BUFFALO, N.Y. — POP Biotechnologies Inc. (POP BIO) received a $2M Phase II STTR under award number 2R42CA243954-02, supported by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), to pursue development of a unique treatment for hepatocellular carcinoma (HCC), or liver cancer.

Patients with inoperable locally advanced hepatocellular carcinoma (HCC) have a 5-year survival rate of just 31%.

The goal of this proposal is to provide a proof of concept that a novel interstitial chemo-phototherapy (I-CPT) with long-circulating, doxorubicin in porphyrin-phospholipid liposome formulation (PhotoDox) is a potent antitumor therapeutic option for locally advanced HCC.

There are limited treatment options for the treatment of locally advanced HCC. Thermal ablation is used to treat non-resectable HCC that involves heating target tumor volumes to temperatures near boiling. Despite the potency of thermal ablation, treatment volumes can be difficult to define, and proximity to blood vessels can induce local cool zones.

This proposed combination of chemotherapy and phototherapy (CPT) involves systemically administering a single agent, PhotoDox, which is a novel long-circulating, porphyrin-phospholipid liposomal formulation of doxorubicin. PhotoDox has numerous characteristics that are conducive to clinical translation of CPT. Photodox combines the well-established advantages of spatially targeted drug delivery with photodynamic therapy, a light-based cancer therapy pioneered at Roswell that has been demonstrated as both a curative and palliative treatment. While numerous recent studies have examined nanocarriers capable of light-triggered drug release, PhotoDox is unique in that it achieves drug release that: 1) is thermally independent; 2) uses 665-nm light, enabling defined tissue penetration; 3) has rapid drug release upon light administration; 4) is long-circulating (~22 hour half-life), serum stable, and otherwise like DOXIL®; and 5) has been demonstrated to be effective in multiple tumor models.

In the NIH-funded project, POP BIO will partner with Gal Shafirstein’s group at the Roswell Park Comprehensive Cancer Center