Research Outcomes: Continuing Towards Cures

by St. Baldrick's Foundation
May 18, 2021

With your continued support, pediatric cancer researchers are making discoveries to help improve treatment and provide hope for more cures. Five examples of research outcomes you’ve made possible are detailed below:

general research lab equipment

Promising Viral Immunotherapy for Pediatric Brain Tumors

May is Brain Tumor Awareness Month, a perfect time to share exciting recent findings in pediatric brain tumor treatment. A viral immunotherapy developed by researchers, including previous St. Baldrick’s Scholar Dr. Gregory Friedman, shows promise for pediatric brain tumor patients.

As published in the New England Journal of Medicine, Dr. Friedman and colleagues delivered a viral immunotherapy (an engineered herpes virus) directly into the tumor of children with brain tumors.

“The key findings thus far are that the approach is safe and well tolerable, and the preliminary evidence of efficacy is promising,” said Dr. Friedman.

The overall survival rate of patients treated was more than double the typical survival rate for children with high-grade-glioma. The next steps are to determine the ideal timing to treat patients and what therapies can be used along with viral immunotherapy to maximize the anti-tumor immune response.

New Drug Targets for Neuroblastoma

A study published in Science Advances looked at why some neuroblastomas are easy to treat while others have relatively low five-year survival rates.

The researchers, including Dr. Sam Behjati, discovered that all neuroblastomas come from a single type of developmental cell called sympathoblasts, which makes sympathoblasts an attractive drug target. Sympathoblasts are not normally found in children after they are born, which leads to hope that less intrusive therapies targeting these cells can be developed.

Dr. Behjati was also the first international recipient of the St. Baldrick’s Robert J. Arceci Innovation Award.

Preventing Relapse in Acute Lymphoblastic Leukemia (ALL)

Another in a steady stream of discoveries from the St. Baldrick’s Foundation-Stand Up to Cancer Pediatric Cancer Dream Team* was published in the Journal of Clinical Oncology. Researchers discovered that pediatric cancer patients who receive CAR T-cell immunotherapy for ALL suffer fewer relapses and are more likely to survive when treatment is paired with a subsequent stem cell transplant.

Initially CAR T-cell therapy results in complete remission in 60%-100% of patients, but relapse rate are high. However, when combined with a stem cell transplant after CAR therapy, the relapse rate was less than 10% two years later.

Previous St. Baldrick’s Scholar, Dr. Daniel ‘Trey’ Lee was part of the study and says, “We should do all we can to maximize the chance for a cure, and right now that means a transplant after CAR therapy for most.”

International Research: Preventing Central Nervous System Relapse in ALL

Another study, recently published in the journal Blood, identified factors to help predict and better manage the risk of central nervous system (CNS) relapse in ALL patients worldwide.

In this study out of China the five-year, overall survival rate showed a dramatic improvement from previous clinical trials, but CNS relapse risk remains.

Researchers, including St. Baldrick’s International Scholar Dr. Hui Zhang, found the following factors are associated with lower CNS relapse and are dependent on clinical resources available: beginning chemotherapy treatment several days earlier, receiving total intravenous anesthesia for spinal taps, and the use of flow cytometry compared to conventional microscopic examination to accurately diagnose the presence of leukemic cells in cerebrospinal fluid.

Working to Bring Approved Adult Cancer Drugs to Kids

A group of drugs known as PARP1 inhibitors (PARPi) have been approved for treatment of some adult cancers. These drugs are currently under investigation in clinical trials for treatment of pediatric tumors and are likely to be approved for kids in the near future.

Recently published in Nature Communications, St. Baldrick’s Scholar Dr. George-Lucian Moldovan, says, “PARPi resistance is broadly relevant for the clinical deployment of these drugs to treat pediatric cancers. This includes the EWS translocation in Ewing sarcoma, the IDH1/2 mutations in gliomas, and the MYCN overexpression in neuroblastomas.”

The lab will continue to explore these mechanisms to develop therapeutics that might prevent cancers from occurring and make cancer cells more susceptible to treatment.

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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