February 10, 2020 12:00 PM

Accelerating Children’s Access to New Treatments for High Risk Brain Tumors

Michigan Medicine joins exclusive, global network that helps press gas on linking children with incurable brain cancer to promising clinical trials.

Doctor in lab
Carl Koschmann, M.D., who serves as Michigan Medicine’s site co-principle investigator for the network.

For children with high risk brain tumors that resist standard therapy, the next step is often enrolling in a clinical trial.

But connecting these young patients with the most promising therapies in the world is often a complicated and lengthy process.

To address this challenge, an elite, global network of children’s hospitals are teaming up to accelerate access to clinical trials for children with incurable brain cancer through the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Michigan Medicine’s C.S. Mott Children’s Hospital has joined the exclusive group of 22 top children’s hospitals from the United States, Canada, Australia and Europe who are leading the way.

The groundbreaking collaboration, which involves more than 225 pediatric brain tumor specialists and 16 clinical trials, allows sites to share research data in real time and provide patients faster access to clinical trial studies.

Michigan Medicine was selected largely because of the transformative work being done at the Chad Carr Pediatric Brain Tumor Center, whose patient referrals have tripled over the last two years.

“Being a PNOC member puts our program in one of the most exciting inner circles of brain tumor research,” says Mott pediatric oncologist Carl Koschmann, M.D., who now serves as a site co-principle investigator with PNOC.

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“Children from Michigan with high-risk, incurable brain tumors now have access to some of the most promising clinical trials in the world. We can push boundaries in an efficient, rapid and collaborative way to seek breakthroughs.”

Other members of the PNOC team at Michigan include Andrea Franson, M.D.,  Patricia Robertson, M.D., and Marcia Leonard, N.P., who provide additional clinical leadership roles in the pediatric neuro-oncology clinic at Mott. Alyssa Paul will serve as the lead clinical research specialist for the center’s clinical trials, including the PNOC trials that open at U-M.

“We are very proud of the folks working on building our program, and the people from around Michigan and beyond who have believed in and supported the Chad Carr Pediatric Brain Tumor Center,” Koschmann says.

“The sky is the limit for where we can take this center from here.”

Koschmann answers more questions about how the collaboration will make an impact on pediatric brain cancer research and clinical care.

Doctor Image
"Children from Michigan with high-risk, incurable brain tumors now have access to some of the most promising clinical trials in the world. We can push boundaries in an efficient, rapid and collaborative way to seek breakthroughs,"
Carl Koschmann, M.D.

How does this model benefit families and research?

PNOC has a very well organized and motivated centralized clinical trial infrastructure in San Francisco and Zurich that allows trials to open quickly, complete quickly, and share data quickly. This allows us to speed up translating discoveries in the lab to the bedside and share our data with colleagues around the world in real time. 

We now know enough about the sub (and sub-sub) types of tumors that we may need to treat them very differently. In order to know if a specific therapy works, with statistical proof, we typically need to enroll at least 20 patients from each sub-group, and that often requires collaboration from a broad number of sites. PNOC trials are based on precision medicine and immunotherapy, tailoring treatment specifically for each individual child’s tumor.

Many trials will require or offer sequencing along with the therapy. This data will be deposited into a central PNOC database that allows researchers within and outside of PNOC to access de-identified data.

We are able to work together to design the future generation of trials while also re-evaluating current trials.

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What positions institutions like Michigan for PNOC membership?

To qualify for PNOC, children’s hospitals and academic centers must have a large amount of experience and research in high-risk pediatric brain tumors, as well as evidence of the infrastructure to carry out complex early phase clinical trials in this patient population.

Over the last five years, the University of Michigan’s translational pediatric brain tumor program has seen unprecedented growth – from clinical and research to financial and infrastructural perspectives. This led to forming a distinct center – the Chad Carr Pediatric Brain Tumor Center. Our program now meets all of the requirements, and even more exciting, our investigators are positioned to be in a leadership role in a number of international multi-site trials in the coming years.

This is a big milestone for the center, both for the growth of the program and the scale at which we can now develop trials. The acceptance into PNOC was also a big step for validating the efforts already put in by our large and growing team.

What does Michigan’s participation mean for the region?

There are no other PNOC sites in Michigan. We wanted to be able to offer patients in these areas access to the current and future PNOC trials. We also were looking for a platform to introduce some of the research concepts coming from translational researchers at U-M into a national consortium. PNOC generally promotes an early phase, outside the box, collaborative approach that we felt was consistent with the values and strengths of our center and the institution.

What is an example of how research results can be applied in real time?

One example is a tool that we developed at U-M called “CNS TAP” (TAP stands for “Targeted Agent Prediction.”) It’s an algorithm we developed for patients at Michigan with sequencing results to help select targeted therapies for patients with brain tumors. PNOC approached us about incorporating it into a trial they just opened for children with high-grade glioma. After multiple conference calls and adjustments, the trial is being amended to incorporate the tool for patients at PNOC sites around the world. This gives all sites with the trial open access to the tool.

What are your hopes for how this collaboration can play a role in improving care?

While brain cancer is the deadliest form of childhood cancer, only three cancer drugs have been approved for use in children since 1980. By embracing the bold, collaborative approach offered through networks like PNOC, I hope that, together, we can make large steps towards improved therapies and moving towards curing some of these devastating tumors.