Challenges in Developing A Treatment for NAFLD/NASH

Nonalcoholic fatty liver disease (NAFLD) is the leading cause of chronic liver disease and the second most common reason for liver transplantation in the US and Europe. NAFLD is characterized by fat accumulation in the liver not due to alcohol. It affects nearly one quarter of the population, yet displays few symptoms.

The disease progresses into an advanced, irreversible stage called nonalcoholic steatoheptatitis (NASH), which can lead to complications such as fibrosis, cirrhosis, hepatocellular carcinoma, or liver failure. These instances of NASH range from 3% to 5% worldwide. Despite significant annual related health expenditures being over $103 billion only in the United States, there are currently no approved treatments for NAFLD or NASH.

Studying NAFDL/NASH In Vitro and In Vivo

There are multiple obstacles towards developing effective therapies to NAFLD and NASH. In preclinical phases, there is a lack of validated in vivo and in vitro models that can elucidate understanding of NASH pathophysiology, identify new therapeutic targets, and characterize novel drug therapies. In vitro models are utilized to study molecular pathways for target identification, and in vivo studies are utilized to study efficacy, pharmacokinetics, and pharmacodynamics. Developing a comprehensive, translatable model poses a challenge for effective treatment.

Biomarker and Diagnosis Challenges

There is an urgent, global need to address NAFLD/NASH because there are no FDA-approved drugs to treat the disease, and the complex pathophysiology is not completely understood. Some potential targets of interest include antifibriotic and anti-inflammatory therapies due to behavior in the liver.

In addition to therapies, there is a need for sensitive, cost-effective, and noninvasive biomarkers for NAFLD/NASH diagnosis. This is because most NAFLD cases stay undetected and thus untreated until they reach irreversible stages. Finding diagnostic biomarkers that solve these problems will help to diagnose NAFDL in early, still reversible stages, to follow disease progression as well as identify those at risk for developing the disease.

Pushing Research Forward

PerkinElmer provides various tools and NASH research solutions that can overcome the challenges of validating disease models in vitro and in vivo. Download our NASH brochure to learn more. The brochure includes examples of novel approaches that lead to greater understanding of pathophysiology, murine NASH models, and development of diagnostic tools.