THERATARGET, INC. — Department of Health and Human Services STTR Phase II: 102

Description

SUMMARY This STTR Phase II proposal addresses the significant need for improved treatment options for patients with liver cancerthe fifth highest incidence of cancer in the worldBecause of the lack of symptomshepatocellular carcinomaHCCis detected at advanced stages inof casesfor which theyear survival rate isand atyears it isThe only curative option for advanced HCC is surgical liver resection and liver transplantationunfortunately not available to most patients due to the lack of donor livers and the rapid progression of the diseaseAs HCC is generally unresponsive to systemic chemotherapytranscatheter arterial chemoemobolizationTACEis the most widely usedlocalized treatment that can slow the progression of the diseaseCurrent embolizing agents are deficient in precision of catheter delivery or compatibility for effective delivery of chemotherapeutic agentsespecially high molecular weight biotherapeuticsThe objective of the proposed work is to characterize the novel liquid embolizing agent composed of the genetically engineered protein polymerSELPsilk elastinlike proteinKwhich based on our previous work has demonstrated properties uniquely suited for this applicationUnlike existing agentsSELPK will be injectable as a liquidable to penetrate into the tumor arteriesand transform to an insoluble hydrogel in situ forming a substantially durable occlusionThe embolizing liquid will be completely aqueous and compatible with drugs and new biotherapeuticsenabling their localized controlled releaseThe protein based SELPK will eventually biodegradeenabling subsequent TACE treatmentsSELPK liquid embolic will enable the controlled delivery of chemotherapeutic drugs and new biotherapeutic agents with increased precision of transcatheter delivery for more selective embolizationreduced off target toxicityand reduced collateral damage to the healthy liverConsequentlyTACE treatment will be offered to a larger patient population having a greater number of tumors and or greater tumor sizeThe aims of the research areto characterize the delivery of single and multiple drugs via the SELPK gel networkto conduct in vivo studies in the McA RHHCC liver tumor rat model to evaluate therapeutic performanceto conduct SELPK manufacturing and analytical methods developmentandto conduct GLP preclinical toxicology and performance testing of manufactured SELPK embolic NARRATIVE This STTR Phase II proposal details the rationale and the research plan for the development of a novel liquid embolizing agent composed of the genetically engineered protein polymerSELPsilk elastinlike proteinKfor treatment of hepatocellular carcinoma by transcatheter arterial chemoembolizationTACESELPK embolic will improve the precision of embolizationthe compatibility with newly developed drugsand the selectivity of tumor specific therapyConsequentlyTACE treatment will be offered to a larger patient population having a greater number of tumors and or greater tumor sizefor which few treatment options exist