Introduction to Targeted Therapy and Small Molecule Inhibitors

A set of vocabulary flashcards covering the definitions of targeted therapies, biologics, companion diagnostics, and specific small molecule inhibitors including their mechanisms and naming conventions.

Small Molecule Inhibitors

Low molecular weight chemical compounds that bind to proteins and inhibit either enzyme activity or protein-protein interactions.

Biologics

Active substances made by or derived from a biological source (e.g., proteins produced using biotechnology in cell culture) rather than being synthesized chemically.

Companion Diagnostic

An in vitro diagnostic device or test carried out before treatment to assess a diagnostic or predictive biomarker, ensuring the safe and effective use of a targeted therapy.

Cytotoxic ADRs

Adverse drug reactions associated with conventional systemic therapies, specifically bone marrow suppression, GI effects, mucositis, alopecia, and infertility.

Immunosuppressant ADRs

Adverse drug reactions including GI effects, renal failure, hepatoxicity, and infections.

Gefitinib

A targeted cancer chemotherapy that binds to the ATP binding site of the tyrosine kinase domain of $EGFR/Her1$ ($ErbB-1$).

Baricitinib

A selective and reversible inhibitor for $JAK1$ and $JAK2 > JAK3$ used as an immunosuppressant to reduce $STAT$-mediated gene expression.

Vemurafenib

A targeted cancer chemotherapy that binds to the ATP binding site of the constitutively active mutant $V600E$ B-Raf when the enzyme is in its active conformation.

L858R

A specific mutation in the kinase domain of the receptor that, along with exon deletions or overexpression of $EGFR$, leads to greater efficacy of Gefitinib.

-ib

The stem used in naming conventions to identify a drug as a small molecule inhibitor.

-tinib

The naming stem indicating a small molecule inhibitor that targets tyrosine kinase (e.g., Gefitinib).

-rafenib

The naming stem indicating a small molecule inhibitor that targets $RAF$ kinase (e.g., Vemurafenib).

-zomib

The naming stem indicating a small molecule inhibitor that targets the proteasome (e.g., ixazomib).

-ciclib

The naming stem indicating a small molecule inhibitor that targets cyclin-dependent kinase (e.g., palbociclib).

Janus Kinase (JAK)

A family of tyrosine kinases that bind to the intracellular domains of tyrosine kinase-linked receptors and phosphorylate $STAT$ transcription factors upon activation.