Question

A new macromolecule was identified that seems to be common in patients having a certain type of tumors, and you are searching for an inhibitor. The functions and structure of the macromolecule are unknown and you do not have time/resources to investigate them

1.1. What will be your strategy to search for the lead small molecule inhibitor?

1.2. What are some of the advantages (name at least 3) and disadvantages (name at least 3) of the approach you suggested to use in 1.1.?

Answer 1

Targeted treatment strategies are being studied in cancer therapies where compounda are administered orally through the plasma membrane and interact with the cytoplasmic contents of the receptors and the signaling molecules.

Advantages: since they are administered orally, there is a less harm to normal cells other than cancerous cells; few side effects; very effective

Disadvantages:; Low affinity and lifetime; side effects like hepatitis and other liver problems; skin problems; expensive