Impact of neurodegenerative diseases

Effective treatment of neurodegenerative diseases remains one of the greatest challenges in modern medicine. Many of these diseases are life-threatening. Even if not fatal, they may cause debilitating symptoms and personality changes that are life‑changing for patients and family members. There are many treatments available aimed at managing symptoms; however, to date, none can stop or reverse disease progression. Our mission is to meet this need by developing therapies that target the root causes of these diseases.

Parkinson’s Disease

Parkinson’s disease is a life-threatening neurodegenerative disease that results from a progressive loss of nerve cells in the brain.1,2 A diagnosis of Parkinson’s disease is life-changing for patients, as increasingly debilitating and distressing problems with movement and mental function can develop.1,2 As the disease progresses, patients may require assistance for most activities of daily living, which places a profound emotional and physical strain on family members.1

Although the exact cause of the disease is not fully understood, there is compelling evidence that abnormal deposition of the α-synuclein protein, which is toxic to nerve cells, plays an important role.3

Impact on patients

The progressive symptoms of Parkinson’s disease have a profound impact on the lives of patients. Symptoms include shaking, slow movement, stiff and inflexible muscles, instability, depression, memory loss, sleeping problems, and personality changes.1,2 While the average age at onset is 60 years, some patients are diagnosed much younger.4 This means that a patient’s ability to work may be compromised, as it becomes increasingly difficult to perform daily tasks. Parkinson’s disease can deprive patients of recreational activities that they previously enjoyed, and eventually of their independence

Prevalence

An estimated 7–10 million people worldwide live with Parkinson’s disease5

Impact on family members

Parkinson’s disease places substantial physical, emotional, and financial strain on family members.1 As the disease progresses, a patient’s personality may change,1 which may be particularly distressing for family members. In the final stages of the disease, patients may need assistance for most activities of daily living1

Current treatments

Current treatments are aimed at managing symptoms and maintaining the quality of life of patients for as long as possible; however, there is no treatment that can stop disease progression.6 Treatments often have side effects, which may also require management.1,6 Over time, the need for medication may increase, and adherence to progressively complex dosing regimens may become difficult as a result1,7

New therapies

New therapies for Parkinson’s disease are urgently needed in order to address the root causes of this debilitating disease. We are investigating the use of SAIT to specifically target oligomeric species of α-synuclein protein, which are thought to be the toxic forms of the protein, with the aim of reducing the progression of Parkinson’s disease

Huntington’s Disease

Huntington’s disease is a life-threatening neurodegenerative disease that has a profound impact on both patients and family members.8–10 Patients with Huntington’s disease suffer from increasingly debilitating problems with movement, cognition, and psychiatric function.8–10 During the later stages of the disease, patients may not be able to walk, speak, or swallow food, as they become dependent on caregivers.8–10 The symptoms usually first appear at 30–50 years of age, and it normally leads to death within 10–30 years of symptom onset.9

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.8,10,11

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.8 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 swings8,11

Prevalence

An estimated 3–7 per 100,000 people of European ancestry are affected by Huntington’s disease10

Impact on family members

Huntington’s disease places substantial physical, emotional, and financial strain on family members.8,10 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.8 Patients may become dependent on caregivers before they reach old age8,10

Current treatments

Current treatments aim to ease the symptoms of Huntington’s disease; however, there is no treatment that can stop disease progression.8 Additionally most of these treatments can cause troublesome side effects12

New therapies

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.

Alzheimer’s Disease

Alzheimer’s disease is a common neurodegenerative disease in the elderly, which causes debilitating and distressing symptoms that have a profound impact on patients and family members.13 As the disease progresses, mental function deteriorates, and patients may become unable to recognize loved ones and may develop changes in personality.13 In the final stages of the disease, patients may become totally dependent on care-givers.13

Changes in the brains of patients with Alzheimer’s disease include the abnormal deposition of β‑amyloid protein, Tau, and α-synuclein, which may disrupt nerve cell function and contribute to cell death.14,15

Impact on patients

The progressive symptoms of Alzheimer’s disease have a profound impact on the lives of patients. Symptoms include memory loss, personality changes, communication difficulties, loss of the ability to reason, and loss of conscious thought.13 In the early stages of the disease, mild memory loss and confusion may merely be frustrating, but as the disease develops, symptoms impact all daily tasks until the patient loses independence13

Prevalence

It has been estimated that nearly 44 million people worldwide are living with Alzheimer’s disease or a related dementia.16 This number is expected to rise due to the general aging of the world’s population17

Impact on family members

Alzheimer’s disease places substantial physical, emotional, and financial strain on family members.13 The decline in mental function is particularly distressing for family members. As the disease develops, patients may not be able to recognize loved ones.13 The personality and behavior of patients may change, causing them to become shadows of their former selves. In final stages of the disease, patients may become bed-bound and dependent on caregivers for basic activities of daily living13

Current treatments

Current treatments may help to slow down memory loss, but none can stop progression of the disease itself13

New therapies

New therapies for Alzheimer’s disease are urgently needed to address the root causes of this debilitating disease. We are investigating the use of SAIT to target the β-amyloid protein, in combination with one or more undisclosed targets, with the aim of reducing the progression of Alzheimer’s disease

References

  1. World Health Organization (WHO). Neurological disorders associated with malnutrition. In: Neurological Disorders: public health challenges. 2006. http://www.who.int/mental_health/neurology/chapter_3_b_neuro_disorders_public_h_challenges.pdf?ua=1. Accessed 15 January 2019
  2. National Health Service (NHS). Overview. Parkinson’s disease. 9 June 2016. https://www.nhs.uk/conditions/parkinsons-disease/. Accessed 15 January 2019
  3. Stefanis L. α-synuclein in Parkinson’s disease. Cold Spring Harb Perspect Med 2012;2(2):a009399
  4. Michael J. Fox Foundation for Parkinson’s research. Parkinson’s disease causes. https://www.michaeljfox.org/understanding-parkinsons/living-with-pd/topic.php?causes. Accessed 15 January 2019
  5. Parkinson’s News Today. Parkinson’s disease statistics. 2018. https://parkinsonsnewstoday.com/parkinsons-disease-statistics/. Accessed 15 January 2019
  6. National Health Service (NHS). Treatment. Parkinson’s disease. 9 June 2016. https://www.nhs.uk/conditions/parkinsons-disease/treatment/. Accessed 15 January 2019
  7. Neurology Times. Parkinson medication adherence is suboptimal. 17 February 2015. http://www.neurologytimes.com/parkinson-disease/parkinson-medication-adherence-suboptimal. Accessed 15 January 2019
  8. National Health Service (NHS). Overview. Huntington’s disease. 13 February 2018. https://www.nhs.uk/conditions/huntingdons-disease/. Accessed 15 January 2019
  9. 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
  10. Huntington’s Disease Foundation. Life with Huntington’s disease. 2017. http://www.huntingtonsdiseasefoundation.org/life-with-hd-1. Accessed 15 January 2019
  11. National Health Service (NHS). Symptoms. Huntington’s disease. 13 February 2018. https://www.nhs.uk/conditions/huntingtons-disease/symptoms/. Accessed 15 January 2019
  12. 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
  13. National Institutes of Health (NIH), National Institute on Aging (NIA). Alzheimer’s disease fact sheet. 17 August 2016. https://www.nia.nih.gov/health/alzheimers-disease-fact-sheet. Accessed 15 January 2019
  14. National Institute on Aging (NIH). What happens to the brain in Alzheimer’s disease. 2017. https://www.nia.nih.gov/health/what-happens-brain-alzheimers-disease. Accessed 15 January 2019
  15. Crews L, Tsigelny I, Hashimoto M, Masliah E. Role of Synucleins in Alzheimer’s disease. Neurotox Res 2009;16(3):307–317
  16. Alzheimers.net. 2017 Alzheimer’s statistics. 2018. https://www.alzheimers.net/resources/alzheimers-statistics/. Accessed 15 January 2019
  17. Verdict Hospital (GlobalData Healthcare). Aging population expected to increase the prevalent cases of Alzheimer’s disease. 2 March 2018. https://www.hospitalmanagement.net/comment/aging-population-expected-increase-prevalent-cases-alzheimers-disease/. Accessed 15 January 2019