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temozolomide (SI 053 / Temodex / SI053)

✓ Approved

Double Bond Pharmaceutical · Small Molecule · Small Molecule

What is temozolomide?

temozolomide is a small molecule developed by Double Bond Pharmaceutical. It is approved for therapeutic indications via intratumoral injection.

Drug Profile

Brand NamesSI 053, Temodex, SI053
CompanyDouble Bond Pharmaceutical
Drug ClassSmall Molecule
RouteIntratumoral Injection
StatusApproved
Sources: ClinicalTrials.gov, Double Bond Pharmaceutical

Therapeutic Indications

temozolomide is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Brain neoplasm malignant✓ Approved

Related Research Articles

PubMedCells2026-10-16

Dexamethasone Modulates Global DNA Methylation and Oxidative DNA Damage in Glioblastoma Cell Lines.

Barciszewska Anna-Maria AM, Belter Agnieszka A, Barciszewski Jakub F JF, Perrigue Patrick M PM et al.

Glioblastoma remains one of the most aggressive primary brain tumors, characterized by rapid progression, therapeutic resistance, and poor prognosis. Dexamethasone is routinely administered to patients with glioblastoma to reduce peritumoral edema and improve neurological symptoms. However, its broader molecular effects on tumor-cell biology and treatment response remain incompletely understood. This study examined the effects of dexamethasone alone and in combination with temozolomide, valproic acid, or metformin on glioblastoma cell viability, real-time proliferation, senescence-associated β-galactosidase activity, cell-cycle distribution, global DNA methylation, and oxidative DNA damage. Dexamethasone induced concentration- and time-dependent changes in global DNA methylation in glioma cells. In T98G cells, shorter exposure increased 5-methylcytosine, whereas longer incubation or selected combined treatment conditions reduced methylation, suggesting dynamic epigenetic remodeling. Low dexamethasone concentrations were associated with increased 5-methylcytosine content, also in temozolomide-containing conditions. In contrast, higher dexamethasone concentrations reduced global methylation. That effect was cell-line dependent. In SW1783 cells, high-dose dexamethasone caused a reduction in 5-methylcytosine amount as well as an increase in 8-oxo-2'-deoxyguanosine level. Dexamethasone also reduced real-time T98G cell proliferation after prolonged exposure, while temozolomide was the main compound associated with cell-cycle changes in T98G cells. Metformin displayed comparatively limited cytotoxicity under the tested conditions. Beyond dexamethasone's well-established anti-edematous and anti-inflammatory role, it may also modify glioma redox and epigenetic states and influence cellular responses to temozolomide, valproic acid, and metformin. When considering these findings it is necessary to take into account a number of limitations. The upper limits of the DEX, TMZ, and MF concentration ranges were one to three orders of magnitude higher than the brain or CSF exposure levels that have been reported, and the experiments were carried out at standard culture oxygen concentrations (about 18-21% O2), which are much higher than the oxygen levels found in brain tissue (about 2-6% O2). Furthermore, the conclusions drawn from the T98G experiments only apply to this cell line and cannot be applied to glioblastoma without first being verified in other models.

PMID 42882443
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PubMedCells2026-10-16

GSC State Plasticity Drives Therapeutic Resistance and Recurrence in Glioblastoma.

Shuai Jinxi J, Chang Cuicui C, Gong Yanju Y

Glioblastoma (GBM) is the most aggressive primary brain tumor in adults, and treatment failure is closely linked to the presence of GBM stem cells (GSCs). Rather than representing a fixed marker-defined state, GSC identity is better understood as a dynamic and reversible stem-like state. GBM cells exhibit transcriptional plasticity, most clearly reflected by the proneural-to-mesenchymal transition, although direct interconversion among neural progenitor cell-like, oligodendrocyte precursor cell-like, astrocyte-like, and mesenchymal-like states remains unproven. GBM cells can also transition bidirectionally between stem-like and differentiated phenotypes, shift between quiescent and proliferative states, and reprogram metabolism in response to nutrient deprivation, hypoxia, and therapy. Perivascular, hypoxic, extracellular matrix, and immunosuppressive niches further regulate these transitions. This plasticity contributes to therapeutic resistance because radiotherapy, temozolomide, and targeted agents can both select preexisting tolerant populations and induce adaptive states. Therapy-persistent GSC-like cells may form residual reservoirs that undergo further remodeling and contribute to recurrence. Emerging strategies, most still preclinical, aim to destabilize stem-like states, broaden coverage across heterogeneous GSC populations and protective niches and block therapy-induced adaptive transitions. Thus, constraining GSC state plasticity may provide a framework for overcoming therapeutic resistance and recurrence in GBM.

PMID 42882455
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PubMedCancers2026-10-16

Real-World Application of PD-1 Inhibitors in High-Grade Glioma: A Case Series.

Ren Yin Y, Guo Xiaopeng X, Xing Hao H, Kang Xiaoman X et al.

Background/Objectives: Patients with high-grade glioma (HGG) have a poor prognosis despite maximal safe resection, radiotherapy, and chemotherapy, partly because of the immunosuppressive tumor microenvironment. Unfortunately, all phase III trials of anti-PD-1 therapy to date have failed to yield positive results in HGGs. Recent early-phase trials have identified promising strategies involving immune checkpoint inhibitors. Here, we share our 8-year experience with anti-PD-1 therapy in patients with HGG in a real-world setting. Methods: All patients with HGG who received PD-1 inhibitor-based therapy at our center between 2018 and 2026 were included. Medical records were reviewed longitudinally for each patient, and the latest follow-up data were collected. Patients lost to follow-up were excluded. Results: Four regimens involving anti-PD-1 treatment were identified in 22 patients with HGG (newly diagnosed = 11, recurrent = 11): (1) VEGFR-targeted therapy plus PD-1 inhibition (9 recurrent HGGs), (2) the "2-THE-TOP" regimen (tumor-treating fields plus temozolomide and PD-1 inhibitor therapy) (5 newly diagnosed HGGs), (3) anti-PD-1 monotherapy (2 recurrent and 2 newly diagnosed HGGs), and (4) neoadjuvant plus adjuvant anti-PD-1 therapy (4 newly diagnosed HGGs). Median overall survival for these four regimens was 32.7, 20.3, 28.6, and 31.8 months, respectively. No grade 3-4 treatment-related adverse events were recorded. Conclusions: Our preliminary observations suggest that PD-1 blockade may remain a viable option for patients with HGG when combined with specific therapies, administered with careful timing, and applied in selected patients.

PMID 42882817
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PubMedBiology2026-10-16

Disrupted Signalling Landscapes in Glioblastoma: Mechanistic Insights and Therapeutic Frontiers.

Ansari Ammar Asif Ali AAA, Haddad Natalia N, Assed Ana Maria AM, Awaad Nada N et al.

Glioblastoma (GBM) is the most prevalent and aggressive malignant primary brain tumour, originating from glial cells known as astrocytes and accounting for over 50% of all central nervous system malignancies. Its aetiology is linked to various factors, including radiation exposure, genetic alterations, and viral influences, which trigger the uncontrolled growth and rapid infiltration of glial cells into surrounding brain tissues. Despite standard multidisciplinary management involving maximal safe surgical resection, radiotherapy, and temozolomide, the prognosis remains poor. The molecular landscape of GBM is characterised by profound dysregulation across highly conserved signalling networks, including the PI3K/AKT/mTOR, ERK/MAPK, Wnt/β-catenin, and Hippo pathways. These pathways are frequently hijacked by genetic and epigenetic alterations such as EGFR amplification and PTEN loss, which drive essential hallmarks of cancer like uncontrolled proliferation, angiogenesis, and apoptosis evasion. A defining feature of GBM is its extreme intra-tumoral heterogeneity and cellular plasticity, where tumour cells dynamically transition between distinct transcriptional states, including mesenchymal, astrocyte-like, and neural progenitor-like, to adapt to therapeutic pressures. This complexity is further exacerbated by microenvironmental factors. Clinical progress is severely hindered by the blood-brain barrier (BBB), which restricts systemic agents from reaching invasive tumour margins. Targeted monotherapies, such as EGFR and mTOR inhibitors, have largely failed in clinical trials due to compensatory feedback loops and pathway redundancy. Consequently, the therapeutic paradigm is shifting toward rational combination therapies and novel immunotherapeutic platforms. Emerging strategies, such as bivalent CAR T-cell therapy targeting both EGFRvIII and IL-13Rα2, have shown early clinical promise in achieving tumour regression, highlighting a potential path toward precision medicine. This review synthesises current insights into signalling dysregulation and explores the future frontiers of multi-targeted therapeutic interventions in GBM.

PMID 42883362
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PubMedAsia-Pacific journal of clinical oncology2026-10-15

Multifocal Glioblastoma Managed With Primary Temozolomide as a Strategy to Delay Radiation Therapy: Clinical and Radiological Outcomes.

Loh Chun Khai CK, Wheeler Helen H, Lee Adrian A, Wong Matthew M et al.

To evaluate the clinical and radiological outcomes of upfront temozolomide (TMZ) monotherapy in deferring radiotherapy for multifocal glioblastoma (GBM). We analysed 18 consecutive adult patients with newly diagnosed, multifocal GBM treated with upfront TMZ monotherapy between 2016 and 2024. The primary endpoint was a 6-month progression-free survival (PFS). Secondary endpoints were overall survival (OS) and radiological tumor response. The median age of the cohort was 69 years. The median follow-up was 12 months. Thirteen (72.2%) patients had O⁶-methylguanine-DNA methyltransferase (MGMT)-methylated tumors. Bi-hemispheric disease was present in 16 patients (88.9%) at baseline. Patients received a median of six TMZ cycles. Disease relapse occurred in 94.4% (n = 17) of cases, with 47.0% of failures involving distant sites. The 6-month PFS was 61% (95% CI: 42.3%-88.3%), and the median OS was 12.2 months (95% CI: 11.2-31.1). MGMT-methylated tumors demonstrated a significant volumetric reduction at 3 months compared to unmethylated cases (median -34.2% vs. +114.0%; p = 0.007). Survival outcomes trended in favour of the methylated cohort (6-month PFS 69.2% vs. 40.0%; median OS 12.3 vs. 11.2 months), though these associations did not reach statistical significance (p = 0.480). This study provides valuable insights into the use of primary TMZ as an exploratory strategy for short-term modulation in highly selected patients with extensive multifocal GBM. Whilst this approach facilitates a strategic delay in large-volume radiation, the high relapse rate underscores that upfront TMZ serves as a temporary window for deferring neurotoxicity rather than achieving durable disease control.

PMID 42878023
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PubMedFrontiers in neurology2026-10-15

Cytokine-induced killer cells combined with temozolomide suppress glioblastoma progression in an orthotopic model and demonstrate clinical feasibility in recurrent glioblastoma.

Park Young Kyu YK, Yang Dong Won DW, Park Kyun Woo KW, Nam Gun He GH et al.

Glioblastoma (GBM) remains an aggressive primary brain tumor with limited therapeutic options after recurrence. Cytokine-induced killer (CIK) cells are ex vivo-expanded cytotoxic lymphocytes with T-cell and natural killer-like properties. We evaluated CIK-cell-based therapy using an orthotopic LN229-luciferase GBM model and a retrospective cohort of patients with recurrent GBM. Twelve independently manufactured patient-derived CIK-cell products were phenotypically characterized. In the orthotopic model, control, temozolomide (TMZ), and TMZ+CIK groups (n = 4 per group) were monitored by longitudinal bioluminescence imaging, with quantitative histological and immunohistochemical analyses. Clinical outcomes and safety were retrospectively evaluated in 12 heavily pretreated patients with recurrent GBM. Expanded CIK-cell products showed a predominant effector-memory phenotype and high NKG2D and DNAM-1 expression. Longitudinal bioluminescence imaging demonstrated a significant treatment-by-time interaction among the three groups through Week 4 (p = 0.0054). At Week 4, both TMZ and TMZ+CIK showed significantly lower radiance than control, whereas TMZ and TMZ+CIK did not differ significantly. An exploratory comparison between TMZ and TMZ+CIK through Week 8 was not significant (p = 0.0663). Quantitative H&E analysis showed an overall group difference in tumor area fraction (p = 0.0376), whereas Granzyme B and CD56 staining did not differ significantly. In the clinical cohort, stable disease was the best recorded response in all evaluable patients; median progression-free survival was 10.5 months and median overall survival was 14.2 months. CIK-cell-based therapy was generally well tolerated. These findings provide quantitative preclinical evidence of treatment-associated antitumor activity and support the clinical feasibility of CIK-cell-based therapy in recurrent GBM. However, the study does not establish a statistically significant incremental preclinical benefit of adding CIK cells to TMZ or clinical therapeutic efficacy. Prospective, adequately powered controlled studies are warranted.

PMID 42877257
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