ABC2 is working with researchers at UCLA to develop the first-ever natural disease history study of adult midline gliomas. Patients in this study will be treated with experimental therapies, such as ONC201, that target their mutations. This type of study will open up the path to accelerated approval based on radiographic responses to new treatments in this rare and immediately life-threatening disease.
Findings from this adult study could open up new treatment options for children diagnosed with diffuse midline glioma, also known as diffuse intrinsic pontine glioma (DIPG).
Diffuse midline gliomas are rare and extremely aggressive. They primarily affect children, but can occasionally be found in adults as well. They are a rare type of astrocytoma that look similar under the microscope to other more common astrocytomas, such as a glioblastoma. Since it can be hard to tell them apart, they require molecular testing. Some diffuse midline gliomas have changes in histone-related genes, the most common is h3K27M.
Diffuse midline gliomas harboring the histone H3 K27M mutation have a dismal prognosis with a median survival from diagnosis consistently reported to be between 8 and 11 months. No drug has been approved for H3 K27M-mutant glioma. Radiation is typically used in newly diagnosed patients and associated with some benefit before the tumor progresses, leading to the dismal survival results.
The UCLA study will follow patients from first diagnosis through a series of therapeutic interventions that for a subset of patients will include ONC201, an experimental drug in clinical trials for H3 K27M-mutant gliomas that has reduced the tumor in some patients, based on imaging tests.
“The goal of the study is to quantify the dynamics of how midline gliomas change over time on clinical imaging to better understand the natural disease progression of this disease and if treatments can alter those dynamics,” said Timothy F. Cloughesy, MD, Clinical Professor and Director of the Neuro-Oncology Program at the University of California, Los Angeles. “The natural disease history could serve as a benchmark for new therapies such as ONC201. We are very thankful to ABC2 for their support that will allow us to research this important and unaddressed topic.”
ONC201 is being developed by Oncoceutics, Inc. - a clinical-stage drug discovery and development company, headquartered in Philadelphia, Pennsylvania. Oncoceutics issued a press release to announce the grant.