Immunotherapy Research & Clinical Trials

The Angie Fowler Adolescent & Young Adult Cancer Institute at UH Rainbow Babies & Children’s Hospital has a rigorous research program with a national reputation for scientific and translational innovation in pediatric oncology and immunotherapy that extends to include teens and young adults (AYA).

The Adolescent & Young Adult Research Initiative at Case Comprehensive Cancer Center at Case Western Reserve University was established in 2014 through the generous $6.7 million gift from the Fowler family to support AYA cancer research, including gaining a basic understanding of the biology of the metastatic process and of therapy resistance in cancers that occur in this age group, and leveraging expertise in imaging and drug development. In 2017, the initiative has awarded funding to numerous projects that hold great promise for developing new and innovative therapies for cancer patients.

Promising projects underway include:

Small molecule oral therapy for treatment of resistant tumors: An existing drug approved for adult cancers that neutralizes the immune-suppressive effects of tumors against host immune cells. Early pre-clinical results show effectiveness in advanced sarcoma.
Cryoablation therapy: Ultra-cold temperatures have been used clinically to destroy bulking tumors, and Dr. Alex Huang’s team has identified a novel biomarker to show why cryoablating certain tumors can elicit an immunotherapy response.
Naturally derived herbal compounds: Borrowing nature’s own immunotherapeutic agents to activate our body’s immune system; effectiveness potential for sarcoma, melanoma and other hard-to-treat cancers. Dr. Huang and Mei Zhang, PhD, an Assistant Professor of Biomedical Engineering at Case Western Reserve University, have identified a naturally derived compound from oat bran as a potential novel cancer therapeutic. It turns out that the constituent of the oat bran, the outer shell of the oat, is highly immunogenic and can stop tumor growth in mouse models of osteosarcoma, melanoma and pancreatic cancer by eliciting robust anti-tumor immune responses.
Monoclonal antibodies: From mouse studies, we’ve developed an understanding of the factors that are very critical for tumor systems to survive. These factors serve as new targets for therapy and UH Rainbow investigators have identified new ways to block these factors. It turns out that one of the agents that can do that, natalizumab, is already FDA-approved for treating multiple sclerosis and Crohn’s disease. Natalizumab works to affect white blood cells and reduce inflammation. Early results show it may be effective in treating pediatric sarcoma. UH Rainbow hematologist/oncologist Kristen VanHeyst, DO, is working to open a phase I clinical trial of natalizumab in recurrent, refractory or progressive pulmonary metastatic osteosarcoma at UH Rainbow Babies & Children’s Hospital in the fourth quarter of 2021.
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Natural killer (NK) cells: A type of white blood cells that can be engineered to destroy tumors. Angie’s Institute would be among the first places in the U.S. to offer this treatment to pediatric patients.

University Hospitals is one of less than a dozen academic medical centers to have successfully manufactured CAR-T cells for human use onsite in our own laboratory and is the cornerstone of the nation's only National Center for Regenerative Medicine.

Employing Immunotherapy Agents Against Sarcoma

Alex Huang, MD, PhD
Leading efforts from UH Rainbow’s new Center for Pediatric Immunotherapy

Evaluating the mechanisms that mediate disease progression

John Letterio, MD and colleagues determine IFN-γ abundance accelerates gastrointestinal epithelial malignancy by promoting epithelial cell transformation.

Targeting the VCAM-1/VLA-4 signaling axis in the pediatric and AYA cancers

Alex Huang, MD and team strive to develop novel and directed therapy approaches for young adults with osteosarcoma with new proof of concept clinical trial.

Survival Benefit for Individuals With CMMRD Undergoing Surveillance

Duncan Stearns, MD and a team of researchers determine surveillance and early cancer detection are associated with improved OS for individuals with CMMRD.

Center of Excellence for Innovation in Pediatric Immunotherapy Structure

The Angie's Institute and UH Rainbow Babies & Children’s established itself globally through the creation of the Center of Excellence for Innovation in Pediatric Immunotherapy (CEIPI). CEIPI leads new scientific discovery and unique investigator-initiated clinical trials that make UH Rainbow a true destination of choice for pediatric and AYA patients suffering from cancers and other rare diseases that could be effectively targeted using immune-based genetic and cellular therapies. The availability of these novel therapies sets UH Rainbow apart among competing institutions in the region, and undoubtedly exert its clinical impact across multiple disciplines within UH Health System as well as regionally, nationally and globally.

The Promise of Immunotherapy for Pediatric Cancer

While immunotherapies have gained significant traction in treating adult cancers and are a first line treatment for some cancers, including lung cancer and melanoma, the evolution to pediatric cancers has been slow. Currently, only a few pediatric patients who have not responded to other treatments have access to immunotherapies. However, treatments that work for adults cannot be assumed to work for children. They must be studied through rigorous clinical trials, and the FDA has a high bar for research involving children. The science must be perfect to ensure safety and efficacy, and more data is needed about immunotherapies.

UH Rainbow Babies & Children’s is striving to fill the research gap with a goal to bring the exceptional promise of immunotherapies to our youngest patients fighting cancer. This emerging type of therapy has shown tremendous promise in treating pediatric cancers, providing options for difficult to treat diagnoses and the possibility of reducing toxicities that cause life-long risks for survivors. Immunotherapy has potential to improve outcomes and reduce side effects.