The Development of Targeted Oral Therapy for Treatment of Low Grade Gliomas
Written By: Lead Researcher, Dr. Nicholas Foreman, Neuro-oncologist, Children's Hospital Colorado
One of the biggest breakthroughs our research team has had in the past 7-8 years, which has been funded by the Olivia Caldwell Foundation, has been the movement away from traditional IV chemotherapy to targeted oral therapy in low grade gliomas (LGG), which is the most common tumor in childhood.
Previously, most children, prior to this "new era" in treatment, had been treated with IV chemotherapy given into veins/lines, necessitating travel of large distances to receive chemo once per week in a hospital setting.
The development of targeted therapy involved understanding the very specific "drivers" of low grade gliomas. The Olivia Caldwell Foundation's funding was vital in this important basic work. The introduction of oral targeted therapy to children was made possible by the identification of the drivers specific to an individual child's tumor. This at first was limited to LGGs driven by Braf mutation, but is expanding to include NF1 and FGFR1 driven mutations. All of this was made possible thanks to OCF.
In children with neurofibramatosis, targeted therapy proved not only effective for LGG, which occur in these children, but also for plexiform, which are feature deforming tumors, especially of the face and neck.
The demonstration that targeted therapy could be identified in most children with LGG and that targeted therapy was successful was a major impetus to the development of standard testing of all brain tumors for drivers by the labs of Children's. This movement from research labs to the clinic to routine testing of drivers owes much to OCF. Now children with LGG, from throughout the widespread region we serve, can expect to have their tumor looked at for its driver and to be treated at home with oral therapy which is specific to their tumor's driver. I would estimate over 40 children with LGG have been treated with targeted agents now.
Previously, most children, prior to this "new era" in treatment, had been treated with IV chemotherapy given into veins/lines, necessitating travel of large distances to receive chemo once per week in a hospital setting.
The development of targeted therapy involved understanding the very specific "drivers" of low grade gliomas. The Olivia Caldwell Foundation's funding was vital in this important basic work. The introduction of oral targeted therapy to children was made possible by the identification of the drivers specific to an individual child's tumor. This at first was limited to LGGs driven by Braf mutation, but is expanding to include NF1 and FGFR1 driven mutations. All of this was made possible thanks to OCF.
In children with neurofibramatosis, targeted therapy proved not only effective for LGG, which occur in these children, but also for plexiform, which are feature deforming tumors, especially of the face and neck.
The demonstration that targeted therapy could be identified in most children with LGG and that targeted therapy was successful was a major impetus to the development of standard testing of all brain tumors for drivers by the labs of Children's. This movement from research labs to the clinic to routine testing of drivers owes much to OCF. Now children with LGG, from throughout the widespread region we serve, can expect to have their tumor looked at for its driver and to be treated at home with oral therapy which is specific to their tumor's driver. I would estimate over 40 children with LGG have been treated with targeted agents now.