Development of a specific targeted therapy for hepatocellular carcinoma – Year two

The global burden of hepatocellular carcinoma (HCC) is increasing as it has become the second leading cause of cancer-related
deaths and the fifth most common cancer worldwide. HCC is heterogenous, arising in the setting of various chronic liver diseases
such as hepatitis C, non-alcoholic fatty liver disease and cirrhosis. Patients with cirrhosis who develop cancer cannot tolerate therapy
due to inadequate liver function. We hypothesize that PorphyHDL, a carrier that behaves like a lipoprotein (fat transporter), will
effectively deliver a small interfering RNA targeting spalt-like transcription factor 4 (SALL4), a fetal oncoprotein expressed in
aggressive HCCs. We will partner with Highland Therapeutics Inc., which has designed a novel oral drug delivery technology known
as DELEXIS® and has successfully commercialized a DELEXIS methylphenidate formulation in the US. Our partner will utilize this
technology, which precisely controls time of release and rate of release of an active ingredient, to design a DELEXIS-PorphyHDLSALL4 formulation. This formulation will be targeted to specific regions of the gastrointestinal (GI) tract to facilitate uptake into the
hepatic portal vein, with the aim of delivering drug directly to HCC tumors and avoiding side effects associated with systemic
circulation.

Faculty Supervisor:

Mamatha Bhat

Student:

Partner:

Ironshore Pharmaceuticals

Discipline:

Life Sciences

Sector:

Health and Related Sciences & Technology; Nanotechnology; Pharmaceuticals

University:

University of Toronto

Program: