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Innovation Grants

These grants are designed to provide critical and significant seed funding for experienced investigators with a novel and promising approach to finding causes and cures for childhood cancers. A Letter of Intent is required. The Innovation Award amount totals $250,000 over two years. The Award may not be renewed, however, one no cost extensions are allowable.

Children's Research Institute at Nationwide Children's Hospital

Osteosarcoma is the most common malignant bone tumor in kids. Osteosarcoma frequently express constantly activated STAT3, which is required for cancer progression. Therefore, the development of STAT3 inhibitors for osteosarcoma therapy is desirable and should have high clinical impact. We have developed a novel STAT3 drug discovery approach using multiple ligand simultaneous docking (MLSD) and drug repositioning. MLSD can check multiple fragments from existing drugs already approved for use in humans for docking to STAT3 binding sites.

Principal Investigator Name: 

Jiayuh Lin, PhD

Project Title: 

Novel STAT3 drug development for childhood osteosarcoma therapy using drug repositioning

Year Awarded: 

2012

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

CASE Western Reserve University

Pediatric brain tumors have among the highest cancer incidence and are major contributor of mortality and morbidity. Among pediatric brain tumors, medulloblastoma constitutes the most common malignant brain tumors with metastatic potential. Despite aggressive therapy consisting of surgery, radiation, chemotherapy and bone marrow transplantation, survival in high-risk medulloblastoma patients is only about 60%.

Principal Investigator Name: 

Alex Huang, MD, PhD

Project Title: 

Developing Immune-modulatory Strategies Against Primary and Metastatic Tumors in the Central Nervous System

Year Awarded: 

2012

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

Dana-Farber Cancer Institute

An emerging concept in cancer drug discovery is that of genotype-specific drugs or drugs that only act in a cancer-specific genetic background.  Osteosarcoma, or bone cancer, is characterized by the deletion or mutation of two major cancer-associated genes called p53 and pRb.  We hypothesize that loss of p53 and pRb, in the human disease and in our mouse model of osteosarcoma, renders cancer cells particularly sensitive to inactivation of other genes or pathways that are less detrimental in non-cancerous cells.

Principal Investigator Name: 

Stuart Orkin, MD

Project Title: 

Searching for genetic vulnerabilities in osteosarcoma through genome-wide RNAi screening

Year Awarded: 

2012

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

Brigham and Women's Hospital

Hepatoblastoma is the most common liver cancer of infancy and early childhood, that has become much more common in recent years. The tumor often requires aggressive chemotherapy and liver transplantation. Hepatoblastomas originate from primitive liver cells, so-called liver stem cells, that are formed during embryonic development. This means that the embryonic period prior to birth holds the key to our understanding of the development and prevention of hepatoblastoma.

Principal Investigator Name: 

Wolfram Goessling, MD/PhD

Project Title: 

The role of vitamin D in hepatoblastoma formation and prevention

Year Awarded: 

2012

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

The Johns Hopkins University School of Medicine

Background
Certain visible changes in the chromosomes and mutations in the genes correlate with good or bad prognosis in leukemia. FLT3 is one of the most frequently mutated genes in pediatric AML and also plays an important role in Infant ALL. In both types of pediatric leukemias the FLT3 protein acts as a gas pedal that is permanently pressed to the floor, telling cells to grow out of control. In order to send this signal, FLT3 needs to bind to an energy molecule called ATP.

Principal Investigator Name: 

Donald Small, MD, PhD

Project Title: 

Development of the Next Generation FLT3 Inhibitors for Pediatric AML and Infant ALL

Year Awarded: 

2012

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

The Children's Hospital of Philadelphia

The failure of cancer treatments to kill tumor cells, called therapy resistance, is responsible for most childhood cancer deaths. The precise mechanisms for this resistance are obscure, but most are thought to disrupt death signals that should be “turned on” when sufficient cell stress is encountered. A family of Bcl2-homology (BH) proteins are responsible for responding to chemotherapy- or radiation-induced stress to activate such a death signal. Accordingly, cancer cells typically alter their BH proteins to block these death signals, leading to resistance.

Principal Investigator Name: 

Michael Hogarty, MD

Project Title: 

BH3 Profiling to define therapy resistance classes in neuroblastoma

Year Awarded: 

2011

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

Sanford-Burnham Medical Research Institute

Medulloblastoma, a tumor of the cerebellum, is the most common malignant brain tumor in children. Although a combination of surgery, radiation and chemotherapy can cure many medulloblastoma patients, 25-30% of patients still die from their disease. Moreover, those who survive suffer devastating side effects from the treatment, including cognitive deficits an increased susceptibility to other cancers later in life.

Principal Investigator Name: 

Robert Wechsler-Reya, PhD

Project Title: 

Modeling Anaplastic Medulloblastoma using Cerebellar Stem Cells

Year Awarded: 

2011

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

Baylor College of Medicine

   High Risk neuroblastoma is the most common abdominal tumor in children and accounts for 15% of all pediatric cancer mortality. This is despite arduous intensive toxic chemotherapy treatments with major long-term side effects. Most deaths result from relapsed neuroblastoma, which grows back after initially responding to therapy. Thus, our major goal is to develop novel treatments to prevent neuroblastoma relapse. This will have a major impact on survival for neuroblastoma (currently less than 40% ).

Principal Investigator Name: 

Jason Shohet, MD, PhD

Project Title: 

Strategies for Epigenetically Reprogramming Neuroblastoma Cancer Stem Cells

Year Awarded: 

2011

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

University of California, San Diego

Until last year, the majority of patients diagnosed with high risk neuroblastoma succumbed to the disease despite intensive chemotherapy and bone marrow transplantation. In 2010, the PI of this proposal reported a major improvement in survival from 46% to 66% using an antibody therapeutic. Unfortunately, we cannot predict who will respond to this treatment which is frequently accompanied by serious side effects.

Principal Investigator Name: 

Alice Yu, MD, PhD

Project Title: 

Predicting Outcome and Ameliorating Toxicities of the Immunotherapy of Neuroblastoma

Year Awarded: 

2011

Cancer Research Category: 

Category of Grant: 

Medical, Nurse Researcher, Quality of LIfe: 

Institution: 

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