Current status and research progress of non-surgical treatments for neurofibromatosis type 1
10.3760/cma.j.cn114453-20240305-00056
- VernacularTitle:神经纤维瘤病1型非手术疗法现状
- Author:
Lingbo DANG
1
;
Zhe YANG
1
Author Information
1. 中国医学科学院北京协和医学院整形外科医院会阴整形与性别重塑科，北京 100144
- Publication Type:Journal Article
- Keywords:
Neurofibromatosis 1;
Non-surgical treatment;
Signaling pathway;
Microenvironment;
Gene therapy
- From:
Chinese Journal of Plastic Surgery
2025;41(4):441-450
- CountryChina
- Language:Chinese
-
Abstract:
Neurofibromatosis type 1 (NF1) is a hereditary tumor syndrome caused by biallelic inactivation of the tumor suppressor gene NF1 in glial cells in the skin, nerve plexus, or brain, resulting in the development of benign tumors such as cutaneous neurofibromas, plexiform neurofibromas, and gliomas. As the first-line treatment for NF1, surgical treatment has its limitations. Finding more effective non-surgical treatment options has always been the direction of researchers' efforts. However, despite more than 40 years of research, only one drug is currently approved to treat plexiform neurofibromas, and there are no drugs specifically designed to treat other tumors with NF1. Research over the past few years has shown that NF1 patients may benefit from inhibiting different cell signaling pathways in tumor cells, targeting cells in the tumor microenvironment, or currently mature gene therapy. In this review, we provide an overview of nonsurgical treatment options that have previously completed clinical trials, those currently undergoing clinical trials, and recent research advances that may translate into new treatment options for patients with NF1 in the future.
