In Huntington’s disease (HD), proving systemic therapy rather than intrathecally or direct brain injection techniques is a challenge. Rajeev Kumar, MD, Rocky Mountain Movement Disorders Center, Englewood, CO, explains the current interest in improving the penetration of large molecules into the brain through focused ultrasound. There is also the possibility of small molecules, as these can penetrate the blood-brain barrier, which was observed to be effective in targeting synuclein in Parkinson’s disease. However, small molecule use in HD is not something that has been looked at as of yet. The PTC compound RNA splicing modulator is an HD therapy that has some exciting preclinical work. This is beginning to enroll in Europe and has been expanded to a 1-year study. The concept of treating HD with systemic therapy is very exciting, especially due to the oral route and low toxicity of the compound. These have not heightened levels of neurofilament, which has caused safety concerns in recent studies. This interview took place at the AAN Annual Meeting 2023 in Boston, MA.
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