The Clock is Ticking - Another perspective on research issues

I asked a member of the KDA's board of directors, who is also a biology professor, to comment on the Newsweek Article that I wrote about in "The Clock is Ticking." The article, "Desperately Seeking Cures - How the road from promising scientific breakthrough to real-world remedy has become all but a dead end," focused on why cures and treatments are so difficult to move from the test tube and lab mouse stage to clinical trials and actual usable drugs/treatments.

The professor was fortunate enough to spend a summer working in a Kennedy's Disease lab at Thomas Jefferson University. He came away with a better understanding of the research process as well as the difficulties in dealing with genetic disorders. I thought you would find his thoughts on the subject interesting.

"The article is intriguing and brings up some interesting points, however, it ignores what, in my opinion, is the biggest reason for the lack of drugs for these diseases. Each of the diseases discussed in the article are difficult to cure. They are generally diseases that have no known cellular mechanisms of causation that can be easily corrected. They are not caused, for example, by pathogens that can be treated with antibiotics or immunizations. Almost all of them deal with genetic changes in which the exact reason for the disease is not well understood. Looking at KD, for example, in 1991 it was discovered that KD was due to an elongated CAG repeat in the gene for the androgen receptor. This causes the cells to make an altered form that, for some reason, causes certain cells to die. Even though we knew the actual cause of KD (the mutated gene), we had no idea why this mutation caused KD, nor how to cure it. Over the past 20 years, the researchers (all the ones we know) have struggled to figure out why nerve cells die in KD (just as researchers of Huntington's Disease struggle doing the same thing for HD) with the hope that if it is understood why the cells die; they will be able to stop it. The research has certainly advanced, but it is still not clear what causes the cell death. It is quite difficult to stop something that is not understood. While a treatment may pop up at any time, the reality of the situation is that it will take time. These are not easy diseases.

I do not know enough about patents and drugs to make a real comment, although I have read complaints that out patents are too lenient!

Also, note that one of the items that the article felt was necessary was the sharing of info between researchers - something I believe 'our' researchers already do (and the KDA encourages)."

As I mentioned in my original article, "The Clock is Ticking," I believe there are three sides to every story. The comments above are probably closer to the truth in regards to Kennedy's Disease research. However, for those of us in the sixties, seventies, and eighties, the clock continues to tick.