Thanks to our generous supporters, since The Lilabean Foundation began fundraising in the fall of 2012, we have raised over $900,000 all of which has been raised for The Brain Tumor Institute at Children’s National Medical Center.
Lila and Dr. Rood of Children’s National Medical Center
CBTTC is a collaborative, multi-institutional research program dedicated to the study and treatment of childhood brain tumors. The mission of the CBTTC is to find cures and improve treatments for children diagnosed with brain tumors. To achieve this mission, the CBTTC collects high quality brain tumor biospecimens and associated clinical data to facilitate the genomic analysis of biospecimens. The CBTTC has established a collaborative, multi-institutional tissue and data repository to enable the collection and analysis of the specimens. For more information on the CBTTC.
This is what the doctors have to say:
“The recent funding will enable us to assess combination therapies for treating childhood cancers such as high grade gliomas. Given our expertise and our unique preclinical models, our laboratory is in a unique position to test these therapeutics. Without the funding from the Lilabean Foundation, we would not have been able to start this innovative project which has a potential for establishing new regimen for treating children with brain cancers.”
– Dr. Javad Nazarian
By identifying and quantifying the proteins in tumor cells of the pediatric brain tumor medulloblastoma, we have been able to create maps of the protein networks operating in these tumors. With funding from the Lilabean Foundation, we will begin to target these pathways with therapeutic compounds and use our protein measurement tools and cell viability tests to assess the effect upon the cells. We will also treat cells until they become resistant and determine the mechanisms of that resistance by measuring how the protein networks of the cell have adjusted.
“The Lilabean Foundation has also funded the application of a new and cutting edge technology called aptamers to the discovery of proteins that are present in the cerebrospinal fluid of children newly diagnosed with a brain tumor and that decline as those tumors are successfully treated. The goal is to discover cerebrospinal fluid proteins that are created by tumor cells whose presence could be used as a measure of tumor burden. Such a tool would help treating physicians to better tailor therapy to an individual child to improve success rates and minimize harmful side effects.”
– Dr. Brian Rood
Specifically, LBF has funded:
The development of a research platform that makes it possible to identify and quantify the complex mixture of proteins in a tissue of biologic fluid. This platform constitutes a new tool for researchers to determine which of the many genetic events present in tumors are translated to the level where the cell manifests its essential nature. This is critical to make optimal use of the massive amount of data generated by genomic technologies and to identify the most exploitable targets for drug therapy.
The creation of a unique resource comprised of the complete set of tumor proteins altered to be slightly heavier than their normal counterparts. Using this reference atlas, investigators are able to compare the abundance of these proteins in multiple tumor samples from children with brain tumors.
Research paper: Proteomic profiling of high risk medulloblastoma reveals functional biology
One application of the quantitative proteomics platform built with support from the Lilabean Foundation is the discovery of biomarkers, proteins that can be used to detect the presence of disease, monitor for disease recurrence or inform clinicians about the biologic characteristics of a tumor.
Training the next generation of scientists to tackle the challenge of curing childhood brain tumors is a vital mission for the Lilabean Foundation. We have partnered with the Brain Tumor Institute at Children’s National to support the research endeavors of both MD and PhD young investigators.
Of course, advancing the understanding of pediatric brain tumors requires continuous investment in reagents and consumable supplies such a cell culture reagents, protective gear, measurement equipment and much more. Similarly, the generation of large amounts of data (e.g. quantitation of thousands of proteins per sample) requires cutting edge software and bioinformatics analysis.
Innovation is a key element of the campaign to improve the way we treat children with brain tumors. True innovation can result in new ways of looking at the problem, develop new tools for research and leap over traditional barriers to progress.
However, innovation is also risky. Not every idea works out. Often the best opportunities for success are the result of previous failures. It is therefore critical to remain nimble and creative, able to follow the data where it leads you, learning from every new result, expected or not.
Traditional research funding mechanisms are not structured to foster this model of research. One writes a research plan for a 2-5 year proposal, attempting to anticipate the results, demonstrating key experimental elements in advance to “de-risk” the proposal, identifying potential pitfalls and illustrating alternative approaches in the event they occur. In short, such proposals are rigid and risk is not welcomed despite the fact that it is inherent in true innovation.
The Lilabean Foundation has overcome these barriers to innovative science with its focus on the shared goal of defeating childhood brain tumors and its approach of funding the building blocks of innovation.
Such a strategy requires determination, vision and faith – qualities displayed by the children we serve and the foundation we support.
–Dr. Brian Rood,
Director of Clinical Neuro-Oncology