A new Ivy Brain Tumor Center study finds that recently-approved drug ribociclib may be an important part of treatment for glioblastoma, one of the deadliest cancers in the world.

Glioblastomas remain incurable due to the complex nature of these tumors, the inability of drugs to penetrate the brain tissue, and the lack of correlation between animal models and the human condition. In an innovative phase 0/2 study led by Dr. Nader Sanai, the Director of the Ivy Brain Tumor Center at the Barrow Neurological Institute, the drug ribociclib, recently approved for breast cancer, was shown to penetrate the brain tumor, providing first evidence of clinically-relevant activity of this drug in human glioblastoma.

Data from this study suggests that even though ribociclib is able to get to the tumor region and stop tumor cells from dividing, some tumor cells are able to escape the treatment.

“This is an important early step in the Center’s journey to creating the first ever drug cocktail that would effectively treat these types of brain tumors, reducing them to a chronic disease,” says Dr. Sanai.

The Ivy Brain Tumor Center arrived at these results through the use of a phase 0 clinical trial, an often misunderstood piece of the clinical trial process. The phase 0 trial design keeps in mind the importance of time for brain tumor patients and spares them from investing precious time, effort and energy in therapies that don’t work. The Center is the first and only research body to do this type of study on this type of drug.

The Ivy Brain Tumor Center was founded in 2018 to provide new treatment avenues and hope for glioblastoma patients and is home to the largest phase 0 clinical trials program in the world for brain tumor patients. These studies are funded by the Ben and Catherine Ivy Foundation, in partnership with the Barrow Neurological Foundation.

The Ivy Brain Tumor Center is an advanced clinical trials and research program that offers rapid, state-of-the-art treatment for patients with the most aggressive brain tumors. The Center runs the largest phase 0 clinical trials program in the world, using exploratory studies to help speed up and streamline the drug testing and approval process.The Center’s singular goal is to develop new therapies and treatments that target individual brain tumors, allowing for unique care to every patient. The Center does this by featuring the most advanced scientific and technical techniques available, including many pioneered by the Center itself.