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When Dr. Vitanza, of Seattle Children’s Hospital, entered the world of DIPG in 2014, researchers were just beginning to learn more about the devastating disease which had remained a mystery for decades. Grieving families had started donating their lost children’s tumor tissue to science, allowing researchers to finally gain some insight into the deadly and aggressive tumors. However, most of the existing preclinical models were developed from DIPG cells that had already been exposed to radiation and chemotherapy, and therefore were not an ideal model of the disease to test potential treatments.

In 2019, the ChadTough Defeat DIPG Foundation awarded Dr. Vitanza a New Investigator grant for his work developing “treatment-naive” DIPG models (models from initial biopsy before the tumor have been exposed to any treatment) and testing new targeted therapies.

In a recent interview with Dr. Vitanza, we learned about the discoveries he has made since receiving the New Investigator grant, as well as the exciting work he has done in the world of CAR T cell therapy.

CTDDF: Can you tell us a bit about yourself and how you decided to work in the world of brain cancer research?

  1. NICK VITANZA: For all of my childhood through medical school, I wanted to be a general pediatrician. It wasn’t until working closely with children with cancer during my pediatric residency that I made the relatively late decision to do a fellowship focused on pediatric oncology. At NYU, I was very lucky to work with two of the world’s best childhood leukemia doctors, Bill Carroll and Elizabeth Raetz, and initially thought I would focus on leukemia laboratory research. This changed when I met a child with DIPG for the first time.

Sitting through that first visit as a trainee and hearing the neuro-oncologist tell a family that there was no cure for their daughter’s tumor changed the direction of my professional life. That day I made the decision to focus my career on pediatric neuro-oncology.

CTDDF: Tell us about the goal of your research and what you have learned so far?

  1. NICK VITANZA: When I first entered the world of DIPG research, working under the extraordinary Dr. Michelle Monje at Stanford, there was still so much basic information about DIPG to learn. One of the issues we encountered was that, while there were children and families who selflessly and generously donated brain tissue at the time of death to develop laboratory models, there were almost no “treatment naïve” models, meaning models that were developed from a DIPG tumor before it had received any treatment. When I arrived at Seattle Children’s – where we frequently biopsy DIPG tumors – my first goal was to establish a platform for DIPG biopsies to come to the lab to be developed into models that can be shared across the world. Fortunately, that program has been a success and we have generated several DIPG laboratory models from initial biopsies and even had those patients visit the lab to see their tumor cells at work.

My second goal was to study the role of biologically targeted drugs in combination with radiation and other conventional drugs. While we had some laboratory success in understanding the way these drugs and radiation interact, the overall benefit of these combinations seemed limited but are deserving of more studies which are currently ongoing.

My third goal was to develop a translational targeted immunotherapy program. Under the mentorship of Drs. Julie Park, Mike Jensen, and Rebecca Gardner, I have now written four CAR T cell trials for children with CNS (central nervous system) tumors, including two trials for children with DIPG. Three of these trials are already open and have enrolled over 65 patients and given over 330 intracranial CAR T cell doses. We have been fortunate to work with incredibly brave patients and families and honored to share our preliminary results in Nature Medicine and Cancer Discovery. So far, we know that we can make enough CAR T cells for patients to receive repeated doses (for example, one 3 year old with DIPG has now received over 30 doses), that these CAR T cells can circulate through the brain and spine, that they are causing immune responses, and at least preliminarily that these may be tolerable treatments. Early phase clinical trials are really designed to understand safety, but we are hopeful that some of our patients are doing much better than we would otherwise expect although it will take many years to know that for sure. Ultimately, I am lucky to work with exceptional mentors, operate at a world-class institution, and conduct research with a great team of scientists including Matt Biery, Carrie Myers, and Michael Meechan, as well as two exciting new hires Dr. Andrea Timpanaro and Dr. Edward Song who work with CAR T cells – and together I am sure we will continue to make important discoveries in the years ahead.

CTDDF: What do you hope the impact will be once your research is completed?

  1. NICK VITANZA: I hope our work has set a foundation for intracranial CAR T cell dosing including, how a pediatric center can design and run these trials, how these doses can be given safely in an outpatient clinic on a repeated basis, and what are the correlative research studies we can perform on the leftover clinical samples (such as blood and CSF) that patients donate to research. My hope is that these studies will teach us what CAR T cells work best, why they may fail, why they may succeed, and how to quickly alert these findings to patients as nothing is as important as time to them.

CTDDF: What do you feel is the importance of private funding, such as grants from the ChadTough Defeat DIPG Foundation, in moving the field forward?

  1. NICK VITANZA: For all practical purposes, our entire CAR T cell program is philanthropically funded. It would take hundreds of NIH grants to run the program we all envisioned. It is just too slow a process. Large private donations have allowed us to build new centers, a state-of-the-art CAR T cell production facility, and provide the funding for patients to enroll on our trials. On a smaller scale, foundation and individual donations have driven my own laboratory research and allowed our plans to be quick, nimble, and collaborative. It would be literally true to say I would not have a lab without private funding and we would not have the broadest CNS CAR T cell program in the country.

CTDDF: Can you comment on the current state of DIPG treatment in general?  For example, how have things changed since Chad Carr was diagnosed in 2014?

  1. NICK VITANZA: In 2014 we were only beginning to learn about the epigenetic alterations in DIPG – which we are still studying. This led to the investigation of entirely new classes of drugs, which led to understanding how they work. I hope this will lead to more targeted drugs that are safer and more effective. We have also learned about the immune landscape of DIPG and vulnerabilities that allow us to use new treatments such as CAR T cells. There has never been a broader range of DIPG clinical trials than there are today and I expect this to only expand over the next 10 years as we begin combining treatments.

ChadTough Defeat DIPG Foundation is proud to have supported Dr. Nick Vitanza’s cutting-edge research, which has led to a deeper understanding of the deadly disease. After years of little development in the field of DIPG, the work conducted by researchers like Dr. Vitanza is finally giving hope to families that a cure will be part of our future.

To learn more about Dr. Vitanza’s work.