Cholesterol is a waxy substance that is needed by the body to build cells or make vitamins. However, high levels of cholesterol can contribute to building up hard deposits, so called plaques, at the walls of arteries. In this way the risk for cardiovascular diseases like heart attack or stroke is increased.1 Cardiovascular diseases are the leading causes of death worldwide, being responsible for 27% of all deaths.2
According to the WHO, the global prevalence of raised total cholesterol among adults was 39%. Raised total cholesterol was estimated to contribute to about 2.6M deaths worldwide.3
Usually, high levels of cholesterol are treated with statins. Some people will not tolerate statins, showing muscle or cognitive adverse events, or will not achieve sufficient reduction in their cholesterol levels. In addition, statins are taken orally and compliance to the daily schedule is only 54%. After one year of treatment only half of the patients are still taking their medication regularly. Recently, two monoclonal antibodies targeting proprotein convertase subtilisin/kexin Type 9 (PCSK9), a protein involved in the regulation of cholesterol in the blood, were licensed by the U.S. FDA. However, these therapies need to be injected every two or four weeks and come with high costs.4
To overcome the issues associated with currently available treatments, new efficient, cost-effective therapies with convenient administration schedules are needed.
Huntington’s disease is a life-threatening neurodegenerative disease that has a profound impact on both patients and family members.5-7 Patients with Huntington’s disease suffer from increasingly debilitating problems with movement, cognition, and psychiatric function.5-7 During the later stages of the disease, patients may not be able to walk, speak, or swallow food, as they become dependent on caregivers.5-7 The symptoms usually first appear at 30–50 years of age, and it normally leads to death within 10–30 years of symptom onset.6
Huntington’s disease results from inheriting a mutated version of the huntingtin gene; the resultant faulty huntingtin protein (mutHTT) induces a progressive loss of nerve cells in certain parts of the brain, leading to the development of symptoms.5,7,8
Impact on patients
The progressive symptoms of Huntington’s disease have a profound impact on the lives of patients. Symptoms include memory lapses, depression, personality changes, and movement difficulties.5 Symptoms may affect a patient’s ability to work, perform daily tasks, and take part in recreational activities that they used to enjoy. The behavior of patients may become erratic and they may suffer from mood swings.5,8
An estimated 3–7 per 100,000 people of European ancestry are affected by Huntington’s disease.7
Impact on family members
Huntington’s disease places substantial physical, emotional, and financial strain on family members.5,7 The knowledge of a familial genetic predisposition and the effects of the disease on mental health can be particularly distressing for family members, as they may see their loved one develop mood swings and personality changes.5 Patients may become dependent on caregivers before they reach old age.5,7
Current treatments aim to ease the symptoms of Huntington’s disease; however, there is no treatment that can stop disease progression.5 Additionally most of these treatments can cause troublesome side effects.9
New therapies for Huntington’s disease are urgently needed to address the root cause of this debilitating disease. We are investigating the use of SAIT to target mutHTT, with the aim of reducing the progression of Huntington’s disease
We are developing not only SAIT but also a novel monoclonal antibody therapy for treating Huntington’s disease. Both these approaches are entering the preclinical phase of development. If you wish to learn more about this approach, please contact us.
- https://www.heart.org/en/health-topics/cholesterol/about-cholesterol. Accessed 15 December 2021
- https://www.who.int/news-room/fact-sheets/detail/the-top-10-causes-of-death. Accessed 15 December 2021
- https://www.who.int/data/gho/indicator-metadata-registry/imr-details/3236. Accessed 15 December 2021
- Trentman TM et al. J Anaesthesiol Clin Pharmacol. 2016 Oct-Dec; 32(4): 440–445.
- National Health Service (NHS). Overview. Huntington’s disease. 13 February 2018. https://www.nhs.uk/conditions/huntingdons-disease/. Accessed 15 January 2019
- National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS). Huntington’s disease information page. 15 June 2018. https://www.ninds.nih.gov/Disorders/All-Disorders/Huntingtons-Disease-Information-Page. Accessed 15 January 2019
- Huntington’s Disease Foundation. Life with Huntington’s disease. 2017. http://www.huntingtonsdiseasefoundation.org/life-with-hd-1. Accessed 15 January 2019
- National Health Service (NHS). Symptoms. Huntington’s disease. 13 February 2018. https://www.nhs.uk/conditions/huntingtons-disease/symptoms/. Accessed 15 January 2019
- National Health Service (NHS). Treatment and support. Huntington’s disease. 13 February 2018. https://www.nhs.uk/conditions/huntingtons-disease/treatment/. Accessed 15 January 2019