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 or by contacting Patient and Professional Advocacy at (866) 282-5873, (908) 912-9256 or

By selecting Confirm, you are leaving the PTC Therapeutics U.S. website. This external link is provided for your convenience. Please note that external links or web sites may not be controlled by PTC Therapeutics, and thus not subject to our Privacy policy and Terms of use.

Thank you for visiting our site.
Cancel Confirm
This site uses cookies to store information on your computer.
Cancel Continue