DMD is a fatal x-linked muscle disease affecting one in 3500 boys and is caused by mutations in the dystrophin gene that result in the absence or severe reduction of the cytoskeletal protein dystrophin.
It is usually treated with corticosteroids, which can have severe side effects, but some surveys show that 80% of caregivers have provided alternative medicine in conjunction with their traditional treatments, academics from Australia state in the journal Nutrients.
“These statistics are concerning given that many supplements are taken based on purely anecdotal evidence,” they stated.
“Many nutraceuticals are thought to have anti-inflammatory or anti-oxidant effects. Given that dystrophic pathology is exacerbated by inflammation and oxidative stress these nutraceuticals could have some therapeutic benefit for Duchenne Muscular Dystrophy,” they said, leading them to assess the peer-reviewed scientific studies that have used nutraceuticals in clinical or pre-clinical trials for DMD to “separate the credible from the conjecture.”
They found that while some nutraceuticals such as co-enzyme Q10, components of soybeans and taurine show promise, others such as some Chinese herbal medicines have limited or no beneficial effects.
With regard to CoQ10, they point to a small study of 12 boys, of which nine showed improvements under Quantitative Muscle Testing (QMT).
This came after taking a daily 400mg dose, with a subsequent 100mg, until they reached a CoQ10 plasma level of 2.5 ug/mL
“Whilst it is unlikely that CoQ10 will replace the current corticosteroids treatment for DMD patients, it could be a valuable addition to help preserve muscle strength and function in patients who have adverse side effects from corticosteroids,” the paper states, adding that a current, larger trial, would hopefully provide more insights.
They also point to some positive trials on mice with soy isoflavones, which appear to show they have the potential to modulate immune response and chronic inflammation in DMD patients.
“While the results in the mice are promising, both trials tested a single dosage and evaluated outcomes at a single time-point and further studies are needed to determine their optimal dosage,” they added.
“No human clinical trials have been reported using soy nutraceuticals in DMD patients and none are recruiting or registered on the Clinical Trials website,” they stated.
The other positive nutraceutical they identified was taurine, with one mice study showing considerable improvements in muscle function and grip.
“Overall these results are highly promising and encourage future studies into the effects of taurine supplementation for treating DMD,” they stated, but warned that little is understood about the safety implications of high-dosage supplementation.
As for Traditional Chinese Medicine, they say that any positive findings speculative and there may be risks associated with their use.
“It is therefore recommended that DMD patients do not supplement with Chinese herbs,” they added.
Lack of consistency
The researchers said the main challenge they found when comparing studies that have used nutraceutical interventions in mice was the lack of consistency, with studies using different dosages, treating mice at different ages and for different periods of time, or assessing different muscles.
“This highlights the need to follow pre-clinical standard operating procedures when assessing any intervention be it nutraceutical or pharmaceutical. In addition, pre-clinical trials would benefit from directly comparing improvements in muscle function and performance with those achieved with corticosteroid treatment, to determine overall efficacy and translational potential,” they said.
They concluded that while nutraceuticals would never cure DMD, they could have significant potential as complimentary therapies in counteracting the damaging effects of chronic inflammation or oxidative stress.
“A number of nutraceuticals have clinical merit and more research into their therapeutic potential to treat DMD is justified,” they wrote.
“If a nutraceutical could produce similar therapeutic benefits to corticosteroids without adverse side effects, it could provide many DMD patients with an improved quality of life as well as reduce costs associated with recurrent hospital visits to monitor and treat corticosteroid-induced side effects.
“For this to occur, research needs to focus on performing good quality peer-reviewed research using compounds that have scientific merit and thus separate the credible from the anecdotal conjecture.”
“Nutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the Conjecture”
Authors: Keryn G. Woodman, et al.