Tamoxifen may be a promising treatment option for patients with Duchenne muscular dystrophy (DMD), according to a systematic literature review published in Frontiers in Pharmacology.

“[Tamoxifen] presents compelling advantages over other therapeutic avenues,” the researchers wrote. “In particular, [tamoxifen] might benefit patients with DMD within a short time and is much less costly than other options. Furthermore, compared to gene therapy, [tamoxifen] treatment could offer access to all DMD patients regardless of their specific genetic profile.”

To analyze the mechanism of action and toxicologic effects of tamoxifen on DMD and explore whether the potential benefits of the treatment should be further evaluated in patients with DMD, a team of researchers led by Davide Staedler, PhD, conducted a systematic review of the literature. 


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The researchers identified and screened 15 articles published between 1996 and 2021 that explored the use of tamoxifen, which revealed promising outcomes in muscular strength recovery and decreases in pathologic biomarkers.

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Two publications recognized tamoxifen as a potential treatment for DMD and stated that the repurposing of drugs is of great importance in finding new ways of treating DMD.

However, apart from its potential pharmacological and clinical benefits, the researchers were unable to gather enough evidence to conclude that the treatment was beneficial in terms of quality of life and survival, especially in the youngest patients with the disease.

Tamoxifen, a cancer drug is a selective estrogen receptor modulator used to prevent and treat breast cancer as well as other types of cancer. Its metabolites have a high affinity for estrogen receptors, which are also found on muscle fibers and play a role in myogenesis and angiogenesis.

Its potential in treating DMD has been known for 20 years, and many studies have shown promising results, while others failed to show any benefit.

Reference

Botti V, Menzel O, Staedler D. A state-of-the-art review of tamoxifen as a potential therapeutic for Duchenne muscular dystrophy. Front Pharmacol. 2022;13:1030785. doi:10.3389/fphar.2022.1030785