Treatment of Lysosomal Storage Diseases and Muccopolysacaharidoses with Tyrosine Kinase Inhibitors

Categories: “Neurological Disorders” 

Reference #: 2017-017

OTC Contact: Ruchika Nijhara, Ph.D., MBA, CLP (Directory Information | Send a Message)


The invention is a method of treating lysosomal storage disorder (LSD) by administering FDA approved tyrosine kinase inhibitors (TKI) to activate the autophagy-lysosome pathway thereby clearing toxic proteins, reducing neuronal death and reversing behavioral deficits associated with LSDs.

Lysosomal diseases are a group of inherited metabolic disorders, that are typically fatal. They are caused by defective proteins that are critical to the function of lysosomes. Impaired lysosomal activity in turn leads to the accumulation of undigested or toxic material with a cell, causing dysfunction and even cell death. Affected children suffer from progressive neurological deterioration. In the present invention Georgetown University researchers demonstrate TKI mediated activation of the autophagy-lysosome pathway to clear toxic and/or undigested material, thereby reducing neuronal cell degeneration or death


  • Treating LSD patients – The effects of TKIs on lysosomal clearance leads to removal of toxic or accumulating proteins


  • TKIs encompassed in the invention can cross can cross the blood brain barrier
  • TKIs increase lysosomal genesis to accelerate lysosomal clearance in the brain and peripheral tissues

Stage of Development

Validating in animal models of the disease


Charbel E. Moussa

Patent Status

Provisional application has been filed.