OnCo
termsTerm

CNS penetration (brain-penetrant drugs)

aka CNS penetration, CNS-penetrant, CNS penetrant, brain-penetrant, brain penetrant, brain penetration, blood-brain barrier penetration, BBB-penetrant, CNS-active, intracranial efficacy, CNS efficacy, CNS progression, CNS protection, CSF concentration, Kp,uu, efflux transporter, P-glycoprotein substrate

How well a drug crosses from the blood into the brain, where most cancer drugs are kept out by the blood-brain barrier. Brain-penetrant pills can treat and prevent brain metastases; those that are not leave the brain as a sanctuary where the cancer regrows.

Third-generation EGFR (osimertinib) and ALK (alectinib, lorlatinib) inhibitors were designed for CNS penetration and cut brain progression dramatically compared with earlier drugs; tucatinib, and unexpectedly T-DXd, are active against HER2-positive brain metastases; temozolomide, lomustine and vorasidenib are brain-penetrant by design. Penetration is measured as unbound brain-to-plasma ratio (Kp,uu) or CSF concentration and is limited by efflux pumps (P-glycoprotein, BCRP). Intracranial response rates and CNS progression-free survival are now standard secondary endpoints in lung and breast cancer trials, and intrathecal or intraventricular delivery bypasses the barrier for leptomeningeal disease.

Category
Treatment jargon

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