in their hindlimb muscles; and showed 50% less fibrosis in their diaphragms.

“The effects they report are very substantial and compare well with genetic methods that restore the expression of dystrophin,” says Dominic Wells, a neuromuscular researcher at the Royal Veterinary College at the University of London. “Importantly, they used a dose in the mice that is within the range already approved for use in children, so it would be possible to rapidly translate these findings into the clinic.”

Whitehead says he and his Washington colleague Stanley C. Froehner are planning possible clinical trials in humans with DMD.

 
Chemical & Engineering News
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Comments
Dr S V S Verma (September 29, 2015 8:17 AM)
In the recent times when lot more emphasis is being given to evolving an effective treatment for the dreaded Duchenne Muscular Dystrophy, this report brings out some hope to the millions of young boys with DMD. I like to heartily congratulate Dr Wells, Dr Whitehead and all those associated with this piece of work. Hope this treatment will be effective in all DMD boys irrespective of exon deletion! I like to urge all concerned to allow the use of the drug to save many a boys undergoing agony, while further research work may continue. Let all the agencies come together to evolve quick way to help all the boys with DMD. Please do understand the agony of such boys and their care givers.Once again I congratulate all concerned with this wonderful piece of research work. With best regards and good wishes.
Anonymous (October 1, 2015 9:08 PM)
When do you think we could give the boys this drug and is it already approved drug for other diseases and if so why should we not use it
Michael Torrice (October 6, 2015 9:59 AM)
So far they've only tested this therapy for DMD in mice. The researchers are starting to plan a clinical trial in people but that is probably a year or more away. Thank you for reading!
James Raffone (September 29, 2015 10:56 AM)
The is fantastic information. I am the CEO of JAR of Hope. We are a non profit that funds medical research such as your study on statins. Please feel free to contact me and let me know how we can help.

Thank you,
Jim Raffone
732.720.6868
Jim@jarofhope.org
Dr.Rasika Wagh (September 30, 2015 1:39 AM)
Hi im DR.Rasika Wagh..my brother have DMD ..he is 18 yrs old..he is wheelchair bound ..he is currently on homeopathic treatment..can I start simvastatin for him..if yes can u please help me with dosage for statins..and any prerequisit tests that should be done before starting therapy.? PLZ help..
Yogi (September 30, 2015 6:01 AM)
As a parent of our DMD/BMD child, we are looking desperately for the treatment specially when we don't have money / facilities to send our kid for clinical trails. Everyday we see our boy and pray to god to get him cure and discover the treatment ASAP before....
amina (September 30, 2015 7:35 AM)
Hi, I am a sufferer of some form of Myopathy/ Muscular Dystrophy looking for possible treatments for weakening muscles. i am on the verge of disability if God's Grace does not come to my rescue. I have read the account with great interest and hope. My cholestrol levels are fine. Can i take this medicine simvastatin (Zocor) to improve the codition of my muscles? Is it safe ? I am a middle aged mother of two grown children and and recently my skeletal muscles have deteriorated sharply. i cannot climb steps , my balance is bad and proximal muscle strength is compromized , though not as bad as the sufferers of DMD which is the worst form of MD.Please can someone tell me if i can try this medicine in hope of some relief / improvement?
Subhas S Nadkarni (September 30, 2015 7:43 AM)
My son 26 year old is suffering with proximal muscular dystropy. I request all to suggest suitable drug or treatment for improving muscle strength.
Walid Itani (September 30, 2015 7:39 PM)
How statins can help for muscles dystrophy and it increases cpk level
anuja (October 1, 2015 6:31 AM)
Thank you for your endless effort. I hope now my brother will be alright.. Please let us know when this drug will be available and how could we get it.. thank you..
mahek (October 16, 2015 1:20 AM)
When will these drugs will be available.please help all dmd patients

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