aerosol gemcitabine

Aerosol gemcitabine is an investigational formulation of the nucleoside analog chemotherapy drug gemcitabine, specifically developed by the M.D. Anderson Cancer Center for delivery via nebulization. This localized delivery method is designed to treat pulmonary metastases from various solid tumors, most notably osteosarcoma, by achieving high drug concentrations directly in the lung tissue while minimizing systemic toxicity. Gemcitabine functions as a pyrimidine antimetabolite; once phosphorylated intracellularly, it inhibits DNA synthesis by competing with cytidine for incorporation into DNA and by inhibiting ribonucleotide reductase, which depletes the pool of deoxynucleotide triphosphates. Research indicates that this aerosolized delivery also triggers apoptosis in metastatic cells through the Fas/FasL pathway. Clinical development has primarily focused on Phase I dose-escalation studies to evaluate safety and tolerability in pediatric and adult patients with refractory lung metastases.

Other names

nebulized gemcitabineinhaled gemcitabinegemcitabine aerosol