Recently Novartis group developed a highly specific inhibitor (called compound 3) against PAK1. Its IC50 in vitro is around 5 nM against PAK1、 but 400 nM against PAK2. Unfortunately,however, its IC50 against the growth of pancreatic cancer cells is around 2000 nM. In other words, only one in 400 of this molecule could pass through cell membranes. Thus, it is very unlikely that the compound 3 could be used clinically for cancer therapy.
However, because of this high specificity and affinity for PAK1, it could be potentially used as a ligand for affinity purification of PAK1 from animal cell extracts, if it could be covalently "immmobilized" to beads such as agarose/sepharose beads without a loss of its high affinity for PAK1.
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