Systematic Review of Pediatric Brain Tumors in Neurofibromatosis Type 1: Status of Gene Therapy

Abstract

As oncology practice is rapidly shifting away from toxic chemotherapy, gene therapy provides a highly specific therapeutic approach for brain tumors. In this systematic review, we investigate gene therapy's status in pediatric brain tumors and future recommendations. The search was conducted systematically using PubMed, Cochrane, Google Scholar, and ClinicalTrials.gov databases. The field search used in the process was selected based on the keywords and Medical Subject Headings (MeSH), depending on the database used. We included cases of neurofibromatosis type 1 (NF1) brain tumors in all age groups with the additional inclusion of English language, free full text, articles published within the last 20 years, randomized controlled trials (RCTs), observational studies, systematic reviews, and meta-analyses. We excluded case reports, case studies, and editorials. The search identified a total of 1,213 articles from the databases. We included 19 studies with 16 narrative reviews, one systematic review, and two randomized clinical trials with 43 patients. After reviewing all data in the articles, we found that gene therapy can improve standard treatment efficacy when used as adjuvant therapy. It can be used to overcome barriers such as chemotherapy resistance by downregulating resistance genes. It is associated with mild toxicity when compared with other available treatment options, but given the overall poor prognosis in pediatric brain tumors, further studies are warranted.

Neurofibromatosis Type 1: Optimizing Management with a Multidisciplinary Approach

“…[24][25][26] Gliomas NF1 incurs a susceptibility to optic pathway gliomas (OPG) and brain stem gliomas, most frequently pilocytic astrocytomas. [27][28][29] Approximately two-thirds of these tumors belong to the OPG category. OPG are World Health Organization (WHO) grade I astrocytic tumors 27 that almost always remain fully benign, but which can lead to blindness or other symptoms associated with tumor location.…”