L: Antineoplastic and immunomodulating agents
Agents used to kill malignant cells and to push the immune system either way, from cytotoxics through targeted small molecules to antibodies and cell products. The endpoint that decides the program, survival, reads out late and is linked to exposure only through intermediates such as tumor size [1].
On 27 August 1942 a Yale team under Louis Goodman and Alfred Gilman gave intravenous nitrogen mustard, a chemical warfare agent, to a factory worker with lymphosarcoma. Ten daily doses left no tumor on biopsy, though it returned by day 49 [2].
| Level 2 code | Description | In plain terms | Example drug |
|---|---|---|---|
| L01 | Antineoplastic agents | drugs that kill or block tumor cells | Pembrolizumab (L01FF02) / trastuzumab (L01FD01) |
| L02 | Endocrine therapy | hormone-blocking therapy, mainly breast and prostate cancer | Tamoxifen (L02BA01) / anastrozole (L02BG03) |
| L03 | Immunostimulants | Filgrastim (L03AA02) / pegfilgrastim (L03AA13) | |
| L04 | Immunosuppressants | Tacrolimus (L04AD02) / adalimumab (L04AB04) |
Modeling notes
Mechanistic extrapolation. PBPK is routine for kinase inhibitors, predicting CYP3A and gastric-pH interactions that reach labels; QSP extrapolates immuno-oncology dosing, notably CD3 bispecific T-cell engagers, from preclinical data [3,4].
E-R Exposure-response for targeted agents is drug-specific, not class-wide: no efficacy relationship was detectable across the osimertinib dose range [5], while trough concentrations separated progression-free survival for crizotinib and alectinib [6].