Preliminary results may open door to gene therapy just a bit wider

Article Abstract:

The use of genetic manipulation as an immunotherapeutic technique has been explored by National Institutes of Health (NIH) investigators. The procedure, pioneered by Steven A. Rosenberg, M.D., has been used in a recent study conducted on patients with advanced malignant melanoma, a particularly virulent variety of cancer of the skin which is usually fatal. The technique involves the use of tumor-infiltrating lymphocytes (white blood cells that insert themselves into tumors) which contain a marker gene whose movements can be followed. The survival rate and the distribution of these cells were studied. The procedure itself involves removing tumor cells from the patient and then cultivating them in vitro (outside the body) to accumulate a large concentration. Genetic manipulation is then performed. These cells, now having new properties, are reinjected into the patient and the cell behavior can be observed. This process was followed in five patients who showed no adverse effects from the inserted gene. Out of these five, only one patient was studied in detail. It was observed that the marker gene survived up to 19 days. This was encouraging as it indicates the possibility of other genes which can inhibit tumor growth. It was concluded that this technique may be promising in the treatment of cancers. Genes that code for mechanisms that demonstrate a variety of disease-inhibiting properties are currently being examined and clinical trials are anticipated to begin within a year. Various compounds are being investigated in an effort to identify the gene therapy which will be the most effective by the use of this technique.

Immunotherapy, Tumor-infiltrating lymphocytes

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Two more cell infusions on schedule for gene replacement therapy patient

Article Abstract:

In 1990, a four-year-old girl was the first recipient of gene therapy, in which genetically defective blood cells are removed, treated by insertion of the correct gene, then returned to the body. The patient suffers from adenosine deaminase (ADA) deficiency, a disorder of T lymphocytes (one cell type of the immune system). In each of the six infusions thus far, the corrected ADA gene has been successfully inserted into between three and eight percent of the patient's lymphocytes. It appears to be expressed, since blood levels of ADA have risen. The head of the study is Dr. Michael Blaese of the National Institutes of Health, in association with Dr. W. French Anderson. Another ADA-deficiency patient has also started gene replacement therapy. The first patient was diagnosed with ADA deficiency at the age of two, and responded to drug therapy with pegademase bovine (the approved treatment); however, a relapse occurred two years later, after which she entered the gene therapy program. After the second gene replacement infusion, the patient's T cell count doubled, and by the time of the third infusion, it had reached normal levels. The infused cells remain in circulation for as long as 30 days, and levels of ADA have increased steadily since the treatments started. Furthermore, the patient's immune system appears to be more capable of manufacturing antibodies. (Consumer Summary produced by Reliance Medical Information, Inc.)

Evaluation, Genetic aspects

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As number of trials increases, gene therapy begins to look promising for medicine's future

Article Abstract:

Eight gene therapy trials have been approved by the National Institutes of Health's Recombinant DNA Advisory Committee. Researchers at the University of Michigan Medical School will insert a gene for the HLA-B7 antigen into liposomes, and then inject the liposomes directly into a patient's melanoma tumor. They hope to stimulate an immune response to the tumor. Another Michigan scientist will try to correct familial hypercholesterolemia using gene therapy. Four gene therapy studies are already in progress, including a treatment for adenosine deaminase deficiency (ADA), the first gene therapy trial to be approved. The two patients treated have improved, but their immune systems are still impaired. Researchers in Italy and the Netherlands will begin similar trials of gene therapy in ADA deficiency. Other studies involve the insertion of the gene for tumor necrosis factor into melanoma tumors and insertion of the gene for factor IX into fibroblasts to treat hemophilia.

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