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From: Dennis Drayna
Date: 13 Oct 2005
Time: 11:03:37 -0500
Remote Name: 165.112.46.123
Hi Alan, Your question may be tongue in cheek, but it's actually a serious one, as the people who support research in stuttering do so for several reasons, one of which is better treatment for this disorders. Having spent 15 years in the pharmaceutical industry, I can also tell you that every day I ask myself the same questions you have posed. The answer depends on what our genetic studies uncover. The power of our approach is that it can pinpoint a specific gene, that encodes a specific component in the body. Such components are what is required to develop a drug, for example. Some of these specific components are what industry scientists refer to as "drug-able", that is, they lend themselves to making a drug in a straightforward manner. A good example here is an enzyme in the body called HMG Co-A Reductase. Don't worry about the name, you just need to know that this enzyme is the key step in our body's synthesis of cholesterol. This component of the body proved to be very "drug-able". Once its role became known, this allowed scientists to produce an entire class of medicines, called statins, that are widely used today to reduce cholesterol levels. However, there are lots of components of the body that have not been suitable to developing a drug, and we don't know what our search for the products of 'stuttering genes" will give us. As for time frame, if we find a gene that shows a straightforward way to develop a drug, the process to do this takes from 10 to 15 years, as it involves basic research, development, manufacturing, clinical trials, and review by the FDA. Just to give you some additional perspective, the cost to do this currently runs about $900 million. Regarding who would benefit from such a drug, the investment would probably only happen if it seemed likely the drug would benefit a large fraction of PWS, not just a single family. Finally, I should mention that finding "stuttering genes" may be helpful in other ways that would be sooner and cheaper. For example, it might allow us to classify PWS to assign them to a particular type of speech therapy that would be most effective. All this is dependent on finding these gene first, however, which is keeping us pretty busy at the moment.