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.

Neurofibromatosis type Ⅰ(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. The search for more effective non-surgical treatments is ongoing. 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.

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.

Neurofibromatosis type Ⅰ(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. The search for more effective non-surgical treatments is ongoing. 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.