PILOT STUDY OF GENE THERAPY FOR LOCALLY ADVANCED PANCREATIC ADENOCARCINOMA WITH INTRATUMOURAL INJECTION OF JetPEI/DNA COMPLEXES WITH ANTITUMOURAL EFFECT AND CHEMOSENSITIZING ACTIVITY FOR GEMCITABINE
This is a gene therapy open non randomized phase I trial for advanced and/or metastatic
pancreatic cancer patients. The protocol is based on the administration of increasing doses
of a plasmid DNA pre-complexed to PEI (polyethylenimine - non-viral vector) that encodes two
genes (somatostatin receptor subtype 2 named sst2 and deoxycitidine kinase ::
uridylmonophosphate kinase named dck::umk) which exhibit complementary therapeutic effects.
Both transgenes induce an antitumor bystander effect and render gemcitabine treatment more
efficient. Intratumor injections of the gene therapy product (CYL-02) will be performed by
transgastric or transduodenal route under endoscopic ultrasound guidance. Each injection
will be followed standard gemcitabine IV administration every week (1000 mg/m2). Two
intratumor injections of a same dose of CYL-02 will be administered at one month interval.
Four increasing doses (125 µg, 250 µg, 500 µg and 1 mg) will be tested by group of 6
patients. The primary objectives are: evaluation of local pancreatic and general tolerance;
the secondary objectives are: possible tumor volume regression, secondary respectability,
evaluation of transgene biodistribution.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Feasability and security : Number of Participants with Adverse Events
Feasibility, security (pancreas and general) of intratumor injections of the gene therapy product (CYL-02 plasmid DNA pre-complexed to PEI encoding sst2 dck::umk) administered under endoscopic ultrasound guidance and followed by gemcitabine treatment at standard doses.
Louis BUSCAIL, MD,PhD
University Hospital of Toulouse
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)