Pharmaceutical companies are rushing to fill the NASH treatment gap
Categories: For Potential Participants, [Nonalcoholic Steatohepatitis, Drug development, Elafibranor (GFT505), Liver disease, Obeticholic acid (OCH)]
Nonalcoholic steatohepatitis (NASH) is a liver disease caused by a buildup of fat in the liver that may result in cirrhosis (liver scarring), liver failure, and possibly cancer. Nonalcoholic steatohepatitis–related cirrhosis is likely to become the main cause for liver transplantation. Nonalcoholic steatohepatitis has drawn more attention in recent years after not being a focus for clinical research for a long time. The reason for this lack of interest was because NASH is largely asymptomatic (i.e. shows no symptoms) and because the disease progression is slow and covert. In addition, the diagnosis relies on liver biopsies that may cause pain and discomfort. Apart from some recommended lifestyle changes (e.g. diet, exercise, and weight loss) and control of diabetes, there is no medication approved for NASH treatment so far, but this may be about to change.
A number of new drugs are being investigated for their potential to reverse liver disease and to reduce the risk for further damage.
A lot of attention surrounds obeticholic acid (OCA), a drug currently in clinical development. Obeticholic acid is a man-made version of a naturally occurring acid normally found in the liver, which can lower the levels of certain liver enzymes associated with inflammation, an early event in cirrhosis. Obeticholic acid positively influences the metabolism of fats and sugars in the body. However, the compound is known to cause itching and elevated “bad cholesterol” at higher doses. This may limit its application and calls for more research. In 2016, OCA received approval from the US Food and Drug Administration for the treatment of another chronic liver disease – primary biliary cholangitis – in which the bile ducts in the liver are slowly destroyed.
Elafibranor (GFT505) is another potential new drug in development, which may help people with severe forms of NASH. Previous clinical studies indicated that elafibranor improves blood sugar balance and increases levels of “good cholesterol.” An ongoing clinical trial in approximately 2000 patients around the world will provide further information about the potential for elafibranor for the treatment of NASH.
Other potential drugs in development include TBE-31, which has been tested in mice so far. TBE-31 was found to activate a protein that can switch on the natural antioxidant defense system within liver cells. In other words, damaging processes such as oxidative stress, inflammation, and fibrosis were neutralized. Further research is now required to figure out if the same outcomes can be replicated in humans and if the use of TBE-31 will treat NASH and liver fibrosis.
An approach combining 2 potential treatments for NASH is also being researched, as 2 big pharmaceutical companies have teamed up to target different components of the disease at the same time – fibrosis and key metabolic processes in the liver. The research is in the early stages of clinical trials and needs to assess how safe the combination treatment is and whether it works.
Although there are no approved drugs for NASH, many pharmaceutical companies are in different stages of developing potential new therapies.
Interested in learning more about NASH? Register on ClinicalResearch.com to receive news about NASH and related IQVIA clinical trials. Check ClinicalResearch.com often for updates and news on a variety of disease areas and to find out more about some of the latest clinical research trials.References