Children’s Brain Tumor Project Lab Update: Spring 2014

Posted in About the CBTP, Research, Updates

By Mark M. Souweidane, MD Co-Director, Children’s Brain Tumor Project I’ll never forget May 1, 2012 — that was the day I performed the very first procedure in a new clinical trial testing convection-enhanced delivery (CED) of a therapeutic agent in a child with DIPG. More than ten years of my prior lab work, including bench research and animal testing, had me absolutely convinced that this was a safe procedure. Still, with anything so completely new there’s just that tiny seed of doubt, the faintest whisper of “what if…” What I was not prepared for were the emotional ties that quickly took hold of me. The little girl in the OR that day was Caitlin Downing, whom I had come to love in just a few short weeks of knowing her and her family. Hugs, insightful questions, and smiles from this 5-year-old were gripping. Caitlin’s sister, Courtney, had even joined her on one of her preoperative visits to New York so that she could approve of the doctor who would offer hope. Imagine my relief when the procedure went exactly as expected, and imagine my heartbreak several months later when the tumor came back to claim Caitlin’s life. But her successful surgery validated every effort that went into this translational project and left no doubt that we should push on. Today we find ourselves in an exciting position: With 13 children treated so far (all of whom have tolerated the treatment well, with no adverse effects), we have answered critical questions about the technical, surgical, and diagnostic aspect of this drug delivery tactic. Early results have raised awareness from oncologists, neuro-surgeons, radiologists, and pharmacologists from all over the world. The momentum is building rapidly. So encouraging are the results that we have decided to request a continuation of the study. If the continuation is approved, we will plan to treat a minimum of 12 more children at escalating doses. The study is being positively viewed on a national level. The preliminary results of this trial were presented to the Pediatric Brain Tumor Consortium (PBTC) in Atlanta last month. That group is highly interested in moving the strategy into a “group-wide” study and has assembled a working group, which I am leading, to formulate the best plan. Even as we modify the current strategy, we are continuing our laboratory and clinical efforts in different arenas. As an example, we are using other strategies for bypassing the blood-brain barrier, which is what prevents us from getting adequate doses of cancer-fighting molecules to a tumor site. In addition to testing CED, we are also pursuing another potential route, using super-selective intra-arterial chemotherapy to deliver medicine directly into the blood vessels of the...

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Family Update: Spring 2014

Posted in About EH, From the Minter Family, Updates

In our December 2013 newsletter we told you how close Elizabeth’s Hope had come to raising its first $1 million, and we urged our supporters to help us reach that milestone. We’re both proud and humbled to say that we made it, thanks to you. We are dedicated to continuing our work supporting the Children’s Brain Tumor Project, and we are so thankful to know that you are behind us every step of the way. Thank...

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