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Pediatric Clinical Trials Update

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Pediatric Studies Explore Drug and Molecular Therapies

When pediatric neuro-oncologist Amar Gajjar, M.D., joined the CERN Foundation leadership team at its inception, he took on a daunting challenge to find new and effective drugs to treat ependymoma.
The disease is typically treated with surgery and radiation because most existing chemotherapy agents are not very effective, explains Dr. Gajjar, also the director of the Neuro-Oncology Division at St. Jude’s Children Research Hospital. Ependymoma is the third most common brain tumor in children.
In collaboration with an international team of investigators, including CERN investigator Richard Gilbertson, M.D., Dr. Gajjar co-led a trial that discovered the drug 5-fluorouracil (5-FU), which is widely used for colon cancer, could have an impact in treating ependymoma if administered rapidly. This discovery in mice led to a phase I clinical trial supported by the CERN Foundation that determined the drug is safe to administer to children. The trial included 26 patients who were 22 years old and younger with ependymoma that recurred after treatment.
“The drug showed modest activity against the tumors that were tested, but it has led to subsequent research that has tested chemotherapy drugs in combination with 5-FU,” Dr. Gajjar says. Such studies are still in development.

Mother Writes Book to Help Others Cope

By Sue (Shawn’s Mom)
CERN Inspirational Story
Our son, Shawn, was diagnosed with an ependymoma brain tumor at 16-months-old. He had been sleepless and irritable and after four different doctor visits in one month, we finally received a diagnosis. Shawn was rushed to SickKids Hospital in Toronto, he had surgery the next day. The surgeon removed 99% of the golf ball-sized tumor and relieved the pressure of fluid that had built up in his brain. A few weeks later, once Shawn was out of intensive care, he started 33 rounds of radiation. We lived a few hours away from the hospital, so I stayed with Shawn and my husband stayed at home with our three-year-old daughter.
Our New Normal
When a child is diagnosed with cancer it affects so many aspects of a family. Not only were we devastated that Shawn has such a serious illness but we were hit with what was called our new ‘normal’. Living apart for days and weeks on end. Our daughter, being only three, did not quite understanding what was going on. The stress on our finances as I could no longer work. It is a whirlwind of change, worry and upheaval.
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