Research Funded

Comprehensive mutational analysis of gastrointestinal stromal tumors lacking activating mutations in the KIT, PDGFRA, and BRAF genes

Martin G. Belinsky, PhD, Fox Chase Cancer Center
Recipient of the: $50,000 Sarcoma Foundation of America Research Award

Abstract: Gastrointestinal stromal tumors (GISTs) are mesenchymal neoplasms that generally harbor activating kinase mutations in either KIT or PDGFRA.  GISTs respond to front-line treatment with the specific kinase inhibitor imatin... Read More

Epigenetic changes and epigenetic therapy in retroperitoneal liposarcomas

Nita Ahuja, MD, Johns Hopkins University
Recipient of the: $50,000 Sarcoma Foundation of America Research Award

Abstract: Retroperitoneal liposarcomas are rare tumors and the only effective treatment is surgery. However, local recurrence is common after surgery and is the major cause of death.  Due to the rarity of these tumors, little is kn... Read More

Hedgehog palmitoylation inhibitors to block sarcoma growth

Marilyn D. Resh, PhD, Memorial Sloan-Kettering Cancer Center
Recipient of the: $50,000 Sarcoma Foundation of America Research Award

Abstract: Hedgehog (Hh) signaling has been implicated in the pathogenesis of several types of sarcomas. The goal of the proposed research is to develop drugs that block modification of Hh proteins with palmitate and thereby prevent ... Read More

Identification of causative mutations for Ewing sarcoma

Mizuki Azuma, PhD, University of Kansas Center for Research
Recipient of the: $25,000 Dominick Rizzi Memorial Research Award

Abstract: Ewing sarcoma is the second most common form of bone childhood cancer, and its genetic hallmark is an aberrant fusion gene known as EWS/FLI1.  It is unclear whether the expression of EWS/FLI1 alone is sufficient, or addit... Read More

Crosstalk between EGFR and IGF1R mediated by polymorphisms in the EGFR promoter as a mechanism for resistance to IGF1R directed therapy in osteosarcoma

E. Anders Kolb, MD, Alfred I. duPont Hospital for Children
Recipient of the: $25,000 Dominick Rizzi Memorial Research Award

Abstract: Osteosarcoma is the most common primary malignant tumor of bone, with a pea incidence in the second decade of life. New therapies are needed to improve survival especially among patients with recurrent or metastatic diseas... Read More

Functional analysis of Mirk/Dyrk 1B in osteosarcoma

Zhenfeng Duan, MD, PhD, Sarcoma Molecular Biology Laboratory, Center for Sarcoma and Connective Tissue Oncology, Massachusetts General Hospital
Recipient of the: $25,000 Alexander Burdo/Ziopharm Research Award

Abstract: Osteosarcoma is the most common primary malignant tumor of bone.  Standard treatment is surgery and chemotherapy, which has significantly improved the survival rate from 11% with surgery alone to 60-70% when surgery is co... Read More

A Quantitative Imaging-based Biomarker for Assessment of Therapy Response in Soft Tissue Sarcomas by Differential Volume Estimation of Viable and Non-viable Tumor Fractions

Anand K. Singh, M.D., Massachusetts General Hospital
Recipient of the: $25,000 research award

Abstract: The rapid developments in imaging techniques and action of newer chemotherapeutic drugs have highlighted the limitations of response evaluation criteria in solid tumors (RECIST) for assessing treatment response in soft tis... Read More

Targeting GLI1 in Ewing Sarcoma

William A. May, M.D., Saban Research Institute, Children’s Hospital Los Angeles
Recipient of the: $25,000 research award, Mark Herzlich Research Award

Abstract: The EWS/FLI1 oncogenic transcription factor is both unique to and indispensable to the development of Ewing’s Sarcoma and peripheral Primitive Neuroectodermal Tumor (collectively known as the Ewing Sarcoma Family Tumors ... Read More

Developing a MicroRNA-Based Strategy for Targeting Uterine Leiomyosarcoma

Matthew L. Anderson MD, PhD, Baylor College of Medicine
Recipient of the: $25,000 Jay V. Jackson Memorial Research Award

Abstract: MicroRNAs (miRNAs) are a novel class of small, non-coding RNA transcripts that broadly regulate patterns of gene expression. Recently, we used Next Generation Sequencing to dentify 49 individual miRNAs differentially expre... Read More

Lipid Metabolism in Liposarcoma: A Novel Target for Therapeutic Intervention

Nancy B. Kuemmerle, PhD, Saban Research Institute, Dartmouth Medical School
Recipient of the: $25,000 Richard and Kathy Lobo Research Award

Abstract: There is no effective medical therapy for patients with liposarcoma. We find that liposarcomas are dependent upon a constant supply of fatty acids to fuel their growth. Tumors may potentially acquire these lipids by a) de ... Read More

How does a sarcoma circumvent fusion oncoprotein-mediated toxicity?

Frederic G. Barr, MD, PhD, University of Pennsylvania School of Medicine
Recipient of the: $25,000 ARIAD and Merck Research Award

Abstract: Alveolar rhabdomyosarcoma (ARMS) is an aggressive soft tissue sarcoma with a 2;13 translocation that generates a PAX3-FKHR fusion oncoprotein. Introduction of PAX3-FKHR into multiple cell types showed that this oncoprotein... Read More

Identification of the target genes of the EWS/NR4A3 fusion protein expressed in extraskeletal myxoid chondrosarcoma

Yves Labelle, Ph.D., Centre Hospitalier Universitaire de Quebec
Recipient of the: $25,000 research award, Mandell/Kropp Run for a Sarcoma Cure Research Award

Abstract: Extraskeletal myxoid chondrosarcoma (EMC) are soft tissue tumors occurring mainly in the extremities, most commonly the thigh and knee. In approximately 75% of these tumors, at (9;22) chromosome translocation is present. T... Read More

A preclinical mouse model for targeted therapy in uterine leiomyosarcoma

Sandra Orsulic, Ph.D., Cedars-Sinai Medical Center
Recipient of the: $25,000 research award, Shelby L. Richter Memorial Research Award

Abstract: Uterine leiomyosarcoma (ULMS) is a rare gynecologic malignancy that has a low survival rate. Currently, there is no effective treatment for ULMS. We have generated a mouse model of ULMS and demonstrated that the loss of BR... Read More

Therapeutically targeting aberrant chromatin remodeling in sarcomas

Charles W. M. Roberts, M.D., Ph.D., Dana-Farber Cancer Institute
Recipient of the: $25,000 research award, Michael Mandell/Run for a Sarcoma Cure Research Award

Abstract: SNF5, a core subunit of the SWI/SNF chromatin remodeling complex, is a potent tumor suppressor that is specifically mutated in several types of sarcoma.  It has been thought that oncogenesis in the absence of SNF5 occurs ... Read More

Evaluation of the oncogenic potential of genes within the 1q23 amplicon

Alexander Beeser, PhD, Kansas State University
Recipient of the: $25,000 Blake Cadkin Memorial Research Award

Abstract: Comparative genomic hybridization identified amplification of 1q23 to be associated with liposarcomas. Further analyses of 1q23 narrowed down the candidate “driver” genes to two; DUSP12 (encoding a dual-specificity pho... Read More

Development of novel dual Insulin-like Growth Factor-1 Receptor (IGF-1R)/ Epidermal Growth Factor Receptor (EGFR) inhibitors for treatment of Ewing’s sarcoma

William Bornmann, PhD, MD Anderson Cancer Center
Recipient of the: $25,000 Mary Beth Knox Memorial Research Award

Abstract: Ewing’s sarcoma, a devastating disease in children and young adults, is in great need for a therapy.  Insulin-like Growth Factor type 1Receptor (IGF-1R) and Epidermal Growth Factor Receptor (EGFR) proved to be interesti... Read More

Targeting kinases to reverse multidrug resistance in human sarcoma

Zhenfeng Duan, M.D., Ph.D., Massachusetts General Hospital
Recipient of the: $25,000 research award, ARIAD and Merck Research Award

Abstract: Protein kinases play an important role in the pathogenesis of cancer.  Furthermore, kinases can be targeted by pharmaceutical agents to decrease tumor growth (i.e. EGFR, Her2, c-kit).  This project examines the role of s... Read More

Identification of aberrantly methylated genes in Ewing sarcoma

Scott Borinstein, MD, PhD, Fred Hutchinson Cancer Research Center
Recipient of the: $25,000 ARIAD and Merck Research Award

Abstract: DNA methylation is part of a complex mechanism that regulates genes.  The goal of this proposal is to identify genes that are “turned off” by DNA methylation and contribute to Ewing Sarcoma tumor formation.  We will ... Read More

Targeting the Ras-pathway for the treatment of embryonal rhabdomyosarcoma

David Langenau, Ph.D., Massachusetts General Hospital
Recipient of the: $25,000 research award, PharmaMar Research Award

Abstract: A transgenic zebrafish model of embryonal rhabdomyosarcoma was recently created that is molecularly similar to human disease and from which the cancer stem cell has been identified, a cell most similar to the normal muscle... Read More

Targeting centrosomes in uterine leiomyosarcoma

Matthew Anderson, M.D., Ph.D., Baylor College of Medicine
Recipient of the: $25,000 research award

Abstract: Uterine leiomyosarcoma (ULMS) is characterized by its early metastases, frequent recurrences and poor outcome.  Using genome-wide transcriptional profiling, we have found that the Aurora A and B centrosomal kinases are ro... Read More

Targeting the Wnt pathway in synovial sarcoma

Josiane Eid, M.D., Vanderbilt University Medical Center
Recipient of the: $25,000 research award

Abstract: Synovial Sarcoma is an aggressive soft tissue tumor (SS) that afflicts young adults. Its high fatality rate warrants the design of more efficient curative approaches. SS is characterized by a unique translocation that crea... Read More