RARE DISEASE
Squamous cell carcinoma of the oropharynx
Squamous cell carcinoma of the oropharynx
Squamous cell carcinoma of the oropharynx
Drug discovery
7
drugs
With orphan designations
Overview
Squamous cell carcinoma of the oropharynx (OPSCC) is a head and neck malignancy primarily linked to HPV infection (70-80% of cases) or tobacco/alcohol use. HPV-positive tumors, often affecting the tonsils/base of tongue, present with cervical lymphadenopathy and have superior prognosis compared to HPV-negative tumors, which typically involve older patients and advanced stages [1][5][9]. Diagnosis relies on biopsy and imaging, with treatment strategies emphasizing organ preservation through surgery, radiotherapy, chemotherapy, or combined modalities [5][15].
Burden
Annual U.S. incidence: ~11.5/100,000 persons, with rising HPV-driven cases in younger cohorts [2][14][18].
5-year survival: >80% for HPV-positive vs. <50% for HPV-negative tumors [9][14].
Economic and functional burdens from treatment toxicities (e.g., dysphagia) and rising healthcare costs [12][16].
Therapies
Early-stage: Transoral surgery (e.g., TORS) or definitive radiotherapy (RT) [3][15].
Locally advanced: Concurrent cisplatin-based chemoradiation (standard) or cetuximab for cisplatin-ineligible patients [11][15][19].
Adjuvant therapy: Postoperative RT ± cisplatin for high-risk features (e.g., extracapsular extension, positive margins) [7][15].
Categories: rare neoplastic diseases, rare otorhinolaryngological diseases
Research Papers
669 drug discovery papers about Squamous cell carcinoma of the oropharynx, with 3 first-in-class and 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
669 drug discovery papers about Squamous cell carcinoma of the oropharynx, with 3 first-in-class and 4 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-08-15 | Durable Complete Response Following Nivolumab Discontinuation in Human Papillomavirus (HPV)-Positive Oropharyngeal Carcinoma Complicated by Bullous Pemphigoid: A Case Report.
Immune checkpoint inhibitors have improved outcomes in recurrent/metastatic head and neck squamous cell carcinoma (HNSCC), although durable complete responses (CRs) remain uncommon. The optimal duration of immunotherapy is not yet established. We report the case of a 69-year-old female smoker diagnosed in November 2015 with human papillomavirus (HPV)16-positive squamous cell carcinoma of the oropharynx (base of tongue), staged cT2N2bM0. Following induction chemotherapy with docetaxel, cisplatin, and 5-fluorouracil, and concurrent chemoradiotherapy with cisplatin, persistent disease was confirmed by biopsy. In December 2016, she underwent extensive surgery that included partial pharyngectomy with tracheostomy + hemiglossectomy + bilateral neck dissection + microvascular free flap reconstruction with positive margins (ypT4aN2R1). Following surgery with positive margins, complementary systemic therapy was administered (paclitaxel, followed by cetuximab combined with methotrexate). In 2019, the patient presented with locoregional cutaneous recurrence, which required hemostatic palliative radiotherapy. Nivolumab was initiated in December 2019, and a CR was achieved about two months later, by February 2020. Treatment was discontinued in February 2021 due to immune-related toxicity (bullous pemphigoid, Common Terminology Criteria for Adverse Events v5.0, grade 3). Remarkably, the patient has maintained a CR for more than four years after discontinuation of immunotherapy, as confirmed by imaging in 2025. This case highlights the potential for durable CRs after immunotherapy discontinuation in heavily pretreated HPV-positive HNSCC, raising important questions regarding optimal treatment duration, patient selection, and the role of immune-related adverse events as a potential marker of response.
2026-05-28 | ERBIOTAX (TTCC-2022-02): A phase II, multicenter, randomized study of cetuximab with or without weekly paclitaxel after progression on first-line pembrolizumab plus platinum/5-FU in recurrent or metastatic head and neck squamous cell carcinoma.
TPS6131 Background: Pembrolizumab, with or without platinum–5FU (PF), is the current first-line (1L) standard of care for PD-L1–positive recurrent or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN). However, no established standard of care exists after progression on immune checkpoint inhibitors (ICI). Emerging evidence suggests that cetuximab administered post-ICI achieves higher objective response rates (ORR) and longer progression-free (PFS) and overall survival (OS) compared with historical second-line (2L) cetuximab outcomes from the pre-ICI era. We hypothesize that 2L treatment with cetuximab +/- paclitaxel may be more effective after ICI failure than previously observed in the pre-ICI era. Methods: This is a multicenter, open-label, randomized, non-comparative, two-arm, investigator-initiated phase 2 trial. Patients will be randomized (2:1) to cetuximab plus paclitaxel (Arm A: ERBITAX) or cetuximab monotherapy (Arm B), administered on Days 1, 8 and 15 of 21-day cycles for 4 cycles, followed in both arms by biweekly cetuximab monotherapy until disease progression, death, unacceptable toxicity, or withdrawal of consent. A total of 65 evaluable patients will be enrolled: 41 in Arm A (H0: ORR 25%, H1: 45%, 80% power, two-sided alpha 0.05) and 24 in Arm B (H0: ORR 10%, H1: 30%, 80% power, two-sided alpha 0.05). The primary endpoint is ORR in each arm. Secondary endpoints include disease control rate (DCR), PFS, OS, health-related quality of life (EORTC QLQ-C30, EORTC QLQ-H&N35 and EuroQol EQ-5D) and safety. Baseline tumor tissue (all patients) and on-treatment samples (between cycles 2-5 in 50% of patients; optionally at progression), as well as serial plasma samples will be collected for translational exploratory studies. Patients must have histologically confirmed SCCHN of oral cavity, oropharynx (HPV-positive or HPV-negative), hypopharynx, or larynx, with confirmed progression per RECIST 1.1. on or after 1L pembro + PF. The study started enrollment in June 2025, with 7 patients enrolled by December 23, 2025. The total accrual period is 18 months. Clinical trial identifiers: NCT06856213; EUDRACT 2024-514953-31-00. Trial supported by Merck, S.L.U., Madrid, Spain, an affiliate of Merck KGaA, Darmstadt, Germany, providing study medication and financial support. Clinical trial information: NCT06856213 .
2026-05-28 | Phase 2 study of intratumoral oncolytic VV1 plus cemiplimab: Simon's stage 1 results from head and neck squamous cell carcinoma (HNSCC) cohort.
e18026 Background: Immune checkpoint inhibitor (ICI) therapy is part of standard of care first-line therapy for recurrent or metastatic (R/M) HNSCC. However, less than 20% of patients (CPS ≥1) have an objective response to ICI monotherapy, and most tumors progress within ~3 months. We sought to improve the proportion of patients who benefit from a chemotherapy-free regimen by combining intratumoral (IT) VV1 oncolytic virus (VSV-IFNβ-NIS) injections with the anti-PD-1 antibody, cemiplimab. We report a pre-planned analysis of Simon's stage 1 results. Methods: We activated a phase 2 study testing the efficacy of first line IT VV1 plus cemiplimab in 1 st line patients with R/M HNSCC. A Simon's two-stage design was employed whereby 10 patients would be accrued in stage 1, and if 3 or more responses were observed, 12 additional patients would be accrued for stage 2. The primary endpoint was objective response rate (ORR) per investigator (RECIST 1.1). Eligible patients had untreated R/M HNSCC from primary oropharynx, oral cavity, hypopharynx, or larynx sites with CPS ≥1. Patients had to have at least one measurable lesion amenable to IT injection. Treatment comprised of IT injections to 1-5 lesions of oncolytic VV-1 virus and IV cemiplimab 350 mg on day 1, repeated every 21 days. Each lesion could be injected 3 times; a 4th injection was allowed if a partial response (PR) was achieved after 3 injections. Cemiplimab could be continued for up to 2 years until progression or unacceptable toxicity. Results: Patient and tumor characteristics, as well as efficacy and toxicity are summarized in the table below. Ten patients were treated at the time of Stage 1 analysis, with 3 showing a confirmed PR for ORR of 30%. Responder CPS scores were 45, 12 and 20%. Two of the responders subsequently showed progression after 6.2 and 9.9 months, the third has maintained PR at 7.7 months to date. Eight of 10 patients showed decrease in the sum of target lesion size (mean decrease of 16%; range -57 to 77%). One grade 2 event of ICI nephritis resolved with corticosteroid treatment. Conclusions: Three patients showed confirmed PR by RECIST 1.1 in Simon's stage 1, meeting criteria to proceed to Simon's stage 2. A total of 22 patients in the HNSCC will be enrolled. VV1 plus cemiplimab shows promise in first line R/M HNSCC based on these initial results. Clinical trial information: NCT04291105 . Patient demographics. N (treated) 10 Sex 20% female Median age (y) 60 (54 – 76) Oropharynx 7 HPV+ 7 Oral cavity 3 PD-L1 CPS median (range) 7.5 (1-45%) Locoregional injection 3 Distant injection 6 Local and distant injection 1 Best response (RECIST 1.1) PR 3 SD 5 PD 2 Adverse Events #pts any VV1/cemiplimab-related G3 AE 4 Lymphopenia 3 Elevated ALK 1
2026-05-28 | NRG-HN015: A phase II randomized trial of neoadjuvant chemotherapy or chemo-immunotherapy in patients with recurrent/persistent PD-L1–positive squamous cell carcinoma of the head and neck undergoing salvage surgery.
TPS6128 Background: Treatment for locoregionally recurrent squamous cell carcinoma of the head and neck (SCCHN) remains a challenge. 30-40% of patients treated with definitive-intent therapy will recur, the majority locoregionally (Ang 2010, Galloway 2016, Tan 2010). Despite improvements in the effectiveness of palliative systemic therapy over the last decade (Vermorken 2008), salvage surgery (SS) remains the modality of choice to achieve cure in patients with locally recurrent disease particularly in patients who have previously received radiation. Thus, there is a pressing need to improve the therapeutic ratio by increasing the benefit of SS and improving oncologic outcomes. One promising means would be early introduction of systemic therapy with or without immune checkpoint inhibition for patients who are candidates for SS. NRG-HN015 proposes to investigate the effect on event-free survival (EFS) of pre-operative chemotherapy or chemo-immunotherapy in comparison to the standard SS approach. Methods: NRG-HN015 (NEOPOLIS) is a randomized, multicenter, controlled, 3-arm, superiority phase II study of neoadjuvant chemotherapy or chemo-immunotherapy in patients with recurrent/persistent PD-L1 enriched SCCHN who have planned SS. The primary objective is to compare investigator-assessed EFS of patients treated with neoadjuvant chemotherapy or chemo-immunotherapy prior to SS versus SS alone. The primary hypothesis is that neoadjuvant chemotherapy or chemo-immunotherapy added to SS will improve EFS. Enrolled patients will be randomized 1:1:1 to receive SS (Arm 1 - control), chemotherapy + SS (Arm 2), or chemo-immunotherapy + SS (Arm 3). Prior to randomization, patients will be stratified by primary tumor site (Oropharynx or oral cavity vs. larynx or hypopharynx, stage (rT4a or rN3a vs. other), and prior use of iPD1/PDL1 inhibitors in the definitive setting. Patients must have locally recurrent or persistent SCCHN arising within the oral cavity, oropharynx, larynx, or hypopharynx and are deemed candidates for salvage surgery. P16-positive oropharynx patients with T2, T3, T4, N0, N1, N2 and all other patients with T2, T3, T4a, N0, N1, N2a, N2b, N2c, N3a are eligible. Patients must have PDL1-positive and measurable disease, with no major vascular involvement (>180° involvement of the common carotid or internal carotid artery), jugular foramen involvement, or prevertebral, paraspinous muscle involvement precluding a curative resection. Patients who are candidates for salvage laryngectomy to treat recurrent laryngeal cancer and who are having SS for curative intent are eligible. The trial is opened to enrollment. Clinical trial information: NCT071953734 .
2026-05-28 | A phase II trial of a novel induction regimen: Polymeric micellar paclitaxel plus cisplatin for locally advanced head and neck squamous cell carcinoma.
e18067 Background: Induction chemotherapy with the TPF regimen is recommended by international guidelines for locally advanced head and neck squamous cell carcinoma. However, its significant toxicities lead to generally poor patient tolerance, limiting its widespread clinical application, particularly among elderly or medically unfit patients. By encapsulating paclitaxel within micelles, pm-Pac enhanced permeability and retention effect, thereby improving pharmacokinetics and tumor-targeted delivery. This study evaluated the efficacy and safety of induction therapy with polymeric micellar paclitaxel plus cisplatin in LA-SCCHN, aiming to determine whether its pharmacologic advantages translate into clinical benefit. Methods: According to clinical trail the Eligible patients were stage III to IVB HNSCC received polymeric micellar paclitaxel 230 mg/m² (Day 1) plus cisplatin 70 mg/m² (Day 1–2) every three weeks for 2–4 cycles. The primary endpoint was objective response rate (ORR). Secondary endpoints included disease control rate (DCR), safety, adverse events (AEs), and completion of subsequent definitive therapy. Results: Thirty patients were enrolled (median age, 66 years), including five HPV-positive cases (16.7%). After induction therapy, 5 patients achieved complete response (CR) and 11 achieved partial response (PR), yielding an ORR of 53.3% and a DCR of 93.3%. ORR reached 80% in HPV-positive patients versus 48% in HPV-negative/unknown patients. Common AEs included myalgia/limb pain (60%), neutropenia (26.7%), and bone marrow suppression (33.3%). Grade ≥3 AEs were mainly hematologic; no solvent-related hypersensitivity reactions occurred. Conclusions: Polymeric micellar paclitaxel plus cisplatin demonstrated promising antitumor activity, high disease control, and favorable tolerability in patients with LA-SCCHN, particularly among elderly or TPF-intolerant individuals. Clinical trial information: ChiCTR2500110591. Variable Number (n=30) Percentage (%) Age, years Median 66 (range 35–77) — <65 12 40.0 ≥65 18 60.0 Sex Male 23 76.7 Female 7 23.3 Primary Tumor Site Hypopharynx 9 30.0 Larynx 8 26.7 Oropharynx 6 20.0 Oral cavity 4 13.3 Other* 3 10.0 Clinical Stage II 2 6.7 III 13 43.3 IVA 14 46.7 IVB 1 3.3 HPV Status Positive 5 16.7 Negative/Unknown 25 83.3 Response Number (n=30) Percentage (%)
2026-08-15 | Durable Complete Response Following Nivolumab Discontinuation in Human Papillomavirus (HPV)-Positive Oropharyngeal Carcinoma Complicated by Bullous Pemphigoid: A Case Report.
Immune checkpoint inhibitors have improved outcomes in recurrent/metastatic head and neck squamous cell carcinoma (HNSCC), although durable complete responses (CRs) remain uncommon. The optimal duration of immunotherapy is not yet established. We report the case of a 69-year-old female smoker diagnosed in November 2015 with human papillomavirus (HPV)16-positive squamous cell carcinoma of the oropharynx (base of tongue), staged cT2N2bM0. Following induction chemotherapy with docetaxel, cisplatin, and 5-fluorouracil, and concurrent chemoradiotherapy with cisplatin, persistent disease was confirmed by biopsy. In December 2016, she underwent extensive surgery that included partial pharyngectomy with tracheostomy + hemiglossectomy + bilateral neck dissection + microvascular free flap reconstruction with positive margins (ypT4aN2R1). Following surgery with positive margins, complementary systemic therapy was administered (paclitaxel, followed by cetuximab combined with methotrexate). In 2019, the patient presented with locoregional cutaneous recurrence, which required hemostatic palliative radiotherapy. Nivolumab was initiated in December 2019, and a CR was achieved about two months later, by February 2020. Treatment was discontinued in February 2021 due to immune-related toxicity (bullous pemphigoid, Common Terminology Criteria for Adverse Events v5.0, grade 3). Remarkably, the patient has maintained a CR for more than four years after discontinuation of immunotherapy, as confirmed by imaging in 2025. This case highlights the potential for durable CRs after immunotherapy discontinuation in heavily pretreated HPV-positive HNSCC, raising important questions regarding optimal treatment duration, patient selection, and the role of immune-related adverse events as a potential marker of response.
2026-05-28 | ERBIOTAX (TTCC-2022-02): A phase II, multicenter, randomized study of cetuximab with or without weekly paclitaxel after progression on first-line pembrolizumab plus platinum/5-FU in recurrent or metastatic head and neck squamous cell carcinoma.
TPS6131 Background: Pembrolizumab, with or without platinum–5FU (PF), is the current first-line (1L) standard of care for PD-L1–positive recurrent or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN). However, no established standard of care exists after progression on immune checkpoint inhibitors (ICI). Emerging evidence suggests that cetuximab administered post-ICI achieves higher objective response rates (ORR) and longer progression-free (PFS) and overall survival (OS) compared with historical second-line (2L) cetuximab outcomes from the pre-ICI era. We hypothesize that 2L treatment with cetuximab +/- paclitaxel may be more effective after ICI failure than previously observed in the pre-ICI era. Methods: This is a multicenter, open-label, randomized, non-comparative, two-arm, investigator-initiated phase 2 trial. Patients will be randomized (2:1) to cetuximab plus paclitaxel (Arm A: ERBITAX) or cetuximab monotherapy (Arm B), administered on Days 1, 8 and 15 of 21-day cycles for 4 cycles, followed in both arms by biweekly cetuximab monotherapy until disease progression, death, unacceptable toxicity, or withdrawal of consent. A total of 65 evaluable patients will be enrolled: 41 in Arm A (H0: ORR 25%, H1: 45%, 80% power, two-sided alpha 0.05) and 24 in Arm B (H0: ORR 10%, H1: 30%, 80% power, two-sided alpha 0.05). The primary endpoint is ORR in each arm. Secondary endpoints include disease control rate (DCR), PFS, OS, health-related quality of life (EORTC QLQ-C30, EORTC QLQ-H&N35 and EuroQol EQ-5D) and safety. Baseline tumor tissue (all patients) and on-treatment samples (between cycles 2-5 in 50% of patients; optionally at progression), as well as serial plasma samples will be collected for translational exploratory studies. Patients must have histologically confirmed SCCHN of oral cavity, oropharynx (HPV-positive or HPV-negative), hypopharynx, or larynx, with confirmed progression per RECIST 1.1. on or after 1L pembro + PF. The study started enrollment in June 2025, with 7 patients enrolled by December 23, 2025. The total accrual period is 18 months. Clinical trial identifiers: NCT06856213; EUDRACT 2024-514953-31-00. Trial supported by Merck, S.L.U., Madrid, Spain, an affiliate of Merck KGaA, Darmstadt, Germany, providing study medication and financial support. Clinical trial information: NCT06856213 .
2026-05-28 | Phase 2 study of intratumoral oncolytic VV1 plus cemiplimab: Simon's stage 1 results from head and neck squamous cell carcinoma (HNSCC) cohort.
e18026 Background: Immune checkpoint inhibitor (ICI) therapy is part of standard of care first-line therapy for recurrent or metastatic (R/M) HNSCC. However, less than 20% of patients (CPS ≥1) have an objective response to ICI monotherapy, and most tumors progress within ~3 months. We sought to improve the proportion of patients who benefit from a chemotherapy-free regimen by combining intratumoral (IT) VV1 oncolytic virus (VSV-IFNβ-NIS) injections with the anti-PD-1 antibody, cemiplimab. We report a pre-planned analysis of Simon's stage 1 results. Methods: We activated a phase 2 study testing the efficacy of first line IT VV1 plus cemiplimab in 1 st line patients with R/M HNSCC. A Simon's two-stage design was employed whereby 10 patients would be accrued in stage 1, and if 3 or more responses were observed, 12 additional patients would be accrued for stage 2. The primary endpoint was objective response rate (ORR) per investigator (RECIST 1.1). Eligible patients had untreated R/M HNSCC from primary oropharynx, oral cavity, hypopharynx, or larynx sites with CPS ≥1. Patients had to have at least one measurable lesion amenable to IT injection. Treatment comprised of IT injections to 1-5 lesions of oncolytic VV-1 virus and IV cemiplimab 350 mg on day 1, repeated every 21 days. Each lesion could be injected 3 times; a 4th injection was allowed if a partial response (PR) was achieved after 3 injections. Cemiplimab could be continued for up to 2 years until progression or unacceptable toxicity. Results: Patient and tumor characteristics, as well as efficacy and toxicity are summarized in the table below. Ten patients were treated at the time of Stage 1 analysis, with 3 showing a confirmed PR for ORR of 30%. Responder CPS scores were 45, 12 and 20%. Two of the responders subsequently showed progression after 6.2 and 9.9 months, the third has maintained PR at 7.7 months to date. Eight of 10 patients showed decrease in the sum of target lesion size (mean decrease of 16%; range -57 to 77%). One grade 2 event of ICI nephritis resolved with corticosteroid treatment. Conclusions: Three patients showed confirmed PR by RECIST 1.1 in Simon's stage 1, meeting criteria to proceed to Simon's stage 2. A total of 22 patients in the HNSCC will be enrolled. VV1 plus cemiplimab shows promise in first line R/M HNSCC based on these initial results. Clinical trial information: NCT04291105 . Patient demographics. N (treated) 10 Sex 20% female Median age (y) 60 (54 – 76) Oropharynx 7 HPV+ 7 Oral cavity 3 PD-L1 CPS median (range) 7.5 (1-45%) Locoregional injection 3 Distant injection 6 Local and distant injection 1 Best response (RECIST 1.1) PR 3 SD 5 PD 2 Adverse Events #pts any VV1/cemiplimab-related G3 AE 4 Lymphopenia 3 Elevated ALK 1
2026-05-28 | NRG-HN015: A phase II randomized trial of neoadjuvant chemotherapy or chemo-immunotherapy in patients with recurrent/persistent PD-L1–positive squamous cell carcinoma of the head and neck undergoing salvage surgery.
TPS6128 Background: Treatment for locoregionally recurrent squamous cell carcinoma of the head and neck (SCCHN) remains a challenge. 30-40% of patients treated with definitive-intent therapy will recur, the majority locoregionally (Ang 2010, Galloway 2016, Tan 2010). Despite improvements in the effectiveness of palliative systemic therapy over the last decade (Vermorken 2008), salvage surgery (SS) remains the modality of choice to achieve cure in patients with locally recurrent disease particularly in patients who have previously received radiation. Thus, there is a pressing need to improve the therapeutic ratio by increasing the benefit of SS and improving oncologic outcomes. One promising means would be early introduction of systemic therapy with or without immune checkpoint inhibition for patients who are candidates for SS. NRG-HN015 proposes to investigate the effect on event-free survival (EFS) of pre-operative chemotherapy or chemo-immunotherapy in comparison to the standard SS approach. Methods: NRG-HN015 (NEOPOLIS) is a randomized, multicenter, controlled, 3-arm, superiority phase II study of neoadjuvant chemotherapy or chemo-immunotherapy in patients with recurrent/persistent PD-L1 enriched SCCHN who have planned SS. The primary objective is to compare investigator-assessed EFS of patients treated with neoadjuvant chemotherapy or chemo-immunotherapy prior to SS versus SS alone. The primary hypothesis is that neoadjuvant chemotherapy or chemo-immunotherapy added to SS will improve EFS. Enrolled patients will be randomized 1:1:1 to receive SS (Arm 1 - control), chemotherapy + SS (Arm 2), or chemo-immunotherapy + SS (Arm 3). Prior to randomization, patients will be stratified by primary tumor site (Oropharynx or oral cavity vs. larynx or hypopharynx, stage (rT4a or rN3a vs. other), and prior use of iPD1/PDL1 inhibitors in the definitive setting. Patients must have locally recurrent or persistent SCCHN arising within the oral cavity, oropharynx, larynx, or hypopharynx and are deemed candidates for salvage surgery. P16-positive oropharynx patients with T2, T3, T4, N0, N1, N2 and all other patients with T2, T3, T4a, N0, N1, N2a, N2b, N2c, N3a are eligible. Patients must have PDL1-positive and measurable disease, with no major vascular involvement (>180° involvement of the common carotid or internal carotid artery), jugular foramen involvement, or prevertebral, paraspinous muscle involvement precluding a curative resection. Patients who are candidates for salvage laryngectomy to treat recurrent laryngeal cancer and who are having SS for curative intent are eligible. The trial is opened to enrollment. Clinical trial information: NCT071953734 .
2026-05-28 | A phase II trial of a novel induction regimen: Polymeric micellar paclitaxel plus cisplatin for locally advanced head and neck squamous cell carcinoma.
e18067 Background: Induction chemotherapy with the TPF regimen is recommended by international guidelines for locally advanced head and neck squamous cell carcinoma. However, its significant toxicities lead to generally poor patient tolerance, limiting its widespread clinical application, particularly among elderly or medically unfit patients. By encapsulating paclitaxel within micelles, pm-Pac enhanced permeability and retention effect, thereby improving pharmacokinetics and tumor-targeted delivery. This study evaluated the efficacy and safety of induction therapy with polymeric micellar paclitaxel plus cisplatin in LA-SCCHN, aiming to determine whether its pharmacologic advantages translate into clinical benefit. Methods: According to clinical trail the Eligible patients were stage III to IVB HNSCC received polymeric micellar paclitaxel 230 mg/m² (Day 1) plus cisplatin 70 mg/m² (Day 1–2) every three weeks for 2–4 cycles. The primary endpoint was objective response rate (ORR). Secondary endpoints included disease control rate (DCR), safety, adverse events (AEs), and completion of subsequent definitive therapy. Results: Thirty patients were enrolled (median age, 66 years), including five HPV-positive cases (16.7%). After induction therapy, 5 patients achieved complete response (CR) and 11 achieved partial response (PR), yielding an ORR of 53.3% and a DCR of 93.3%. ORR reached 80% in HPV-positive patients versus 48% in HPV-negative/unknown patients. Common AEs included myalgia/limb pain (60%), neutropenia (26.7%), and bone marrow suppression (33.3%). Grade ≥3 AEs were mainly hematologic; no solvent-related hypersensitivity reactions occurred. Conclusions: Polymeric micellar paclitaxel plus cisplatin demonstrated promising antitumor activity, high disease control, and favorable tolerability in patients with LA-SCCHN, particularly among elderly or TPF-intolerant individuals. Clinical trial information: ChiCTR2500110591. Variable Number (n=30) Percentage (%) Age, years Median 66 (range 35–77) — <65 12 40.0 ≥65 18 60.0 Sex Male 23 76.7 Female 7 23.3 Primary Tumor Site Hypopharynx 9 30.0 Larynx 8 26.7 Oropharynx 6 20.0 Oral cavity 4 13.3 Other* 3 10.0 Clinical Stage II 2 6.7 III 13 43.3 IVA 14 46.7 IVB 1 3.3 HPV Status Positive 5 16.7 Negative/Unknown 25 83.3 Response Number (n=30) Percentage (%)
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Drug Discovery Landscape
7 orphan drug designations for Squamous cell carcinoma of the oropharynx, including 1 approved therapy.
7 orphan drug designations for Squamous cell carcinoma of the oropharynx, including 1 approved therapy.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Adenovirus serotype-5 (Ad5) vector that contains a modified non-oncogenic fused early 6 (E6) and early 7 (E7) gene of the human papillomavirus (HPV); (Ad5 [E1-, E2b-]-E6/E7) | gene therapies | FDA | 2014-09-04 | — | Etubics Corporation |
live attenuated bioengineered Listeria monocytogenes immunotherapy | vaccines | FDA | 2013-11-04 | — | Ayala Pharmaceuticals, Inc. |
HPV-16 cancer therapeutic trojan peptide vaccine | vaccines | FDA | 2009-01-12 | — | Gliknik, Inc. |
MAGE-A3 cancer therapeutic Trojan peptide vaccine | vaccines | FDA | 2008-11-24 | — | Gliknik, Inc. |
Humanised antibody fragment (Ep-CAM)-truncated Pseudomonas exotoxin A fusion protein | antibodies | EMA | 2005-06-20 | — | Viventia Biotech (EU) Limited |
recombinant fusion protein with a truncated form of the cytotoxic protein Pseudomonas exotoxin | proteins | FDA | 2005-01-28 | — | Sesen Bio, Inc. (Sesen Bio) |
cetuximab [Erbitux] | antibodies | FDA | 2000-07-03 | 2006-03-01 | ImClone Systems Incorporated |
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