This article is from Wired Science a blog network.
By Brandon Keim August 27, 2007 | 4:41:32 PMCategories: Gene Therapy, History, Medicine & Medical Procedures
Murray When something goes wrong in a gene therapy trial, it’s front page news — yet when something goes wrong in a trial involving a drug or surgical technique, people hardly even notice.
This double standard is something that frustrates many researchers, and with good reason. They’re worried that the field will wither before it has a chance to succeed. Even though gene therapy research is nearly two decades old, other now-mainstream treatments took just as long if not longer to develop.
As Theodore Friedmann, former head of the American Society of Gene Therapy, wrote in the Hastings Center’s Bioethics Forum:
Modern medicine relies on many forms of therapy that, during their development, went through long periods of setbacks and failure before the underlying conceptual and technical unknowns were solved. These periods often included patient injury and even death. But today’s medicine would be impoverished if it lacked these important technologies – disease-fighting techniques such as bone marrow transplantation, cancer chemotherapy, and the use of monoclonal antibodies for the design of targeted drugs. Bone marrow transplantation – which now saves so many lives – began clinical trials in 1958. By the early 1970s, the survival rate from the procedure was only around 1 percent. It was 32 years before the technology earned its principal discoverer, Professor Donnel Thomas, a Nobel Prize in medicine. Similarly, chemotherapy for childhood leukemia began in 1948, but the cure rate remained below 10 percent well into the 1960s.
I’m just finishing up a feature on gene therapy and its use in diseases that aren’t immediately life threatening. This double standard is something that came up a few times in interviews, and — frustratingly — there wasn’t space in the article to do it justice.
But that’s the beauty of blogs, and I wanted to take a moment to talk about this issue.
Now, I don’t mean to imply that tragedies like that of Jesse Gelsinger or — quite possibly — Jolee Mohr should be downplayed or written off as inevitabilities. Just because experimental therapies are are risky doesn’t mean those risks should be minimized.
Scientists are right, however, that the public fear of gene therapy and other biotech therapies is exaggerated. Unfortunately, that’s just the way it is — unfair, but unavoidable.
“Like it or not, gene therapy has fallen into a situation where it’s been high-profile, heavily hyped for promise, and wound up with no real demonstrable successes after more than a decade of effort,” University of Pennsylvania bioethicist Arthur Caplan told me during an inteview for the story.
As a result, said Caplan, gene therapy is at a crossroads. Will its promise be fulfilled, or be abandoned just short of success? It’s quite possible that gene therapy, treated with suspicion by investors and the public and journalists, will lose funding and interest, with researchers electing to pursue other, less-controversial fields.
If that happens, it would likely be tragic. But at the same time, the examples — particularly that of organ transplantation — that are used by scientists and doctors who counsel patients involve life-or-death conditions. If early organ transplants had been tested on people who had other options, the resulting outcry could have halted research altogether.
However frustrating it might be, the prudent course is to focus gene therapy research on critical conditions, and take extra care when designing trials for treating less-severe conditions. In some cases — such as moderate rheumatoid arthritis — regulators might be well-advised to temporarily reject gene therapy altogether.
This is bound to frustrate many people, and perhaps it will slow gene therapy research even more. But in an environment where mistakes are inevitable and high-profile, it may be the only way for gene therapy to survive long enough to succeed.
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