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- Title
Advances in targeted strategies for managing neurofifibromatosis type 1-related tumors.
- Authors
Zhang Li; Bhavana, Rajbanshi; Surendra, Shrestha; Li Xiuli; Zhao Jingjun
- Abstract
Neurofibromas are the most common and disfiguring feature of neurofibromatosis type 1 (NF1). The treatment options for NF1 have been limited to surgical removal, yet in some cases, the growth pattern of neurofibroma may make its complete resection unpractical. Practitioners are attempting to determine the treatment options for NF1-related tumors that may shrink tumor size, which may cause local organ compression or even decrease the potential long-term risk of undergoing malignant transformation. Several clinical trials evaluating targeted therapeutics reported to have achieved promising results, including Raf inhibitors (sorafenib), MEK inhibitors (selumetinib and trametinib), mammalian target of rapamycin (mTOR) inhibitors (rapamycin), and those targeting the tumor environment (imatinib mesylate and pirfenidone). In 2018, due to high efficacy and low side effects of selumetinib symptomatically and progressively for inoperable plexiform neurofibromas, it was granted orphan drug designation by the FDA and the European Medicine Agency. In this review, we discuss the most common types of NF1-related tumors and the possible mechanisms of tumorigenesis, including the contributions of different signaling pathways and the tumor microenvironment for its management. We also focus on the recent notable advances in the development of therapeutic strategies for NF1-related tumors, including the compounds that have completed their clinical trials and the promising drugs still in clinical trials that have not shown their outcomes to provide perspective to researchers for future studies.
- Subjects
UNITED States. Food &; Drug Administration; NEUROFIBROMA; NEUROFIBROMATOSIS 1; TUMOR microenvironment; ORPHAN drugs; TUMORS
- Publication
Our Dermatology Online / Nasza Dermatologia Online, 2023, Vol 14, Issue 3, p311
- ISSN
2081-9390
- Publication type
Article
- DOI
10.7241/ourd.20233.18