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Published on: April 11, 2016
Future strategies toward the cure of indolent B-cell malignancies. Molecular genetic approaches
1Division of Hematology/Oncology, University of California, San Diego, La Jolla 92093-0663, USA.
Abstract:
Cancer is a genetic disease caused by somatic mutation(s) in essential genes that regulate cell growth or survival. Advances in genetics have identified molecular defects that can be targeted by novel pharmaceutics that exploit the altered genetic machinery of tumor cells. Gene transfer also can be used to potentiate the host immune response to weak tumor antigens that may arise during the process of neoplastic transformation. Advances in our understanding of the mechanics of immune activation have led to development of immune-based strategies for gene therapy of cancer. Preliminary findings in a phase I trial of one such approach in patients with chronic lymphocytic leukemia (CLL) are encouraging. Conceivably, we soon may see the advent of effective gene therapy for patients with indolent B-cell malignancies.
Insights
Cancer gene therapy is advancing, targeting genetic defects to boost immune response. Early trials show promise for treating chronic lymphocytic leukemia (CLL) and other indolent B-cell malignancies.
Area of Science:
- Oncology
- Genetics
- Immunology
Background:
- Cancer arises from somatic mutations in genes controlling cell growth and survival.
- Genetic discoveries enable targeted therapies exploiting tumor cell genetic alterations.
- Gene transfer can enhance anti-tumor immune responses against tumor antigens.
Purpose of the Study:
- To explore immune-based strategies for cancer gene therapy.
- To evaluate the potential of gene therapy for indolent B-cell malignancies.
Main Methods:
- Utilizing advances in understanding immune activation mechanics.
- Developing novel pharmaceutics targeting cancer's genetic machinery.
- Employing gene transfer to potentiate host immune response.
Main Results:
- Preliminary findings from a phase I trial are encouraging.
- Demonstrated potential for immune-based gene therapy in cancer treatment.
Conclusions:
- Immune-based gene therapy strategies are emerging for cancer treatment.
- Effective gene therapy for indolent B-cell malignancies may be forthcoming.
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