A Fast-Paced Search for Targeted DIPG Therapies

Title: Rapid preclinical development of a targeted clinical therapy for DIPG
Investigators: Charles Keller and Paul Spellman
Grantees: Oregon Health & Science University (+ 7 others in consortium)

Discovering, testing, and obtaining FDA approval for new drugs to treat specific types of brain tumors is a difficult process that can take many years.  This process is even harder for diffuse intrinsic pontine glioma (DIPG) because we lack good laboratory tools with which to test whether potential new chemotherapies work.  This technology gap delays the discovery and clinical development of much-needed, targeted DIPG treatments.

However, a consortium of scientists recently generated just the sort of tool we need to test DIPG treatments in the lab.  Using brain tumor samples taken from living human patients, these scientists discovered how to grow DIPG cancer cells outside of the human body in a dish.  In addition, they were able to grow different versions of human DIPG tumor cells with widely varying genetic features reflecting the different kinds of DIPG tumors found in patients.

Recognizing the power of this new model, ABC2 established a consortium with The Cure Starts Now, CureSearch for Children’s Cancer, and the Lyla Nsouli Foundation to support a fast-paced, creative search for effective DIPG chemotherapies.  This search campaign involves testing drugs already approved by the FDA to see if they also have “bonus” efficacy against DIPG.  The search will determine whether certain drugs or drug combinations – all of which have FDA approval for human use in other indications – can effectively suppress the growth of laboratory-grown, human DIPG tumor cells with specific genetic features.  This type of focused search has the potential of dramatically cutting down the time needed to develop targeted DIPG chemotherapies to the point where they can be tested in patients.

An important goal of this project is to identify specific genetic features of a DIPG tumor that predict its vulnerability to specific drugs – a key part of matching the right patient with the right treatment.  If this project is successful, it will identify existing drugs or drug combinations that can be quickly progressed into animal and then human clinical trials, thereby taking a rapid first step toward identifying effective, personalized DIPG chemotherapies.

UPDATE:  The most recent research update was published in the June 2015 issue of Nature MedicinePDF iconFunctionally defined therapeutic targets in diffuse intrinsic pontine glioma.