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Researchers and Pediatric Brain Tumor Nonprofits Gather in San Francisco for 2019 Translating Discoveries to Cure the Kids Conference


Forty-three researchers representing academic centers in the United States and Europe, industry and government agencies and 19 leaders from the nonprofit community gathered in San Francisco, California earlier this month for the Pediatric Brain Tumor Foundation’s annual Translating Discoveries to Cure the Kids (TD2) conference.

As the world’s leading nonprofit dedicated to children and teens with brain tumors, the PBTF’s TD2 offers pediatric brain tumor researchers and nonprofits an annual opportunity to forge new partnerships and increase the momentum for better treatments.

This year’s annual scientific meeting explored the unique challenges of delivering drugs to central nervous system (CNS) tumors and how we can overcome them.

Breaking Through the Blood Brain Barrier

For a drug or therapy to effectively treat pediatric brain tumors, there are numerous conditions that must be met. First, the drug must be able to simply reach the tumor or site of treatment. Then, it must remain in its active form in effective concentrations for a long enough period of time.

TD2 2019 began by examining one significant challenge to achieving these conditions – the blood-brain barrier, a semipermeable membrane that surrounds the brain and prevents harmful toxins and bacteria in the blood stream from entering. While it generally acts as a lifesaving defense, it can do the opposite for kids with brain tumors by preventing treatment from reaching the brain or allowing them to act as intended.

The blood-brain barrier is one of the many reasons treating brain tumors is so uniquely challenging. As stated by Dr. Kathy Warren, National Cancer Institute, “the drug development paradigm does not work for children with CNS tumors…the blood-brain barrier limits delivery of systematically-administered agents to the CNS."

During the meeting, researchers discussed ways to measure and enhance drug entry past the blood-brain barrier and the potential impact of new delivery approaches, such as convection-enhanced delivery (CED), intrathecal drug delivery, nanoparticles and MRI-guided focused ultrasound, to overcome biological and clinical barriers to brain tumor drug delivery and penetration.

Imagine the impact if an active therapy could actually reach its target in effective concentrations. These researchers aren’t just imagining – they are committed to breaking through these barriers so that one day soon, all children with brain tumors will thrive.

Collaborating to Cure DIPG

Diffuse intrinsic pontine glioma (DIPG), a highly aggressive brain cancer that has no cure, was another focus of the meeting.

In children, DIPG is the leading cause of death from brain tumors. The PBTF is committed to and currently helping fund two critical DIPG research projects that were discussed during the meeting, Project OPEN DIPG and “ALL IN” DIPG.

Project OPEN DIPG is a joint venture by the Pacific Pediatric Neuro-Oncology Consortium and the Children's Brain Tumor Tissue Consortium. The PBTF-funded project uses state-of-the-art technology to determine the potency of candidate drugs belonging to the novel class of molecules called HDAC inhibitors.

In the meeting, researchers presented different case scenarios, demonstrating what they’ve found to be successful and the progress being made. Dr. Adam Resnick, of Children’s Hospital of Philadelphia and a principal investigator on a PBTF-funded Project OPEN DIPG grant, also discussed the importance of convergence research, which involves deep integration across disciplines, usually driven by a specific and compelling problem, and how Project OPEN DIPG embraces it.

“As experts from different disciplines pursue common research challenges, their knowledge, theories, methods, data, research communities and languages become increasingly intermingled or integrated. New frameworks, paradigms or even disciplines can form sustained interactions across multiple communities,” says Dr. Resnick.

Next, Dr. Kathy Warren, NCI, Dr. Michelle Monje, Stanford University, funders and grantees spoke about “ALL IN” DIPG. Having these representatives from multiple perspectives provided a unique opportunity for productive discussion.

“ALL IN” DIPG is a novel public-private partnership initiated by the National Cancer Institute to coordinate DIPG studies and better qualify drug candidates for clinical trials. The PBTF’s Opportunity Grant in support of ALL IN’s projects at NCI is precedent-setting. “ALL IN” DIPG also includes a component that’s specifically designed to distribute funding as extramural research project requests are received and reviewed, making the process more efficient. The extramural research projects are funded by the Musella Foundation, McKenna Claire Foundation and Prayers from Maria Children’s Glioma Cancer Foundation.

Dr. Warren and Dr. Monje provided an update on the project, how to bring new effective therapies to the clinic, and what’s needed to keep it moving forward. Grantees shared updates on their research, how they’ve been able to successfully collaborate, as well as the efficiency of receiving funding due to this novel model.

Saving Children, Not Statistics

Kristine Wetzel from McKenna Claire Foundation and Megan McNamara from Prayers from Maria also spoke about the “why” of the “ALL IN“ DIPG research project, having lost children to this devastating disease themselves.

Both founders drove home that “these are children, not statistics,” and “we’re all better when we’re working together” – two good reminders of why we strive to find ways to collaborate through TD2 and other initiatives.

Representatives from nonprofits Dragon Master Foundation, Friends of Jaclyn Foundation, Heroes for Hope, Inc., MaxLove Project, Michael Mosier Defeat DIPG Foundation, Mithil Prasad Foundation, The Morgan Adams Foundation, Swifty Foundation and Unravel Pediatric Cancer joined McKenna Claire Foundation and Prayers from Maria in attending TD2 to meet with researchers and discuss ways the pediatric brain tumor community can come together to better meet families’ needs.

Each group emphasized that no one person – or institution, consortium, industry or foundation – is going to cure pediatric brain cancer alone. But collectively, we can.

During the nonprofit group meeting, Swifty Foundation, Mithil Prasad Foundation, Friends of Jaclyn and Heroes for Hope, Inc. each spoke about the critical work they’re doing across the country, such as the Swifty Foundation’s Gift from a Child tissue donation initiative.

This convening of organizations demonstrated that by leveraging our unique strengths, all of us can weave a stronger fabric of support for the pediatric brain tumor community to change the treatment landscape and improve outcomes for children with cancer.

By bringing together researchers and nonprofits at TD2, different perspectives led to enriched discussion, which continued at the weekend’s SNO Pediatric Neuro-Oncology conference. As presenting sponsor, PBTF president and CEO Bill Tiller spoke about moving cures forward through collaboration and community, and the PLGA Fund at PBTF hosted a series of pediatric low-grade glioma sessions. Continuing these meaningful conversations will be crucial to achieving our one shared vision: a world without childhood brain tumors.

The PBTF won't stop until there's a cure for every childhood brain tumor. Since 1991, we have funded and partnered with organizations to fund more than $44 million in scientific discovery to increase survivorship, improve quality of life, and ultimately eliminate pediatric brain tumors. Current projects include our Early Career Development program, Immunotherapy Research Initiative, the PLGA Fund at PBTF, and Opportunity Grants. Click here to learn more about the PBTF’s research funding strategy and donate today to help make this research possible.