Research Outcomes: Novel Discoveries

by St. Baldrick's Foundation
February 15, 2022

Scientific research continues at a great pace thanks to your tireless support. Pediatric cancer researchers proceed to make new discoveries and provide hope for children with cancer. See five examples of the many research outcomes you’ve made possible below:

Lab Equipment with text: Research Outcomes

New Cancer Therapy Developed

St. Baldrick’s Foundation Stand Up To Cancer Pediatric Cancer Dream Team* researchers have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival. Historically these proteins have been impossible to reach, until now.

Published in Nature, the researchers were able to use a new class of engineered T cells to stimulate an immune response that irradicates tumors in models. Researchers completed this in neuroblastoma tumor models, but it’s especially exciting that this method could be used in any cancer, expanding the number of kids who can benefit from with immunotherapy. In the coming months researchers will bring this therapy to a clinical trial for kids with cancer.

New Test to Detect Remaining Cancer in Medulloblastoma Patients

Researchers including St. Baldrick’s supported Dr. Paul Northcott have developed a test aimed at identifying residual disease sooner than other current methods for children with medulloblastoma. Currently, by the time doctors discover the cancer has returned – through MRI or spinal tap – it is usually advanced and difficult to treat. Unfortunately for children with relapsed medulloblastoma, almost all ultimately die from the disease.

This new test detects specific changes in DNA shed from medulloblastoma tumor cells into the fluid surrounding the brain and spinal cord. Based on their findings published in Cancer Cell, they believe this test would identify children who still have evidence of disease shortly after completing treatment and are at high risk of relapse. Additional studies are needed to validate their results before the test can be used for kids with medulloblastoma, but the hope is that this will ultimately give doctors precious time to treat these patients with more aggressive therapies, to save lives.

Identifying Children at Risk for Serious Side Effects from Chemotherapy

Chemotherapy-related toxicities (chemotoxicity) remain poorly understood in children. One important consideration is body composition, which goes beyond the commonly known BMI measurement by assessing skeletal muscle and adipose (fat) tissue. This allows for doctors to see differences in individuals that have the same BMI, but different muscle and adipose tissue make-up. In adults with cancer, body composition has an established association with chemotoxicity.

Using CT measured body composition scans (an emerging predictor of chemotoxicity in adult cancers), St. Baldrick’s Fellow Dr. Aman Wadhwa and colleagues measured body composition in children with lymphoma and rhabdomyosarcoma. Their findings published in Cancer suggest that measuring body composition at diagnosis may provide information that could identify children at risk for serious side effects from chemotherapy. Larger studies are needed, but researchers are hopeful that identifying patients at greatest risk for chemotoxicity could lead to chemotherapy dose reductions or other interventions, and improved quality of life.

Immunotherapy Treatment Possible for some Brain Tumors Previously Thought to be Non-Responsive

When a cell divides, the DNA duplicates. Any errors in this process are corrected by a natural repair mechanism. If not corrected, this may lead to mutations. The repair mechanism itself may be faulty in some children with an inherited condition. Children and young adults with a repair mechanism deficiency can develop brain, intestine, and blood cancers with a very high number of mutations. These hypermutant cancers are difficult to diagnose, do not respond to standard chemotherapy and radiation, and are more common in developing countries. Unfortunately, almost all children with this condition that have relapsed cancers will succumb to the disease.

St. Baldrick’s International Scholar, Dr. Anirban Das and colleagues, developed an inexpensive tool called ‘signatures’ which will help better diagnose these cancers, and benefit many children in underserved areas across the globe.

While these tumors were not previously known to respond to immunotherapy treatment, when treated with a specific type of immunotherapy researchers saw improved survival in relapsed disease compared to traditional chemotherapy and radiation, as published in Nature. In addition to increased survival rates, identifying immunotherapy as an alternative treatment could mean fewer late effects from chemotherapy and/or radiation, and improved quality of life for these children. Dr. Das and colleagues are now working to open a clinical trial to further validate these findings.

Focusing on Data Sharing to Fight Pediatric Cancer

The pediatric oncology community has a long history of research collaboration. With so few cases of childhood cancer compared to other cancers, it’s important for researchers to share data to achieve the sample size, genotypic diversity, and statistical power necessary for new discoveries. Sharing data on this level has increased in adult oncology, but there is a need for a pediatric-oncology-specific data commons that makes clinical (demographic and treatment) genomic, pathology, and imaging data available to promote collaborative international research efforts.

Since 2015, the St. Baldrick’s Foundation supported Pediatric Cancer Data Commons (PCDC) at the University of Chicago has been developing such a commons, to streamline the collection and sharing of pediatric cancer data. The PCDC now has developed, or is developing, data commons for data from patients with neuroblastoma, pediatric soft tissue sarcomas, acute myelogenous leukemia, acute lymphocytic leukemia, Ewing sarcoma, osteosarcoma, Hodgkin lymphoma, central nervous system tumors, retinoblastoma, and germ cell tumors. Working with representatives from international pediatric cancer cooperative groups, the PCDC is harmonizing the largest set of clinical trials and registry data ever collected (> 25,000 children with cancer). Recently, in this spirit of sharing, the PCDC published six critical features of commons design and implementation to serve as a blueprint for others wishing to develop similar resources.

Not every publication of research supported by St. Baldrick’s makes the news, but each one adds to the body of scientific knowledge that takes us one step closer to better outcomes for kids with cancer. Your continued support will make more research possible to Conquer Kids’ Cancer. 

Donate now and help support research into better treatments for kids with cancer


*Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).

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