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Molecular Oncology and Experimental Therapeutics

The Pediatric Molecular Oncology and Experimental Therapeutics research team is focused on translating the basic science understanding of genes and protein mechanisms into new therapeutic modalities while developing better models for tumor studies and a greater understanding of tumor biology. The group puts emphasis on all types of pediatric cancers and related tumors, including sarcomas (osteosarcoma and rhadbomyosarcoma). Basic and preclinical studies facilitate clinical trials as well as understanding mechanism of the diseases.

Areas of Focus

The current areas of research for the Pediatric Molecular Oncology and Experimental Therapeutics group include DNA repair, epigenetics, signaling pathways, oncoproteins, tumor suppressors, angiogenesis and development of novel therapeutics, tumor-microenvironment interaction, and chemotherapy-induced peripheral neuropathy (CIPN).

Learn more about the program's active research

  • Pediatric precision medicine
    Personalized cancer therapy entails targeting and optimizing treatments to the patient. This includes clinical pharmacology of anti-cancer drugs, genetic assessment of each patient’s ability to tolerate treatment and identification of genetic biomarkers in DNA and proteins that predict therapy response.
  • New cancer therapy
    Researchers in the Pediatric Molecular Oncology and Experimental Therapeutics research program are working on developing and optimizing new drugs to treat patients with cancer.

    The Fishel and Kelley labs have worked on the development of APE1/Ref-1 (AP endonuclease1 / Redox effector factor-1) inhibitors based on its role in response to stress, inflammation, and chemoresistance. These studies are built on the structure-affinity relationship (SAR) analysis on parent compound, APX3330 which has completed phase I clinical trials in solid tumors in adults.

  • Pediatric sarcoma
    Active studies on a number of sarcomas have a focus on relapsed osteosarcoma and Ewing’s sarcoma. These highly aggressive tumors are in the bones or in tissue around the bones. Novel tumor models are being developed directly from tumor samples donated by young patients at Riley Hospital for Children at IU Health.
  • Neurofibromatosis tumors
    Ongoing research in the molecular oncology program involved subsets such as malignant peripheral nerve sheath tumor (MPNST). The collaborative group from the Herman B Wells Center discovered that a protein with DNA repair and transcriptional regulatory activity (APE1/Ref-1) increases as NF1 tumors progress to MPNST. Efforts are ongoing to develop and test new inhibitors of this protein in mouse models in order to inhibit the growth of these tumors. The program's interrogation of public and internal MPNST gene expression data resulted in the identification of genes that are upregulated specifically in MPNST and can then be downregulated with inhibition of APE1/Ref-1 in MPNST cells.
  • Glioblastoma multiforme (GBM)
    Glioblastoma multiforme (GBM) is a cancer with no known cure. In pediatric patients 15 years of age or younger, malignant brain tumors represent approximately one-quarter of all pediatric cancers and are associated with the most significant cancer morbidity. Researchers at the Wells Center are focused on understanding the complex mechanisms involved in GBM progression and finding therapeutic agents that interfere with these complex processes.
  • Cancer treatment toxicity
    Investigators are working to identify cellular defects that lead to the the toxicity of cancer treatments. In many people who survive cancer, treatment-related damage can result in lifelong issues such as neuropathy (pain, numbness, hearing loss). This is a major concern for children who survive cancer treatments.
  • Human papillomavirus (HPV)
    Human papillomavirus (HPV) is a common infection that affects more than 75 percent of the adult population and is typically acquired during adolescence and early adulthood. Nearly 5 percent of cancers worldwide are caused by HPV. Based on their association with cancer, different types of HPV are categorized as high-risk or low-risk. The molecular oncology research group studies the protein partnerships that drive cancer development and progression in certain high-risk HPV viral oncogenes.
7 principal investigators
12 trainees
$6 million annually in research funds

Cross Collaboration

Faculty members from the Pediatric Molecular Oncology and Experimental Therapeutics program work in partnership with like-minded faculty and researchers across IU School of Medicine and beyond to forge innovative cancer research.

Team members are proud to collaborate with the Melvin and Bren Simon Comprehensive Cancer Center, Center for HPV Research, the Eugene and Marilyn Glick Eye Institute, and other advocates committed to advancing transformational research.

Featured News

A portrait of a smiling man.

Commitment to excellence

D. Wade Clapp, MD, and Mark R. Kelley, PhD, from the Pediatric Molecular Oncology and Experimental Therapeutics program were two of six Indiana University faculty members elected to the 2021 class of fellows of the American Association for the Advancement of Science. This is one of the most distinguished honors within the scientific community, which recognizes outstanding contributions to the progress of science and research.

tyler-trent-home

Improving outcomes

Karen E. Pollok, PhD, is the lead researcher for two new grants totaling nearly $2M to support osteosarcoma research which was built on previous research made possible by Tyler Trent's tumors. Tyler was a Purdue University student and football superfan who died in 2019, after waging a long and valiant fight against an aggressive form of bone cancer.

Wade Clapp, MD

Bench to bedside

D. Wade Clapp, MD, and his team of scientists are among the world’s foremost experts on neurofibromatosis type 1 (NF1), a rare and potentially fatal genetic condition. Clapp’s work has lead to several of clinical trials and improved quality of life for children with NF1 and their families.

Passion and purpose

Early losses led to a career in cancer research for Melissa Fishel, PhD. Now, she uses this inspired purpose to uncover the mysteries of cancerous cells by developing improved model systems.

HPV and its role in cancer

Rachel Katzenellenbogen, MD, joined IU School of Medicine as the inaugural Chuck and Tina Pagano Scholar in 2018. Her research is focused on human papillomavirus (HPV), a very common infection that affects more than 75 percent of the adult population. Learn more about Dr. Katzenellenbogen.