MANAGEMENT OF ENDOCRINE DISEASE: Precision medicine in neuroendocrine neoplasms: an update on current management and future perspectives

Gaudenzi, Germano; Dicitore, Alessandra; Carra, Silvia; Saronni, Davide; Pozza, Carlotta; Giannetta, Elisa; Persani, Luca; Mantovani, Giovanna

doi:10.1530/eje-19-0021

Cited by 17 publications

(14 citation statements)

References 73 publications

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“…Owing to the high heterogeneity of these types of tumors in the clinical aggressiveness and response to the therapy, the clinical efficacy of current treatment options is limited. For these reasons, a precision medicine (PM)-based strategy could be useful for the management of NENs 100…”

Section: Resultsmentioning

confidence: 99%

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<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

Fallahi¹,

Ferrari²,

Elia³

et al. 2019

CMAR

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Medullary thyroid cancers (MTCs) are neuroendocrine tumors, which secrete calcitonin and carcinoembryonic antigen, both of which can serve as tumor markers. Extensive and accurate surgical resection is the primary treatment for MTC, whereas the use of external beam radiotherapy is limited. Moreover, since MTC is derived from thyroid parafollicular cells or C cells, it is not responsive to either radioiodine or thyroid-stimulating hormone suppression, and therefore, they cannot be considered as treatment strategies. Traditional therapies for advanced or metastatic progressive medullary thyroid cancer (pMTC) are poorly effective. Among the new approaches tested in clinical trials, targeted chemotherapies with tyrosine kinase inhibitors (TKIs) are now available and they represent effective interventions for progressive disease, with additional investigational options emerging. This paper reviews the efficacy and safety of vandetanib in patients with a pMTC, as it has been shown to improve progression-free survival (30.5 vs 19.3 months in controls). Vandetanib is approved by the FDA and EMA for symptomatic or progressive MTC in patients with unresectable locally advanced or metastatic disease in adults, adolescents, and children older than 5 years. The most common adverse events in vandetanib-treated patients are diarrhea, rash, folliculitis, nausea, QTc prolongation, hypertension, and fatigue. More data are required to deepen our knowledge on molecular biology of tumor and host defense, with the aim to achieve better prognosis and higher quality of life for affected patients.

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Section: Resultsmentioning

confidence: 99%

“…Recently, CANscript™, a human platform technology, has been developed to predict the response to anti-cancer therapies in patients with head and neck squamous cell carcinoma. Moreover, patient-derived xenografts in mice are considered the strongest and most evaluated experimental platform for the development of PM applications 100…”

Section: Resultsmentioning

confidence: 99%

<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

Fallahi¹,

Ferrari²,

Elia³

et al. 2019

CMAR

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“…Although embryonic organs and systems are completely defined, their differentiation is incomplete in embryos. This aspect together with the physiological differences between fish and mammals may influence drug metabolism in zebrafish, which may be different from that in mammals (Gaudenzi et al 2019). Advantages and limitations in performing tumor xenografts in zebrafish embryos are summarized in Table 1.…”

Section: Zebrafish Model In Cancer Researchmentioning

confidence: 99%

“…Taking also into consideration the permeability of embryo to small molecules dissolved in the fish water, zebrafish/NET xenograft represents an attractive, fast and technically simple platform to perform drug screening. Moreover, larger or not water-soluble molecules can be injected into the blood stream to ensure drug uptake (Gaudenzi et al 2019). Due to the low proliferation rate of some NETs, the possibility to observe tumor progression in implanted zebrafish embryos in a small temporary window results particularly suitable to test the anti-angiogenic and the anti-metastatic potential of selected drugs, while it may limit the analysis of their anti-proliferative effects.…”

Section: Net Xenografts In Zebrafish Embryosmentioning

confidence: 99%

Fishing for neuroendocrine tumors

Gaudenzi

Carra

Dicitore

et al. 2020

Endocrine-Related Cancer

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Neuroendocrine tumors (NETs) are a class of rare and heterogeneous neoplasms that originate from the neuroendocrine system. In several cases, these neoplasms can release bioactive hormones leading to characteristic clinical syndromes and hormonal dysregulations with detrimental impact on the quality of life and survival of these patients. Only few animal models are currently available to investigate pathogenesis, progression and functional syndromes in NETs and to identify new therapeutic strategies. The tropical teleost zebrafish (Danio rerio) is a popular vertebrate model system that offers unique advantages for the study of several biological processes, ranging from embryonic development to human diseases such as cancer. In this review, we summarize recent advances on zebrafish models for NET preclinical research that take advantage of modern genetic and transplantable technologies. In the future, these tools may have a role in the treatment decision-making and tertiary prevention of NETs.

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“…Preclinical in vitro and in vivo models have recently been developed to capture the heterogeneity of human NENs aiming at delineating the biological behaviour of these tumours as well as to investigate the efficacy of new antitumour agents. The patient-derived xenografts (PDX) in mice and zebrafish embryos are considered as the most promising preclinical models for the introduction of personalized medicine [71].…”

Section: Future Perspectivesmentioning

confidence: 99%

Medical Treatment of Gastrointestinal Neuroendocrine Neoplasms

et al. 2020

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Neuroendocrine neoplasms (NENs) are rare tumours that arise mainly in the gastrointestinal or pulmonary system. Most NENs are well-differentiated and may obtain prolonged survival besides the presence of metastatic disease; however, a subset (poorly differentiated NENs) may display a truly aggressive behaviour exhibiting a poor prognosis. The recently developed classification systems along with advances in functional imaging have helped stratify patients to the administration of appropriate therapeutic options. Surgery is the mainstay of treatment of NENs, but in recent decades there has been a considerable evolution of medical treatments that are used for locally advanced or metastatic disease not amenable to surgical resection. Long acting somatostatin analogues are the main therapeutic modality for patients with functioning and well-differentiated low grade NENs exhibiting symptomatic control and mainly stabilisation of tumour growth. Other systemic treatments include chemotherapy, molecular targeted agents, interferon-α, peptide receptor radionuclide therapy (PRRT), and immunotherapy. In addition, new agents such as telotristat may be used for the control of symptoms of carcinoid syndrome. The choice and/or sequence of therapeutic agents should be individualized according to tumour origin and differentiation, disease burden, presence of clinical symptoms and patients’ performance status in the context of a multidisciplinary approach. Recent advances in the molecular pathogenesis of NENs set the field for a more personalised treatment approach.

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MANAGEMENT OF ENDOCRINE DISEASE: Precision medicine in neuroendocrine neoplasms: an update on current management and future perspectives

Cited by 17 publications

References 73 publications

<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

<p>Evaluating vandetanib in the treatment of medullary thyroid cancer: patient-reported outcomes</p>

Fishing for neuroendocrine tumors

Medical Treatment of Gastrointestinal Neuroendocrine Neoplasms

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