Rare Disease Report

Dietary Supplement Boosts Muscle-Force Production in Duchenne Muscular Dystrophy Model

JULY 23, 2018
Kristi Rosa
A progressive form of muscular dystrophy that occurs primarily in males, Duchenne muscular dystrophy (DMD), currently has no cure; the goal of treatment is to control symptoms and complications associated with the disease in order to improve quality of life.

Now, researchers from the Université Laval’s Faculty of Medicine and Centre hospitalier universitaire (CHU) de Québec Research Centre-Université Laval have found that a glucose-derived dietary supplement was capable of boosting muscle-force production by 50% in a Duchenne muscular dystrophy mouse model in just 10 days.

The researchers believe that their promising findings could potentially pave the way for a clinical trial set to evaluate the supplement’s safety and efficacy in humans with the disease.

“People have a lot of hope for gene therapy, but it will still take years of research before we find an effective treatment,” professor Sachiko Sato stressed in a recent statement. “That’s why it’s important to find other treatments to help preserve the muscular strength of patients as long as possible.”

Failure of the muscle membrane, sarcolemma, to properly attach to the basal lamina results in muscle injury, which serves as the underlying cause of DMD, in which mutations occurring in the dystrophin gene hinder firm adhesion.

“In patients with DMD, even moderate contraction causes damage, leading to progressive muscle degeneration,” the study authors write. Damaged muscles are repaired through a process referred to myogenesis, which happens a lot in patients with DMD; however, the repeated activation of this process is known to lead to the exhaustion of myogenic stem cells. As such, treatment approaches that will decrease the risk of this exhaustion, strengthen the interaction between the sarcolemma and the basal lamina, and increase the effectiveness of myogenesis are needed.

In the study, researchers found that galectin-3—an oligosaccharide-binding protein known to be involved in cell-cell interactions and to be expressed in myoblasts and skeletal muscle—and the glucose derivative used as a dietary supplement—N-acetylglucosamine—promotes myogenesis in vitro.

The supplement is “a simple sugar whose structure differs from that of glucosamine, which is sold to treat joint problems,” Professor Sato explained.
Known to boost the synthesis of binding partners of galectin-3, the supplement was also found to increase muscle-force production in the DMD mouse model by 50% after 10 days of treatment.

“We don’t know yet whether the molecule increases the production of muscular fiber or improves its survival rate, but we found that the mice’s muscular strength was better preserved,” Professor Sato added.

Despite the fact that the study was conducted in laboratory animals, the results are promising, suggesting that treatment with the supplement might mitigate the burden of DMD, according to the study authors. Furthermore, Professor Sato stressed that the treatment is not only cost-effective, but it can be synthesized in a laboratory or even extracted from crustacean shells. Furthermore, N-acetoglucosamine is found in human milk as the sugar with the second highest concentration following lactose.

“Everything indicates that it is worth testing its effectiveness in improving the quality of life of DMD patients,” Professor Sato concludes. “We now need to conduct clinical trials in order to confirm the substance’s effectiveness on humans and determine the treatment’s duration and dosage.”

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