BETHESDA -- The Johns Hopkins School of Medicine received preliminary approval yesterday to conduct its first gene therapy experiment on 26 kidney cancer patients who have little chance of survival.
The National Institutes of Health advisory committee gave the Hopkins researchers the approval to do an experiment to determine whether the therapy was safe.
The committee also approved other gene therapy experiments for brain cancer and leukemia in children, which will be done in Des Moines, Iowa, and Memphis, Tenn.,in collaboration with a Maryland company, Genetic Therapy Inc. of Gaithersburg.
The Hopkins researchers will take cancer cells from the patients, insert a cancer-fighting gene into the cells and irradiate them to help ensure that they are safe to put back into the patient. Theoretically, the genetically engineered cells would help the immune system fight the cancer.
Committee members praised the Hopkins researchers for the study's design, but complained that one of the consent forms that patients would sign before the treatment has been so simplified that it was inaccurate. One committee member abstained from voting on the experiment on the basis that patients would not understand the treatment.
The Recombinant DNA Advisory Committee's recommendations are only advisory, and all of the researchers are required to receive Food and Drug Administration approval as well. However, the RAC is considered the most rigorous review of gene therapy trials. Its decisions are rarely contradicted by the FDA.
Hopkins officials said they did not expect the FDA to act on the proposal until June and that the earliest the experiment could begin was August. Another experiment, one to treat up to 40 brain cancer patients at the Iowa Medical Center in Des Moines, was also approved. A similar treatment at the National Institutes of Health began last December, but researchers have not announced whether the five patients treated there were improving.
The gene therapy experiment for leukemia in Memphis was intended to find the best way to rid bone marrow cells of leukemia in children who are in remission.
Both the brain cancer and the leukemia experiments are conducted using genetically engineered viruses made by Genetic Therapy to place the genes into cells which are then injected into the patients.
Somatix Therapy Inc., a California-based biotechnology company, will genetically engineer the kidney cells taken from patients for Hopkins researchers.