Angela C. Hirbe
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View article: EXTH-26. Targeting Amino Acid Transporter in Malignant Peripheral Nerve Sheath Tumors: A Strategy to Enhance Efficacy of Chemotherapy and Targeted Therapy
EXTH-26. Targeting Amino Acid Transporter in Malignant Peripheral Nerve Sheath Tumors: A Strategy to Enhance Efficacy of Chemotherapy and Targeted Therapy Open
OBJECTIVES Malignant peripheral nerve sheath tumors (MPNST) are aggressive soft tissue sarcomas with limited treatment options and poor prognosis. Similar to other high-grade sarcomas, MPNSTs exhibit elevated metabolic demands and frequent…
View article: CTNI-06. UPDATE FROM THE LONG-TERM FOLLOW-UP (LTFU) PHASE OF RENEU: A PIVOTAL PHASE 2B TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED SYMPTOMATIC PLEXIFORM NEUROFIBROMA (PN)
CTNI-06. UPDATE FROM THE LONG-TERM FOLLOW-UP (LTFU) PHASE OF RENEU: A PIVOTAL PHASE 2B TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED SYMPTOMATIC PLEXIFORM NEUROFIBROMA (PN) Open
BACKGROUND Mirdametinib is the first FDA-approved MEK1/2 inhibitor for both adults and children ≥2 years with NF1 who have symptomatic PN not amenable to complete resection. We report results from the phase 2b ReNeu trial (NCT03962543), in…
View article: CTNI-55. EXPLORING THE RELATIONSHIP BETWEEN PATIENT BASELINE CHARACTERISTICS WITH RESPONSES TO MIRDAMETINIB IN THE RENEU TRIAL OF CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED PLEXIFORM NEUROFIBROMA (PN)
CTNI-55. EXPLORING THE RELATIONSHIP BETWEEN PATIENT BASELINE CHARACTERISTICS WITH RESPONSES TO MIRDAMETINIB IN THE RENEU TRIAL OF CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED PLEXIFORM NEUROFIBROMA (PN) Open
BACKGROUND Mirdametinib, a MEK1/2 inhibitor, is the first FDA-approved medicine for both adults and children (≥2y) with NF1-PN not amenable to complete resection. In the phase 2b ReNeu trial (NCT03962543), confirmed objective response rate…
View article: DDDR-64. MTA-Cooperative PRMT5 Inhibitors Demonstrate Efficacy in MTAP-Deleted MPNST Models and Enhance Chemotherapy Response
DDDR-64. MTA-Cooperative PRMT5 Inhibitors Demonstrate Efficacy in MTAP-Deleted MPNST Models and Enhance Chemotherapy Response Open
Malignant peripheral nerve sheath tumors (MPNST) are highly aggressive soft tissue sarcomas with limited treatment options and poor prognosis, underscoring the urgent need for novel targeted therapies. Approximately 25-50% of MPNSTs harbor…
View article: DDDR-59. Focal Adhesion Kinase (FAK) Inhibitor Synergizes with RAF/MEK inhibitor in Malignant Peripheral Nerve Sheath Tumors with Chromosome 8 Gain
DDDR-59. Focal Adhesion Kinase (FAK) Inhibitor Synergizes with RAF/MEK inhibitor in Malignant Peripheral Nerve Sheath Tumors with Chromosome 8 Gain Open
OBJECTIVES Aneuploidy is a common hallmark of cancer and associated with worse prognosis. Gain of chromosome 8 (Chr8) is a common genetic alteration in various cancers, including breast, prostate, colorectal, and malignant peripheral nerve…
View article: OS04.4.A RENEU LONG-TERM FOLLOW-UP (LTFU) UPDATE: A PIVOTAL, PHASE 2B TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED SYMPTOMATIC PLEXIFORM NEUROFIBROMA (PN)
OS04.4.A RENEU LONG-TERM FOLLOW-UP (LTFU) UPDATE: A PIVOTAL, PHASE 2B TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1 (NF1)-ASSOCIATED SYMPTOMATIC PLEXIFORM NEUROFIBROMA (PN) Open
BACKGROUND Mirdametinib is the first FDA-approved MEK1/2 inhibitor for both adults and children (≥2 years) with NF1 who have symptomatic PN not amenable to complete resection. We report updated results from the LTFU phase of the pivotal, p…
View article: Integrated Multiclass Driver ctDNA Profiling Enables MPNST Detection and Monitoring in NF1 Patients
Integrated Multiclass Driver ctDNA Profiling Enables MPNST Detection and Monitoring in NF1 Patients Open
We developed and cross‑validated an integrated circulating tumor DNA assay incorporating SNVs, indels, CNAs, and SVs to distinguish neurofibromatosis type 1 patients with malignant peripheral nerve sheath tumors from those with benign plex…
View article: Mirdametinib, an FDA-Approved MEK1/2 inhibitor for adult and pediatric NF1-associated plexiform neurofibromas
Mirdametinib, an FDA-Approved MEK1/2 inhibitor for adult and pediatric NF1-associated plexiform neurofibromas Open
Mirdametinib offers an effective, targeted, and orally available treatment for NF1-associated plexiform neurofibromas, with the added advantage of CNS penetration and durable clinical benefit. However, resistance and toxicity remain challe…
View article: Integrated genomic analysis of NF1-associated peripheral nerve sheath tumors: an updated biorepository dataset
Integrated genomic analysis of NF1-associated peripheral nerve sheath tumors: an updated biorepository dataset Open
Neurofibromatosis type 1 (NF1) is an inherited neurocutaneous condition that predisposes to the development of peripheral nerve sheath tumors (PNST) including cutaneous neurofibromas (CNF), plexiform neurofibromas (PNF), atypical neurofibr…
View article: MTA-Cooperative PRMT5 Inhibitors Are Efficacious in MTAP-Deleted Malignant Peripheral Nerve Sheath Tumor Models
MTA-Cooperative PRMT5 Inhibitors Are Efficacious in MTAP-Deleted Malignant Peripheral Nerve Sheath Tumor Models Open
Purpose: Malignant peripheral nerve sheath tumors (MPNST) are highly aggressive sarcomas with poor prognosis. The enzyme methylthioadenosine phosphorylase (MTAP) is lost in ∼25% to 50% of MPNSTs, which is associated with loss of the tumor …
View article: YAP signaling promotes resistance to MEK and AKT inhibition in <i>NF1</i> -related MPNSTs
YAP signaling promotes resistance to MEK and AKT inhibition in <i>NF1</i> -related MPNSTs Open
Neurofibromatosis type 1 (NF1) is a tumor predisposition syndrome caused by loss of function of the neurofibromin protein. Malignant peripheral nerve sheath tumors (MPNSTs) are a rare and deadly sarcoma with few therapeutic options that ar…
View article: Collaborative Frontiers in Pediatric Neuro-Oncology: Establishing an International Tumor Board for Enhanced Care and Global Impact
Collaborative Frontiers in Pediatric Neuro-Oncology: Establishing an International Tumor Board for Enhanced Care and Global Impact Open
Background Central nervous system tumors are the leading cause of cancer-related mortality in children, with significant disparities in diagnostic and treatment capabilities between low- and middle-income countries and high-income countrie…
View article: Immortalization and Characterization of Schwann Cell Lines Derived from NF1 Associated Cutaneous Neurofibromas
Immortalization and Characterization of Schwann Cell Lines Derived from NF1 Associated Cutaneous Neurofibromas Open
Neurofibromatosis type 1 (NF1) is an autosomal dominant condition in which patients are heterozygous for a disruptive pathogenic variant in the NF1 gene. The most characteristic feature of the condition NF1 is the neurofibroma, a benign, m…
View article: Augmented expression of superoxide dismutase 2 mitigates progression and rupture of experimental abdominal aortic aneurysm
Augmented expression of superoxide dismutase 2 mitigates progression and rupture of experimental abdominal aortic aneurysm Open
Rationale: Oxidative stress is implicated in the pathogenesis and progression of abdominal aortic aneurysm (AAA). It is suggested that an excess in reactive oxygen species (ROS) over endogenous antioxidant activities can lead to endothelia…
View article: Phase II Study of Pexidartinib Plus Sirolimus in Unresectable Malignant Peripheral Nerve Sheath Tumors Identifies M2 Macrophage Activation
Phase II Study of Pexidartinib Plus Sirolimus in Unresectable Malignant Peripheral Nerve Sheath Tumors Identifies M2 Macrophage Activation Open
PURPOSE To evaluate the preliminary efficacy and safety of the combination of pexidartinib, an inhibitor of colony-stimulating factor-1 receptor (CSF1R), and sirolimus, a mammalian target of rapamycin inhibitor, to target infiltrating M2 m…
View article: A Sequencing Overview of Malignant Peripheral Nerve Sheath Tumors: Findings and Implications for Treatment
A Sequencing Overview of Malignant Peripheral Nerve Sheath Tumors: Findings and Implications for Treatment Open
Malignant peripheral nerve sheath tumors (MPNSTs) are rare but aggressive malignancies with a low 5-year survival rate despite current treatments. MPNSTs frequently harbor mutations in key genes such as NF1, CDKN2A, TP53, and PRC2 componen…
View article: UBR5 in Tumor Biology: Exploring Mechanisms of Immune Regulation and Possible Therapeutic Implications in MPNST
UBR5 in Tumor Biology: Exploring Mechanisms of Immune Regulation and Possible Therapeutic Implications in MPNST Open
Malignant peripheral nerve sheath tumor (MPNST) is a rare but aggressive soft-tissue sarcoma characterized by poor response to therapy. The primary treatment remains surgical resection with negative margins. Nonetheless, in the setting of …
View article: SS18-SSX drives TYK2 expression to activate STAT3/Bcl2 axis, facilitating apoptosis evasion and advancing synovial sarcoma progression
SS18-SSX drives TYK2 expression to activate STAT3/Bcl2 axis, facilitating apoptosis evasion and advancing synovial sarcoma progression Open
Synovial sarcoma (SS) is a rare soft tissue sarcoma characterized by high-grade malignancy and poor prognosis. Preliminary research indicates that apoptosis evasion is a key factor in SS progression, primarily attributed to the overexpress…
View article: UBR5 in the Tumor Microenvironment: Exploring Mechanisms of Immune Regulation and Possible Therapeutic Implications in MPNST
UBR5 in the Tumor Microenvironment: Exploring Mechanisms of Immune Regulation and Possible Therapeutic Implications in MPNST Open
Malignant peripheral nerve sheath tumor (MPNST) is a rare but aggressive soft-tissue sarcoma characterized by poor response to therapy. The primary treatment remains surgical resection with negative margins. Nonetheless, in the setting of …
View article: ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma
ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma Open
PURPOSE Pharmacologic therapies for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PNs) are limited; currently, none are US Food and Drug Administration–approved for adults. METHODS ReNeu is an open-label, multicenter, pi…
View article: Brain Metastases in Sarcomas: A Multicenter Retrospective Cohort Study
Brain Metastases in Sarcomas: A Multicenter Retrospective Cohort Study Open
Purpose: Brain metastases (BM) in sarcomas occur rarely and are associated with poor prognosis. This study is a large retrospective cohort describing the demographic and clinical characteristics of these patients, treatment strategies, and…
View article: EXTH-62. FOCAL ADHESION KINASE (FAK) IN CHROMOSOME 8 GAIN-ASSOCIATED MALIGNANT PERIPHERAL NERVE SHEATH TUMORS (MPNST)
EXTH-62. FOCAL ADHESION KINASE (FAK) IN CHROMOSOME 8 GAIN-ASSOCIATED MALIGNANT PERIPHERAL NERVE SHEATH TUMORS (MPNST) Open
OBJECTIVES Malignant peripheral nerve sheath tumors (MPNST) are aggressive sarcomas often associated with Neurofibromatosis type 1 (NF1), a genetic syndrome driven by overactivation of the RAS pathway. MEK inhibitors (MEKi) target downstre…
View article: QLTI-20. BRIDGING THE GAP: UNDERSTANDING THE CURRENT TREATMENT RESPONSES TO ENHANCE THE STANDARD OF CARE AND OUTCOMES FOR MALIGNANT PERIPHERAL NERVE SHEATH TUMORS
QLTI-20. BRIDGING THE GAP: UNDERSTANDING THE CURRENT TREATMENT RESPONSES TO ENHANCE THE STANDARD OF CARE AND OUTCOMES FOR MALIGNANT PERIPHERAL NERVE SHEATH TUMORS Open
Malignant peripheral nerve sheath tumor (MPNST) are a rare and aggressive cancer that make up 5-10% of all sarcomas. There is currently no effective targeted therapy to treat MPNST and surgical resection remains the mainstay of treatment, …
View article: BIOM-37. EXPLORING L-TYPE AMINO ACID TRANSPORTER 1 (LAT1) AS A DIAGNOSTIC MARKER AND THERAPEUTIC TARGET IN MALIGNANT PERIPHERAL NERVE SHEATH TUMORS (MPNST)
BIOM-37. EXPLORING L-TYPE AMINO ACID TRANSPORTER 1 (LAT1) AS A DIAGNOSTIC MARKER AND THERAPEUTIC TARGET IN MALIGNANT PERIPHERAL NERVE SHEATH TUMORS (MPNST) Open
OBJECTIVES Malignant peripheral nerve sheath tumors (MPNST) are aggressive soft tissue sarcomas that frequently arise from benign plexiform neurofibromas (PN). Approximately half of MPNST are associated with Neurofibromatosis type 1 (NF1).…
View article: QOL-08. HEALTH-RELATED QUALITY-OF-LIFE (HRQOL) IN ADULTS AND CHILDREN WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMA (NF1-PN) TREATED WITH MIRDAMETINIB: PIVOTAL, PHASE 2B RENEU TRIAL
QOL-08. HEALTH-RELATED QUALITY-OF-LIFE (HRQOL) IN ADULTS AND CHILDREN WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMA (NF1-PN) TREATED WITH MIRDAMETINIB: PIVOTAL, PHASE 2B RENEU TRIAL Open
Patients with NF1-PN experience pain and other symptoms that negatively impact functioning and quality-of-life. We present patient- and parent proxy-reported HRQoL outcomes from ReNeu (NCT03962543), a pivotal, phase 2b trial of mirdametini…
View article: CNSC-13. CROSSTALK BETWEEN SENSORY NEURONAL ACTIVITY AND TUMOR CELLS PROMOTES MALIGNANCY AND CANCER PAIN
CNSC-13. CROSSTALK BETWEEN SENSORY NEURONAL ACTIVITY AND TUMOR CELLS PROMOTES MALIGNANCY AND CANCER PAIN Open
BACKGROUND Nerve infiltration of solid tumors is associated with poor prognosis in multiple cancer types. Understanding the role of neuronal activity in cancer progression offers innovative approaches for treating malignancies and cancer-a…
View article: CTNI-20. ADDRESSING SKIN ADVERSE EVENTS (AES) DURING MIRDAMETINIB TREATMENT IN PATIENTS WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMAS (NF1-PN): GUIDANCE FROM A MULTIDISCIPLINARY GROUP OF EXPERTS ON THE MANAGEMENT OF MEK INHIBITOR-ASSOCIATED SKIN AES
CTNI-20. ADDRESSING SKIN ADVERSE EVENTS (AES) DURING MIRDAMETINIB TREATMENT IN PATIENTS WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMAS (NF1-PN): GUIDANCE FROM A MULTIDISCIPLINARY GROUP OF EXPERTS ON THE MANAGEMENT OF MEK INHIBITOR-ASSOCIATED SKIN AES Open
ReNeu (NCT03962543) is a trial of the investigational, oral, selective MEK1/2 inhibitor (MEKi) mirdametinib in adults (≥18 years) and children (2-17 years) with NF1-PN. Skin adverse events (AEs) were common in ReNeu, and some discontinuati…
View article: CTNI-21. PIVOTAL, PHASE 2B RENEU TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMA (NF1-PN): A SPOTLIGHT ON PATIENTS ACHIEVING DEEP RESPONSE
CTNI-21. PIVOTAL, PHASE 2B RENEU TRIAL OF MIRDAMETINIB IN CHILDREN AND ADULTS WITH NEUROFIBROMATOSIS TYPE 1-ASSOCIATED PLEXIFORM NEUROFIBROMA (NF1-PN): A SPOTLIGHT ON PATIENTS ACHIEVING DEEP RESPONSE Open
BACKGROUND Patients with NF1-PN in the phase 2b ReNeu trial (NCT03962543) of mirdametinib in adults (N=58, ≥18y) and children (N=56, 2-17y) had a deeper median (range) best reduction (adults: -41% [-90% to 13%]; children: -42%, [-91% to 48…
View article: TMOD-26. GENERATION OF A CHROMOSOME 8 GAIN;NF1-/- HIPSC-DERIVED SCHWANN CELL PRECURSOR MODEL
TMOD-26. GENERATION OF A CHROMOSOME 8 GAIN;NF1-/- HIPSC-DERIVED SCHWANN CELL PRECURSOR MODEL Open
Neurofibromatosis type 1 (NF1) is an autosomal dominant cancer predisposition syndrome caused by a germline mutation in the NF1 gene. The loss of the second copy of NF1 within Schwann cell precursors (SCPs) contributes to the formation of …