New Gene Therapy May Reverse Hemophilia

New Gene Therapy May Reverse Hemophilia

12.12.11 | By

Saw this story about possible new therapy to reverse hemophilia in a couple of outlets over the weekend and wanted to pass it along. One of the stories in the New York Times struck me as particularly interesting because the potential advance against hemophilia using gene therapy was written about as an example of how, after much un-fulfilled promise, innovative potential gene therapies are again exciting researchers.
Certainly, the news that there may be a great advance in treating and effectively curing hemophilia in the offing is great and important. However, what I also see in this and other stories like it - and am frustrated by - is a failure by reporters to fully communicate that research and development always takes time. There are always scientific, technological and other setbacks along the way as any potential therapy or new medical technology is researched and developed, but if the concept is right it will, it seems to me, eventually result in progress.
I keep thinking, for example, of HIV/AIDS treatments. 30 years ago, there were none and the disease was undefined and un-described in the medical literature. Today, there are medicines that for many patients effectively make HIV/AIDS a chronic though treatable condition and there is real hope that a cure can be found. That took 30 years. There were lots of scientific ups and downs along the way. But today progress is undeniable.
In the end, my point is that we should all cheer-on this apparent progress in the fight to find a treatment for hemophilia and in reviving research into potential genetic therapies. At the same time, while we all also want big scientific, game-changing therapeutic breakthroughs, we shouldn't ever lose sight of the fact that most medical progress is incremental. It takes time and it takes biopharmaceutical research companies and others willing to invest in research for the long haul.

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