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Gene Modified Stem Cells Cure Melanoma in Mice

Kathleen Blanchard's picture

Researchers have found a way to cure melanoma in mice using gene therapy. Adding a potent anti-tumor gene to hematopoietic (bone marrow and blood producing) cells resulted in complete remission of melanoma for the lifetime of the mice used in the study.

T Cell Receptor Gene Boosts Immune Response for Melanoma Treatment

According to Christopher E. Touloukian, M.D., an assistant professor of surgery and immunology at the Indiana University School of Medicine where the study was conducted, "To date, cancer immunotherapies have been hampered by limited and diminishing immune responses over time. We believe this type of translational model opens new doors for patients with melanoma and potentially other cancers by taking advantage of the potent regenerative capacity of hematopoietic stem cells and new advances in gene therapy."

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The scientists used a modified lentivirus to introduce the T-cell receptor gene into the stem cells of mice that was cloned from a patient with melanoma. Touloukian says, “We found that the transplantation of gene-modified hematopoietic stem cells results in a new host immune system and the complete elimination of tumor."

A Phase I trial is expected, beginning late 2011 that will recruit 12 patient. Immunotherapy has created exciting options for patients with melanoma, but the immunity doesn't always last. The T-cell gene added to stem cells that boost immunity binds to specific proteins found on melanoma tumor. For mice, the gene therapy resulted in complete remission of cancer.

J Clin Invest. doi:10.1172/JCI43274