Unmet need
Neurofibromatosis 1 (NF1) is a common genetic disorder affecting 1 in 3000 individuals worldwide (see CTF). NF1 patients are at a markedly increased risk for the development of benign and malignant tumors that often disfigure the faces of children by the time they are teenagers. Furthermore, when malignant tumors develop they are often resistant to treatments, difficult to resect, and associated with poor survival. Despite this, there are no chemo-preventative strategies for children born with NF1.
Technology Overview
Johns Hopkins researchers have discovered a globally accessible, inexpensive, FDA-approved drug with the potential to serve as a chemo-preventative regimen for NF1. This drug increases survival and prevent malignancies from developing in mouse model of NF1.
Stage of Development
Using a cis Nf1+/-;Tp53+/- (NPcis) mouse model of NF1, researchers show substantially delayed formation of solid malignancies and significantly increased median survival in mice given the drug. In addition to overall increase in survival and reduction of malignant tumor formation, treatment with the drug also showed a reduction in size of benign tumors in the mice. This would translate to a substantial improvement in quality of life for patients suffering from NF1 in addition to prolonging their life and preventing tumor formation.
Publication:
Staedtke, Verena, et al. "Preventative effect of mebendazole against malignancies in neurofibromatosis 1." Genes 11.7 (2020): 762.
