RARE DISEASE
Rare head and neck tumor
Rare head and neck tumor
Rare head and neck tumor
Drug discovery
4
drugs
With orphan designations
Overview
Rare head and neck tumors (R-HNCs) encompass diverse malignancies such as salivary gland cancers, sinonasal tumors, mucosal melanomas, neuroendocrine neoplasms, and sarcomas. These tumors represent <5% of all head and neck cancers and pose diagnostic challenges due to histological heterogeneity and overlapping features with common subtypes. Management requires specialized molecular profiling, multidisciplinary collaboration, and tailored therapies addressing unique molecular drivers [1][6][14].
Population
Annual incidence ranges from 0.26–48 cases per million depending on subtype (e.g., 13/million for salivary gland tumors vs <5/million for nasal/paranasal cancers) [1][15]
Higher nasopharyngeal cancer rates in Asian populations vs sinonasal tumors in Europe [6][9]
Pediatric rhabdomyosarcoma accounts for 3% of childhood cancers, with median diagnosis at age 10 [5]
Burden
Global incidence increased by 35% from 1990–2019, with highest DALYs in Asia [4]
5-year survival ranges from 17% (metastatic rhabdomyosarcoma) to 90% (early-stage nasopharyngeal cancer) [5][19]
Significant treatment-related morbidity: 60% of patients develop chronic dysphagia or voice impairment post-chemoradiation [3][14]
Categories: rare neoplastic diseases
Research Papers
206 drug discovery papers about Rare head and neck tumor, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
206 drug discovery papers about Rare head and neck tumor, with 1 first-in-class and 1 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
categories:
Small molecules
antibodies
2026-06-22 | Integrating Immunotherapy Into Head and Neck Surgery: Bridging Tumor Biology to Perioperative Decision‐Making, a Review
OBJECTIVE: To provide a comprehensive review of the biological rationale, clinical evidence, and practical perioperative management of immunotherapy for the head and neck surgeon. SUMMARY BACKGROUND DATA: Standard treatment for resectable head and neck squamous cell carcinoma (HNSCC) has reached a survival plateau, with over 50% of patients experiencing recurrence. The integration of immune checkpoint inhibitors (ICIs) into the neoadjuvant window represents a paradigm shift toward biologically adapted surgical intervention. RESULTS: Neoadjuvant immunotherapy capitalizes on an intact immune substrate to create an in situ vaccine, avoiding the post-surgical immune desert that limits adjuvant efficacy. Emerging phase III data confirm that perioperative ICI significantly improves event-free survival. Successful implementation requires the surgeon to navigate unique diagnostic challenges, such as distinguishing rare but anatomically risky pseudoprogression from true progression. While combination therapies (chemoimmunotherapy or immunoradiotherapy) yield higher pathologic complete response rates, they also increase toxicity. Intraoperatively, ICI monotherapy generally preserves tissue planes without increasing surgical delays or major wound complications. Standard biomarkers like PD-L1 and TMB, alongside emerging tools such as liquid biopsy (ctDNA), are essential for patient selection and dynamic monitoring. CONCLUSIONS: The transition to neoadjuvant immunotherapy facilitates future surgical de-escalation and function-preserving approaches. To optimize outcomes, the modern surgeon must act as a surgical immunologist, interpreting translational data to guide real-time operative planning.
2026-04-14 | The Molecular Landscape of CASTLE: A Rare Thymus-like Head and Neck Cancer
Carcinoma showing thymus-like differentiation (CASTLE) is a rare malignancy arising in the thyroid or neck, with an uncertain cellular origin that complicates both diagnosis and treatment. To better understand its molecular underpinnings and identify potential therapeutic avenues, we conducted integrated whole-exome and transcriptome sequencing on six CASTLE and six thymic carcinoma samples. Whole-exome sequencing (WES) was performed on all 12 samples, while RNA sequencing was successful for 1 CASTLE and 6 thymic carcinoma samples. Our analysis included somatic mutation profiling, mutational signature deconvolution, differential gene expression, and characterization of tumor microenvironment for the cases with available data, with comparisons to genomic data from other thyroid cancers. CASTLE tumors demonstrated a higher median tumor mutational burden than thymic carcinoma and lacked the common BRAF and RAS mutations typically found in thyroid cancers. They harbored alterations in genes such as TRHDE, cilia-associated genes (ANKS6, CFAP46, DNAH6), and Wnt signaling components (TRRAP, BCL9L), as well as mutational signatures suggestive of mismatch repair deficiency and oxidative damage. MSIsensor-pro analysis of the WES data provided support for the potential for mismatch repair deficiency in a subset of CASTLE samples. Exploratory transcriptomic analysis from a single CASTLE case showed downregulation of thyroid follicular markers and an “immune-hot”, lymphocyte-rich microenvironment, closely resembling that of thymic carcinoma. While these findings require validation in larger cohorts, they support a thymic origin for CASTLE and establish its molecular distinction from follicular-derived thyroid cancers. The immunogenic tumor landscape suggests that immune checkpoint inhibitors, particularly those targeting PD-1/PD-L1, may be a promising therapeutic strategy, alongside emerging targets for precision oncology.
2026-01-27 | Immune checkpoint inhibitors in nuclear protein in testis carcinoma treatment: evidence of limited clinical benefits from a case series
Nuclear protein in testis (NUT) carcinoma is an extremely rare and aggressive malignancy characterized by NUTM1 gene rearrangement. It frequently develops in the lungs or the head and neck region as a poorly differentiated squamous cell carcinoma. The prognosis is generally poor and particularly dismal in cases with a pulmonary origin. Given the lack of an established standard treatment and the rapid disease progression, recently, immune checkpoint inhibitors (ICIs) have attracted attention, particularly in combination with platinum-based chemotherapy. We report a case series of four patients with pulmonary NUT carcinoma who received ICI-containing regimens as first-line therapy. Three patients received ICIs in combination with platinum-based chemotherapy and showed transient tumor shrinkage, although all ultimately experienced disease progression and died. One patient received ICI monotherapy owing to poor performance status and showed no clinical response. The median progression-free and overall survival were 53 and 108 days, respectively. Given the limited treatment options for NUT carcinoma, a combination of ICIs with platinum-based chemotherapy may represent a potential first-line treatment option. However, their efficacy remains limited.
2026-01-09 | Quality of life in patients with pheochromocytoma and paraganglioma: influence of tumor type, genetics and surgical resection
CONTEXT: Pheochromocytomas and paragangliomas (PPGL) are rare endocrine tumors with high heritability. Carriers of pathogenic variants (PVs) in susceptibility genes face a lifelong risk of recurrence or metastatic disease. Quality of life (QoL) in patients with PPGL, a history of PPGL and PV carriers, remains insufficiently studied. METHODS: We assessed patient-reported QoL in patients with PPGL before and after surgery and in carriers of susceptibility PVs for the development of PPGLs within the prospective ProsPheo study. QoL was evaluated using standardized questionnaires (SF-12, PHQ-D, and GAD-7). RESULTS: A total of 202 participants were analyzed. SF-12 physical component summary scores (PCS) differed significantly across subgroups (P = .006), with the lowest PCS scores in patients with metastatic PPGL (40.3) and unresected head and neck paragangliomas (HNPGL) (40.0), compared with PV carriers after curative PPGL resection (50.2). Within metastatic disease, hormonally active tumors showed lower PCS scores than inactive tumors (36.9 vs 46.2; P = .03). Perceived physical health in carriers of a PV without history of a PPGL was significantly lower compared to the age-matched general population. Mental health was not significantly impaired across all subgroups. Clinically relevant anxiety was reported in 15.2% of PV carriers and 21.7% of patients with unresected HNPGLs, compared with about 5.9% in the general population. CONCLUSIONS: Patient-reported physical health was reduced in patients with metastatic PPGLs, unresected HNPGLs, and PV carriers without prior PPGL, whereas patients after curative PPGL resection reported QoL comparable to the general population, regardless of PV status, suggesting that annual follow-up has minimal impact on QoL.
2026-01-01 | Biomarker-guided precision immunotherapy in head and neck cancer: Harnessing toripalimab-tpzi as a PD-1 checkpoint inhibitor
The recent humanized anti-programmed death-1 (PD-1) monoclonal antibody, toripalimab-tpzi, has now become a promising immunotherapeutic agent in the control of head and neck cancer (HNC), especially head and neck squamous cell carcinoma (HNSCC), the most common form of the disease, constituting approximately 90% of all cases. In this conceptual review, the authors critically examine the molecular processes, clinical activity, safety, biomarker-guided precision of their approach, resistance biology, and combination regimens of toripalimab-tpzi in HNSCC. Toripalimab-tpzi offers antitumor effects due to selective inhibition restricting the PD-1/programmed death-ligand 1 (PD-L1) axis, which restores cytotoxic T-cell activity and intensifies the antitumor immune responses. In the Phase I-III clinical trials on recurrent or metastatic HNSCC, the objective response rates of toripalimab-tpzi were around 20-36, the median progression-free survival (PFS) was 2.8-4.5 months, and the median overall survival was 10-15 months, especially with PD-L1-enriched populations. The safety profile is comparable to the PD-1 class effects, and the majority of the immune-related adverse events (irAEs) were grade 1-2 and grade 3-4 toxicities that were rare in less than 20 percent of the patients. The new evidence is pointing to the significance of patient selection according to biomarker to maximize therapeutic response. The single predictive utility of such markers as PD-L1 expression, tumor mutational burden, microsatellite instability, tumor-infiltrating lymphocytes, or immune gene signatures is variable, with the deficiency of single-biomarker systems. Integration therapy involving toripalimab-tpzi and platinum-based chemotherapy, radiotherapy, targeted therapies, or other immunomodulators has been shown to result in better response rates (up to 4060% in select studies) and may result in resistance being overcome by intrinsic or acquired immunomodulatory mechanisms. Unlike previous generalized scientific reviews of PD-1/PD-L1 blocking in HNSCC, the article summarizes the toripalimab-tpzi-specific clinical and translational data and suggests a precautionary immunotherapy paradigm and a biomarker-driven combination-resistance management plan. Taken together, the above data favor toripalimab-tpzi as a clinically significant supplement to precision immunotherapy in HNSCC, whose optimal role is determined by the biologically informed selection of patients and the rational combination of multimodal treatment. • Toripalimab-tpzi enables biomarker-guided precision immunotherapy in HNSCC • Clinical benefit is enriched in PD-L1–positive and immune-inflamed tumors. • Composite biomarkers outperform PD-L1 alone in predicting response • Rational combination strategies help overcome intrinsic and acquired resistance. • Precision algorithms are key to optimizing efficacy and minimizing toxicity.
small molecules
2026-06-01 | Advances in Systemic Therapies for Rare Head and Neck Malignancies: Spotlight on Adenoid Cystic Carcinoma, Differentiated Thyroid Cancer, and Sinonasal/Paranasal Tumors
Rare head and neck cancers, including adenoid cystic carcinoma (ACC), differentiated thyroid cancer (DTC), and sinonasal/paranasal tumors, represent a diverse pathology with shared clinical challenges, including heterogenous biology and limited prospective evidence. Although these tumors may present commonly as locally aggressive disease, there is distant metastatic potential, underscoring the complexities of treatment selection. Precision medicine has broadened the landscape of treatment modalities for these rare tumors; nevertheless, there are persistent critical needs for more robust molecular predictors to refine risk stratification, enhance prognostic accuracy, and drive innovative targeted therapies. In ACC, multikinase inhibitors remain a central systemic therapy strategy; however, emerging options targeting MYB and NOTCH pathways, theranostics by prostate-specific membrane antigen‑directed radioligand therapy, and immunotherapy are providing novel alternatives of care for patients with these malignancies. In radioactive iodine‑refractory DTC, redifferentiation approaches using an antineoplastic agent for 4-6 weeks have restored I131 uptake in selected patients. Clinical trials and real-world experiences with BRAF, MEK, RET, and NTRK inhibitors have revealed that a subset of patients may benefit from this approach to delay long-term systemic therapy initiation. Molecular testing has enabled better classification of sinonasal tumors, in which multimodal therapeutic approaches have been the cornerstone, with newer biomarkers guiding the consideration of immunotherapy implementation among other alternatives. In these rare malignancies, patient-centered care, including timely access to expert multidisciplinary teams in high-volume centers, prioritization for clinical trial enrollment, and balancing options on the basis of molecular profiles, remains essential to advance the field.
2026-05-14 | Endovascular embolization in the treatment of patients with complicated malignant neoplasms of the head and neck organs
Malignant neoplasms of the head and neck organs are quite rare, but they are the leading causes of death in cancer patients. High mortality rates are associated with the peculiarities of the clinical course of tumors of this localization, their pronounced effect on the patient’s somatic status, as well as the highly toxic methods of antitumor treatment used. Due to the unique topographic and anatomical features of the structures of the head and neck, malignant tumors in the process of their growth can give a wide range of complications, significantly aggravating the clinical prognosis. Bleeding in the head and neck prevents the initiation or continuation of antitumor treatment, and in some cases is the reason for its complete rejection. The main method of hemostasis in such situations for a long time was open ligation of the branches of the external carotid artery or its trunk, which often led to the development of irreversible ischemic disorders and frequent recurrence of bleeding due to the opening of collateral blood flow. These disadvantages required the development and widespread implementation of new methods of hemostasis, which make it possible to stop bleeding in cancer patients with a lower risk of complications. Vascular embolization is used as one of these methods today. The purpose of this work is to review the scientific literature data on the methodology of vascular embolization of tumors of the head and neck, modern devices used for endovascular embolization and embolizing agents, complications of intraarterial embolization of tumor vessels during tumor bleeding, as well as the clinical significance of this method in the treatment of complications of the course of the tumor process in the head and neck.
2026-02-23 | Genetic mutations in recurrent and/or metastatic nasal carcinoma: A pathological comparison based on the Japanese national genomic profiling database
Comprehensive genome profiling (CGP) has been reimbursed under Japan's national health insurance since 2019 for patients with advanced solid tumors, including head and neck cancers. Despite this, genomic insights into rare nasal carcinoma subtypes remain limited, hindering subtype-specific therapeutic strategies. To characterize the genetic mutational profiles of recurrent and/or metastatic nasal carcinoma across histological subtypes using data from the Japanese National Genomic Profiling Database. Materials and methods: Genomic data from 135 patients with nasal carcinoma were analyzed, including squamous cell carcinoma (SCC) (n = 48), neuroendocrine tumor (NET) (n = 52), adenocarcinoma (n = 20), and undifferentiated carcinoma (n = 15). All cases were registered in the Center for Cancer Genomics and Advanced Therapeutics (C-CAT), National Cancer Center Japan, between June 2019 and May 2025. Mutations were identified via FoundationOne CDx next-generation sequencing. Survival analysis employed the log-rank test and Cox proportional hazards model. Results: TP53 and KMT2D were frequently mutated across all subtypes. In SCC, KMT2D (p = 0.03), EGFR (p = 6.4 × 10-), and RICTOR (p = 0.03) mutations correlated with poor prognosis. No significant differences were found among subtypes in mutation count or tumor mutational burden (TMB). Discussion: Shared and distinct mutational patterns were observed, with prognostic relevance in SCC. These findings underscore the importance of genomic stratification in rare nasal carcinomas. Conclusions: CGP reveals key genetic drivers in nasal carcinoma, supporting its role in precision oncology and informing future subtype-specific therapeutic approaches.
2026-01-29 | Accuracy of Sentinel Lymph Node Biopsy to Predict Nodal Metastasis in Head and Neck Merkel Cell Carcinoma
Importance: Merkel cell carcinoma (MCC) is a rare neuroendocrine malignant neoplasm often affecting the head and neck. Sentinel lymph node biopsy (SLNB) is routinely performed; however, the accuracy and reliability of SLNB for head and neck MCC remains unknown. Objective: To determine the accuracy of SLNB in head and neck MCC and inform pre- and post-SLNB risk stratification. Design, Setting, and Participants: This was a single-center cohort study of patients with clinically node-negative head and neck MCC who underwent SLNB from January 2006 to January 2025; median (IQR) follow-up was 1.9 (0.8-4.6) years. Data analysis was conducted from April to November 2025. Interventions: SLNB outcome and nodal disease status. Main Outcomes and Measures: SLNB accuracy measured by sensitivity, negative predictive value (NPV), and false negative (FN) rate. FN SLNB was defined as regional nodal recurrence at the first site of recurrence after a negative SLNB without concurrent local or in-transit recurrence. Factors associated with failed SLNB (defined as nonvisualization on lymphoscintigraphy, inability to identify the sentinel lymph node intraoperatively, or absence of nodal tissue in the pathology specimen) were evaluated using effect-size measures. Cox proportional hazards model identified factors associated with nodal disease, comparing patients who developed nodal disease (identified by SLNB or subsequent clinical and/or imaging evaluation) with those who did not develop nodal disease during follow-up. Results: Of 86 participants (mean [SD] age, 75.6 [9.6] years; 36 female [41.9%] and 50 male [58.1%] individuals) with clinically node-negative head and neck MCC, 69 (80.2%) underwent SLNB. Occult nodal disease was identified in 19 participants (27.5%). SLNB sensitivity was 52.6% (10 of 19 patients), NPV was 88.6% (31 of 35 patients), and FN rate was 26.7% (4 of 15 patients). SLNB failed in 29.0% (20 of 69 patients), with tumor location, female sex, and older age having medium to large effect on SLNB failure. Lymphovascular invasion, infiltrative growth pattern, and tumor size had the largest effect size on nodal disease with Cox analysis. Conclusions and Relevance: In this large single-site cohort study, SLNB remained a useful tool in head and neck MCC, identifying more than half of occult nodal disease. Given reduced performance in this region, patients with failed SLNB or negative SLNB plus high-risk features may benefit from tailored management, including intensified therapy and surveillance.
2026-01-01 | “Primary Chronic Granulomatous Sialadenitis of the Parotid Gland: A Rare Case Report”
Parotid gland tumors account for 1-3% of head and neck tumors and approximately 70-80% of all salivary gland neoplasms, most of which are benign and predominantly occur in adults. However, non-neoplastic parotid lesions, such as chronic granulomatous sialadenitis, are rare entities with an estimated incidence of 1.7%. They may present as slow-growing, painless masses that are clinically indistinguishable from benign tumors, representing a diagnostic challenge. CASE REPORT: We report the case of a patient with a left parotid mass of two years' evolution, initially evaluated by fine-needle aspiration biopsy (FNAB) (reported as Milan category II, non-neoplastic) and subsequently treated with surgical excision. Histopathological analysis revealed chronic granulomatous sialadenitis, with no evidence of infectious agents or apparent systemic involvement, suggesting a possible autoimmune or idiopathic origin. DISCUSSION: FNAB is the initial diagnostic method of choice due to its low cost, accuracy, and minimal complication rate. It shows a sensitivity of 80%, specificity of 97%, positive predictive value of 90%, and negative predictive value of 94%. However, when cytological findings do not allow confirmation of the nature of the lesion, or when the clinical course is atypical, surgical excision with subsequent histopathological evaluation becomes necessary. Ideally, intraoperative biopsy analysis should be available to establish the definitive diagnosis. CONCLUSION: Granulomatous reactions in the parotid gland may result from ductal obstruction, specific infections, or systemic granulomatous diseases, making clinico-pathological and immunohistochemical correlation essential. This case highlights the importance of considering inflammatory lesions in the differential diagnosis of parotid masses and the need for a stepwise approach that combines cytology, imaging, and selective surgery.
cell therapies
2025-09-23 | Pectoralis Major Myocutaneous Flap Reconstruction in Locally Advanced Head and Neck Malignant Solitary Fibrous Tumors: Functional and Oncologic Outcomes
BACKGROUND: Malignant solitary fibrous tumor (MSFT), a rare mesenchymal malignancy with aggressive local infiltration and metastatic propensity, poses significant therapeutic challenges in the head and neck region. Locally advanced cases requiring extensive resection necessitate reliable reconstruction strategies to balance oncologic control and functional preservation. METHODS: We conducted a retrospective analysis of 7 consecutive patients with T3/T4N0-2M0 head and neck MSFT undergoing radical resection and immediate pedicled pectoralis major myocutaneous flap (PMMF) reconstruction (2017-2024). Outcomes included surgical parameters, University of Washington Quality of Life (UW-QoL) scores at 6 months, and disease-free survival (DFS). RESULTS: All patients achieved R0 resection with 100% flap survival. Mean operative time was 276±24 minutes, with no major complications (Clavien-Dindo ≥III). UW-QoL scores demonstrated significant functional recovery: pain control (75.6±3.2), swallowing (78.6±5.1), and speech (95.7±2.4), with total scores exceeding 800/1200 in all cases. Over a mean follow-up of 35.1 months (range: 6-84), DFS was 100%. Four high-risk patients received adjuvant radiotherapy (50-70 Gy) without acute toxicity. CONCLUSIONS: PMMF reconstruction provides a technically accessible solution for locally advanced head and neck MSFT, enabling radical tumor clearance while optimizing functional rehabilitation and QoL. The absence of microvascular anastomosis and a low morbidity profile make it particularly suitable for resource-constrained settings. Prospective multicenter studies are needed to validate these findings.
2025-09-17 | Tumor–Immune Interactions in Pediatric Oral Rhabdomyosarcoma: A Narrative Review on Immuno-Oncology and Emerging Therapies
Pediatric oral rhabdomyosarcoma (RMS) is a rare and aggressive cancer of the head and neck, characterized by a complex and mostly immunosuppressive tumor-immune microenvironment. Unlike adult cancers, pediatric RMS typically exhibits a "cold" immune profile, characterized by minimal T-cell infiltration, a low mutational burden, and resistance to immune checkpoint blockade. The tumor's location in the oral cavity adds difficulty to treatment because of anatomical and functional limitations. Additionally, the presence of fusion oncogenes, such as PAX3:FOXO1, hampers immunogenicity and treatment response by disrupting antigen presentation and reducing immune cell infiltration. Advances in immuno-oncology have introduced new strategies, including immune checkpoint inhibitors, chimeric antigen receptor (CAR) therapies, cancer vaccines, and oncolytic viruses. However, these approaches face specific challenges in the pediatric population due to developmental immune factors. This narrative review highlights recent findings on the immunobiology of pediatric oral RMS, focusing on tumor-immune interactions and their impact on disease progression and treatment resistance. We reviewed the cellular components of the TIME, the mechanisms of immune evasion, and the expression of immune checkpoints, including PD-L1 and B7-H3. Emerging immunotherapies, including CAR-T, CAR-NK, and CAR-CIK cell therapies; checkpoint inhibitors; oncolytic viruses; and cancer vaccines, are discussed, with an emphasis on their current limitations and potential to transform the pediatric RMS immune landscape.
2025-07-11 | Multimodal delineation of a layer of effector function among exhausted CD8 T cells in tumors
Undescribed functional axes may intersect with the trajectory of T cell exhaustion (TEX) to contribute to the antitumoral functions of CD8 T cells. By leveraging fluorescent transcriptional reporting of the T cell activation marker Cd69, we defined a classifier for potent versus suboptimal CD69+ activation states arising from T cell stimulation. In tumors, this delineation provided an additional functional readout among TEX subsets, marked by enhanced effector molecule production. The more potent Cd69-TFPhi state was the most prominent in a T cell-mediated tumor clearance model, displaying increased engagement and superior tumor cell killing. Simultaneous analysis of gene and protein expression in human head and neck tumors enabled a similar strategy to identify Cd69RNAhiCD69+ cells with enhanced functional features compared with Cd69RNAloCD69+ cells among intratumoral CD8 T cell subsets. Thus, refining the T cell functional landscape in tumors potentiates the identification of rare, potent effectors that could be leveraged for improving cancer treatment.
2025-05-09 | Features and Management of Osteoradionecrosis of the Skull Base A Systematic Review and Meta‐Analysis
BACKGROUND: Skull base osteoradionecrosis (SBORN) is a rare but potentially devastating consequence of radiotherapy (RT) for head and neck tumors. This study aims to synthesize existing literature and analyze treatment outcomes among patients with SBORN. METHODS: A systematic literature search was conducted following PRISMA guidelines. Adult patients with SBORN were included. Random effects modeling was used for meta-analysis. RESULTS: Overall, 29 studies with 593 patients were included. Mean time to presentation of SBORN following RT was 84.3 months. Analysis of treatment modalities revealed 165 patients underwent conservative management, 258 surgery without free flap reconstruction, and 101 surgery with free flap reconstruction. Meta-analysis demonstrated a significantly lower death rate in patients who underwent free tissue transfer, compared to those who were managed conservatively (p < 0.01). CONCLUSIONS: SBORN represents a challenging complication following radiotherapy of head and neck tumors. The consequences can be catastrophic; therefore, prompt identification and treatment are key. The use of vascularized free tissue transfer for patients with significant structural defects from SBORN may improve survivorship compared to conservative treatment measures; however, further research on treatment outcomes is warranted.
2023-09-15 | Abstract PO-061: Utilizing T cell receptor-based therapy to treat anaplastic thyroid cancer
Abstract Anaplastic thyroid cancer (ATC) is considered to be among the most aggressive solid tumors in thyroid tissue, with a 6-month medium overall survival. The effectiveness of conventional treatments, such as surgery, chemotherapy, and radiation therapy, in extending the survival of patients is limited. Therefore, there is an urgent need to develop novel systemic therapeutic interventions that can induce long-lasting responses. Adoptive cell therapy has revolutionized cancer immunotherapy by successfully treating B-cell malignancies through chimeric antigen receptor-based approaches. For solid tumors, there is a need to identify a broader spectrum of tumor target antigens, and the field has now shifted towards leveraging T cell receptors (TCRs) which can recognize both intracellular and extracellular antigen-derived epitopes displayed by major histocompatibility complex (MHC) molecules. Our team has established an immunogenic epitope discovery pipeline, followed by a specialized protocol for empiric validation of candidate epitopes. We characterized immunogenic epitopes by coupling tandem mass spectrometry of MHC-eluted peptides with tumor proteome and MHC-peptide binding prediction databases. By integrating multi-omics data (genomics, transcriptomics, and immunopeptidomics) from both in-house and publicly available ATC databases, we find that ATC exhibits significant upregulation of pathways associated with microtubule-based movement, chromosome segregation, kinetochore complex components, and mitotic cytokinesis. We have identified several HLA-A2- and A24-restricted immunogenic peptides representing these pathway-associated proteins that covered more than 60% of the patient population. NDC80 kinetochore complex components (NDC80) and PDZ binding kinase (PBK) have been selected as top candidates. Peptides derived from NDC80 and PBK were subsequently utilized to isolate antigen-specific T cells from peripheral blood and identify cognate TCR. These peptide-specific T lymphocytes elicit cytotoxicity toward multiple HLA-matched ATC cell lines and tumor samples in vitro and in vivo. Furthermore, understanding the role of these genes in ATC tumorigenesis may identify novel combination strategies with adoptive cell therapy to yield synergistic effects. In conclusion, our studies contribute to the development of a new treatment paradigm for treating ATC, with the prospect of improving survival and outcome in this rare cancer patient population. Citation Format: Shao-Hsi Hung, Ying Henderson, Ke Pan, Yulun Chiu, Shailbala Singh, Yunyun Chen, Stephen Lai, Cassian Yee. Utilizing T cell receptor-based therapy to treat anaplastic thyroid cancer [abstract]. In: Proceedings of the AACR-AHNS Head and Neck Cancer Conference: Innovating through Basic, Clinical, and Translational Research; 2023 Jul 7-8; Montreal, QC, Canada. Philadelphia (PA): AACR; Clin Cancer Res 2023;29(18_Suppl):Abstract nr PO-061.
proteins
2026-03-19 | Rare Presentation of Myxoid Pleomorphic Liposarcoma in Cervical Prevertebral Region
Myxoid pleomorphic liposarcoma (MPLS) is a rare subtype of liposarcoma, with the mediastinum being the most common site.Prevalence in the head and neck region is extremely rare.These tumors are very aggressive due to the presence of TP53 mutation and widespread loss of heterozygosity (LOH).These tumors are usually asymptomatic and therefore diagnosed at advanced stages.Surgery followed by adjuvant chemoradiation remains the mainstay of treatment.We report a case of MPLS at a very rare location, the cervical prevertebral region (C4-D3).
2026-03-09 | A Rare and Giant Dedifferentiated Liposarcoma of Buccal Fat Pad
Although liposarcoma is one of the most common soft-tissue sarcomas, facial localization is extremely rare. The buccal fat pad is an important anatomic structure located in the face that recently gained interest as a result of increasing research on facial anatomy. In this paper, we report a case of giant liposarcoma originating from the buccal fat pad. The precise localization of the tumor was determined preoperatively with computed tomography examination. The liposarcoma that invaded the body· and the extensions of the buccal fat pad was resected completely. The pathological examination revealed a sclerosing, dedifferenciated liposarcoma, which is known to be very rare in the head and neck region. Only 51 cases of dedifferentiated liposarcoma of the head and neck have been reported, of theses only 5 in the cheek. In this report we present the only case of a dedifferentiated liposarcoma originating in the left buccal fat pad of the cheek.
2026-02-26 | Clinicopathological Features of Extranodal Head and Neck Lymphomas
Objective: Primary extranodal lymphomas of the head and neck region relatively rare and represent a biologically distinct subset. The diagnosis and differential diagnosis of head and neck lymphomas are important and deserve special attention. The aim of the present study was to retrospectively evaluate patients diagnosed with primary head and neck lymphomas at the Department of Pathology between January 2020 and January 2026. Histopathological subtypes, localization, relative frequencies, overall survival were analyzed. Material and Method: This retrospective study included 31 cases diagnosed with lymphoma involving the head and neck region. Medical records were reviewed. Histopathological slides re-evaluated under light microscopy by experienced pathologists. All cases were classified according to the current World Health Organization (WHO) classification of tumors of haematopoietic and lymphoid tissues. An extensive immunohistochemical panel was applied. Statistical analyses were performed using SPSS statistical software. Results: The study group included 31 patients with head-and-neck lymphoma. The most common histological type was diffuse large B-cell lymphoma (DLBCL) (54.8%). Other histological subtypes included follicular lymphoma (FL), mantle cell lymphoma (MCL), extranodal natural killer/T-cell lymphoma (NKTCL), anaplastic large cell lymphoma (ALCL), and Hodgkin lymphoma (HL). Most common location was tonsil (38.7%). Other locations were nasopharynx, oral cavity, nasal cavity, salivary gland and thyroid. Epstein–Barr virus (EBV) positivity was detected in two patients (6.5%), and human immunodeficiency virus (HIV) infection was identified in two patients (6.5%). At the time of last follow-up, 27 patients (87.1%) were alive, whereas four patients (12,9%) had died. The mortality rate of 6.5%. The median overall survival was 28 months (95% CI: 10–45). Conclusions: Malignant lymphoma should be kept in mind when evaluating head and neck masses, and histopathological assessment of the affected tissue remains the cornerstone of diagnosis.
2026-01-12 | Solitary Fibrous Tumors of the Head and Neck: A Report of Two Cases and Review of the Literature
Solitary fibrous tumors (SFTs) are rare mesenchymal neoplasms, particularly uncommon in the cheek and floor of the mouth. We report two such cases to emphasize diagnostic and management considerations. A retrospective review at a tertiary academic hospital included two female patients aged 40 and 49 years. Both underwent clinical evaluation, radiologic imaging, surgical excision, and histopathologic assessment with immunohistochemistry, including STAT6, CD34, BCL2, and CD99. Risk stratification was based on mitotic index and histological features. The first case involved a 40-year-old woman with a 3.5 × 2 × 1.5 cm lesion in the floor of the mouth. Histopathology confirmed SFT with STAT6, CD34, BCL2, and CD99 positivity; the mitotic rate was 1/10 HPF, consistent with low risk. The second case involved a 49-year-old woman with a 1.5 × 1.5 cm right cheek mass, also confirmed as SFT with a similar immunoprofile and negligible mitotic activity, indicating very low risk. Both patients underwent complete excision and recovered uneventfully, with no recurrence on a two-week follow-up. These cases illustrate that SFTs, though rare in the cheek and floor of the mouth, should be considered in the differential diagnosis of soft tissue tumors. Diagnosis depends on histopathology and immunohistochemistry, particularly STAT6. Complete excision is usually curative, but long-term surveillance remains essential.
2025-11-20 | Malignant peripheral nerve sheath tumor of the head and neck in the pericarotid plexiform: a case report of a rare disease entity
Abstract Malignant peripheral nerve sheath tumors (MPNST) are rare high-grade sarcomas that arise from cells of the peripheral nerve sheaths. They are considered as one of the most aggressive malignant lesions in the head and neck region, with a notably high rate of local recurrence. In the current case report, we described A 28-year-old female known to have neurofibromatosis type 1 (NF1) with a rare case of MPNST in the right sided neck and reviewed the relevant literature. Computed tomography (CT) scan of the head and neck with contrast was initially obtained and showed right sided soft tissue neck mass that was predominantly cystic. Head and neck magnetic resonance imaging (MRI) with contrast demonstrated a large right pericarotid plexiform heterogeneously enhancing tumor. Subsequently surgical excision of the mass under general anesthesia was done. Histopathology of the mass was reported to be malignant peripheral nerve sheath tumor (low grade). The patient was kept on regular follow-up in the head and neck combined clinic, and the surgical site healed well in six weeks, with no sign of local recurrence or metastasis.
other
2026-01-05 | Ganglioneuroma has a potential for lymph node metastasis, not impacting recurrence
Ganglioneuromas are rare, benign tumors arising from neuroblastic cells in the autonomic sympathetic nervous system. While generally considered indolent, limited case reports suggest their potential for regional metastasis. This retrospective study analyzed the clinical, demographic, and pathological features of 25 adult patients diagnosed with ganglioneuroma at Bursa Uludag University, Faculty of Medicine, between April 2007 and November 2023. The cohort comprised 18 females (72%) and seven males (28%), with a median age of 42 years (range: 19-79). Tumors were most commonly located in the abdomen (64%), followed by the thoracic (24%) and head and neck regions (12%), with the adrenal gland being the primary site in 32% of cases. Symptoms were present in 56% of patients, including pain, vision loss, hypertension, and palpable masses, while the remaining were asymptomatic. Surgical resection was performed in 92% of cases, with a median tumor size of 7.5 cm (1.5-18 cm). Median follow-up time was 88.3(16.2 - 217.8) months. Regional lymph node metastases were identified in 8% of patients, but no distant metastases or recurrences were observed during follow-up. These findings, including the novel observation of regional metastases, contribute valuable insights to the limited literature on ganglioneuromas. Despite its benign nature, this study highlights the potential for lymph node metastasis. However, the relationship between lymph node metastasis and recurrence has not been documented. In this context, further research is essential to better understand the risk factors, tumorigenesis, and the optimal management strategies for this rare tumor.
2023-12-26 | NUT carcinoma in pediatric patients: Characteristics, therapeutic regimens, and outcomes of 11 cases registered with the German Registry for Rare Pediatric Tumors (STEP)
Abstract Background and aims Nuclear protein of the testis (NUT) carcinoma (NC) is a rare and highly aggressive tumor defined by the presence of a somatic NUTM1 rearrangement, occurring mainly in adolescents and young adults. We analyzed the clinical and biological features of German pediatric patients (≤18 years) with NC. Methods This study describes the characteristics and outcome of 11 children with NC registered in the German Registry for Rare Pediatric Tumors (STEP). Results Eleven patients with a median age of 13.2 years (range 6.6–17.8) were analyzed. Malignant misdiagnoses were made in three patients. Thoracic/mediastinal tumors were found to be the primary in six patients, head/neck in four cases; one patient had multifocal tumor with an unknown primary. All patients presented with regional lymph node involvement, eight patients (72.7%) with distant metastases. Seven patients underwent surgery, eight radiotherapy with curative intent; polychemotherapy was administered in all patients. Novel treatment strategies including immunotherapy, targeted therapies, and virotherapy were applied in three patients. Median event‐free survival and overall survival were 1.5 and 6.5 months, respectively. Conclusions Every undifferentiated or poorly differentiated carcinoma should undergo testing for the specific rearrangement of NUTM1, in order to initiate an intense therapeutic regimen as early as possible. As in adults, only few pediatric patients with NC achieve prolonged survival. Thus, novel therapeutic strategies should be included and tested in clinical trials.
2023-02-28 | Knockdown of lncRNA SNHG16 Attenuates the Proliferation and Radioresistance of Nasopharyngeal Carcinoma Cells by Mediating miR-31-5p/SFN Axis.
Nasopharyngeal carcinoma (NPC) is a rare head and neck tumor that threatens people's health. Radiotherapy is a major treatment for NPC, however, radioresistance of the NPC cells may contribute to treatment failure. LncRNA SNHG16 was upregulated in NPC; however, the function of SNHG16 in radioresistant NPC cells remains unexplored. RT-qPCR was applied for detecting SNHG16, miR-31-5p and SFN levels. MTT assay and colony formation assay were applied to assess the cell viability and proliferation. Dual luciferase was applied for assessing the relation among SNHG16, miR-31-5p and SFN. SFN level in NPC cells was examined by Western blot. The level of SNHG16 and SFN in NPC cells was significantly upregulated by exposure to radiation. In addition, silencing of SNHG16 or miR-31-5p mimics notably attenuated radioresistance of NPC cells. SNHG16 could positively regulate the expression of SFN in NPC cells through binding with miR-31-5p. Furthermore, SNHG16 downregulation obviously attenuated the proliferation and radioresistance of NPC cells by regulation of miR-31-5p/SFN axis. Knockdown of lncRNA SNHG16 attenuates radioresistance of nasopharyngeal carcinoma cells by miR-31-5p/SFN axis. Thus, our research data show a novel method for improving the efficacy of radiotherapy for NPC.
antibodies
2026-06-22 | Integrating Immunotherapy Into Head and Neck Surgery: Bridging Tumor Biology to Perioperative Decision‐Making, a Review
OBJECTIVE: To provide a comprehensive review of the biological rationale, clinical evidence, and practical perioperative management of immunotherapy for the head and neck surgeon. SUMMARY BACKGROUND DATA: Standard treatment for resectable head and neck squamous cell carcinoma (HNSCC) has reached a survival plateau, with over 50% of patients experiencing recurrence. The integration of immune checkpoint inhibitors (ICIs) into the neoadjuvant window represents a paradigm shift toward biologically adapted surgical intervention. RESULTS: Neoadjuvant immunotherapy capitalizes on an intact immune substrate to create an in situ vaccine, avoiding the post-surgical immune desert that limits adjuvant efficacy. Emerging phase III data confirm that perioperative ICI significantly improves event-free survival. Successful implementation requires the surgeon to navigate unique diagnostic challenges, such as distinguishing rare but anatomically risky pseudoprogression from true progression. While combination therapies (chemoimmunotherapy or immunoradiotherapy) yield higher pathologic complete response rates, they also increase toxicity. Intraoperatively, ICI monotherapy generally preserves tissue planes without increasing surgical delays or major wound complications. Standard biomarkers like PD-L1 and TMB, alongside emerging tools such as liquid biopsy (ctDNA), are essential for patient selection and dynamic monitoring. CONCLUSIONS: The transition to neoadjuvant immunotherapy facilitates future surgical de-escalation and function-preserving approaches. To optimize outcomes, the modern surgeon must act as a surgical immunologist, interpreting translational data to guide real-time operative planning.
2026-04-14 | The Molecular Landscape of CASTLE: A Rare Thymus-like Head and Neck Cancer
Carcinoma showing thymus-like differentiation (CASTLE) is a rare malignancy arising in the thyroid or neck, with an uncertain cellular origin that complicates both diagnosis and treatment. To better understand its molecular underpinnings and identify potential therapeutic avenues, we conducted integrated whole-exome and transcriptome sequencing on six CASTLE and six thymic carcinoma samples. Whole-exome sequencing (WES) was performed on all 12 samples, while RNA sequencing was successful for 1 CASTLE and 6 thymic carcinoma samples. Our analysis included somatic mutation profiling, mutational signature deconvolution, differential gene expression, and characterization of tumor microenvironment for the cases with available data, with comparisons to genomic data from other thyroid cancers. CASTLE tumors demonstrated a higher median tumor mutational burden than thymic carcinoma and lacked the common BRAF and RAS mutations typically found in thyroid cancers. They harbored alterations in genes such as TRHDE, cilia-associated genes (ANKS6, CFAP46, DNAH6), and Wnt signaling components (TRRAP, BCL9L), as well as mutational signatures suggestive of mismatch repair deficiency and oxidative damage. MSIsensor-pro analysis of the WES data provided support for the potential for mismatch repair deficiency in a subset of CASTLE samples. Exploratory transcriptomic analysis from a single CASTLE case showed downregulation of thyroid follicular markers and an “immune-hot”, lymphocyte-rich microenvironment, closely resembling that of thymic carcinoma. While these findings require validation in larger cohorts, they support a thymic origin for CASTLE and establish its molecular distinction from follicular-derived thyroid cancers. The immunogenic tumor landscape suggests that immune checkpoint inhibitors, particularly those targeting PD-1/PD-L1, may be a promising therapeutic strategy, alongside emerging targets for precision oncology.
2026-01-27 | Immune checkpoint inhibitors in nuclear protein in testis carcinoma treatment: evidence of limited clinical benefits from a case series
Nuclear protein in testis (NUT) carcinoma is an extremely rare and aggressive malignancy characterized by NUTM1 gene rearrangement. It frequently develops in the lungs or the head and neck region as a poorly differentiated squamous cell carcinoma. The prognosis is generally poor and particularly dismal in cases with a pulmonary origin. Given the lack of an established standard treatment and the rapid disease progression, recently, immune checkpoint inhibitors (ICIs) have attracted attention, particularly in combination with platinum-based chemotherapy. We report a case series of four patients with pulmonary NUT carcinoma who received ICI-containing regimens as first-line therapy. Three patients received ICIs in combination with platinum-based chemotherapy and showed transient tumor shrinkage, although all ultimately experienced disease progression and died. One patient received ICI monotherapy owing to poor performance status and showed no clinical response. The median progression-free and overall survival were 53 and 108 days, respectively. Given the limited treatment options for NUT carcinoma, a combination of ICIs with platinum-based chemotherapy may represent a potential first-line treatment option. However, their efficacy remains limited.
2026-01-09 | Quality of life in patients with pheochromocytoma and paraganglioma: influence of tumor type, genetics and surgical resection
CONTEXT: Pheochromocytomas and paragangliomas (PPGL) are rare endocrine tumors with high heritability. Carriers of pathogenic variants (PVs) in susceptibility genes face a lifelong risk of recurrence or metastatic disease. Quality of life (QoL) in patients with PPGL, a history of PPGL and PV carriers, remains insufficiently studied. METHODS: We assessed patient-reported QoL in patients with PPGL before and after surgery and in carriers of susceptibility PVs for the development of PPGLs within the prospective ProsPheo study. QoL was evaluated using standardized questionnaires (SF-12, PHQ-D, and GAD-7). RESULTS: A total of 202 participants were analyzed. SF-12 physical component summary scores (PCS) differed significantly across subgroups (P = .006), with the lowest PCS scores in patients with metastatic PPGL (40.3) and unresected head and neck paragangliomas (HNPGL) (40.0), compared with PV carriers after curative PPGL resection (50.2). Within metastatic disease, hormonally active tumors showed lower PCS scores than inactive tumors (36.9 vs 46.2; P = .03). Perceived physical health in carriers of a PV without history of a PPGL was significantly lower compared to the age-matched general population. Mental health was not significantly impaired across all subgroups. Clinically relevant anxiety was reported in 15.2% of PV carriers and 21.7% of patients with unresected HNPGLs, compared with about 5.9% in the general population. CONCLUSIONS: Patient-reported physical health was reduced in patients with metastatic PPGLs, unresected HNPGLs, and PV carriers without prior PPGL, whereas patients after curative PPGL resection reported QoL comparable to the general population, regardless of PV status, suggesting that annual follow-up has minimal impact on QoL.
2026-01-01 | Biomarker-guided precision immunotherapy in head and neck cancer: Harnessing toripalimab-tpzi as a PD-1 checkpoint inhibitor
The recent humanized anti-programmed death-1 (PD-1) monoclonal antibody, toripalimab-tpzi, has now become a promising immunotherapeutic agent in the control of head and neck cancer (HNC), especially head and neck squamous cell carcinoma (HNSCC), the most common form of the disease, constituting approximately 90% of all cases. In this conceptual review, the authors critically examine the molecular processes, clinical activity, safety, biomarker-guided precision of their approach, resistance biology, and combination regimens of toripalimab-tpzi in HNSCC. Toripalimab-tpzi offers antitumor effects due to selective inhibition restricting the PD-1/programmed death-ligand 1 (PD-L1) axis, which restores cytotoxic T-cell activity and intensifies the antitumor immune responses. In the Phase I-III clinical trials on recurrent or metastatic HNSCC, the objective response rates of toripalimab-tpzi were around 20-36, the median progression-free survival (PFS) was 2.8-4.5 months, and the median overall survival was 10-15 months, especially with PD-L1-enriched populations. The safety profile is comparable to the PD-1 class effects, and the majority of the immune-related adverse events (irAEs) were grade 1-2 and grade 3-4 toxicities that were rare in less than 20 percent of the patients. The new evidence is pointing to the significance of patient selection according to biomarker to maximize therapeutic response. The single predictive utility of such markers as PD-L1 expression, tumor mutational burden, microsatellite instability, tumor-infiltrating lymphocytes, or immune gene signatures is variable, with the deficiency of single-biomarker systems. Integration therapy involving toripalimab-tpzi and platinum-based chemotherapy, radiotherapy, targeted therapies, or other immunomodulators has been shown to result in better response rates (up to 4060% in select studies) and may result in resistance being overcome by intrinsic or acquired immunomodulatory mechanisms. Unlike previous generalized scientific reviews of PD-1/PD-L1 blocking in HNSCC, the article summarizes the toripalimab-tpzi-specific clinical and translational data and suggests a precautionary immunotherapy paradigm and a biomarker-driven combination-resistance management plan. Taken together, the above data favor toripalimab-tpzi as a clinically significant supplement to precision immunotherapy in HNSCC, whose optimal role is determined by the biologically informed selection of patients and the rational combination of multimodal treatment. • Toripalimab-tpzi enables biomarker-guided precision immunotherapy in HNSCC • Clinical benefit is enriched in PD-L1–positive and immune-inflamed tumors. • Composite biomarkers outperform PD-L1 alone in predicting response • Rational combination strategies help overcome intrinsic and acquired resistance. • Precision algorithms are key to optimizing efficacy and minimizing toxicity.
small molecules
2026-06-01 | Advances in Systemic Therapies for Rare Head and Neck Malignancies: Spotlight on Adenoid Cystic Carcinoma, Differentiated Thyroid Cancer, and Sinonasal/Paranasal Tumors
Rare head and neck cancers, including adenoid cystic carcinoma (ACC), differentiated thyroid cancer (DTC), and sinonasal/paranasal tumors, represent a diverse pathology with shared clinical challenges, including heterogenous biology and limited prospective evidence. Although these tumors may present commonly as locally aggressive disease, there is distant metastatic potential, underscoring the complexities of treatment selection. Precision medicine has broadened the landscape of treatment modalities for these rare tumors; nevertheless, there are persistent critical needs for more robust molecular predictors to refine risk stratification, enhance prognostic accuracy, and drive innovative targeted therapies. In ACC, multikinase inhibitors remain a central systemic therapy strategy; however, emerging options targeting MYB and NOTCH pathways, theranostics by prostate-specific membrane antigen‑directed radioligand therapy, and immunotherapy are providing novel alternatives of care for patients with these malignancies. In radioactive iodine‑refractory DTC, redifferentiation approaches using an antineoplastic agent for 4-6 weeks have restored I131 uptake in selected patients. Clinical trials and real-world experiences with BRAF, MEK, RET, and NTRK inhibitors have revealed that a subset of patients may benefit from this approach to delay long-term systemic therapy initiation. Molecular testing has enabled better classification of sinonasal tumors, in which multimodal therapeutic approaches have been the cornerstone, with newer biomarkers guiding the consideration of immunotherapy implementation among other alternatives. In these rare malignancies, patient-centered care, including timely access to expert multidisciplinary teams in high-volume centers, prioritization for clinical trial enrollment, and balancing options on the basis of molecular profiles, remains essential to advance the field.
2026-05-14 | Endovascular embolization in the treatment of patients with complicated malignant neoplasms of the head and neck organs
Malignant neoplasms of the head and neck organs are quite rare, but they are the leading causes of death in cancer patients. High mortality rates are associated with the peculiarities of the clinical course of tumors of this localization, their pronounced effect on the patient’s somatic status, as well as the highly toxic methods of antitumor treatment used. Due to the unique topographic and anatomical features of the structures of the head and neck, malignant tumors in the process of their growth can give a wide range of complications, significantly aggravating the clinical prognosis. Bleeding in the head and neck prevents the initiation or continuation of antitumor treatment, and in some cases is the reason for its complete rejection. The main method of hemostasis in such situations for a long time was open ligation of the branches of the external carotid artery or its trunk, which often led to the development of irreversible ischemic disorders and frequent recurrence of bleeding due to the opening of collateral blood flow. These disadvantages required the development and widespread implementation of new methods of hemostasis, which make it possible to stop bleeding in cancer patients with a lower risk of complications. Vascular embolization is used as one of these methods today. The purpose of this work is to review the scientific literature data on the methodology of vascular embolization of tumors of the head and neck, modern devices used for endovascular embolization and embolizing agents, complications of intraarterial embolization of tumor vessels during tumor bleeding, as well as the clinical significance of this method in the treatment of complications of the course of the tumor process in the head and neck.
2026-02-23 | Genetic mutations in recurrent and/or metastatic nasal carcinoma: A pathological comparison based on the Japanese national genomic profiling database
Comprehensive genome profiling (CGP) has been reimbursed under Japan's national health insurance since 2019 for patients with advanced solid tumors, including head and neck cancers. Despite this, genomic insights into rare nasal carcinoma subtypes remain limited, hindering subtype-specific therapeutic strategies. To characterize the genetic mutational profiles of recurrent and/or metastatic nasal carcinoma across histological subtypes using data from the Japanese National Genomic Profiling Database. Materials and methods: Genomic data from 135 patients with nasal carcinoma were analyzed, including squamous cell carcinoma (SCC) (n = 48), neuroendocrine tumor (NET) (n = 52), adenocarcinoma (n = 20), and undifferentiated carcinoma (n = 15). All cases were registered in the Center for Cancer Genomics and Advanced Therapeutics (C-CAT), National Cancer Center Japan, between June 2019 and May 2025. Mutations were identified via FoundationOne CDx next-generation sequencing. Survival analysis employed the log-rank test and Cox proportional hazards model. Results: TP53 and KMT2D were frequently mutated across all subtypes. In SCC, KMT2D (p = 0.03), EGFR (p = 6.4 × 10-), and RICTOR (p = 0.03) mutations correlated with poor prognosis. No significant differences were found among subtypes in mutation count or tumor mutational burden (TMB). Discussion: Shared and distinct mutational patterns were observed, with prognostic relevance in SCC. These findings underscore the importance of genomic stratification in rare nasal carcinomas. Conclusions: CGP reveals key genetic drivers in nasal carcinoma, supporting its role in precision oncology and informing future subtype-specific therapeutic approaches.
2026-01-29 | Accuracy of Sentinel Lymph Node Biopsy to Predict Nodal Metastasis in Head and Neck Merkel Cell Carcinoma
Importance: Merkel cell carcinoma (MCC) is a rare neuroendocrine malignant neoplasm often affecting the head and neck. Sentinel lymph node biopsy (SLNB) is routinely performed; however, the accuracy and reliability of SLNB for head and neck MCC remains unknown. Objective: To determine the accuracy of SLNB in head and neck MCC and inform pre- and post-SLNB risk stratification. Design, Setting, and Participants: This was a single-center cohort study of patients with clinically node-negative head and neck MCC who underwent SLNB from January 2006 to January 2025; median (IQR) follow-up was 1.9 (0.8-4.6) years. Data analysis was conducted from April to November 2025. Interventions: SLNB outcome and nodal disease status. Main Outcomes and Measures: SLNB accuracy measured by sensitivity, negative predictive value (NPV), and false negative (FN) rate. FN SLNB was defined as regional nodal recurrence at the first site of recurrence after a negative SLNB without concurrent local or in-transit recurrence. Factors associated with failed SLNB (defined as nonvisualization on lymphoscintigraphy, inability to identify the sentinel lymph node intraoperatively, or absence of nodal tissue in the pathology specimen) were evaluated using effect-size measures. Cox proportional hazards model identified factors associated with nodal disease, comparing patients who developed nodal disease (identified by SLNB or subsequent clinical and/or imaging evaluation) with those who did not develop nodal disease during follow-up. Results: Of 86 participants (mean [SD] age, 75.6 [9.6] years; 36 female [41.9%] and 50 male [58.1%] individuals) with clinically node-negative head and neck MCC, 69 (80.2%) underwent SLNB. Occult nodal disease was identified in 19 participants (27.5%). SLNB sensitivity was 52.6% (10 of 19 patients), NPV was 88.6% (31 of 35 patients), and FN rate was 26.7% (4 of 15 patients). SLNB failed in 29.0% (20 of 69 patients), with tumor location, female sex, and older age having medium to large effect on SLNB failure. Lymphovascular invasion, infiltrative growth pattern, and tumor size had the largest effect size on nodal disease with Cox analysis. Conclusions and Relevance: In this large single-site cohort study, SLNB remained a useful tool in head and neck MCC, identifying more than half of occult nodal disease. Given reduced performance in this region, patients with failed SLNB or negative SLNB plus high-risk features may benefit from tailored management, including intensified therapy and surveillance.
2026-01-01 | “Primary Chronic Granulomatous Sialadenitis of the Parotid Gland: A Rare Case Report”
Parotid gland tumors account for 1-3% of head and neck tumors and approximately 70-80% of all salivary gland neoplasms, most of which are benign and predominantly occur in adults. However, non-neoplastic parotid lesions, such as chronic granulomatous sialadenitis, are rare entities with an estimated incidence of 1.7%. They may present as slow-growing, painless masses that are clinically indistinguishable from benign tumors, representing a diagnostic challenge. CASE REPORT: We report the case of a patient with a left parotid mass of two years' evolution, initially evaluated by fine-needle aspiration biopsy (FNAB) (reported as Milan category II, non-neoplastic) and subsequently treated with surgical excision. Histopathological analysis revealed chronic granulomatous sialadenitis, with no evidence of infectious agents or apparent systemic involvement, suggesting a possible autoimmune or idiopathic origin. DISCUSSION: FNAB is the initial diagnostic method of choice due to its low cost, accuracy, and minimal complication rate. It shows a sensitivity of 80%, specificity of 97%, positive predictive value of 90%, and negative predictive value of 94%. However, when cytological findings do not allow confirmation of the nature of the lesion, or when the clinical course is atypical, surgical excision with subsequent histopathological evaluation becomes necessary. Ideally, intraoperative biopsy analysis should be available to establish the definitive diagnosis. CONCLUSION: Granulomatous reactions in the parotid gland may result from ductal obstruction, specific infections, or systemic granulomatous diseases, making clinico-pathological and immunohistochemical correlation essential. This case highlights the importance of considering inflammatory lesions in the differential diagnosis of parotid masses and the need for a stepwise approach that combines cytology, imaging, and selective surgery.
cell therapies
2025-09-23 | Pectoralis Major Myocutaneous Flap Reconstruction in Locally Advanced Head and Neck Malignant Solitary Fibrous Tumors: Functional and Oncologic Outcomes
BACKGROUND: Malignant solitary fibrous tumor (MSFT), a rare mesenchymal malignancy with aggressive local infiltration and metastatic propensity, poses significant therapeutic challenges in the head and neck region. Locally advanced cases requiring extensive resection necessitate reliable reconstruction strategies to balance oncologic control and functional preservation. METHODS: We conducted a retrospective analysis of 7 consecutive patients with T3/T4N0-2M0 head and neck MSFT undergoing radical resection and immediate pedicled pectoralis major myocutaneous flap (PMMF) reconstruction (2017-2024). Outcomes included surgical parameters, University of Washington Quality of Life (UW-QoL) scores at 6 months, and disease-free survival (DFS). RESULTS: All patients achieved R0 resection with 100% flap survival. Mean operative time was 276±24 minutes, with no major complications (Clavien-Dindo ≥III). UW-QoL scores demonstrated significant functional recovery: pain control (75.6±3.2), swallowing (78.6±5.1), and speech (95.7±2.4), with total scores exceeding 800/1200 in all cases. Over a mean follow-up of 35.1 months (range: 6-84), DFS was 100%. Four high-risk patients received adjuvant radiotherapy (50-70 Gy) without acute toxicity. CONCLUSIONS: PMMF reconstruction provides a technically accessible solution for locally advanced head and neck MSFT, enabling radical tumor clearance while optimizing functional rehabilitation and QoL. The absence of microvascular anastomosis and a low morbidity profile make it particularly suitable for resource-constrained settings. Prospective multicenter studies are needed to validate these findings.
2025-09-17 | Tumor–Immune Interactions in Pediatric Oral Rhabdomyosarcoma: A Narrative Review on Immuno-Oncology and Emerging Therapies
Pediatric oral rhabdomyosarcoma (RMS) is a rare and aggressive cancer of the head and neck, characterized by a complex and mostly immunosuppressive tumor-immune microenvironment. Unlike adult cancers, pediatric RMS typically exhibits a "cold" immune profile, characterized by minimal T-cell infiltration, a low mutational burden, and resistance to immune checkpoint blockade. The tumor's location in the oral cavity adds difficulty to treatment because of anatomical and functional limitations. Additionally, the presence of fusion oncogenes, such as PAX3:FOXO1, hampers immunogenicity and treatment response by disrupting antigen presentation and reducing immune cell infiltration. Advances in immuno-oncology have introduced new strategies, including immune checkpoint inhibitors, chimeric antigen receptor (CAR) therapies, cancer vaccines, and oncolytic viruses. However, these approaches face specific challenges in the pediatric population due to developmental immune factors. This narrative review highlights recent findings on the immunobiology of pediatric oral RMS, focusing on tumor-immune interactions and their impact on disease progression and treatment resistance. We reviewed the cellular components of the TIME, the mechanisms of immune evasion, and the expression of immune checkpoints, including PD-L1 and B7-H3. Emerging immunotherapies, including CAR-T, CAR-NK, and CAR-CIK cell therapies; checkpoint inhibitors; oncolytic viruses; and cancer vaccines, are discussed, with an emphasis on their current limitations and potential to transform the pediatric RMS immune landscape.
2025-07-11 | Multimodal delineation of a layer of effector function among exhausted CD8 T cells in tumors
Undescribed functional axes may intersect with the trajectory of T cell exhaustion (TEX) to contribute to the antitumoral functions of CD8 T cells. By leveraging fluorescent transcriptional reporting of the T cell activation marker Cd69, we defined a classifier for potent versus suboptimal CD69+ activation states arising from T cell stimulation. In tumors, this delineation provided an additional functional readout among TEX subsets, marked by enhanced effector molecule production. The more potent Cd69-TFPhi state was the most prominent in a T cell-mediated tumor clearance model, displaying increased engagement and superior tumor cell killing. Simultaneous analysis of gene and protein expression in human head and neck tumors enabled a similar strategy to identify Cd69RNAhiCD69+ cells with enhanced functional features compared with Cd69RNAloCD69+ cells among intratumoral CD8 T cell subsets. Thus, refining the T cell functional landscape in tumors potentiates the identification of rare, potent effectors that could be leveraged for improving cancer treatment.
2025-05-09 | Features and Management of Osteoradionecrosis of the Skull Base A Systematic Review and Meta‐Analysis
BACKGROUND: Skull base osteoradionecrosis (SBORN) is a rare but potentially devastating consequence of radiotherapy (RT) for head and neck tumors. This study aims to synthesize existing literature and analyze treatment outcomes among patients with SBORN. METHODS: A systematic literature search was conducted following PRISMA guidelines. Adult patients with SBORN were included. Random effects modeling was used for meta-analysis. RESULTS: Overall, 29 studies with 593 patients were included. Mean time to presentation of SBORN following RT was 84.3 months. Analysis of treatment modalities revealed 165 patients underwent conservative management, 258 surgery without free flap reconstruction, and 101 surgery with free flap reconstruction. Meta-analysis demonstrated a significantly lower death rate in patients who underwent free tissue transfer, compared to those who were managed conservatively (p < 0.01). CONCLUSIONS: SBORN represents a challenging complication following radiotherapy of head and neck tumors. The consequences can be catastrophic; therefore, prompt identification and treatment are key. The use of vascularized free tissue transfer for patients with significant structural defects from SBORN may improve survivorship compared to conservative treatment measures; however, further research on treatment outcomes is warranted.
2023-09-15 | Abstract PO-061: Utilizing T cell receptor-based therapy to treat anaplastic thyroid cancer
Abstract Anaplastic thyroid cancer (ATC) is considered to be among the most aggressive solid tumors in thyroid tissue, with a 6-month medium overall survival. The effectiveness of conventional treatments, such as surgery, chemotherapy, and radiation therapy, in extending the survival of patients is limited. Therefore, there is an urgent need to develop novel systemic therapeutic interventions that can induce long-lasting responses. Adoptive cell therapy has revolutionized cancer immunotherapy by successfully treating B-cell malignancies through chimeric antigen receptor-based approaches. For solid tumors, there is a need to identify a broader spectrum of tumor target antigens, and the field has now shifted towards leveraging T cell receptors (TCRs) which can recognize both intracellular and extracellular antigen-derived epitopes displayed by major histocompatibility complex (MHC) molecules. Our team has established an immunogenic epitope discovery pipeline, followed by a specialized protocol for empiric validation of candidate epitopes. We characterized immunogenic epitopes by coupling tandem mass spectrometry of MHC-eluted peptides with tumor proteome and MHC-peptide binding prediction databases. By integrating multi-omics data (genomics, transcriptomics, and immunopeptidomics) from both in-house and publicly available ATC databases, we find that ATC exhibits significant upregulation of pathways associated with microtubule-based movement, chromosome segregation, kinetochore complex components, and mitotic cytokinesis. We have identified several HLA-A2- and A24-restricted immunogenic peptides representing these pathway-associated proteins that covered more than 60% of the patient population. NDC80 kinetochore complex components (NDC80) and PDZ binding kinase (PBK) have been selected as top candidates. Peptides derived from NDC80 and PBK were subsequently utilized to isolate antigen-specific T cells from peripheral blood and identify cognate TCR. These peptide-specific T lymphocytes elicit cytotoxicity toward multiple HLA-matched ATC cell lines and tumor samples in vitro and in vivo. Furthermore, understanding the role of these genes in ATC tumorigenesis may identify novel combination strategies with adoptive cell therapy to yield synergistic effects. In conclusion, our studies contribute to the development of a new treatment paradigm for treating ATC, with the prospect of improving survival and outcome in this rare cancer patient population. Citation Format: Shao-Hsi Hung, Ying Henderson, Ke Pan, Yulun Chiu, Shailbala Singh, Yunyun Chen, Stephen Lai, Cassian Yee. Utilizing T cell receptor-based therapy to treat anaplastic thyroid cancer [abstract]. In: Proceedings of the AACR-AHNS Head and Neck Cancer Conference: Innovating through Basic, Clinical, and Translational Research; 2023 Jul 7-8; Montreal, QC, Canada. Philadelphia (PA): AACR; Clin Cancer Res 2023;29(18_Suppl):Abstract nr PO-061.
proteins
2026-03-19 | Rare Presentation of Myxoid Pleomorphic Liposarcoma in Cervical Prevertebral Region
Myxoid pleomorphic liposarcoma (MPLS) is a rare subtype of liposarcoma, with the mediastinum being the most common site.Prevalence in the head and neck region is extremely rare.These tumors are very aggressive due to the presence of TP53 mutation and widespread loss of heterozygosity (LOH).These tumors are usually asymptomatic and therefore diagnosed at advanced stages.Surgery followed by adjuvant chemoradiation remains the mainstay of treatment.We report a case of MPLS at a very rare location, the cervical prevertebral region (C4-D3).
2026-03-09 | A Rare and Giant Dedifferentiated Liposarcoma of Buccal Fat Pad
Although liposarcoma is one of the most common soft-tissue sarcomas, facial localization is extremely rare. The buccal fat pad is an important anatomic structure located in the face that recently gained interest as a result of increasing research on facial anatomy. In this paper, we report a case of giant liposarcoma originating from the buccal fat pad. The precise localization of the tumor was determined preoperatively with computed tomography examination. The liposarcoma that invaded the body· and the extensions of the buccal fat pad was resected completely. The pathological examination revealed a sclerosing, dedifferenciated liposarcoma, which is known to be very rare in the head and neck region. Only 51 cases of dedifferentiated liposarcoma of the head and neck have been reported, of theses only 5 in the cheek. In this report we present the only case of a dedifferentiated liposarcoma originating in the left buccal fat pad of the cheek.
2026-02-26 | Clinicopathological Features of Extranodal Head and Neck Lymphomas
Objective: Primary extranodal lymphomas of the head and neck region relatively rare and represent a biologically distinct subset. The diagnosis and differential diagnosis of head and neck lymphomas are important and deserve special attention. The aim of the present study was to retrospectively evaluate patients diagnosed with primary head and neck lymphomas at the Department of Pathology between January 2020 and January 2026. Histopathological subtypes, localization, relative frequencies, overall survival were analyzed. Material and Method: This retrospective study included 31 cases diagnosed with lymphoma involving the head and neck region. Medical records were reviewed. Histopathological slides re-evaluated under light microscopy by experienced pathologists. All cases were classified according to the current World Health Organization (WHO) classification of tumors of haematopoietic and lymphoid tissues. An extensive immunohistochemical panel was applied. Statistical analyses were performed using SPSS statistical software. Results: The study group included 31 patients with head-and-neck lymphoma. The most common histological type was diffuse large B-cell lymphoma (DLBCL) (54.8%). Other histological subtypes included follicular lymphoma (FL), mantle cell lymphoma (MCL), extranodal natural killer/T-cell lymphoma (NKTCL), anaplastic large cell lymphoma (ALCL), and Hodgkin lymphoma (HL). Most common location was tonsil (38.7%). Other locations were nasopharynx, oral cavity, nasal cavity, salivary gland and thyroid. Epstein–Barr virus (EBV) positivity was detected in two patients (6.5%), and human immunodeficiency virus (HIV) infection was identified in two patients (6.5%). At the time of last follow-up, 27 patients (87.1%) were alive, whereas four patients (12,9%) had died. The mortality rate of 6.5%. The median overall survival was 28 months (95% CI: 10–45). Conclusions: Malignant lymphoma should be kept in mind when evaluating head and neck masses, and histopathological assessment of the affected tissue remains the cornerstone of diagnosis.
2026-01-12 | Solitary Fibrous Tumors of the Head and Neck: A Report of Two Cases and Review of the Literature
Solitary fibrous tumors (SFTs) are rare mesenchymal neoplasms, particularly uncommon in the cheek and floor of the mouth. We report two such cases to emphasize diagnostic and management considerations. A retrospective review at a tertiary academic hospital included two female patients aged 40 and 49 years. Both underwent clinical evaluation, radiologic imaging, surgical excision, and histopathologic assessment with immunohistochemistry, including STAT6, CD34, BCL2, and CD99. Risk stratification was based on mitotic index and histological features. The first case involved a 40-year-old woman with a 3.5 × 2 × 1.5 cm lesion in the floor of the mouth. Histopathology confirmed SFT with STAT6, CD34, BCL2, and CD99 positivity; the mitotic rate was 1/10 HPF, consistent with low risk. The second case involved a 49-year-old woman with a 1.5 × 1.5 cm right cheek mass, also confirmed as SFT with a similar immunoprofile and negligible mitotic activity, indicating very low risk. Both patients underwent complete excision and recovered uneventfully, with no recurrence on a two-week follow-up. These cases illustrate that SFTs, though rare in the cheek and floor of the mouth, should be considered in the differential diagnosis of soft tissue tumors. Diagnosis depends on histopathology and immunohistochemistry, particularly STAT6. Complete excision is usually curative, but long-term surveillance remains essential.
2025-11-20 | Malignant peripheral nerve sheath tumor of the head and neck in the pericarotid plexiform: a case report of a rare disease entity
Abstract Malignant peripheral nerve sheath tumors (MPNST) are rare high-grade sarcomas that arise from cells of the peripheral nerve sheaths. They are considered as one of the most aggressive malignant lesions in the head and neck region, with a notably high rate of local recurrence. In the current case report, we described A 28-year-old female known to have neurofibromatosis type 1 (NF1) with a rare case of MPNST in the right sided neck and reviewed the relevant literature. Computed tomography (CT) scan of the head and neck with contrast was initially obtained and showed right sided soft tissue neck mass that was predominantly cystic. Head and neck magnetic resonance imaging (MRI) with contrast demonstrated a large right pericarotid plexiform heterogeneously enhancing tumor. Subsequently surgical excision of the mass under general anesthesia was done. Histopathology of the mass was reported to be malignant peripheral nerve sheath tumor (low grade). The patient was kept on regular follow-up in the head and neck combined clinic, and the surgical site healed well in six weeks, with no sign of local recurrence or metastasis.
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2026-01-05 | Ganglioneuroma has a potential for lymph node metastasis, not impacting recurrence
Ganglioneuromas are rare, benign tumors arising from neuroblastic cells in the autonomic sympathetic nervous system. While generally considered indolent, limited case reports suggest their potential for regional metastasis. This retrospective study analyzed the clinical, demographic, and pathological features of 25 adult patients diagnosed with ganglioneuroma at Bursa Uludag University, Faculty of Medicine, between April 2007 and November 2023. The cohort comprised 18 females (72%) and seven males (28%), with a median age of 42 years (range: 19-79). Tumors were most commonly located in the abdomen (64%), followed by the thoracic (24%) and head and neck regions (12%), with the adrenal gland being the primary site in 32% of cases. Symptoms were present in 56% of patients, including pain, vision loss, hypertension, and palpable masses, while the remaining were asymptomatic. Surgical resection was performed in 92% of cases, with a median tumor size of 7.5 cm (1.5-18 cm). Median follow-up time was 88.3(16.2 - 217.8) months. Regional lymph node metastases were identified in 8% of patients, but no distant metastases or recurrences were observed during follow-up. These findings, including the novel observation of regional metastases, contribute valuable insights to the limited literature on ganglioneuromas. Despite its benign nature, this study highlights the potential for lymph node metastasis. However, the relationship between lymph node metastasis and recurrence has not been documented. In this context, further research is essential to better understand the risk factors, tumorigenesis, and the optimal management strategies for this rare tumor.
2023-12-26 | NUT carcinoma in pediatric patients: Characteristics, therapeutic regimens, and outcomes of 11 cases registered with the German Registry for Rare Pediatric Tumors (STEP)
Abstract Background and aims Nuclear protein of the testis (NUT) carcinoma (NC) is a rare and highly aggressive tumor defined by the presence of a somatic NUTM1 rearrangement, occurring mainly in adolescents and young adults. We analyzed the clinical and biological features of German pediatric patients (≤18 years) with NC. Methods This study describes the characteristics and outcome of 11 children with NC registered in the German Registry for Rare Pediatric Tumors (STEP). Results Eleven patients with a median age of 13.2 years (range 6.6–17.8) were analyzed. Malignant misdiagnoses were made in three patients. Thoracic/mediastinal tumors were found to be the primary in six patients, head/neck in four cases; one patient had multifocal tumor with an unknown primary. All patients presented with regional lymph node involvement, eight patients (72.7%) with distant metastases. Seven patients underwent surgery, eight radiotherapy with curative intent; polychemotherapy was administered in all patients. Novel treatment strategies including immunotherapy, targeted therapies, and virotherapy were applied in three patients. Median event‐free survival and overall survival were 1.5 and 6.5 months, respectively. Conclusions Every undifferentiated or poorly differentiated carcinoma should undergo testing for the specific rearrangement of NUTM1, in order to initiate an intense therapeutic regimen as early as possible. As in adults, only few pediatric patients with NC achieve prolonged survival. Thus, novel therapeutic strategies should be included and tested in clinical trials.
2023-02-28 | Knockdown of lncRNA SNHG16 Attenuates the Proliferation and Radioresistance of Nasopharyngeal Carcinoma Cells by Mediating miR-31-5p/SFN Axis.
Nasopharyngeal carcinoma (NPC) is a rare head and neck tumor that threatens people's health. Radiotherapy is a major treatment for NPC, however, radioresistance of the NPC cells may contribute to treatment failure. LncRNA SNHG16 was upregulated in NPC; however, the function of SNHG16 in radioresistant NPC cells remains unexplored. RT-qPCR was applied for detecting SNHG16, miR-31-5p and SFN levels. MTT assay and colony formation assay were applied to assess the cell viability and proliferation. Dual luciferase was applied for assessing the relation among SNHG16, miR-31-5p and SFN. SFN level in NPC cells was examined by Western blot. The level of SNHG16 and SFN in NPC cells was significantly upregulated by exposure to radiation. In addition, silencing of SNHG16 or miR-31-5p mimics notably attenuated radioresistance of NPC cells. SNHG16 could positively regulate the expression of SFN in NPC cells through binding with miR-31-5p. Furthermore, SNHG16 downregulation obviously attenuated the proliferation and radioresistance of NPC cells by regulation of miR-31-5p/SFN axis. Knockdown of lncRNA SNHG16 attenuates radioresistance of nasopharyngeal carcinoma cells by miR-31-5p/SFN axis. Thus, our research data show a novel method for improving the efficacy of radiotherapy for NPC.
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Drug Discovery Landscape
4 orphan drug designations for Rare head and neck tumor.
4 orphan drug designations for Rare head and neck tumor.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 | peptides | EMA | 2026-06-19 | — | Curadel Surgical Innovations B.V. |
Replication defective recombinant adenovirus serotype 5 vector carrying the p53 gene in its E1 deleted region | gene therapies | FDA | 2003-01-27 | — | Introgen Therapeutics, Inc. |
tirapazamine | small molecules | FDA | 2002-10-23 | — | Sanofi-Aventis US, Inc. |
Porfiromycin | small molecules | FDA | 1995-09-19 | — | Boehringer Ingelheim Pharmaceuticals, Inc. |
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