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Alzheimer's disease (AD); neurology

3-Phosphoinositide-dependent protein kinase-1 (PDPK1)

Mouse and cell culture studies suggest inhibiting PDPK1 activity could help treat AD and prion-associated diseases. In prion-infected, cultured mouse neurons and neurons from mice with amyloid plaques, a PDPK1 inhibitor led to higher levels of neuroprotective amyloid precursor protein (APP) and cellular prion protein (PRNP; PrP; CD230) cleavage products than no treatment. In mouse models, the PDPK1 inhibitor decreased both AD-specific behavioral impairments and prion disease-specific motor impairments compared with no treatment. Next steps include testing other PDPK1 inhibitors and analyzing how PDPK1 is activated by pathogenic prions and amyloid peptides.
Arno Therapeutics Inc.'s AR-12, a small molecule inhibitor of PDPK1, is in Phase I testing to treat lymphoma and solid tumors.
Phusis Therapeutics Inc.'s PHT-427, a small molecule inhibitor of PDPK1, is in preclinical development to treat cancer.

SciBX 6(36); doi:10.1038/scibx.2013.1001
Published online Sept. 19, 2013

Patent and licensing status undisclosed

Pietri, M. et al. Nat. Med.; published online Aug. 18, 2013;
Contact: Benoit Schneider, University Paris Descartes, Paris, France
Contact: Jean-Marie Launay, Lariboisière Hospital, Paris, France