In vitro and in vivo modeling systems of supratentorial ependymomas.
Publication Year:
2024
PubMed ID:
38469231
Funding Grants:
Public Summary:
Ependymomas are rare brain and spinal cord tumors that affect both children and adults. When these tumors form in the upper part of the brain (called supratentorial ependymomas or ST-EPNs), they often involve specific gene changes, such as ZFTA or YAP1 fusions.
Some types of ST-EPN respond well to treatment, but others are much harder to treat. The more aggressive forms often come back after therapy and show a lot of genetic and cellular differences, making them difficult to target with current treatments.
Because real patient samples are rare, scientists are developing laboratory and animal models that can mimic how these tumors behave. These models are crucial for studying how ependymomas develop and for testing potential new treatments.
Scientific Abstract:
Ependymomas are rare brain tumors that can occur in both children and adults. Subdivided by the tumors' initial location, ependymomas develop in the central nervous system in the supratentorial or infratentorial/posterior fossa region, or the spinal cord. Supratentorial ependymomas (ST-EPNs) are predominantly characterized by common driver gene fusions such as ZFTA and YAP1 fusions. Some variants of ST-EPNs carry a high overall survival rate. In poorly responding ST-EPN variants, high levels of inter- and intratumoral heterogeneity, limited therapeutic strategies, and tumor recurrence are among the reasons for poor patient outcomes with other ST-EPN subtypes. Thus, modeling these molecular profiles is key in further studying tumorigenesis. Due to the scarcity of patient samples, the development of preclinical in vitro and in vivo models that recapitulate patient tumors is imperative when testing therapeutic approaches for this rare cancer. In this review, we will survey ST-EPN modeling systems, addressing the strengths and limitations, application for therapeutic targeting, and current literature findings.