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Precision Medicine

“Precision medicine” — sometimes referred to as “individualized medicine,” “personalized medicine,” or “targeted therapy” — is, in basic terms, matching the right patients, to the right medicines, at the right time. This means gaining a deeper understanding of the specific biological, genetic, and/or molecular characteristics that are driving a patient’s tumor growth and finding or developing drugs to target those particular molecular abnormalities that are responsible for the disease.

This approach is potentially very important for brain tumors, which are often very heterogeneous – meaning individual tumors might be made up of cells that have a wide variety of molecular alterations and/or mutations.

Funded Research Highlights

NBTS has maintained an acute focus on funding precision medicine-related research over the past decade. Particularly through past NBTS initiatives like the Defeat GBM and Defeat Pediatric Brain Tumors Research Collaboratives, scientific discovery has advanced our understanding of brain tumors to an extraordinary degree, allowing never-before-seen glimpses into the secrets that tumors had been hiding for centuries. Critically, we’ve been able to identify a variety of molecular culprits that lead to uncontrolled tumor growth as well as potential ‘Achilles Heels’ that may leave certain tumors vulnerable to new, emerging treatment strategies. 

NBTS-funded research has contributed, or directly led, to the discovery of a number of the most important “biomarkers,” or tumor characteristics, that are essential for researchers and doctors to better diagnose patients, plan treatment strategies, and make sure patients can choose a tailored treatment and/or clinical trial, including:

  • Discovery of the mutation IDH mutation, which has become a critical means for neuro-oncologists to distinguish between different types of gliomas.
  • Discovery of the EGFR mutations, which are the most common mutations found in glioblastoma and represent key targets for which many GBM treatment strategies are being developed.
  • Discovery of the 1p/19q co-deletion, which is the key characteristic for identifying and diagnosing low-grade gliomas, including oligodendrogliomas.
  • Discovery of the histone 3.3 mutations, now considered the key driver of pediatric high-grade gliomas.

Featured Research Project: Developing a Novel Therapy to Treat Adult and Pediatric Brain Cancers

NBTS is providing at least $200,000 to support a forthcoming phase II clinical trial of the investigational therapy, ONC201, with the potential for multiple years of funding.

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Featured Research Project: Grant Seeks to Establish New Pipeline of Promising Meningioma Treatments

NBTS is providing $400,000 to a world-class collaboration of researchers who are evaluating potential targeted therapies for meningioma patients that could quickly be translated into an ongoing clinical trial.

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Informed in 30: Precision Medicine

NBTS’s Informed in 30 educational video series took a deep dive into precision medicine.

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