Cancer stem cells have been identified in numerous tumor types. These cells have the ability to initiate a tumor, produce other cancer cell types, move freely and proliferate throughout the body and resist chemotherapy and radiotherapy. PTC596 was developed to target those chemotherapy and treatment- resistant tumors.

PTC596 is being evaluated in children with newly diagnosed diffuse intrinsic pontine glioma (or DIPG) and high- grade gliomas. This is a phase 1b trial that is divided into two parts. The goal of the first part is to find a safe dose of PTC596 that can be given together with radiation, which is the standard of care for DIPG. The second part is a surgical study in high-grade gliomas that will determine the amount of PTC596 that may be found in the tumor and blood.  PTC596 will be given prior to and during a planned surgery, which is part of the standard of care for removal of the recurrent tumor.

The study will enroll 30 patients between the ages of 3 and 21 years. For complete eligibility criteria, click here.

More information on this trial can be found at clinicaltrials.gov or by contacting Patient and Professional Advocacy at (866) 282-5873, (908) 912-9256 or PatientInfo@ptcbio.com

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