Non-alcoholic Steatohepatitis (NASH)
What is NASH?
NASH is a progressive form of chronic liver disease that is one of the leading causes of liver cirrhosis in the U.S. NASH constitutes about 10% of newly diagnosed cases of chronic liver disease in the U.S. and affects 2% to 5% of the population. Diagnosis occurs mostly in overweight adults and children, yet NASH can also occur in individuals with normal weight.
Generally, there are no symptoms in the beginning stages of NASH, so testing typically occurs after a blood panel shows elevated liver enzyme levels that cannot be explained by the use of medication or excessive alcohol. NASH is suspected when x-rays or other imaging procedures show excessive fat in the liver. Final diagnosis is made when a liver biopsy confirms inflammation and liver cell damage. NASH can lead to scarring of the liver, cirrhosis and eventually liver failure. This process can take years or even decades, at which point a liver transplant may be necessary.
Current Standard of Care
At the present time, there are no therapies to treat NASH. General lifestyle changes that are recommended to stop progression of the liver disease include:
- Weight loss
- Healthy diet
- Exercise
- Avoidance of alcohol
Research and Development for NASH
Because of its rapidly growing incidence in developed countries, NASH is an area of active research and development by clinicians and pharmaceutical companies. Some studies have shown that reduction of oxidative stress in the liver with anti-oxidants may result in improvements in NASH patients as determined by imaging and liver enzyme measurements.1,2,3
Raptor is investigating the use of its DR Cysteamine for the potential treatment of NASH. Cysteamine, which is readily taken up in the liver, is a precursor of glutathione, a potent liver anti-oxidant. Recent data from the Phase 2a clinical trial indicated that the study achieved the primary endpoint: mean blood levels of alanine aminotransferase ("ALT"), a common biomarker for NASH, were reduced by over 50%. Additionally, over half of the study participants had achieved normalized ALT levels by the end of the treatment phase.
Additional Resources
- National Liver Foundation
- Non-alcoholic Steatohepatitis Clinical Research Network
- National Digestive Diseases Information Clearinghouse (NDDIC)
- NIH List of NASH Studies
References:
- Contos MJ, Sanyal AJ. The clinicopathologic spectrum and management of nonalcoholic fatty liver disease. Adv Anat Pathol 2002;9(1):37-51.
- Franzese A, Vajro P, Argenziano A, Puzziello A, Iannucci MP, Saviano MC, et al. Liver involvement in obese children. Ultrasonography and liver enzyme levels at diagnosis and during follow-up in an Italian population. Dig Dis Sci 1997;42(7):1428-32.
- Lavine JE. Vitamin E treatment of nonalcoholic steatohepatitis in children: a pilot study. J Pediatr 2000;136(6):734-8.