BASE:PREVENT PRECLINICAL DRUG DEVELOPMENT PROGRAM: PRECLINICAL EFFICACY&INTERMEDIATE BIOMARKERS TASK ORDER: TARGETING STAT3 TO PREVENT HEPATOCELLULAR

NIH RePORTER · NIH · N01 · $843,496 · view on reporter.nih.gov ↗

Abstract

Non-alcoholic steatohepatitis (NASH) is a type of non-alcoholic fatty liver disease (NAFLD) that primarily arises in obese and diabetic individuals. About a quarter of people with NASH ultimately develop cirrhosis, which is the main risk factor for hepatocellular carcinoma (HCC). HCC is the second cause of cancer-related mortality worldwide, and HCC associated with NASH is one of the fastest growing causes of cancer-related deaths in the United States, due in large part to the burgeoning obesity and diabetes epidemics. Prevention is, therefore, an urgent unmet need and an important priority to reverse the current trend of increasing incidence in this country. Signal transducer and activator of transcription 3 (STAT3) is one of the seven members of a family of transcription factors that regulates cell proliferation, differentiation, apoptosis, and the immune response. Although the activation of STAT3 is transient and highly regulated in normal cells, it is constitutively active in several types of cancer, including 50% of HCC. Recent findings from Jung et al. support a role for STAT3 in NASH-associated HCC and suggest that targeting STAT3 with the small molecule C188-9 (also known as TTI-101) could be a successful preventive intervention. TTI-101, developed by Dr. David Tweardy and colleagues and licensed to Tvardi Therapeutics, binds to the SH2 domain of STAT3 with high affinity and inhibits the protein’s dimerization and phosphorylation. It does not target other tyrosine kinases, provides excellent plasma exposure following oral administration, and produces no detectable toxicity when administered for a period of 28 days in rats and dogs. It is currently being evaluated in a Phase I clinical trial in patients with advanced cancers. The purpose of this Task Order is to evaluate TTI-101 for the prevention of HCC associated with NASH in a mouse of model of NASH-associated HCC.

Key facts

NIH application ID
10201174
Project number
75N91019D00021-0-759102000001-1
Recipient
UNIVERSITY OF TX MD ANDERSON CAN CTR
Principal Investigator
DAVID TWEARDY
Activity code
N01
Funding institute
NIH
Fiscal year
2020
Award amount
$843,496
Award type
Project period
2020-06-01 → 2022-11-30