Research

Research Outcomes: Your Dollars at Work

by St. Baldrick's Foundation
August 8, 2022

Read on to learn about some of the research that – thanks to you – is changing the world of childhood cancer care.

lab equipment with text Research Outcomes

A Key Gene Is Turned On In Most Cancer Types

St. Baldrick’s Fellow Dr. Jessica Tsai and colleagues discovered that a gene called FOXR2 that is normally turned off in most tissues is activated in at least 70% of cancer types. Their study, recently published in Cancer Research, may help researchers understand how cancer develops. For instance, they found that osteosarcoma shows FOXR2 expression and that FOXR2 boosts the growth rate of brain tumors, including diffuse midline gliomas. There is still a lot to learn about how the gene is activated and they are already working to figure out how to target this gene with new treatments.

Did you see it? Dr. Tsai was featured on a recent Impact Series – watch it here

Developing CAR T Cells Faster

CAR T cell therapy is a type of immunotherapy, fighting cancer with a patient’s own altered immune cells. A new approach from researchers including St. Baldrick’s Scholar Dr. Saba Ghassemi, has drastically cut the time it takes to alter patients’ immune cells for infusion back into the body to find and attack cancer. This process typically takes 9-14 days, but as shown in a recent publication in Nature Biomedical Engineering, Dr. Ghassemi and colleagues generated functional CAR T cells in just 24 hours. This demonstrates the potential for a substantial reduction in the time, materials, and labor required to generate CAR T cells, which could be especially beneficial in patients with rapidly progressive disease and in resource-poor healthcare environments.

Potential New Drug For Ewing Sarcoma

Results from the St. Baldrick’s Foundation Martha’s BEST Grant for All were presented at the recent American Association of Cancer Research (AACR) Special Conference. This research showed that when tested in models, a new drug candidate was 25 times stronger than current FDA approved drugs in trials for Ewing sarcoma treatment. “These newly presented in-vitro data suggest promising activity for our novel kt-3000 series drug candidates as a potential treatment for Ewing sarcoma and other treatment-resistant cancers,” said St. Baldrick’s supported researcher Mads Daugaard, PhD.

The St. Baldrick’s Martha’s BEST Grant for All is funded through an anonymous $1 million donation aimed at developing new treatments for Ewing sarcoma, an aggressive bone and soft tissue cancer in children and young adults. This grant is named for a special teenager who passed away from Ewing sarcoma.

Recent FDA Approval

The FDA recently approved the combination of 2 targeted drugs for the treatment of adults and children ages 6 years or older with nearly any type of advanced solid tumor that has a specific mutation in a gene called BRAF. This mutation can increase the growth and spread of cancer cells. Results from three clinical trials, including one with pediatric patients, laid the groundwork for the approval. Data from the pediatric trial was also used to adapt the use of Trametinib in a phase 2 trial for pediatric patients with relapsed or refractory Juvenile Myelomonocytic Leukemia (JMML). The St. Baldrick’s Foundation is providing support for this JMML phase 2 COG study, and we are eager to see results from this trial.

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

Donate

Read more on the St. Baldrick’s blog:


Research

Research Outcomes: Incredible Impact and Hope

by St. Baldrick's Foundation
May 13, 2022

Your generosity makes a difference for children and young adults with cancer. Read on to see a few recent examples of the incredible impact you have on pediatric cancer research.

image of lab equipment with text Research Outcomes

Immunotherapy for DIPG

Diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas are universally fatal pediatric brain tumors. Researchers on the St. Baldrick’s Foundation Stand Up to Cancer Pediatric Cancer Dream Team are taking what they have learned from treating blood cancers with CAR-T cell immunotherapy and are applying it to these solid tumors.

It’s not often that thousands of scientists break into enthusiastic applause during a presentation of research outcomes, but that’s what happened in April at the annual meeting of the American Association for Cancer Research. It was during a presentation by St. Baldrick’s Scholar and member of the St. Baldrick’s Foundation – Stand Up to Cancer Pediatric Cancer Dream Team, Dr. Robbie Majzner, reporting the following.

Results published in Nature from the first 4 patients enrolled in a clinical trial show consistent effectiveness, and some trial patients have seen their tumors shrink by 95% or more—a dramatic achievement never before seen in DIPG. Though some have since died, most survived far longer than expected and with a greatly improved quality of life. While more research is needed, these findings provide much-needed hope for families.

Using Nanoparticles to Improve Medulloblastoma Treatment

While most medulloblastoma patients are cured with standard treatment, they are typically left with debilitating side effects, so better treatments are needed. A new study published in Science Advances by St. Baldrick’s Foundation Scholar Dr. Timothy Gershon shows that placing a cancer drug, palbociclib, in nanoparticles helps the drug reach tumors better and stay in the body longer. Palbociclib is currently used as a breast cancer treatment.

What are nanoparticles? In medicine, nanoparticles can be used to carry antibodies, drugs, imaging agents, or other substances to certain parts of the body — similar to a tiny soap bubble with the drug cradled in the center.

This study showed palbociclib on its own did not shrink tumors, but when combined with another drug, sapanisertib, and placed in nanoparticles, the cancer models showed better results. While these results are promising, more work is needed to bring this to human clinical trials and researchers are currently working towards that goal.

Clinical Trial Shows Exciting Results for Kids with T-LL and T-ALL

Results from an international phase 3 Children’s Oncology Group (COG) clinical trial could change the standard of care for patients with T-cell lymphoblastic lymphoma (T-LL) and T-cell acute lymphoblastic leukemia (T-ALL).

Researchers found that adding the drug bortezomib to chemotherapy significantly improved overall survival in children and young adults with newly diagnosed T-LL. Additionally, this study found that radiation treatment could be eliminated in 90% of children with T-ALL when the chemotherapy regimen was intensified, decreasing harmful long-term effects of treatment. These exciting findings were recently published in the Journal of Clinical Oncology.

Since becoming an independent foundation in 2005, the St. Baldrick’s Foundation’s largest grant recipient has been the COG, with funds distributed to each COG member institution to subsidize the cost of treating children in clinical trials. St. Baldrick’s has awarded more than $90 million to the COG.

Repurposing Drugs for Pediatric AML

There are numerous subtypes of pediatric acute myeloid leukemia (AML), some with an extremely poor prognosis. Precision medicine is one way to drive progress in pediatric AML. Supported in early stages by the St. Baldrick’s Foundation, the Target pediatric AML (TpAML) group has been performing genetic sequencing to identify promising drug targets.

In the best-case scenario, through sequencing, a new target is found for which a targeted drug already exists. Researchers can then repurpose these existing drugs to treat AML.

After performing genetic sequencing AML researchers have found 4 existing drugs show promise for pediatric AML treatment. In one case, a well-tolerated ovarian cancer drug was identified.

Recently, two of these drugs have been used to treat patients via compassionate use and have shown positive results. The researchers will next work to complete clinical trials to further evaluate the drugs as therapeutic options.

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

Donate

Read more on the St. Baldrick’s blog:


Research

St. Baldrick’s and the Children’s Oncology Group: An Ambitious Relationship

by St. Baldrick's Foundation
October 11, 2021

Did you know: The St. Baldrick’s Foundation and Children’s Oncology Group have been partners in the fight against childhood cancer since our founding.

graphic of St Baldrick’s and COG logos

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Research

Dramatic Progress for Medulloblastoma Patients

by St. Baldrick's Foundation
August 2, 2021

Want to know how St. Baldrick’s donors are the saving lives of kids with a common brain tumor? This isn’t just an example of progress – it’s the biggest increase in survival rates many researchers have ever seen from one clinical trial! And that trial was supported by St. Baldrick’s.


Grace was diagnosed with medullobalstoma at age 5. She is an almost-14-year survivor.

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