Read on to learn about some of the research that – thanks to you – is changing the world of childhood cancer care.
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
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Your generosity makes a difference for kids with cancer. This edition of the St. Baldrick’s Foundation Research Outcomes recognizes research that is making treatments less toxic, evaluating new drugs, and working to prevent late effects. Thank you for making research possible.
Research progress and hope go hand in hand.
Thanks to your continued support, pediatric cancer researchers are making progress towards better treatments and providing hope for more cures. Here are just four examples of the many research outcomes (and reasons for hope!) you have made possible:
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.
For childhood cancer survivors, treatment helps them to live, but often that survival comes at a cost. But what are these costs? And how big is the problem? That’s what St. Baldrick’s Fellow Dr. Nickhill Bhakta wanted to figure out. And as it turns out, that data could be a lifesaver.
St. Baldrick’s Fellow Dr. Nickhill Bhakta works at his desk in St. Jude Children’s Research Hospital. With a portion of the grant supported by the St. Baldrick’s Friends for Hope Fund, he developed a special statistical tool to help capture the true volume and complexity of chronic health conditions faced by childhood cancer survivors because of the long-term consequences of their treatment — something that hadn’t been done before. Photos courtesy of St. Jude Children’s Research Hospital
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:
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:
In a year when it feels like most things have stopped due to the pandemic, research continues. While most research labs were affected by shutdowns for a time, researchers have found ways to carry on, working hard to find cures for childhood cancers and give survivors long and healthy lives. Read on to see a few interesting outcomes you’ve made possible.
Editor’s Note: Last August, we began a series of blog posts on the outcomes of research that you, our donors, help to fund. Becky Chapman Weaver, Chief Mission Officer, St. Baldrick’s Foundation, provides us with an update.
If you think of a new cure as the top of an enormous mountain, imagine all the work that comes before anyone can stand on that summit for the first time. It takes discovering which routes lead to the top and which are dead ends or sheer cliffs. It takes knowing how to avoid mountain lions, grizzly bears and avalanches. And it takes putting one foot in front of another thousands of times – and often mounting new ascents year after year.
In the world of research, we can think of basic science or laboratory work as the discovery that tells our climber which way to go. Without that, no one gets anywhere. Translational research is our climber using that knowledge to get uphill or to bring those discoveries to the benefit of patients. And a clinical trial tells us whether we’ve achieved the summit – or whether a treatment is more effective for patients than the previous standard of care.
Your support of the St. Baldrick’s Foundation makes every step of the climb possible. Already we have funded more than 1,500 grants, covering every stage of research. Here are a few recent examples of outcomes you can be proud of.
In August of 2019, we blogged about several research outcomes that were fueled by St. Baldrick’s donations and published in scientific journals so that other researchers can build upon them. Research publications are a major way that science moves forward.
With more than 200 new publications a year resulting from research supported by St. Baldrick’s, we’ve decided to make this an occasional series, to highlight some of the most interesting outcomes you’ve made possible.