NF Clinical Trials Consortium Reports Vision Stabilization Following Everolimus Therapy

The NF Clinical Trials Consortium recently published a study examining vision in children with NF1 and optic pathway gliomas (OPGs) following treatment with Everolimus.

Leveraging preclinical studies from the Gutmann laboratory demonstrating that mouse Nf1 optic glioma growth is controlled by activated RAS operating through the mechanistic target of rapamycin (mTOR) protein.  Everolimus is an inhibitor of mTOR. Following treatment with Everolimus, vision (visual acuity) was stable or improved in over 90% of children with NF1-OPG.

Future studies involving larger numbers of patients are currently being planned.


Ullrich NJ, Prabhu SP, Packer RJ, Goldman S, Robison NJ, Allen JC, Viskochil DH, Gutmann DH, Perentesis JP, Korf BR, Fisher MJ, Kieran MW; NF Clinical Trials Consortium. Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1-associated optic pathway gliomas in children. Pediatr Blood Cancer. 2020 Dec 18:e28833. doi: 10.1002/pbc.28833. Online ahead of print. PMID: 33336845

Heidary G, Fisher MJ, Liu GT, Ferner RE, Gutmann DH, Listernick RH, Kapur K, Loguidice M, Ardern-Holmes SL, Avery RA, Hammond C, Hoffman RO, Hummel TR, Kuo A, Reginald A, Ullrich NJ. Visual field outcomes in children treated for neurofibromatosis type 1-associated optic pathway gliomas: a multicenter retrospective study. JAAPOS. 2020 Nov 20:S1091-8531(20)30228-7. doi: 10.1016/j.jaapos.2020.07.013. Online ahead of print. PMID: 33221469 

Ullrich NJ, Prabhu SP, Reddy AT, Fisher MJ, Packer R, Goldman S, Robison NJ, Gutmann DH, Viskochil DH, Allen JC, Korf B, Cantor A, Cutter G, Thomas C, Perentesis JP, Mizuno T, Vinks AA, Manley PE, Chi SN, Kieran MW. A phase II study of continuous oral mTOR inhibitor everolimus for recurrent, radiographic-progressive neurofibromatosis type 1-associated pediatric low-grade glioma: a Neurofibromatosis Clinical Trials Consortium study. Neuro Oncol. 2020 Oct 14;22(10):1527-1535. doi: 10.1093/neuonc/noaa071. PMID: 32236425