There are some drugs on the market that achieve promising results in comparable diseases. These include CPHPC, flupirtin and memantine. These drugs have been used either in diseases with similar symptoms or in cell cultures of NCL patients. However, so far it has only been possible to demonstrate that the biochemical symptoms of the diseases are reduced.

An enzyme replacement therapy would be conceivable only for infantile (CLN1) and late infantile (CLN2) NCL because the affected enzymes are soluble. This concept is not feasible for the juvenile form, since the deficient protien is situated in the lysosomal membran.

 

To directly combat the cause, it will ultimately be necessary to repair the gene defect on chromosome 16. This could be done with a gene therapy.

 

Initial promising results suggest that immune suppression possibly could have a positive effect on the course of the disease. Inflammatory phenomena in the brain apparently contribute to the pathogenesis of lysosomal storage diseases in general.

The following figure shows were possible targets of treatments might be found.