Therapeutic Approaches


There are several major therapeutic approaches that may ultimately be useful in the treatment of Niemann-Pick Disease and other similar lysosomal storage diseases. These treatments include enzyme replacement therapy, gene therapy, neural stem cell therapy, and molecular or pharmacological chaperone therapy.  All of these are aimed at restoring enzyme activity.

Other options being explored are substrate deprivation and metabolic bypass therapy which are not aimed at restoring enzyme activity, but at the reduction in the levels of the compounds that accumulate in the lysosomes. These are currently more theoretical approaches to therapy, and researchers still have much to learn. 

Diseases that affect the central nervous system such as Niemann-Pick Type A are the most difficult to treat, and many experts believe that no single approach will be the solution for treating this devastating disease.

Instead, the hope is that some combination of these approaches mixed together will be able to halt, or even reverse, the ravaging effects of NPA and others that ravage the brain.