It has come to the knowledge in the recent days that through the utilization of a quantitative -throughput screening strategy, the eminent researchers of the National Institutes of Health or the NIH have been able to categorize three new classes of small molecules that has the potential to be proved useful for the treatment of the Gaucher disease. It is to be noted in this respect, that the Gaucher disease is an inherited disorder, quite efficient in the dislocation of a cell's ability to break down and also to organize certain cellular waste products. However the ongoing treatment of the Gaucher disease has the need of expensive and inconvenient intravenous enzyme infusions that happen to be in the command of many symptoms.

In general, the Gaucher disease occurs within an individual when he/she become heir to two defective copies of the gene that incorporates the code for an enzyme known as the glucocerebrosidase. In due course of action, the enzyme operates in a specified part of the cell   known as the lysosome. In this sphere the cellular components are broken down, or metabolized, for the purpose of recycling. It is also perceived that this specific enzyme deals with the normal metabolism of the glucocerebroside, a glycolipid comprising both a fatty acid and a sugar. On the contrary, with the discovery of the flawed enzyme the glycolipid is found amassed in some parts of the body. This include the spleen and liver which in its turn tend to rise painful and disruptive swelling, and the other being the bone marrow which results in stumpy blood counts and bone fragility and pain some of them are also at times found to affect the brain resulting into the commencement of the neurological problems.       

Dr. Elias A. Zerhouni, M.D., Director of the National Institutes of Health said in this respect, "This discovery is exactly the kind of advance that I envisioned when we launched the NIH Roadmap for Medical Research. Until the NIH Chemical Genomics Center (NCGC) was created as part of the Molecular Libraries Screening Center Network, researchers in the public and academic sector lacked the tools commonly used by the pharmaceutical industry to find leads for possible new treatments such as these. The discovery of three new classes of compounds to potentially reverse Gaucher disease is a proof of principle."