What We’ve Done

The Caroline Symmes Cancer Endowment has:

  • Helped to develop research infrastructure and model development to begin unraveling the molecular underpinnings that drive aggressive solid tumors.
    • Riley is starting to see patients with solid tumors and develop personalized plans that are creating successful results
  • Helped to develop novel preclinical models for investigation of aggressive pediatric solid tumors
    • The models allow the team to better understand the Wilms Tumor and now ask scientific questions and explore new therapeutic options that are CRITICALLY need for patients with aggressive cancers
    • The researchers have learned and tested multiple inhibitors including JAK1/2 and exportin 1
    • They are learning to identify better molecular markers for Wilms Tumor
  • Allowed the Cancer Center research team to investigate targeted and combination therapies for better clinical outcomes through extensive analysis of genomes of the tumor
    • The Riley researchers are able to work with relapsed and aggressive cancer patients through genetic sequencing to provide answers and combination therapies to patient families faster
  • Facilitated the cryo-preservation and use of tumors in research from the cell lines generated by researchers
    • This is an important step in developing therapeutic options as pediatric cancers are different than adult cancers
    • Bio-banking helps to look at tumors in various stages of cancer and regression
    • Genetic changes will happen even after getting treatment and genetic changes contribute to the difficulty in treating aggressive cancers.
    • Using relapsed tumors and developing models in animals and cell lines to study combinations help to get the best outcomes

The Precision Genomics Team at Riley has:

  • Been seeing patients for 2 years
  • Seen 160 patients from a goal of 100
  • Received reports back on 128 of the 160
  • Made tailored treatment recommendations on 96
  • Seen 35 to 40% of patients with non-CNS tumors

Successes:

  • The Precision Genomics Team tested a tumor in a 6 year old and started a targeted therapy. They received a dramatic response, and the patient has been stable for 24 months. They are now back in school and doing much better.

Your continued support will help:

  • Focus investigation of aggressive renal tumors and rhabdomyosarcoma
  • Implement novel clinical trials aimed at establishing effective strategies to improve outcomes for progressively smaller subsets of drug resistant cases
  • Help to understand and predict why certain patients relapse and others don’t.

For a detailed look at the Caroline Symmes Endowment Update from Riley Children’s Health on July 26, 2018, please click here.