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PLGA Fund at PBTF

The PLGA Fund at the Pediatric Brain Tumor Foundation fuels the most promising pediatric low-grade glioma and astrocytoma research in addition to equipping, educating and empowering PLGG/PLGA families with resources and a community so that they can thrive.

Following the merger of A Kids’ Brain Tumor Cure with the Pediatric Brain Tumor Foundation in 2018, the PLGA Fund at PBTF continues AKBTC’s work in funding research that will lead to non-toxic, more effective treatments and, ultimately, a cure. The research strategy for the PLGA Fund at PBTF is guided by an external Scientific Advisory Board and two internal Scientific Advisors.

Researchers - Apply for a Research Grant

The PLGA Fund at PBTF accepts PLGG/PLGA research grant proposals on a rolling basis. Projects may vary in length from 1 – 3 years. Novel concepts/hypotheses are encouraged and a premium is given to projects which involve collaboration between institutions and investigators. Eligible projects include basic science, translational and clinical trial initiatives as well as survivorship/outcomes studies. Click here to learn more and email PLGAresearch@curethekids.org with questions.

Families – Find the Support You Need

No family should have to face a child's brain tumor diagnosis alone. The Pediatric Brain Tumor Foundation provides informational, logistical and financial support to help families navigate their journey across the full spectrum of pediatric brain tumor diagnoses. Some resources include the Starfolio Resource Notebook for newly diagnosed families, emergency financial assistance Butterfly Fund and the award-winning Imaginary Friend Society films.

Additionally, the PLGA Fund at PBTF participates in and recommends the following online support groups for those seeking comfort and support following a child’s PLGG/PLGA diagnosis:

Families - Get Involved

More than half of childhood primary central nervous system tumors are gliomas, and, unlike gliomas in adulthood, low-grade gliomas constitute the majority of pediatric gliomas. Pediatric low-grade gliomas arise throughout the nervous system. Despite their slower progression, the impact of the tumors have lasting effects on patients’ quality of life due to the lack of accessibility for safe resection and complications from current toxic medical treatments

The PLGA Fund at PBTF is accelerating the efforts of researchers at top institutions around the globe to find more effective, less toxic treatments. This research is made possible by the generosity of fundraisers, donors, and community events across the country.

Find a fundraiser or learn more about how you can support the PLGA Fund here. You can also follow the PLGA Fund at PBTF on Facebook to stay up-to-date on news and events!

Research We Fund

Over the past decade, the PLGA Fund at PBTF’s support has provided seed funding to researchers around the world and these projects have not only resulted in new targeted therapies being used in the clinics today, but has also enabled scientists to apply and win multi-million-dollar grants from the National Cancer Institute which has resulted in multiple clinical trials that are shaping the direction for targeted therapies.

Keep reading to learn more about our currently funded projects.

Research Grant to Optimize Drug Dosing Strategies for Pediatric LGA/LGG Patients

Award: $180,000 over 2 years
Project Announcement Date: Spring 2020

The Pediatric Brain Tumor Foundation’s PLGA Fund and the American Association for Cancer Research (AACR) are proud to announce the inaugural Research Grant to Optimize Drug Dosing Strategies for Pediatric LGA/LGG Patients. This grant represents a joint effort to promote and support innovative and collaborative research focused on the most common forms of pediatric brain cancer – low grade glioma/astrocytoma. The research proposed for funding should be translational in nature (though basic science and clinical proposals will also be considered) and should be focused on improving the understanding and identification of effective dosing parameters for treating children with PLGA brain tumors, with indications for children battling other types of brain tumors. Proposals should also include at least one collaborator from an institution separate from the applicant’s institution.

Researchers in the field have been invited to apply, as well as investigators with experience in other areas of cancer or biomedical research who have promising ideas and approaches that can be applied to PLGA.

Projects must be implemented by a collaborative research team, composed of one principal investigator and at least one collaborator from different institutions. This grant provides $180,000 over two years for expenses related to the research project, which may include salary and benefits of the grant recipient and any collaborators, postdoctoral or clinical research fellows, graduate students and/or research assistants; research/laboratory supplies; equipment; travel; publication charges for manuscripts that pertain directly to the funded project; and other research expenses.

Phase I/II and Target Validation Study of TAK-580 (MLN2480) for Children with Low-Grade Gliomas and Other RAS/RAF/MEK/ERK Pathway Activated Tumors

Award: $500,000 over 2 years
Principal Investigators: Dr. Karen Wright, Director of Neuro-Oncology, Assistant Professor-Pediatrics, Dana-Farber Cancer Institute, Dr. Daphne Haas-Kogan, Chair-Radiation Oncology, Dana-Farber Cancer Institute/Brigham and Women’s Hospital, and Sabine Mueller, Associate Professor of Oncology, University of California, San Francisco
Funding Partners: Thea's Star of Hope, Starry Night Knoxville, Think Fit for Kids

The RAS/RAF/MEK/ERK pathway is implicated in a number of adult cancers and NF1-associated tumors. Similarly, the majority of pediatric LGGs possess abnormal signaling through the RAS/RAF/MEK/ERK pathway as do a variety of other CNS tumor types including high grade glioma, pleomorphic xanthoastrocytoma and ganglioma. Complete resection is often not feasible and currently available therapies have limited efficacy, leading to an increased morbidity for these patients. More effective therapies using novel mechanisms of action are needed.

Target Validation: To assess the penetration of TAK-580 into the tumor and assessment of the target as defined by suppression of ERK signaling. This trial has finished Phase I and has shown early evidence of response in patients, thereby satisfying the government and agency requirement regarding lack of toxicity in the pediatric population. In addition, pharmacokinetic analysis is complete and 18 PNOC sites are poised to open the Phase II trial once funding is secured.

Phase I/II study of MEK162 for children with progressive or recurrent low-grade gliomas and other central nervous system tumors

Award: $250,000 over 1 year
Principal Investigators: Dr. Nathan Robison, Pediatric Neuro-Oncology Attending, Children’s Hospital Los Angeles, Dr. Mariella Gruber, Department of Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, Dr. Susan Chi, Pediatric Neuro-Oncologist, Dana-Farber Cancer Institute
Funding Partners: Taylor Matthews Foundation, WhyNotMe? Foundation, Making Headway

The target validation component of the study will allow us to determine the ability of MEK162 to penetrate into the tumor as well as assess the ability of MEK162 to affect its target. A short course pharmacokinetics and pharmacodynamic evaluation on these patients for correlation is also planned.

Primary Endpoint:

To quantify concentration of investigational compound in tumor tissue after treatment with MEK162 for 7-21 days and correlate with PK assessment in blood
To assess RAS-RAF-MEK-ERK pathway inhibition, as measured by ERK phosphorylation, in LGG after treatment with MEK162 for 7-21 days. Blood pharmacodynamic assessment will also be performed and correlated with tumor results.

MEK162 continues to demonstrate a positive response; with such promising initial results, researchers are expanding the enrollment by 33% (from 75 patients to 100) and allowing current patients to remain on the trial for an additional year.