The brain of a terminally ill cancer patient was injected with genetically engineered mouse cells Monday in the first experiment in treating brain tumors with gene therapy.
Neurosurgeons at the National Institutes of Health performed the treatment, which involves injecting foreign genes into tumor cells and then killing those cells with a drug, on a brain cancer patient who had not been helped by conventional treatments.
The operation was an important step for researchers who are seeking a gateway to cure a host of fatal diseases, from cystic fibrosis to cancer and AIDS, by tapping the basic building blocks of the human body.
"It is very significant not only for treating malignant brain tumors, but it [also] opens up the door to a whole new approach to treating tumors," said Dr. Henry Brem, director of neurosurgical oncology at the Johns Hopkins School of Medicine. "It is the first step to seeing the fulfillment of gene therapy."
The doctors said they will know within several weeks whether the treatment has shrunk the tumor in the patient, but it will be several months before they can tell whether the tumor will grow back.
Researchers also plan to treat two more patients within six weeks with the same method, and 20 patients in the near future.
If the method succeeds in many patients, doctors could ask the Food and Drug Administration next year for permission to use it more widely.
The experiment, conceived 20 months ago, has attracted the attention of cancer patients throughout the country, thousands of whom have telephoned the NIH, asking to be among the first patients to be treated. About 15,000 patients who are diagnosed with primary brain cancer each year typically face a poor prognosis.
This is not the first time that gene therapy has been used to treat patients. Researchers have asked the FDA for permission to conduct dozens of gene therapy experiments. But only a handful have begun.
Last week, a federal advisory committee, the Recombinant DNA Advisory Committee of the NIH, approved gene therapy treatment for cystic fibrosis, the most common inherited fatal disease. In October, the NIH granted researchers and GTI approval to treat lung cancer patients by inserting genes in their tumors.
Monday's experimental treatment was an important step in the furtherance of gene therapy, a potentially powerful treatment for all kinds of diseases.
