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Can Gene Therapy Cause Complete Remission in Metastasis Melanoma ?

Though melanoma is a less known type of skin cancer, it is responsible for 75 percent of deaths. The ailment is caused by the development of malignant cells in melanocytes. These are the cells which produce the pigment melanin.

Exposure to the harmful ultra-violet rays is mainly responsible for development of this cancer. The treatment techniques presently available are toxic by nature. Most of the patients lose their lives within 6-12 months after the diagnosis of the ailment.

Latest statistics reveal more than 68,000 patients will be diagnosed and approximately 9,000 deaths will be caused by melanoma in 2010. Researchers at the Indiana University School of Medicine have developed a powerful anti-tumor gene and successfully introduced it into the hematopoietic stem cells of laboratory mice suffering from melanoma. The result was complete remission of the ailment. The results of the study will be published in the December 2010 issue of the Journal of Clinical Investigation.

How Remission of Melanoma was Achieved by Gene Therapy

  • Blood and immune system cells are produced in the soft and spongy central section of bones called the bone marrow.
  • Special types of cells called hematopoietic stem cells have the potential of getting converted to either of these cells depending on the requirement of the body.
  • The researchers added a new immune system gene into the malignant hematopoietic stem cells of experimental mice suffering from melanoma.
  • This gene had the ability to detect the presence of a toxic protein found on the surface of the rogue melanoma cells.
  • Genes are the chemical instructions followed by cells to perform their tasks. Proteins are the chemicals which actually carry out these functions.
  • The toxic protein was separated by the researchers from the body of a melanoma patient and genetically modified.
  • On its introduction, the modified gene developed a new immune system in the cancer affected mice and killed all the rogue cells.
  • These mice were found to be free from the clutches of this cancer type for their lifetime.

Conclusion: Gene therapy is a treatment method which is still in its infancy. It involves adding, changing and removing genes from the cells and tissues of individuals to treat diseases.

The research received funding from the National Institutes of Health. The extension of the study to humans will commence from late 2011. Primarily, the human trial will involve treating 12 patients, focusing on their safety and the efficacy of the therapy.

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