By Bill Thomas | May 28th
With the already limited amount of federal funds available for pediatric cancer research in the U.S. at risk from NIH budget cuts, it’s easy to feel pessimistic about the future of childhood cancer treatment. Even in the face of such adversity, though, significant advancements continue to be made. All across the world, researchers are hard at work coming up with newer, safer, better treatment options to help pediatric cancer patients overcome their disease and achieve their full potential so they can realize happy, healthy, productive futures.
One of the most promising pediatric cancer developments in recent years has been the increased use and ongoing refinement of CAR T-cell therapy, a revolutionary form of immunotherapy that empowers patients’ own immune systems to better target and eradicate cancer cells.
For more than 20 years, Pediatric Cancer Research Foundation has been at the forefront of CAR T-cell therapy research, funding the work of numerous researchers looking to expand our understanding of and ability to harness the power of CAR T- cells as a potential treatment for childhood cancers. In fact, it was the work of PCRF-funded researchers, including Dr. Laurence Cooper and others, that helped prove that a patient’s pre-programmed CAR T– cells are capable of mounting immune system attacks on tumor cells when infused back into a patient, thus laying the foundations for all subsequent CAR T therapy cancer research.
Among the most exciting developments built on those foundations is the work of Dr. Nicholas Vitanza, a pediatric neuro-oncologist at the Seattle Children’s Hospital Cancer and Blood Disorders Center.
“When we think about tumors and what ways to target them, for generations we focused on killing rapidly dividing cells through a variety of means and then we transitioned to targeted therapies. But most of that was molecularly targeted, which has challenges because [most cancers] are not clonal diseases like leukemia,” Dr. Vitanza explained in a Profectus blog published in 2024.
“CAR T- cells offer a targeted approach by leveraging somebody’s own immune system to better fight a tumor, with the thinking that some things that the immune system should be doing besides fighting infection, which it’s most famous for, is also eliminating abnormal cells of your own that could become cancer.”
Dr. Vitanza and his team have had success using intracerebroventricular B7-H3-targeting CAR T- cells to treat diffuse intrinsic pontine glioma (DIPG), an aggressive type of pediatric brain tumor with a median survival of just 11 months. In a Phase 1 clinical trial, the results of which were recently published in the journal Nature Medicine, 21 children and young adults diagnosed with DIPG received CAR T-cell infusions every two to four weeks. Notably, the median survival for trial participants nearly doubled, with three patients still alive 3 ½ to 4 ½ years after starting treatment.
One of those patients, six-year-old Gavin Nielsen, was recently the subject of an NBC News article highlighting Dr. Vitanza’s research. Diagnosed with DIPG at the tender age of two, Gavin has now lived four times longer than his doctors predicted.
“It’s the biggest miracle that I could ever ask for, just to have time,” Gavin’s mother Ashlee told NBC News.
Though additional research is still needed to determine exactly how viable CAR T therapy is as a treatment for DIPG, Dr. Vitanza’s trial results are heartening. When taken alongside similar success stories, like the one we reported on earlier this year about a neuroblastoma patient treated with CAR T-cell therapy being in remission for a record-breaking 18 years, it’s clear there are still reasons to be optimistic about the future of pediatric cancer research.
None of these developments would be possible, however, without the long line of foundational studies that preceded them. Nor would they be possible if researchers were deprived of the resources that are vital in enabling them to conduct these kinds of trials in the first place. That’s why, even as federal funding for childhood cancer research threatens to shrink, Pediatric Cancer Research Foundation remains committed to powering research that accelerates cures and transforms futures.
If you would like to support our efforts to better the lives of pediatric cancer patients everywhere, please consider becoming a donor. It’s important that we all take action to make sure federal funding for pediatric cancer research is a priority. To make sure our voices are heard, the AACR website now offers a helpful online tool making it easy to contact your representatives. Click here to let Congress how important NIH funding is for pediatric cancer research.
To stay up-to-date with all the latest news shaping the future of pediatric cancer treatment, don’t forget to follow Pediatric Cancer Research Foundation’s Profectus Blog!
