Dr. Mark Souweidane today treated the final patient in his Phase I clinical trial of convection-enhanced delivery (CED) for diffuse intrinsic pontine glioma (DIPG). The trial, which had enrolled 31 patients over the past four years, was designed to test the safety of CED as a means of delivering a cancer-fighting drug directly to the site of a DIPG tumor. (More about the DIPG trial.)
The trial received FDA approval in late 2011, and the first patient was treated in May 2012. (Read “Cheering for Caitlin,” a profile of the first patient.) Since then, 30 children have received infusions of a therapeutic agent called 124I-8H9, which consists of the 8H9 antibody (produced by mice and effective against many kinds of tumors) combined with the radioactive substance 124I. The dosage of the drug was increased over the course of the trial, also testing safety, but no dose-limiting side effects occurred in any patient.
The infusions, which were done at Memorial Sloan Kettering Cancer Center, were able to deliver the drug directly to the brain stem tumor and were not blocked by the body’s protective blood-brain barrier, which normally prevents chemotherapy drugs from crossing from the bloodstream into the brain in sufficient concentrations to attack a tumor. In this trial, Dr. Souweidane was able to achieve concentrations of 1,000 times or more than what can be achieved with IV chemotherapy. He was also able to design and test new ways to measure those concentrations at the tumor site and monitor how long the drug stayed in the tumor.
Dr. Souweidane and his team will spend the next several months evaluating the data and preparing the results for publication. In the meantime, researchers in the Children’s Brain Tumor Project laboratory have been working to pave the road for the next stages of the trial. Other drugs and drug combinations are being tested to determine what the best agents are to infuse, and at what dose levels. DIPG cell lines are also being grown in the lab, providing a rich source of information about how the tumor mutates over time and responds to different treatments in vitro as well as in animal models. (More about the Children’s Brain Tumor Project.)
The Children’s Brain Tumor Project (CBTP) has been given the opportunity to add another researcher to our ambitious “Summer Sprint.” Carlos Colon, a committee member of the Cristian Rivera Foundation, has pledged $5,000 to support this additional summer research position to supplement our already ambitious effort.
Thanks to our generous supporters, the CBTP lab had already hired six summer researchers to work on testing new drugs and drug combinations against DIPG, GC, and other rare brain tumors; using PET imaging to evaluate these drugs; studying the transformation of low-grade gliomas into malignancies; and procuring samples of thalamic gliomas from other labs around the country. (See more about these projects.) The seventh researcher, Fatima Nathalia Morales, will be working on the team supporting Dr. Souweidane’s DIPG clinical trial, validating the method for monitoring brain tumor and distribution volumes in patients enrolled in the trial.
Fatima, an undergraduate student at Columbia University majoring in biology with a minor in mathematics, is spending the summer in the CBTP lab as part of the Travelers Summer Research Fellowship Program. The support from Carlos Colon and the Cristian Rivera Foundation makes it possible for us to provide her with the equipment and supplies she needs to do her work.
We are extremely grateful to Carlos Colon for the gift, and to John Rivera and the Cristian Rivera Foundation for the tireless efforts on our behalf.
Clinical Trial Update: Now treating patients at its seventh and final dose level, the trial of convection-enhanced delivery (CED) should conclude this summer. Fatima will be helping to evaluate data from more than four years of that trial.
The Cristian Rivera Foundation who is one of the top donors of The Children’s Brain Tumor Project is proud to attend the Children’s Brain Tumor Project Second Annual Family Council Meeting Today Tuesday April 28th, 2015. It’s an honor to come together with other families and foundations who all share the same passion towards helping fund Dr. Mark Souweidane and all the other Doctors who work tirelessly towards finding a cure for inoperable brain tumors.