Gene Therapy for Melanoma
Indiana University school of medicine researchers used a changed lentivirus to introduce a potent anti-melanoma T cell receptor gene into the hematopoietic stem cells of mice. Hematopoietic stem cells are the cells that give rise to all blood and immune system cells.
The T cell gene, that recognizes a particular protein found on the surface of melanoma, was isolated and cloned from a patient with melanoma. The gene-modified stems cells were transplanted back to hosts and found to eradicate metastatic melanoma for the lifetime of the animals.
"We found that the transplantation of gene-modified hematopoietic stem cells ends up in a new host immune system and therefore complete elimination of tumor," reported Christopher E. Touloukian, M.D., an assistant professor of surgery and immunology at the IU school of medicine. "To date, cancer immunotherapies have are hampered by restricted and diminishing immune responses over time. We have a tendency to believe this sort of translational model opens new doors for patients with melanoma and probably other cancers by taking advantage of the potent regenerative capability of hematopoietic stem cells and new advances in gene therapy."
It has paved the way for a brand new clinical trial in humans funded by the V Foundation for Cancer research. The pilot section I trial will involve treatment of twelve patients and focus totally on the security and efficacy of the therapy, said Dr. Touloukian. The clinical trial is anticipated to start accruing patients by late 2011.
In 2010, 68,000 patients are going to be diagnosed with melanoma and therefore disease will be associated with approximately 9,000 deaths. Current treatments for metastatic melanoma are highly toxic and mostly unsuccessful with the most patients dying within six to twelve months after diagnosis.
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