Current Cancer Drug Targets - Volume 25, Issue 8, 2025

Central nervous system tumors are abnormal proliferations of neuronal cells within the brain and spinal cord. They can be primary or secondary and place a heavy financial, psychological, and physical burden on individuals. The highly selective blood-brain barrier, which only permits specific molecules to flow into the brain parenchyma, inhibits the efficacy of pharmacological medicines. Treatment options include surgery, chemoradiotherapy, and targeted therapy. Despite advances in therapy over the past few decades, the overall morbidity and mortality rates are still high, emphasizing the need for improved therapeutic choices to improve survival and quality of life further. Nano pharmaceuticals have demonstrated encouraging outcomes in in vivo trials using microscopic particles to enhance bioavailability and selectivity. The most successful clinical results to date have been achieved by liposomes, extracellular vesicles, and biomimetic nanoparticles; nevertheless, clinical trials are required to confirm their safety, efficacy, affordability, long-term impact, and success in patients from various demographics. Nano pharmaceuticals have the potential to change the paradigm of therapy for brain tumors, allowing better outcomes as primary and adjunctive therapy.

The research provides an in-depth exploration of gene therapy, covering fundamental principles, diverse implementation strategies, and innovative gene delivery vectors. Further implementations of gene therapy, such as apoptosis induction, anti-angiogenesis, and nucleic acid therapy, have been described. Gene delivery vectors, encompassing viral methods such as adenovirus, retroviral, foamy viral, adeno-associated viral, herpes simplex virus (HSV), and vaccinia virus vectors and nonviral methods, such as physical and chemical approaches have been extensively discussed. Further, a significant focus is placed on novel drug carriers, including nanoparticles, such as iron oxide, calcium carbonate, gold, carbon nanotubes, graphene oxide, quantum dots, nanogels, ceramic nanoparticles, calcium phosphate, and metal-organic frameworks. Additionally, lipids, peptides, and polymeric materials, featuring liposomes, exosomes, polymeric micelles, hydrogels, polymersomes, and dendrimers are explored as promising avenues for gene delivery. Finally, the key findings and insights underlining the dynamic landscape of gene therapy research have been summarized, which may offer a comprehensive understanding of current methodologies and potential future directions in the field of gene therapy.

Epigenetic mechanisms have been shown to play a critical role in the development and progression of gastrointestinal [GI] cancers. These mechanisms involve modifications to DNA and histones that can alter gene expression patterns and may contribute to the initiation and progression of cancers. In recent years, epigenetic therapies have emerged as a promising approach to treating GI cancers. These therapies target specific epigenetic modifications, such as DNA methylation and histone acetylation, to restore normal gene expression patterns and inhibit cancer cell growth. Several epigenetic drugs have been approved for the treatment of GI cancers. Moreover, the use of epigenetic therapies in combination with other treatments, such as chemotherapeutic agents, is being studied to improve treatment outcomes.

We have provided an overview of the role of epigenetic mechanisms in GI cancer treatment aimed to focus on recent evidence of the use of epigenetic agents in clinical and preclinical GI cancer studies, including gastric, esophageal, hepatic, pancreatic, and colorectal cancers. Overall, the role of epigenetic mechanisms in GI cancer treatments is an active area of research with the potential to improve patients' treatment outcomes and advance cancer treatment strategies.

Epigenetic alterations are implicated in the early stages of tumorigenesis and are widely recognized as a ubiquitous phenomenon in cancer development. Aberrant epigenetic modifications can alter the expression of target genes, induce heterochromatin formation, and gradually drive normal cells towards immortalized tumor cells with significant consequences. SETDB1 (SET domain bifurcated histone lysine methyltransferase 1), a typical histone methyltransferase, promotes the formation of heterochromatin and inhibits the transcription of genes by modifying the methylation of lysine 9 of histone 3. SETDB1 is usually highly expressed in tumors with high copy numbers, accompanied by poor prognosis and low patient survival rates, which is a typical case of abnormal epigenetic modification. We discuss the mechanism of SETDB1 in a variety of cancers and review the epigenetic inhibitors that have been reported in recent years, along with their anti-tumor effects. In addition, we summarize the role of SETDB1 in a variety of diseases and cell functions.

The advent of mRNA vaccines has heralded a transformative era in oncology, exemplified by the BNT116 mRNA lung cancer vaccine. Leveraging the same groundbreaking technology as COVID-19 vaccines, BNT116 delivers tumor-specific genetic instructions to the immune system, targeting non-small cell lung cancer (NSCLC), the most prevalent lung cancer subtype. This approach contrasts with conventional therapies that lack precision and often damage healthy tissues. By encoding tumor antigens, BNT116 educates cytotoxic T cells to recognize and eradicate malignant cells, aligning with the principles of precision medicine. Early-phase clinical trials (e.g., NCT05142189) have demonstrated a favorable safety profile and promising antitumor activity, with ongoing research exploring its use in combination therapies, such as checkpoint inhibitors. Despite logistical challenges, such as mRNA instability and cold chain requirements, advances in lipid nanoparticle delivery systems are enhancing vaccine stability and efficacy. The adaptability of mRNA technology positions it as a cornerstone for personalized oncology, with potential applications extending to other cancers. Success in the BNT116 trials could redefine NSCLC treatment paradigms, offering a targeted, less cytotoxic alternative. This innovation can not only improve therapeutic outcomes, but also pave the way for preventive cancer vaccines, signaling a new dawn in cancer treatment.

Colorectal cancer (CRC) is currently the third most common malignancy worldwide, with an increasing mortality rate and treatment resistance. Due to the lack of effective biomarkers and therapeutic targets, the early diagnosis and treatment of colorectal cancer remain suboptimal. Circular RNAs (circRNAs) are a novel class of non-coding RNAs with covalent closed-loop structures that are well stabilized and conserved and are involved in multiple pathological conditions in humans. CircRNAs have been identified to be enriched and stable in exosomes. In addition, there is growing proof that exosomal circRNAs that have been identified as oncogenes or tumor suppressors regulate CRC growth, migration, and sensitivity to radiotherapy and chemotherapy. Exosomal circRNAs represent promising candidates as diagnostic biomarkers and anti-tumor targets. In this article, we explore recent studies on exosomal circRNAs in CRC and describe their biological functions in colorectal cancer development, illustrating their potential as biomarkers and targeted therapeutic capabilities.

Crucial for understanding liver cancer patients overall health outcomes. This research aimed to assess the CVM risk of them.