How is Gene Therapy Treatments needed ?

gene therapy

Mesothelioma is a rare cancer in which doctors speculate asbestos exposure through inhalation or ingestion is the contributing environmental factor. Asbestos can affect cells in the lungs (pleural mesothelioma), abdomen (peritoneal mesothelioma), heart (pericardial mesothelioma), and tissue membranes of other organs (epithelial mesothelioma).

The Department of Defense has designated $50 million for research related to mesothelioma. Scientists have allocated research funds to gene therapy in an effort to treat patients diagnosed with mesothelioma. Mesothelioma research enables scientists to understand why genes respond to certain environmental factors that result in cancer.

Experts speculate that gene mutation is partly responsible for the development of cancerous tumors. When genes mutate, the genes may communicate false instructions to other developing cells. The perpetual development of mutating cells may contribute to growth of cancerous tumors. Other unhealthy behaviors, such as exposure to hazardous materials and chemicals, smoking, and extended sun exposure may also be contributing factors.

Several types of gene therapy treatments are available for the mesothelioma patient. Anti-angiogenesis, gene replacement, and gene knockout therapy are current solutions for slowing or eradicating cancer cell growth. Currently, gene therapy remains primarily in research phase. However, experts recommend doctors use gene therapy in conjunction with other treatments, such as chemotherapy and radiation.


Anti-angiogenesis introduces genes into the bloodstream that inhibit the growth of blood vessels. By eliminating oxygen and blood supply to the tumor, scientists have found that cancerous growths will cease or become eliminated in some instances.

Replacement Gene Therapy

One form of gene replacement focuses on replacing the gene responsible for cancerous cell growth. Gene replacement slows or stops the gene responsible for abnormal rapid cell division associated with cancer.

Knock-Out Gene Therapy

University of Pennsylvania is researching another form of gene therapy that introduces a virus which renders cancerous cells vulnerable to cancer medication. When the adenovirus is introduced into the affected area of the body, the “suicide genes” interact with all cells in the vicinity. This occurrence causes the cancerous cells to produce an enzyme called thymidine kinase (TK). This enzyme essentially renders the cancerous cells vulnerable by marking them for death.

A drug called Ganciclovir is administered intravenously. This drug views the cancer cells producing thymidine kinase as a viral infection. Ganciclovir attacks and destroys marked cancer cells, while healthy cells remain unharmed. This type of gene therapy is often referred as “knock out” gene therapy.

Gene Therapy Research and Clinical Trials

German scientists have begun extensive research of mesothelioma gene therapy. The experiments conducted on animal models have yielded significant tumor reduction. According to the research, mesothelioma gene therapy slowed the growth of cancerous cells by fifty percent and increased animal life expectancy by forty percent.

Studies in the United States have increased due to funds allocated to mesothelioma research. The Center for Biologics Evaluation and Research (CBER) is currently conducting research regarding gene therapy. Some applications are applicable to mesothelioma.

Risks of Gene Therapy

Gene therapy is still in clinical trial phase. Therefore, mesothelioma gene therapy may have associated harmful risks. The adenovirus vectors utilized in knock out gene therapy are successful in targeting cancerous tumors. However, the vectors have also been cited as cause for liver damage and inflammation. The liver functions to rid the body of harmful substances. Many of the vectors do not reach the cancerous tumors as a result. Large amounts of vectors must be administered to be effective. However, the abundance of vectors may cause significant harm to the liver. Potential candidates should be aware of such risks before participating in gene therapy.

After prognosis, most mesothelioma patients are given less than two years to live. Most patients are willing to endure experimental gene therapy with hopes that the benefits will outweigh the risks. If successful, gene therapy may prolong the life of a mesothelioma patient. - 100.00%

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