Pharmacological Approaches in Neurofibromatosis Type 1-Associated Nervous System Tumors

Rabab’h, Omar; Gharaibeh, Abeer; Al-Ramadan, Ali; Ismail, Manar M.; Shah, Jawad

doi:10.3390/cancers13153880

Cited by 8 publications

(3 citation statements)

References 132 publications

(164 reference statements)

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“…Neurofibromatosis type 1 is an essential autosomal dominant genetic disorder resulted from loss-of-function mutations in gene neurofibromatosis type 1 that encodes a negative regulator of Ras GTPases under physiological condition [ 339 ] and influences the MAPK signaling. The loss-of-function mutation is usually found in anaplastic astrocytoma [ 340 ], which NF1 subsequently leads to the diffuse or pilocytic phenotype of pLGG [ 341 ]. When treated with carboplatin and vincristine, LGG patients with NF1 experienced prolonged PFS, OS and decreased toxicity [ 342 ].…”

Section: Low-grade Glioma (Lgg)mentioning

confidence: 99%

Glioma targeted therapy: insight into future of molecular approaches

et al. 2022

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Gliomas are the common type of brain tumors originating from glial cells. Epidemiologically, gliomas occur among all ages, more often seen in adults, which males are more susceptible than females. According to the fifth edition of the WHO Classification of Tumors of the Central Nervous System (WHO CNS5), standard of care and prognosis of gliomas can be dramatically different. Generally, circumscribed gliomas are usually benign and recommended to early complete resection, with chemotherapy if necessary. Diffuse gliomas and other high-grade gliomas according to their molecule subtype are slightly intractable, with necessity of chemotherapy. However, for glioblastoma, feasible resection followed by radiotherapy plus temozolomide chemotherapy define the current standard of care. Here, we discuss novel feasible or potential targets for treatment of gliomas, especially IDH-wild type glioblastoma. Classic targets such as the p53 and retinoblastoma (RB) pathway and epidermal growth factor receptor (EGFR) gene alteration have met failure due to complex regulatory network. There is ever-increasing interest in immunotherapy (immune checkpoint molecule, tumor associated macrophage, dendritic cell vaccine, CAR-T), tumor microenvironment, and combination of several efficacious methods. With many targeted therapy options emerging, biomarkers guiding the prescription of a particular targeted therapy are also attractive. More pre-clinical and clinical trials are urgently needed to explore and evaluate the feasibility of targeted therapy with the corresponding biomarkers for effective personalized treatment options.

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Section: Low-grade Glioma (Lgg)mentioning

confidence: 99%

Glioma targeted therapy: insight into future of molecular approaches

et al. 2022

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“…The diagnosis of NF-1 can be made from clinical symptoms alone. 5,6 Cognitive deficit is the most common complication in children with NF-1. The frequency of intellectual disability in children with NF-1 is between 30 to 69%.…”

Section: Introductionmentioning

confidence: 99%

Case report: a child with type 1 neurofibromatosis and intellectual disability

Elice Wijaya,

Ni Luh Putu Ratih Vibriyanti Karna,

Ida Ayu Uttari Priyadarshini

2023

BDVAJ

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Background: Neurofibromatosis is a genetic disorder that manifests as a tumor surrounding the nerves and several other pathologic presentations. Case: Female, 11 years old, according to history, physical examination and supporting the investigation, was consistent with the diagnostic criteria of type 1 neurofibromatosis (NF-1). The patient also has an intellectual disability. A tumor on the foot region that was functionally disabling was found on the patient, and surgical treatment was performed. Cognitive disorder is the most common neurological complication in individuals with NF-1 and usually presents with a low intelligence quotient (IQ). There is no specific treatment to prevent the development of NF-1. However, early treatment can minimize and prevent further complications. Treatment for the patient involves multiple clinical disciplines. Conclusion: A neurofibromatosis case can be diagnosed clinically alone, since many other systemic involvements require a multi-disciplinary approach.

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“…A novel approach uses the injectable measles virus Edmonston vaccine strain engineered to express the human sodium–iodide symporter and induce cell death. 71 .…”

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confidence: 99%

Clinical trials targeting neurofibromatoses-associated tumors: a systematic review

Souza

Abdalla

Mahadevan

2022

Neuro-Oncology Advances

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Background There is a paucity of literature that comprehensively analyzes previous and current clinical trials targeting neurofibromatoses-related tumors. This article aims to provide readers of drug development efforts targeting these tumors by analyzing translational and clinical findings. Methods This systematic review was written according to the PRISMA guidelines. Inclusion criteria were clinical trials involving patients with neurofibromatosis type 1, type 2, or schwannomatosis that were treated with therapies targeting neurofibromatoses-associated tumors and that were registered on clinicaltrials.gov. In addition, a search was performed in PubMed, Web of Science, Google Scholar, and Embase European for articles fully describing these clinical trials. Results A total of 265 clinical trials were registered and screened for eligibility. Ninety-two were included in this systematic review involving approximately 4,636 participants. The number of therapies analyzed was more than 50. Drugs under investigation mainly act on the MAPK/ERK and PI3K/AKT/mTOR pathways, tumor microenvironment, or aberrantly over-expressed cell surface receptors. Selumetinib was the most effective medication for treating a neurofibromatosis type 1-associated tumor with approximately 68-71% partial response for inoperable or progressive plexiform neurofibromas in children 2 years of age and older and bevacizumab for a neurofibromatosis type 2-related tumor with approximately 36-41% partial response for vestibular schwannomas in patients 12 years of age and older. Conclusions This systematic review presents the results of previous clinical investigations and those under development for neurofibromatoses-associated tumors. Clinicians may use this information to strategize patients to appropriate clinical trials.

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Pharmacological Approaches in Neurofibromatosis Type 1-Associated Nervous System Tumors

Cited by 8 publications

References 132 publications

Glioma targeted therapy: insight into future of molecular approaches

Glioma targeted therapy: insight into future of molecular approaches

Case report: a child with type 1 neurofibromatosis and intellectual disability

Clinical trials targeting neurofibromatoses-associated tumors: a systematic review

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