Research Outcomes: Progress from Bench to Bedside

by St. Baldrick's Foundation
February 24, 2021

With your help researchers continue to answer questions, seek out cures, and reduce long-term effects of treatment. Four exciting research outcomes you made possible are detailed below: 

test tubes

Using Immunotherapy to Tackle Fatal Brain Tumors 

Reported in late 2020 in Forbes, as a result of the St. Baldrick’s Foundation-Stand Up to Cancer Pediatric Cancer Dream Team* funding, two new trials are underway to test the effectiveness of immunotherapy on diffuse midline glioma (DMG). DMG is a fatal disease with standard therapies failing to provide any chance of long-term survival, similar to Diffuse intrinsic pontine glioma (DIPG). In this type of immunotherapy, called CAR T cell therapy, a patient’s own immune cells are modified and turned into special CAR T cells, which identify and attack cancer cells. The goals of the studies are to determine if enough CAR-T cells can be created to complete the treatment plan, as well as to test the safety of the CAR-T cells. If CAR T-cell therapies are proven to be safe and effective in patients with DIPG/DMG, this could present a new treatment option for these fatal tumors.  

“Leukemia-on-a-chip” — A New Model for Research 

Recently published in Science Advances a group of researchers, including St. Baldrick’s Research Grant recipient Dr. Iannis Aifantis of New York University School of Medicine, have created a new tool to model B cell acute lymphoblastic leukemia (B-ALL). It is known that B-ALL takes over patients’ bone marrow which facilitates chemoresistance and ultimately disease relapse. This take-over has been difficult for scientists to model and study. This group has created a “leukemia-on-a-chip” model that mimics B-ALL pathology. They were also able to demonstrate the preclinical use of their chip to test regimens, which may translate to patient-specific therapy screening and response prediction. 

Reducing Neurological Side Effects of Treatment in Survivors 

Current treatment strategies for brain and central nervous system tumors include surgery, chemotherapy, and radiation therapy. Radiation therapy has contributed to increased survival rates, but is also associated with adverse neurological side effects. Treatment protocols started in the 90s began to use high dose chemotherapy over a short period of time in order to reduce radiation therapy for the youngest patients. St. Baldrick’s supported researcher, Dr. Sharon O’Neil of Children’s Hospital Los Angeles, recently published positive findings in Neuro-oncology showing excellent survival and preservation of IQ and memory for young children with medulloblastoma using high-dose chemotherapy, with most patients surviving without irradiation. 

FDA Approves Crizotinib for Some Lymphoma Patients  

In January 2021, the FDA approved crizotinib for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is ALK-positive. This approval is based on a clinical trial supported in part by the St. Baldrick’s Foundation. Crizotinib has been used in adults to treat lung cancer. St. Baldrick’s Foundation has supported research into its use in pediatric cancers. This is an exciting new treatment option to add to the list of FDA approvals for pediatric cancers.   

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. 

Help Ensure Research Continues by Donating Today.


*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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