4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) is a polychlorinated pyridyl
cholesterol carbonate that is lipophilic, electrically neural, crosses the blood brain
barrier (BBB), ability to localize in intracranial tumor tissue, lacks neurotoxicity and not
transported out of the brain via Pgp (p-glycoprotein). DM-CHOC-PEN has completed a Phase I
Adolescent and Young Adult (AYA) trial in humans, some of which possessed primary and
secondary tumors involving the brain. Complete remissions in both primary (astrocytoma, GBM)
and metastatic lung cancers were reported.
This Phase II trial is open for adolescent and young adults (AYA) subjects with advanced
cancer - brain involvement is required.