HR 653 & S 305 National Childhood Brain Tumor Prevention Network Act of 2009

1. HR 653 & S 305National Childhood Brain Tumor Prevention Network Act of 2009 Lloyd Morgan, Brain Tumor Survivor (bilovsky@aol.com) Dr. Tarik Tihan, Pediatric Neuro-pathologist (Tarik.Tihan@ucsf.edu)

2. Why Is This Bill Needed? • Almost nothing is known about the causes of Childhood Brain Tumor (CBT) • No single institutions can possibly do a CBT study on their own • There has never been a comprehensive investigation into the cause of CBT IF WE DON’T LOOK, WE WILL NEVER KNOW

3. Childhood Brain Tumor Facts • Leading cause of death from solid tumors in US children • 1stmost common form of solid tumor in US children • 2ndmost common malignancy among US children • Still considered an orphan disease

4. CBT is an Orphan Disease with a costly profile Cost of Initial Treatment per child; calculated for the first line of treatment options. Source :California Childhood Brain Tumor Consortium Study. Dr. Paul Fisher, Stanford University, 2006

5. CBT results in loss of many years of potential life Source :Average years of Potential Life Lost for Childhood Brain Tumors. Thuppal et al. Neuroepidemiology. 2006;27(1):22-7.

6. The incidence of the most common childhood glioma is increasing.Childhood (0-19 yr) Age Adjusted Incidence Rates for Pilocytic Astrocytoma

7. There are numerous types of CBTs and some have been recently defined Newly Described Tumor Types by WHO (2007) • Angiocentric Glioma • Pilomyxoid Astrocytoma • Papillary Glioneuronal Tumor • Rosette-forming Glioneuronal Tumor of the 4th Ventricle (RGNT) • Papillary Tumor of the Pineal Region • Pituicytoma • Spindle Cell Oncocytoma

8. Causes of Childhood Brain Tumors • Therapeutic X-ray to the head <2% • A small fraction of all childhood tumors. • Genetic diseases <5% • Tumor predisposition syndromes such as neurofibromatosis, tuberous sclerosis, nevoid basal cell carcinoma syndrome, Turcot syndrome, Li-Fraumeni syndrome • <5% of childhood brain tumors • UNKNOWN >90% IF WE DON’T LOOK, WE CANNOT LEARN

9. The current research process • Each study is a piece of a 5,000 piece jig-saw puzzle • 32 studies of childhood ependymomas funded • 32 pieces of the puzzle • 1990-2005 • 1,444 children • No common protocol • What does the jig-saw picture look like? • Impossible to see the picture

10. Challenges to Studies on Causes and Risk Factors for Childhood Brain Tumors • Insufficient number of children to study risk factors • The causes/risk factors are likely to be multiple with complex interactions • Without common protocol results cannot be combined • Example: 32 studies of ependymomas • Number of children in studies • Minimum= 11 children ;maximum=92 children • No study use same protocol • Single factor studied: prognostic factor • 32 studies; little to nothing learned!

11. 32 Studies: What Is The Picture?

12. The Rationale • Childhood Brain Tumor (CBT) is an Orphan Disease with a costly profile. • The incidence of the most common type of CBT is increasing • There are new histological types of CBT in the new WHO 2007 • No single institution can accrue sufficient number of “similar” CBT patients in a reasonable period • Causal associations are not likely to be direct • All aspects of CBT needs to be studied together • Genetics, epigenetics, environment, nutrition, pathology, viruses, and clinical

13. Study Design-National Consortium • Regions with sufficient patient population for epidemiological study • Study the “whole picture” rather than one part at a time • All relevant disciplines (multiple experts/resources ) • Environmental exposure analysis (Is there an environmental cause?) • Nutritional analysis (Is there something in the diet?) • Genetic/Genomic analysis (What role do genes play?) • Epigenetic analysis (What turns genes on or off?) • Pathological evaluation/archival information (Are CBT types changing?) • Guthrie cards/perinatal information (Has the child’s DNA mutated since birth?) • Correlation with clinical treatment groups (What are prognostic factors and which treatment can address the causative events?)

14. CBT Study Plan • Case-Control design (1:2 or greater ratio) • Regional Consortia: possibly 5 (>2,500 children) • California/Northwest • Texas/Southwest • Midwest • Florida/Southeast • New York/Northeast • Same procedures and analyses • Designated Central Laboratories for specialized testing • Central Data Repository to collect all research data • Available to all researchers whether or not a study investigator • Provisions for consensus reporting

15. Study Coordination • Funding is additional money to NIH budget • Consortia and study coordination by NCI • Awards grants to regional consortium • Coordinates consortia • Coordinates central statistics and data management • Existing NIH Research Priority • Plan and support a multicenter case-control study of the etiology of childhood brain cancer through the Brain Tumor Epidemiology Consortium (BTEC) • NIH Research Plan for Children’s Brain Tumors, 2008

16. Legislative Strategy for House and SenateBased onprior experience in passing the Benign Brain Tumor Cancer Registries Amendment Act

17. 2009 (111th Congress) • Contact members of House of Representatives • Request they co-sponsor HR 653 • Contact member of Senate • Request they co-sponsor S 305 • Work with House and Senate Committees • Host briefings • Hold meetings

18. 2009-2010 (111th Congress) • Early January • Introduce House and Senate Bills • DONE • February • Meet with NIH/NCI Leadership • Hold Congressional briefings • Push for passage in sub-committee • Meet with sub-committee members and staffers • Identify sub-committee champions • Push for passage in Committee • Meet with sub-committee members and staffers • Identify sub-committee champions • Push for passage in House and Senate • Celebrate

19. Need More Information?Want to Help? • Lloyd Morgan, “Chief Cheerleader” • 510 528-5302; bilovsky@aol.com