SARM for Kennedy's disease (SJ-18-0012)

St. Jude Reference #SJ-18-0012

Description

Researchers at St. Jude have invented a small molecule, selective androgen receptor modulator (SARM) for the treatment of spinobulbar muscular atrophy (SBMA, also known as Kennedy’s disease). This small molecule, abbreviated MEPB, targets a specific interaction surface (called BH3) and alters co-regulator binding to the activation domain (the “AF2” domain).

SBMA is a progressive debilitating neurodegenerative disorder resulting in muscle cramps and progressive weakness due to degeneration of motor neurons in the brainstem and spinal cord. The estimated prevalence of this disease is 1 in 40,000.  The condition is associated with mutation of the androgen receptor (AR) gene and is inherited in an X-linked recessive manner. It is also related to other neurodegenerative diseases caused by similar mutations, such as Huntington's disease. As with many genetic disorders, no cure is known, although research continues. Because of its endocrine manifestations related to the impairment of the AR gene, SBMA can be viewed as a variation of the disorders of the androgen insensitivity syndrome (AIS). Strategies being pursued involving androgen ablation exacerbate androgen insensitivity and have other adverse effects.

This new approach is effective in mice and androgen-sparing. In addition to the possible treatment of spinobulbar muscular atrophy, it is conceivable that this compound could be beneficial for prostate cancer.

Keywords

Small molecule selective androgen receptor modulator (SARM), spinal and bulbar, spinal-bulbar, spinobulbar muscular atrophy (SBMA, Kennedy’s disease), MEPB, BH3, AF2, neurodegenerative, X-linked, prostate cancer, neurodegenerative.

Granted patents or published applications

Patent application pending in the US, published as WO2019/084365

Related scientific references

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