Complete remission of a high risk metastatic rhabdomyosarcoma in an adolescent treated with a liposomal (Myocet) regimen

Abstract

We report the case of a very high risk metastatic rhabdomyosarcoma teenager treated with an induction therapy using only liposomal doxorubicin (Myocet ® ) because of his extremely bad health condition. A near complete remission was obtained after 2 courses. Then he received conventional chemotherapy according to the EpSSG protocol, radiotherapy and a reduced-intensity conditioning regimen followed by pheno-identical allogeneic stem cell transplantation. He is in continuous complete remission with a follow-up of nine years.

Liposome nano‐formulation with cationic polar lipid DOTAP and cholesterol as a suitable pH‐responsive carrier for molecular therapeutic drug (all‐ trans retinoic acid) delivery to lung cancer cells

“…Achieving good entrapment and sustained gradual release of drugs at the target site in a pH‐dependent manner in addition to the EPR effect are the major goals of this study. Many clinical formulations and research studies have used the 5:4:1 ratio for lipo‐drug formulations and also proved effective in many FDA‐approved cancer drugs [ 26 , 27 ]. Cationic liposomes are accepted as pH‐sensitive drug release nanocarriers as they were used in lipoplex and even the neutral lipids like DOPE is conjugated with cationic lipids due to its membrane destabilising at reduced pH, thereby escaping endolysosomal degradation [ 28 ].…”