Clinical Trials and
Research Funding

Due to a lack of funding for pediatric and young adult sarcoma  cancer research, treatment options have remained unchanged for over 40 years. Our hope is to improve outcomes for young patients by supporting research and clinical trials focused on safer, more effective treatments.

Patient Research

Getting to the Heart of the Matter

When Carley learned that her cancer had returned, she desperately wanted a better treatment than more chemo and radiation. She asked her mother to find something, anything other than more chemo and radiation – immune compromising and debilitating treatment that would keep her from going to college and living a normal life. Luckily, Laura found an immunotherapy vaccine trial that helped her body fight the Ewing sarcoma for eight years and live a fulfilled college and post college life. Unfortunately, the cancer returned in 2020, and Carley passed away at 27, fueling the drive more than ever, to find a curative and less toxic sarcoma treatment.

Luckily, her mom, Laura, found a immunotherapy vaccine trial that helped her body fight the Ewing sarcoma for eight years. Unfortunately, the cancer returned in 2020, and Carley passed away at 27.

Funding Guidelines

Our Research Advisory Team focuses on identifying promising research and new drug developments for pediatric sarcomas that are advancing toward FDA approval and clinical trials. 

When selecting projects, we look for three key factors: (1) the treatment must be less toxic and tailored to the individual (2) it must target cancer cells while protecting healthy tissues and organs, and (3) it must boost the immune system to help the body fight the cancer.

Current Research Projects

A Collaborative Effort to Target Ewing Sarcoma

This is a collaborative research project with Qana Therapeutics in partnership with Alex Bishop, PhD at Department of Cell Systems and Anatomy and Greg Aune, MD, PhD, Associate Professor of Pediatric Hematology and Oncology at Greehey Children’s Cancer Research Institute and Jason Yustein, MD, PhD, Professor of Pediatrics in Hematology/Oncology and Pediatric Hematologist/Oncologist at Children’s Emory Department of Pediatrics.

The goal is to help advance two targeted therapies for Ewing Sarcoma.

We are in our fifth year of funding this project. The first part of our funding provided for the development of two targeted therapies for Ewing Sarcoma: Myr5A.AD198 (QA102P) treatment developed by Dr. Jason Yustein and GPX4 developed by Dr. Bishop.

The next phase of the project is getting the two treatments ready for clinical trials.

Currently, AD198 is nearly complete and is currently in the preclinical mouse model testing phase.

The GPX4 formulation is undergoing a dose escalation test to figure out the most effective and least toxic dosing. Once the dosing is complete, Qana will develop the drug and send it to Dr. Bishop’s lab to test its effectiveness in mouse models.

This study, being conducted at MD Anderson, is focused on a sarcoma treatment using attIL12-T cell therapy. This therapy is designed to boost the immune system’s ability to attack the tumor directly within its environment (the tumor microenvironment) while minimizing harmful side effects on the rest of the body.

The therapy is built upon previous successful experiments using a similar treatment called tumor-targeted IL12 therapy, but it aims to address issues such as harmful side effects and limited effectiveness when treating larger tumors.

We are funding the Phase I clinical trial to test attIL12-T cell therapy for patients with advanced or metastatic soft tissue and bone sarcomas. Since this is the first time the therapy is being tested in humans, the goal is to assess its safety, identify the highest dose that can be given without serious side effects, and determine the optimal dose to use in the next phase of testing (Phase II).

Phase 1 of the trial is conducted in two parts:

Part A: Dose Finding

The study will test up to six different doses of the treatment. The doses will be evaluated using a method called Bayesian optimal interval design, a statistical approach that helps determine the most effective dose with the least risk of side effects.

Part B: Expansion

The efficacy of the treatment will be assessed in 10 additional patients with osteosarcoma treated at the recommended Phase II dose.

They are currently recruiting candidates for the clinical trial. Click here for more information to see if you qualify.

We are supporting Phase II of a clinical trial being conducted at MD Anderson focused on the efficacy and the safety Zanzalitinib.

Zanzalitinib is a new type of cancer treatment for adolescents and young adults with advanced or metastatic bone sarcomas, including osteosarcoma, Ewing sarcoma, chondrosarcoma, and other bone sarcomas. The medication is taken orally and specifically targets particular molecules within cancer cells, aiming to disrupt their growth and spread.

The goals of the trial are:

  • Evaluate the progression-free survival rate, a measure of how long the treatment can keep a patient’s cancer from getting worse, once they start taking the medication.
  • Conduct research to better understand how Zanzalitinib works at the molecular level and how specific biomarkers (indicators in the body that can predict disease progression or response to treatment) influence its effects. This information will help guide the proper use of this treatment, potentially improving its effectiveness and targeting the right patients.
  • Provide a new treatment option for patients with a poor prognosis caused by locally advanced, unresectable, or metastatic bone sarcomas.

With our funding support, Dr. Paul Sorenson and his research team at the British Columbia Cancer Research Centre are working to improve immunotherapy treatments for Ewing sarcoma. Initially, they used several strategies to enhance the targeting of IL1RAP, a key protein involved in the spread of Ewing sarcoma.

Results showed that IL1RAP antibody-drug conjugates (ADCs), a type of targeted therapy that combines antibodies and chemotherapy drugs to treat cancers and other diseases, proved to be the most effective option in mice, with minimal side effects.

Multiple testing methods have of this treatment have been conducted, including:

  • In Vitro Testing: Researchers tested the effectiveness (IC50) of IL1RAP-ADC on cancer cell lines from a mouse model. While the therapy was less effective on these mouse cells compared to human Ewing sarcoma cells, it still showed potential, as the IC50 was in the nanomolar range, indicating targeted therapy efficiency.
  • In Vivo Experimentation: The team conducted an experiment with syngeneic (genetically identical) sarcoma tumors in mice. Initial results showed that a low dose of IL1RAP-ADC did not prevent tumor growth. When they increased the dose, the therapy significantly reduced tumor size compared to untreated mice.
  • Patient-Derived Xenografts (PDX): They received patient-derived tumor samples and implanted them into mice (NSG and Nude mice). Early results showed that two PDX successfully grew in NSG mice, and the researchers are now testing how well these tumors respond to IL1RAP-ADC.

The next steps are discussing clinical trial options for Ewing sarcoma patients while continuing to develop alternative approaches.

RCF Research Partners

Bridging the Gap

In addition to supporting current research projects and clinical trials, Rutledge Cancer Foundation (RCF) is focused on improving efficiencies in the research and biotech communities by bringing together researchers, medical institutions and pharmaceutical companies to work together and more effectively to bring novel therapies to patients more quickly.

Cancer Researches

Help RCF Make a Difference for AYA's Living with Cancer

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Raise Survival Rates

We work as a catalyst to increase awareness and early detection.

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We support research for less toxic, personalized cancer therapies.

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