Huntington’s disease is a progressive neurodegenerative disorder, caused by inheritable mutations in the huntingtin (HTT) gene. The mutation produces a toxic form of the HTT protein that aggregates in and ultimately kills nerve cells.
This results in various symptoms, including movement, cognitive, and psychiatric problems.
There is no cure for Huntington’s disease. However, there are several approved therapies to help manage the symptoms of the disease and maintain patients’ quality of life for as long as possible.
Furthermore, research is continually ongoing into the mechanism of the disease, which could lead to the development of new and innovative treatments. There are a number of experimental therapies currently being investigated in clinical trials.
The approved therapies for Huntington’s disease mainly focus on managing the symptoms of the disease. Movement problems, such as chorea, for example, are a common Huntington’s symptom. Xenazine (tetrabenazine) is the only medication specifically approved for Huntington’s chorea. Others, such as antipsychotics and benzodiazepines, have also demonstrated a benefit and can be used off-label.
Physical therapy can help maintain mobility and prevent falls through tailored exercises for the patient. This can be complemented by occupational therapy that helps the patient establish coping strategies and identify ways to make his or her life easier, either through simple changes or the introduction of assistive devices. Occupational therapy and speech therapy can also help deal with communication issues that may arise due to the disease affecting the muscles of the mouth and throat.
Researchers have identified several avenues to further explore for the treatment of Huntington’s disease. Many of these have now progressed to the clinical trial stage in humans, and more are still being developed.
Several therapies are being investigated to manage the symptoms of the disease more effectively. For example, SRX246, a Huntington’s-specific therapy for depression, is currently in a Phase 2 clinical trial.
Gene silencing therapies act to reduce the levels of toxic HTT protein being produced. It is hoped that this could slow the progression of the disease, and not just manage the symptoms.
Neuroinflammation is an abnormal immune response that is common in Huntington’s disease and can lead to further damage and cell death in the brain. Therapies intended to reduce inflammation in the brain are being developed for Huntington’s. Examples of experimental anti-inflammatory therapies include VX15/2503 and laquinimod. Neuroprotective therapies aimed at reducing nerve cell death in the brain are also an option. Examples include Huntexil (prodopidine) and SBT-20.
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