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Published on: June 2, 2016
Antisense therapy for cancer--the time of truth
Burkhard Jansen1, Uwe Zangemeister-Wittke
1Prostate Centre and the Division of Dermatology Vancouver General Hospital, University of British Columbia, BC, Vancouver, Canada. bjansen@interchange.ubc.ca
Abstract:
The recent acceleration in the identification and characterisation of new molecular targets for cancer and the limited effectiveness of conventional treatment strategies has focused considerable interest on the development of new types of anticancer agents. These new drugs are hoped to be highly specific for malignant cells with a favorable side-effect profile due to well-defined mechanisms of action. Antisense oligonucleotides are one such class of new agent--they are short, synthetic stretches of DNA which hybridise with specific mRNA strands that correspond to target genes. By binding to the mRNA, the antisense oligonucleotides prevent the sequence of the target gene being converted into a protein, thereby blocking the action of the gene. Several genes known to be important in the regulation of apoptosis, cell growth, metastasis, and angiogenesis, have been validated as molecular targets for antisense therapy. Furthermore, new targets are rapidly being uncovered through coordinated functional genomics and proteomics initiatives. Phosphorothioate oligonucleotides are the current gold standard for antisense therapy; they have acceptable physical and chemical properties and show reasonable resistance to nucleases. Recently, new generations of these phosphorothioate oligonucleotides that contain 2'-modified nucleoside building blocks to enhance RNA binding affinity and decrease indirect toxic effects have been developed. Antisense therapeutics are, after decades of difficulties, finally close to fulfilling their promise in the clinic.
Insights
Antisense oligonucleotides offer a promising new cancer therapy by targeting specific messenger RNA to block cancer-promoting genes. Advances in phosphorothioate oligonucleotides enhance their effectiveness and reduce toxicity, bringing this approach closer to clinical success.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Conventional cancer treatments have limitations, driving the need for novel, targeted anticancer agents.
- Antisense oligonucleotides represent a new class of therapeutic agents designed for high specificity against cancer cells.
- Identifying and characterizing new molecular targets is crucial for developing effective cancer therapies.
Purpose of the Study:
- To explore the potential of antisense oligonucleotides as a novel therapeutic strategy for cancer.
- To highlight the advancements in antisense oligonucleotide technology and their application in oncology.
- To discuss the role of genomics and proteomics in uncovering new targets for antisense therapy.
Main Methods:
- Antisense oligonucleotides bind to specific messenger RNA (mRNA) strands, preventing protein synthesis.
- Target genes involved in apoptosis, cell growth, metastasis, and angiogenesis are validated for antisense therapy.
- Development of next-generation phosphorothioate oligonucleotides with 2'-modified nucleosides.
Main Results:
- Antisense therapy targets genes crucial for cancer progression, including those regulating apoptosis, cell growth, metastasis, and angiogenesis.
- Functional genomics and proteomics initiatives are continuously identifying new molecular targets.
- Next-generation phosphorothioate oligonucleotides demonstrate enhanced RNA binding and reduced toxicity.
Conclusions:
- Antisense oligonucleotides are nearing clinical realization as a targeted cancer therapy.
- Advancements in oligonucleotide chemistry are improving efficacy and safety profiles.
- This approach holds significant promise for overcoming the limitations of conventional cancer treatments.
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