Selumetinib for treating symptomatic, inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in adults

Selumetinib is currently in clinical development for the treatment of symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in adults. Neurofibromatosis type 1 (NF1), caused by changes in NF1 gene, is a type of genetic disorders that primarily affects the cell growth of neural substance. Due to these changes, the gene is unable to carry out its regulatory function, thereby leading to rapid cell multiplication and development of tumours. Plexiform neurofibromas (PNs) develop along the nerve fibres and can infiltrate into surrounding tissues, casing both neurological and space-occupying symptoms. 30-50% of patients with NF1 will develop PN. Inoperable PNs are those that cannot be completely removed surgically, without affecting adjacent critical structures, such as surrounding nerves and vascular architecture, which can lead to further damage. PNs are typically located in challenging areas such as the head, neck, chest and spine) and can affect large and irregularly shaped. Most PN associated with NF1 are symptomatic and can cause pain, disfigurement, and difficulties with physical functioning.