In a recently published article from Washington University in St. Louis, researchers propose a new treatment approach for NPC.

The article talks about a discovery that has led researchers to suggest that chemical compounds could potentially “chaperone” mutant protein molecules though cells. The chemical compounds, which act as chaperones, allow the mutant protein to pass through the cells quality control system, and do its job of moving cholesterol out of cells.

Whilst this is very exciting news and may lead the way to potential new treatments for NPC, it is unlikely that these treatments would be available in the short term.

As with all these things it is not possible to give an exact time scale. Suitable compounds would need to be identified and then these compounds would have to be developed by a pharmaceutical company. Before a drug could be used on patients, it would need to go through animal testing and then Phase I studies, which is the first stage of use in humans. This would initially be done using healthy volunteers. This is a very important part of the process and it can take quite a long time to ensure that the compound is safe and well tolerated. The correct dosage would also need to be ascertained. Following this, a clinical trial would have to be developed and patients would need to be recruited. When a trial is finished and the data collected, then the pharmaceutical company would need to apply for a licence to market the drug. All this can take quite a long time!

The news of chemical chaperone therapy as a possible treatment of NPC is, however, very positive and exciting news for all of us who are hoping and praying for an effective therapy for this disease.

You can be sure that the Niemann-Pick Disease Group (UK) will continue to keep up to date with developments in this area and will give you feed back on any information we have.

You can read the article at http://mednews.wustl.edu/news/page/normal/10988.html