Cancer Gene Therapy
At the University of Pennsylvania, a cold virus wages war on Mesothelioma
Karen Marcum, 65, made her way from Texas to the University of Pennsylvania to wage battle against Mesothelioma. The deadly lung cancer, hard to detect and one of the most merciless, is primarily caused by exposure to asbestos or similar materials, although Marcum says she's not sure where it came from.Marcus was the 5th member of her family to be diagnosed with the deadly disease, which was discovered in both lungs. Treatment options were limited. She underwent chemotherapy while awaiting entry into a clinical trial utilizing a gene drug developed by Biogen Idec that utilizes a cold virus genetically altered to carry the gene for interferon beta, a natural human protein that marshals the immune system.
Marcum spent four months in Philadelphia, where she had no side effects to the treatment, and in January of this year was told she will not require evaluation for another six months. She is alive and well.
In Texas, a lung cancer survivor thanks gene therapy for her life
Bunny Morrow of Texas, at 72 years of age, has found a new lease on life. Nearly four years after she discovered lung cancer so advanced it had wrapped around her vocal chords, she is doing so well that she volunteers helping patients navigate their treatment at the Mary Crowley Oncology Center at Baylor University, where she successfully participated in a cancer gene therapy trial.There was no cancer in Morrow's family history and although she had smoked at one time, she quit many years earlier. Given the spread of the cancer and positive lymph nodes, surgery was not the best option, although her upper left lobe was removed and she underwent early chemotherapy to shrink the tumor so she might meet the trial protocol.
Her cancer cells were removed and irradiated and then injected back into her body so the immune system would kick in. She had 16 injections and no side effects beyond minor allergic reaction. She currently has an annual scan but no medical limitations, which makes it possible for her to bakes muffins for other trial patients [her specialty is cranberry].
At the NIH, gene therapy successfully destroys advanced Melanoma
At the National Cancer Institute [NCI] of the National Institutes of Health [NIH] a team of researchers successfully utilized a form of immune-mediated therapy to destroy cancer cells in melanoma, a deadly form of skin cancer. The trial demonstrated sustained regression of advanced metastatic cancer using genetically engineered white blood cells from patients in the study. These blood cells were able to recognize, attack and destroy cancer cells, the very essence of gene therapy, without any side effects or contraindications."These results represent the first time gene therapy has been used successfully to treat cancer," said NIH Director, Elias A. Zerhouni, MD. "Moreover, we hope it will be applicable not only to melanoma, but also for a broad range of common cancers."
In a process called adoptive cell transfer, the most aggressive tumor-killing cells are isolated from the patient's lymphocytes, multiplied in the laboratory, and reintroduced to patients.
"We are currently treating advanced melanoma patients using adoptive transfer of genetically altered lymphocytes, and we have now expressed other lymphocyte receptors that recognize breast, lung and other cancers," said Steven A. Rosenberg, MD, PhD, lead researchers in the NCI study. The approach is being used on advanced melanoma patients, and researchers say they hope to soon use the same technique to fight breast, lung and other cancers. Regulatory approval is being sought for further trials.
At M. D. Anderson, dual gene therapy suppressors pack twice the punch against cancer
At the University of Texas M. D. Anderson Cancer Center, a trial utilizing combined targeted gene therapy vastly reduced human non-small cell lung cancer tumors in mice. Two tumor-suppressing genes given together intravenously proved far more powerful than separately, reducing the number of tumors by 75% and the weight by 80%. The genes, wrapped in nanoparticles, were p53, well-known tumor suppressors that cause defective cells to commit suicide, a process known as apoptosis, which is often inhibited in cancer cells."We certainly hope this approach will be more effective, but we also think it's likely to be much less toxic, with fewer side effects, than other types of combined cancer therapy," said Dr. Jack Roth, chair of the M. D. Anderson Department of Thoracic and Cardiovascular Surgery, and member of the ACGT Scientific Advisory Council. "These genes don't have much effect on normal tissue or normal cells when they are overexpressed, only cancer cells."
Gene therapy trial approved for chronic lymphocytic leukemia
Michel Sadelain, MD, PhD, a 2004 ACGT Research Fellow at Memorial Sloan Kettering Cancer Center in New York, received in April final approval to a Phase I clinical trial for victims of chronic lymphocytic leukemia. Sadelain is currently enrolling patients.The trial is based on successful results of a project funded by ACGT to assess the therapeutic potential of genetically targeted T-cells. Sadelain's team demonstrated that T-cells can be redirected to recognize the CD19 antigen and eradicate established systemic tumors borne by immune-deficient mice, demonstrating for the first time that genetically modified human T-cells can induce durable remissions in an in-vivo setting.
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