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Research Outcomes: Fighting Cancer & Helping Survivors

May 18, 2023
3 min read
general lab equipment with text Research Outcomes

This quarterly edition of the St. Baldrick’s Foundation Research Outcomes features work focused on fighting neuroblastoma, rhabdomyosarcoma, and acute lymphoblastic leukemia as well as research to improve identification of survivors at risk for cardiotoxicity.

Thank you for making these outcomes – and more – possible.

Results Show Promise For Kids With Neuroblastoma

Results were recently published on the first in-child study evaluating lorlatinib with and without chemotherapy in children and adults with relapsed ALK-driven neuroblastoma. Supported in part by the St. Baldrick’s Foundation, this New Approaches to Neuroblastoma Therapy (NANT) Consortium phase 1 study found lorlatinib is safe and tolerable in pediatric and adolescent patients with relapsed neuroblastoma. These findings not only provide hope for patients, they support lorlatinib’s rapid translation into active phase 3 trials for patients with newly diagnosed high-risk, ALK-driven neuroblastoma.

Identifying Survivors At Risk For Cardiovascular Dysfunction

The best cardiovascular screening strategy for survivors of childhood cancer 39 years old or younger has not been determined. St. Baldrick’s Scholar, Dr. Wendy Bottinor, is working to fix that. She recently found that measuring strain on parts of the heart through an echocardiogram can likely improve the identification of survivors at risk for cardiovascular dysfunction, and provide an opportunity for early intervention. Dr. Bottinor found that patients who developed cardiotoxicity had apparent changes in strain measurements about five years after cancer diagnosis. Next steps include validating these findings in a larger cohort to support using strain measurements to stratify risk among survivors.

This grant was supported by To-morrow’s Research Fund, a St. Baldrick’s Hero Fund.

Understanding Rhabdomyosarcoma Subtypes

Rhabdomyosarcoma is the most common sarcoma in children. There are two major subtypes; embryonal rhabdomyosarcoma (ERMS) and alveolar rhabdomyosarcoma (ARMS). Unfortunately, children with ARMS have worse outcomes compared to ERMS patients. A St. Baldrick’s supported study evaluated if a chemotherapy commonly used to treat relapsed (or returning) rhabdomyosarcomas is more or less successful based on subtype. After analyzing the results from five phase 2 clinical trials, researchers found that patients with ARMS had significantly higher response to this type of chemotherapy than those with ERMS. Next steps include investigating the use of this chemotherapy in newly diagnosed patients, particularly those with ARMS.

Outstanding Results for Kids With Low-Risk B-ALL

recent report from the Children’s Oncology Group (COG) shows that low risk B-ALL children enrolled on a COG clinical trial  supported in part by St. Baldrick’s Foundation – experience outstanding survival on low intensity regimens. This trial treated children with two different low intensity regimens, and both were favorable. This finding allows physicians and families to select the treatment approach based on preference and still experience the same survival rates. Next steps include exploring further ways to reduce treatment by eliminating unnecessary therapies.

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.

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