Repurposing AKT Inhibitors for the Treatment of Protein Misfolding and Protein Trafficking Diseases

Case ID:

WSU researchers have generated a reagent to understand the degenerative processes occurring in Pelizaeus‑Merzbacher disease (PMD) and to study the intracellular signaling that occurs because of these diseased processes.  They have deduced that the use of AKT inhibitors may reduce disease severity in patients with a wide range of degenerative diseases.

Background & Unmet Need:

Currently there is no treatment for PMD (Pelizaeus‑Merzbacher disease), a neurodegenerative disease that affects motor and intellectual function.  Treatments for many other protein misfolding/trafficking diseases are relatively ineffective (multiple sclerosis), highly invasive and risky (Parkinson’s disease), or non-existent (amyotrophic lateral sclerosis).  Chemical inhibitors of the pro-survival protein kinase, AKT, are currently in pre-clinical trials for the treatment of cancers.  Inhibiting AKT, which is a central player in cell growth, is hypothesized to reduce or eliminate cancer growth.  Inhibition of AKT may also be important in preventing protein misfolding/trafficking commonly seen in neurodegenerative disorders.


Technology Description:

Our researchers have introduced the protein inhibitor of AKT, called Trb3, into a mouse model using a transgene.  They have also introduced Trb3 into an animal model for PMD.  The treated animals have fewer disease symptoms than the untreated animals, showing that by inhibiting AKT, one can ameliorate the symptoms of PMD and other diseases with similar or analogous pathophysiology.  Our researchers are also looking to see which AKT pathway inhibitors might be best at minimizing retinal cell damage and loss.


Commercial Applications:

  • Potential to develop chemical or protein-based inhibitors of ATK to treat neurodegenerative diseases


Stage of Development:



Competitive Advantages:

  • Potential treatments may be less invasive and less risky than existent therapies


Intellectual Property Status:

Patent application filed


Related Publications or Citations of Work:

Flores, AI, Mallon BS, Matsui T, Ogawa W, Rosenzweig A, Okamoto T, Macklin WB. (2000) Akt-mediated survival of oligodendrocytes induced by neuregulins. J. Neurosci. 20:7622-30.


Patent Information:
For Information, Contact:
Ken Massey
Wayne State University
Alexander Gow
Cherie Southwood
Alzheimer's Disease