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Nucleic acid-based drugs for patients with solid tumours
Sebastian G Huayamares1,2, David Loughrey1,2, Hyejin Kim1,2
1Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA.
Abstract:
The treatment of patients with advanced-stage solid tumours typically involves a multimodality approach (including surgery, chemotherapy, radiotherapy, targeted therapy and/or immunotherapy), which is often ultimately ineffective. Nucleic acid-based drugs, either as monotherapies or in combination with standard-of-care therapies, are rapidly emerging as novel treatments capable of generating responses in otherwise refractory tumours. These therapies include those using viral vectors (also referred to as gene therapies), several of which have now been approved by regulatory agencies, and nanoparticles containing mRNAs and a range of other nucleotides. In this Review, we describe the development and clinical activity of viral and non-viral nucleic acid-based treatments, including their mechanisms of action, tolerability and available efficacy data from patients with solid tumours. We also describe the effects of the tumour microenvironment on drug delivery for both systemically administered and locally administered agents. Finally, we discuss important trends resulting from ongoing clinical trials and preclinical testing, and manufacturing and/or stability considerations that are expected to underpin the next generation of nucleic acid agents for patients with solid tumours.
Insights
Novel nucleic acid therapies, including gene therapies and mRNA nanoparticles, show promise for treating advanced solid tumors. These treatments offer new hope for patients with refractory cancers, alone or with standard care.
Area of Science:
- Oncology
- Molecular Biology
- Drug Development
Background:
- Advanced-stage solid tumors often resist conventional multimodality treatments (surgery, chemotherapy, radiotherapy, targeted therapy, immunotherapy).
- Nucleic acid-based drugs are emerging as potent therapeutic options for refractory tumors.
Purpose of the Study:
- To review the development and clinical activity of viral and non-viral nucleic acid-based treatments for solid tumors.
- To discuss mechanisms of action, tolerability, efficacy, and the impact of the tumor microenvironment on drug delivery.
- To highlight future trends, clinical trial outcomes, and manufacturing considerations for next-generation nucleic acid agents.
Main Methods:
- Review of existing literature on viral vectors (gene therapies) and non-viral nucleic acid therapies (mRNA, other nucleotides).
- Analysis of clinical activity, mechanisms of action, tolerability, and efficacy data from solid tumor patients.
- Examination of the tumor microenvironment's influence on systemic and local drug delivery.
Main Results:
- Viral and non-viral nucleic acid therapies demonstrate potential in treating advanced solid tumors, including refractory cases.
- Approved gene therapies and emerging nanoparticle-based treatments are expanding therapeutic options.
- Tumor microenvironment significantly affects drug delivery and therapeutic outcomes.
Conclusions:
- Nucleic acid-based drugs represent a significant advancement in oncology, offering new avenues for patients with limited treatment options.
- Ongoing research and development are crucial for optimizing delivery, efficacy, and manufacturing of these novel therapies.
- Future nucleic acid agents are expected to further improve outcomes for solid tumor patients.
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