GENE THERAPY researchers are to run before they can walk, says an expert panel advising the US National Institutes of Health. Many researchers try out treatments before they understand the basic science behind the diseases they hope to cure. The panel, commissioned to advise the NIH on its spending, says less money should go on clinical trials and more on basic science.
In a report published last week, the panel says that many researchers have rushed to start clinical trials prematurely. As a result, not one of the 100 trials so far has resulted in an effective treatment. 鈥淭oday, the announcement of a gene being discovered is tantamount to the belief that gene therapy exists for the condition. We鈥檝e seen an extrapolation from hope to hype. In the long run this will be destructive to basic clinical science,鈥� says Harold Varmus, director of the NIH.
Last year, the NIH spent $181 million on research into gene therapy, $32 million of it on clinical trials. Industry is estimated to have spent another $200 million on research.
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In the long term gene therapy will almost certainly come up with effective treatments, says Stuart Orkin, a researcher at the Howard Hughes Medical Institute who is one of the panel鈥檚 chairmen. But in their haste to begin clinical trials, researchers 鈥渞isk losing sight of other strategies for treating the disease鈥�. Often, they need to spend more time determining how the faulty gene causes a disease, and if other treatments could be effective.
The panel also says many clinical studies are poorly designed, with no clear hypothesis to be tested. The NIH should spend more time and money investigating possible new ways of ferrying healthy genes into a patient鈥檚 body. Most teams use one of a few genetically engineered viruses as shuttles. But these vectors are often ineffective, passing along very few or even none of the replacement genes.
Orkin and Varmus both called for researchers to be 鈥渕ore circumspect鈥� when talking to the media. They warned that if gene therapy is overhyped, the public will feel let down when it sees no immediate benefits.
William Guggino, director of the Hopkins Gene Therapy Center in Baltimore, agrees that more basic research is needed. But he warns that the importance of clinical trials should not be underestimated. 鈥淪ome of the clinical trials are very worthwhile in that they did point out some of the problems. Very useful information came out of these trials,鈥� he says.