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Treatmenting Lysosomal Storage Diseases
A new study reports a possible breakthrough for treatment for some lysosomal storage diseases. The study finds two drugs -- diltiazem and verapamil -- could prove to be beneficial to patients.
The research was conducted at Scripps Research Institute in La Jolla, California. Lysosomes are organelles that break down macromolecules in a cell and this process is critical for maintaining healthy cells. A lysosomal storage disease results from deficient activity of the hydrolytic enzyme. This enzyme is responsible for the breakdown of defunct molecules. Currently, lysosomal storage diseases are treated by enzyme replacement therapy. The problem is the enzyme has to find a proper way into cells and lysosome to function. Also, enzyme replacement therapy will not work in the brain.
In the latest research, investigators show that diltiazem and verapamil can restore activity of enzymes of three distinct lysosomal storage diseases. These drugs are FDA approved channel blocking drugs. Study authors say also diltiazem crosses the blood-brain barrier, so it may also be useful for neuropathic lysosomal storage diseases.