Schedule of Events

Sunday, September 15, 2013

12:00 - 1:30 PM
Opening Lunch

Awarding of 2013 CureSearch Acceleration Grants
CureSearch for Children's Cancer will announce the recipients for the 2013 Acceleration Initiative grants. Applications were accepted in three areas: metastatic disease, refractory relapse disease, and scientific barriers to accelerating treatments.

2:00 - 5:00 PM
The Changing Face of Pediatric Oncology - Clinical Trials in the 21st Century

Medulloblastoma - Translating Genomics to Define Clinical Outcomes and Refine Therapy
This talk will describe the rapidly evolving understanding of medulloblastoma consisting of different molecular subtypes, which vary in different age groups, and which impact the outcome of treatment. Additionally, the talk will address the introduction of molecularly targeted therapy for the SHH subtype of medulloblastoma and introduction of a combined molecular and clinical stratification that will be used in contemporary prospective clinical protocols both in the US and in Europe.

Clinical Genomics in Pediatric Oncology
The genomic features of pediatric malignancies and pediatric-specific issues in clinical investigation may make translating genomic discoveries to the clinic more difficult. However, through large scale molecular profiling of pediatric tumors, continued coordinated efforts to evaluate novel therapies in the pediatric population, thoughtful phase II and III trial design and continued drug development, genomically-based therapies will become more common in the pediatric oncology clinic in the future. Consequently, it is essential to rigorously evaluate the clinical utility of advanced molecular diagnostics in pediatric oncology. The design of clinical studies to evaluate the feasibility of identifying actionable alterations and making individualized cancer therapy recommendations using currently available clinically applicable tumor profiling technologies will be discussed.

Opportunities and Challenges of ALK Inhibition for Treatment of Patients with Neuroblastoma
This talk will address the biology of ALK in neuroblastoma, preclinical and clinical progress in treatment modalities specifically targeting ALK, challenges associated with resistance and steps being taken to overcome such hurdles.

The Role of the FDA in Assuring Effectiveness and Safety of New Drugs: The Evolving Impact on Pediatric Cancer Clinical Trials
This presentation will provide an introduction to the particular legislative protections for children involved in human subject research. The presentation will also address how maximizing the legislative authority provided under the Best Pharmaceuticals for Children Act (BPCA), now permanently re-authorized under the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 and exercising regulatory flexibility could result in approval of more new drugs for pediatric cancer indications.

6:00 - 7:30 PM

Poster Presentations by CureSearch Young Investigators

Monday, September 16, 2013

7:30 - 8:30 AM

The Future of Cellular Therapy in Managing Pediatric Cancer

Historic Success Story for Cellular Immunotherapy
Cellular immunotherapy was successful through the treatment of EBV-associated lymphoid and epithelial malignancies by tumor specific T cells. Lessons learned from analyzing the mechanisms underlying this success can be implemented to treat a broader array of malignancies.

Highly Active Cell Therapy for Leukemia

Tuning the Next Generation of CAR T cells for Pediatric Cancer Applications
The potential of immunotherapy to become a mainstream component of treatment for pediatric oncology patients is substantial. Emerging clinical data is depicting both the impressive potency as well as toxicities of CAR T cell therapy for ALL. The Jensen lab is focused on developing next generation T cells that are engineered with components that facilitate precise control of their function and fate. Our goal is to deliver on precision therapy that is controlled from the bedside to enhance both efficacy and safety of this therapeutic modality.

Genetically engineered T cells for Childhood Cancers: Progress and Challenges
Technologies are now available to target essentially any cell surface molecule on cancer cells using potent, killer T cells. Impressive antitumor effects have been seen in refractory acute lymphoblastic leukemia, which now challenges the pediatric oncology community to determine how best to integrate this new therapy into treatment regimens. Similar progress for solid tumors is possible in principle, but several challenges remain in this arena and will be discussed.

12:00 - 12:45 PM
Lunch and Keynote Address
The Molecular Pathology of Pediatric Cancer: Lessons Learned from DNA Sequence Analysis and their Clinical Implications

1:00 - 4:00 PM
What is on the Horizon for Metastatic and Drug Resistant Diseases?

The Genetic and Molecular Evolution of Relapsed ALL – Clues to Drug Resistance Pathways
The talk will highlight recent work characterizing the genomic landscape of relapsed blasts and options for prevention and novel treatment approaches.

A Comparative Approach to Improve Understanding of Osteosarcoma Metastasis
Using a cross-species and comparative approach to study metastatic progression we have developed a consensus hypothesis that suggests that single metastatic cells that arrive at secondary microenvironments experience a variety of cellular stresses. We believe that the ability of unique metastatic cells to respond or adapt to these stresses is a critical determinant of the metastatic phenotype. This knowledge has allowed the consideration and development of novel therapeutic approaches that target this uniquely vulnerable and clinically relevant part of the metastatic cascade.

Cellular Plasticity in Ewing Sarcoma - Implications for Metastasis and Opportunities for Therapeutic Intervention
Laboratory models are demonstrating that cancer cells are highly plastic and have the capacity to switch back and forth between proliferative and invasive cell states. This talk discuss how understanding how microenvironmental cues drive cellular plasticity and phenotypic conversion in Ewing sarcoma is providing both novel insights into tumor pathogenesis and new opportunities for metastasis-targeted therapy.

Searching for Biologic Drivers of Metastasis in High-Risk Childhood Tumors

4:00 PM
Closing Remarks