Experimental treatment borrowed from blood cancer shows promise for pediatric brain tumors

An experimental version of CAR-T therapy doubled the survival time for children a deadly and aggressive form of brain cancer called DIPG.
Gavin Nielsen was 2 years old when he was diagnosed with a rare and aggressive brain cancer.
The smiley toddler had diffuse intrinsic pontine glioma, or DIPG, a cancer that occurs in the part of the brain stem that controls vital functions including breathing, blood pressure and heart rate. Very few treatment options exist, and the prognosis for children diagnosed with the disease is, on average, less than one year.
“When we have a child newly diagnosed with this disease, we walk into the room and tell the child’s parents their child has a terminal disease and the only option is palliative radiation,” said Dr. Robbie Majzner, director of the pediatric and young adult cancer cell therapy program at Dana-Farber Cancer Institute and Boston Children’s Hospital.
Gavin’s parents started him on radiation. For 30 days, he had to be sedated daily for the treatment. But they also started looking for clinical trials to enroll Gavin in.
At the time, many of the trials for DIPG had a minimum age requirement of 3 years old, his parents, Nate and Ashlee, said.
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