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Muscular Dystrophy

Targeted Natural Interventions

Coenzyme Q10. Coenzyme Q10 (CoQ10) is a strong antioxidant and plays a central role in cellular energy production. Evidence from human and animal studies implicates CoQ10 deficiency in the development of some forms of muscular dystrophy (Folkers 1995; Siciliano 2001; Tedeschi 2000). 

A study that examined CoQ10 and a combination of CoQ10 and resveratrol in an animal model revealed that high-dose CoQ10 and the CoQ10/resveratrol combination therapy decreased muscle damage and increased muscle integrity (Potgieter 2011). In a pilot study conducted on 12 children with DMD undergoing treatment with corticosteroids, addition of CoQ10 supplementation sufficient to achieve a serum CoQ10 level of 2.5 mg/mL led to an 8.5% increase in muscle strength (Spurney 2011). Furthermore, a 12-month clinical trial that administered idebenone, a synthetic analogue of CoQ10, to 13 children with DMD found improved cardiac and respiratory markers compared to placebo (Buyse 2011).

Two additional clinical trials, cumulatively comprising 27 patients with various forms of muscular dystrophy including DMD, DM, BMD and LGMD, revealed that 100 mg of CoQ10 daily for 3 months improved heart parameters and subjective measures of physical performance compared to placebo. In the first trial, functional improvements corresponded with increasing blood levels of CoQ10 from 0.5 – 0.85 µg/mL prior to treatment to 1.1 – 2.9 µg/mL after treatment. Additional benefits may have been seen with higher dosing since some patients’ blood levels of CoQ10 did not increase significantly. The authors conclude, “Patients suffering from these muscle dystrophies and the like, should be treated with vitamin Q10 indefinitely” (Folkers 1995). Life Extension® suggests an optimal CoQ10 level of at least 3 µg/mL.

Resveratrol. Resveratrol is a phytochemical found in grape skins, Japanese knotweed, and red wine. Its administration for 32 weeks in an animal model of muscular dystrophy led to significantly less muscle loss compared to a control group. Oxidative damage in muscle tissue also decreased significantly with resveratrol supplementation (Hori 2011). Another study conducted on experimental mice that do not express dystrophin revealed that resveratrol supplementation decreased inflammation and increased the genetic coding of a protein called utrophin, which can functionally replace dystrophin (Gordon 2012; ParentProjectMD 2012). Additional evidence suggesting resveratrol may represent an important intervention in muscular dystrophy derives from a 2013 animal model wherein oral administration of resveratrol to genetically dystrophin-deficient mice protected the animals’ hearts against enlargement and fibrosis and restored cardiac function (Kuno 2013).

Creatine. Long used as a supplement by athletes to enhance strength, endurance, and muscle recovery after exercise, creatine may also benefit people with muscular dystrophy. Creatine is a naturally occurring amino acid-like compound that helps provide energy for muscle cells. Evidence suggests it has musculoskeletal and neuroprotective effects (Pearlman 2006; Radley 2007; Tarnopolsky 2011). When creatine is metabolized by the body, it enters muscle cells and promotes protein synthesis and reduces protein breakdown (Hespel 2001; Persky 2001). In addition, it functions as an antioxidant and activates stem cells in muscles that have the ability to self-renew and contribute to regeneration after injury and damage (Tarnopolsky 2011; Relaix 2012). Studies in an animal model of DMD revealed that creatine supplementation improves mitochondrial function (mitochondria are the “powerhouses” of cells), increases muscle health, and decreases muscle cell death (Passaquin 2002).

A clinical trial in which creatine monohydrate (0.1 g/kg/day) was administered to boys with DMD for 4 months found an increase in their fat-free mass and hand grip strength, which occurred independently of steroid usage (Radley 2007). Another study looking at patients with DMD and BMD reported that supplementation with 3 g/day of creatine for 3 months almost doubled the length of time it took for subjects to fatigue (Pearlman 2006). Supplementation was well tolerated in both children and adults, and the benefits also extended to patients undergoing treatment with corticosteroids (Tarnopolsky 2011). In another clinical trial, children and adults with muscular dystrophy were given creatine at doses of 5 and 10 g/day, respectively, for 8 weeks. The treatment was well tolerated and the researchers reported a modest improvement in muscle strength and day-to-day activities.  Benefits were evident in all types of muscular dystrophy studied, which included DMD, BMD, FHMD, and LGMD (Walter 2000). A study that employed technologically advanced methods to monitor muscle physiology in children with DMD found that daily supplementation with 5 g of creatine for 8 weeks led to enhanced cellular energy metabolism; the effect was more pronounced in subjects under 7 years of age (Banerjee 2010).

Omega-3 fatty acids. Omega-3 fatty acids are essential components of cellular membranes. In an animal model of muscular dystrophy, degeneration of skeletal muscle was prevented in animals fed a diet enriched in omega-3 fatty acids from birth until death. The animals fed omega-3 fatty acids had larger muscle cells and were able to more efficiently repair injured muscle. In fact, the preservation of skeletal and heart muscle structure was so pronounced that it improved the animals’ longevity. This study only found benefits when omega-3 supplementation was initiated at weaning, before muscle damage started; it did not appear to be useful when supplementation was initiated in adulthood. This finding, which reveals that results can be obtained solely by dietary intervention, supports the idea that affecting the essential components of cell membranes may also provide a strategy to ameliorate muscular dystrophy in humans (Fiaccavento 2010).

Vitamin D. Vitamin D and calcium are essential for muscle and bone growth and function. Vitamin D supplementation is especially important for patients with severe forms of muscular dystrophy, such as DMD, because: 1) The patients’ bone density is often decreased as a result of decreased mobility, 2) osteoporosis is more frequent in these patients as a result of the adverse effects of corticosteroid treatment, and 3) a decreased exposure to sunlight decreases vitamin D levels (Beytía 2012). A prospective study that included 33 boys with DMD showed that two years of treatment with calcidiol (25-hydroxy vitamin D), combined with an adjusted dietary calcium intake equal to the internationally recommended daily allowance, corrected the vitamin D deficiency and increased bone mass in about two-thirds of the participants (Bianchi 2011).

Taurine. The organic compound taurine is distributed throughout the body and is especially abundant in skeletal muscle, where it functions as an antioxidant and is essential for cellular growth and function (Silva 2011). In fact, mice genetically prone to taurine deficiency display incomplete and abnormal muscular development and decreased capacity for exercise (Miyazaki 2013). Moreover, evidence from an animal experiment shows that taurine supplementation improves muscle performance and protects against damage during electrical stimulation (Goodman 2009).

A few animal studies suggest taurine may confer benefits in muscular dystrophy. In one animal model, mice with muscular dystrophy were given a glucocorticoid (prednisolone) or taurine alone or in combination for 4 – 8 weeks. While both treatments improved functional measures of muscle health, combined treatment with both compounds acted synergistically to augment the functional improvement beyond what was achieved with either alone (Cozzoli 2011). Another mouse model of muscular dystrophy found that taurine supplementation countered the negative effects of excessive exercise over 4 – 8 weeks (De Luca 2003).

In a small human trial on nine patients with myotonic dystrophy, taurine administration led to a significant improvement in myotonia and improved function of muscle cell membranes. The researchers observed no significant side effects of taurine treatment (Durelli 1983).

Glutamine. In an animal model of muscular dystrophy, supplementation with L-glutamine was found to decrease the ratio of oxidized to total skeletal muscle glutathione, indicating that glutamine may be protective against oxidative stress (Mok 2008). In a study on boys with DMD, 13 boys receiving 0.5 g/kg/day glutamine orally for 10 days were compared to a control group of 13 boys who received a nonspecific amino acid mix. Overall, glutamine supplementation was associated with an inhibition of protein degradation (Mok 2006). Another group of researchers analyzed the rate of glutamine synthesis in six children with DMD in comparison with healthy controls. They found that glutamine synthesis was significantly reduced in children with DMD and concluded glutamine “might therefore be a 'conditionally essential' amino-acid in DMD” (Hankard 1999).

L-Carnitine.  L-carnitine is an amino-acid-like compound important for fat metabolism. It is evolving as a promising new potential therapy, based on laboratoryexperiments with DMD patient muscle cells, where it seems to restore muscle cell membrane fluidity (Le Borgne 2012). This is important because a deficiency of dystrophin is known to cause alterations in membrane fluidity and permeability that lead to an increase in reactive oxygen species and muscle damage (Malik 2012).

Melatonin. Melatonin is a hormone most frequently associated with the sleep-wake cycle. It is also a powerful antioxidant that protects cells from the damage caused by free oxygen radicals. In an animal model of muscular dystrophy, melatonin administration increased the total amount of glutathione (a powerful endogenous antioxidant), lowered the ratio of oxidized to reduced glutathione, and lowered the activity of plasma creatine kinase, all actions that work to protect cells from damage (Hibaoui 2011). Melatonin also reduced plasma creatine kinase levels in boys with DMD, and decreased their markers of inflammation (Chahbouni 2010). Furthermore, levels of oxidative stress in red blood cells, which are normally increased in DMD, were lowered after 3 months of melatonin treatment (Chahbouni 2011).

Green tea. Green tea contains powerful phytochemicals that benefit health in several ways; one of its major active constituents is the polyphenol epigallocatechin gallate (EGCG). In an animal model of muscular dystrophy, 1 week of supplementation with green tea extract reduced muscle deterioration by approximately 35% (in a leg muscle). The same researchers later found that green tea extract greatly improved muscle force and resistance to fatigue with 1 week of supplementation. However, these effects were not seen after 5 weeks of supplementation, suggesting that green tea may prevent, but not counteract, muscle degeneration (Dorchies 2006). A study that subcutaneously administered EGCG to mice (4 times a week for 8 weeks) found a delay in the onset of muscle damage, without adverse effects. The benefits included a decrease in serum creatine kinase activity levels back to normal, an increase in the area occupied by muscle fibers relative to damaged tissue in the diaphragm and certain leg muscles, and a positive effect on muscle contraction (Nakae 2008). Another animal model of muscular dystrophy showed that 21-day-old male mice that had their diet supplemented with green tea extract had a 128% increase in their total running distance over 3 weeks (Call 2008).

Vitamin E and Selenium. Vitamin E and selenium are antioxidants that protect cells against damage. In several animal species, selenium deficiency causes disorders that resemble muscular dystrophy; supplementation prevents these disorders. In boys with DMD who received selenium and vitamin E for one year, followed by a one-year observation period without treatment, a slightly more rapid deterioration of muscle strength was reported in the second year when they did not receive any treatment. This finding may suggest that treatment with selenium and vitamin E provided a slight delay in deterioration (Gamstorp 1986). Another study identified a new selenoprotein, a type of protein that contains a special amino acid residue called selenocysteine, which was involved in a congenital type of muscular dystrophy (Moghadaszadeh 2001). This finding supports the idea that the variable effectiveness observed from supplementation may actually be explained by different underlying disease causes (Moghadaszadeh 2001; Rederstorff 2006).

N-acetylcysteine. N-acetylcysteine (NAC) is an antioxidant and has been shown to decrease muscle damage and increase muscle force in an animal model of muscular dystrophy. NAC added to the drinking water of animals was found to reverse the dysregulation of dystrophin-associated proteins caused by oxidative stress and reduce the expression NF-κB, a factor involved in inflammation and muscle damage. The authors conclude, “Our data show that NAC can provide considerable protection against the ongoing muscle degeneration in intact mdx mice [an animal model of DMD] and against damage resulting from stretched contractions. The logical extension of these findings is to combine NAC, or other antioxidants, with blockers of parallel damage pathways … in order to provide a more effective therapeutic approach for DMD.” Although encouraging, clinical trials are needed to test its potential utility in humans (Malik 2012; Whitehead 2008).