SAN FRANCISCO -- A federal review panel has cleared the way for the first commercial test of gene therapy, a potentially revolutionary technique that could cure hereditary diseases and might also help in fighting diseases such as AIDS and cancer.
The Recombinant Advisory Committee of the National Institutes of Health, which oversees gene therapy experiments, gave permission Tuesday to researchers associated with Seattle-based Targeted Genetics Corp. to implant genetically modified cells into patients with acquired immune deficiency syndrome in hopes of helping them fight the disease. The Food and Drug Administration had already approved by the
Targeted Genetics, a unit of Immunex Corp., a biotechnology company, said the test would be the first clinical trial of gene therapy to be run by a company. The test would mark a milestone on the way to commercialization, which could come in the second half of this decade.
There have been several previous tests of gene therapy, but they have been run by individual physicians, such as those associated with the National Institutes of Health.
Another company, Gaithersburg, Md.-based Genetic Therapy Inc., has not organized its own clinical trials but has collaborated with and supplies materials to most of those researchers, including Dr. W. French Anderson at the National Heart, Lung and Blood Institute.
Gene therapy involves implanting genes, which provide the hereditary instructions that control the body's features and functions, into patients. A hemophiliac, for instance, lacks the ability to produce factors that cause the blood to clot.
With gene therapy, such a patient could be given genes to allow him to produce the clotting factor, thereby doing away with periodic injections.
"If you're really doing the right kind of job, one kind of gene therapy application would cure a patient for a lifetime," said K. Michael Forrest, president of Transkaryotic Therapies Inc., a gene therapy company in Cambridge, Mass.
Sickle cell anemia and cystic fibrosis are other hereditary diseases that gene therapy could potentially cure. Even for non-hereditary diseases, genes could be implanted to allow the body to make chemicals that would otherwise have to be given as drugs.
"This is a whole new way of delivering medicine," said John Archer, executive vice president of Somatix Therapy Corp., a gene therapy company in Alameda, Calif.
Gene therapy has attracted many start-up companies, of which two -- Somatix and Genetic Therapy -- have already issued stock publicly. And some big drug companies, led by Sandoz Ltd., are also getting involved.
But gene therapy faces many safety, technical and marketing issues before it can become a successful treatment.