In what could be a major step toward gene therapy, a federal advisory board has recommended that researchers be allowed for the first time to inject genetically altered cells into human patients.
"If this protocol were to work, I'd expect that within six to 12 months there could be a proposal to do a gene therapy experiment," said Dr. William J. Gartland, the executive secretary of the Recombinant DNA Advisory Committee to the National Institutes of Health.The goal of gene therapy - a much more controversial matter - is to treat a genetic disease by replacing a defective or missing gene that causes an inherited illness. The proposed experiment would not be gene therapy but would use many of the same techniques.
The advisory committee on Monday voted 16-5 in favor of the proposal to inject genetically altered cells into patients to determine how well a certain form of cancer therapy is working. A so-called marker gene would be put into a patient's cells in the lab and then placed back inside the patient.
The experiment would not attempt to fight the cancer, but would track the genetically altered cells carrying the altered genes, Gartland said.
The experiment would be limited to 10 patients, and those patients would have life expectations of only about 90 days due to the advancement of their cancer, he said.
NIH Director Dr. James B. Wyngaarden and the Food and Drug Administration still must approve the proposal before it is carried out.
Due to the split in the vote, a decision from Wyngaarden "will probably take a couple of months," Gartland said. "The NIH director will probably want to know why those five people voted against it."