The Limitations of Current Treatment Options for Huntington’s Disease and Spinocerebellar Ataxia
Scott Schobel, MD, chief medical officer at Vico Therapeutics provides an overview of the current treatment options available for Huntington’s disease and spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3).
Huntington's disease is a rare neurodegenerative disorder characterized by choreatic movements, behavioral and psychiatric disturbances, and dementia. It is a hereditary autosomal-dominate disorder that usually begins to appear when a person is 30-50 years old, but earlier cases have been reported. The gene involved is the huntingtin gene, in which a CAG expansion causes neuronal damage, especially in the basal ganglia.
SCA is an inherited, progressive, neurodegenerative disease that mainly affects the cerebellum. To date, over 40 genetic SCAs have been classified, including SCA1 and SCA3. Both SCA1 and SCA3 have a CAG expansion associated with their pathophysiologies.
As noted by Dr. Schobel, treatment options for both Huntington’s disease and SCA are limited but Vico Therapeutics is developing treatments that preferentially target expanded CAG repeats in the mutant mRNA transcript and inhibit mRNA translation, thereby leading to the reduction of mutant protein in those two conditions. The therapy in development, VO659, is an antisense oligonucleotide investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including Huntington's disease, SCA1, and SCA3.
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