Research Outcomes: Inspiring Hope

by St. Baldrick's Foundation
November 16, 2023

Our St. Baldrick’s Foundation Research Outcomes blogs highlight examples of the progress your donations are supporting. This quarterly edition focuses on promising new treatments for two types of pediatric sarcoma, protecting kids’ brains while treating brain tumors, and the ease of treating retinoblastoma with chemotherapy.

Thank you for making this research possible.

general lab equipment with text Research Outcomes

Protecting Kids’ Brains During Radiotherapy Treatment

Radiation therapy is commonly used to treat central nervous system (CNS) tumors in children and adults. While radiation therapy doesn’t usually harm the brains of adults, it can cause serious problems for kids. St. Baldrick’s funded researcher, Dr. Kristopher Sarosiek and colleagues discovered that the younger the patient, the more damage the radiation does to their brain. Additionally, they discovered that radiation triggers a process that leads to brain cells dying in kids but not adults. Digging further, they identified a specific protein called BAX that is responsible for this cell death. When they blocked BAX, it prevented brain cells from dying in models. This finding could lead to new ways to protect kids’ brains while undergoing radiation therapy.

This grant is named for the Making Headway Foundation whose mission for the past 20 years has been to provide care and comfort for children with brain and spinal cord tumors through a continuum of services and programs while also funding medical research for cures.

A Promising New Target for Ewing Sarcoma

Ewing sarcoma is an aggressive pediatric bone or soft tissue cancer. Unfortunately, current treatments are not very effective, especially when the cancer has spread or doesn’t respond to treatment. To discover better treatment options for kids with Ewing sarcoma, Dr. Poul Sorensen and colleagues are studying proteins on the surface of Ewing sarcoma cells.

They discovered a new protein not previously known to be associated with this cancer. This protein, called ENPP1, is found only in Ewing sarcoma and not in normal tissues. Because the protein is only in Ewing sarcoma cancer cells it makes for a promising target because therapies could potentially find this protein, leaving normal tissues alone. The researchers are hopeful this will open the door to new immunotherapy treatments for Ewing sarcoma.

This 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.

New CAR T-Cell Therapy Eliminates Rhabdomyosarcoma

Rhabdomyosarcoma is the most common soft tissue sarcoma affecting kids. A recent study from the St. Baldrick’s Foundation EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer) showed that CAR T-cell therapy effectively eliminated the vast majority of rhabdomyosarcoma tumors in models in a matter of weeks.

CAR T-cell therapy is a type of immunotherapy that involves taking cancer-fighting immune cells, called T cells, from a patient and modifying them to target a specific protein found on the surface of cancer cells. The modified T cells are then injected back into the patient, with a boosted ability to fight cancer. Next steps will move this therapy into humans in a phase I clinical trial. “We have a great deal of work ahead, but the hope is that this therapy is going to work for people with this type of cancer that has been unresponsive to other treatments,” says lead investigator Dr. Javed Khan.

Chemotherapy for Retinoblastoma, A Feasibility Study

Retinoblastoma is the most common type of eye cancer in kids. Delivering chemotherapy directly into the eye as a treatment to save the eye in kids with advanced retinoblastoma is used more and more, based on internationally reported success mainly through retrospective studies. To study the feasibility of this treatment (how easily it can be provided) with St. Baldrick’s Support, the Children’ Oncology Group (COG) initiated a clinical trial at 9 institutions. Findings showed it is not feasible due to trouble accessing the appropriate location in the eye to deliver the medicine in some patients. These results do not mean the treatment is not effective, but suggest doctors use caution when considering this treatment to acknowledge the difficulty of delivering the chemotherapy, and invest in specialized training in order to make the treatment easier to administer. Feasibility studies are important to ensure the best treatments are available for kids with cancer.

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.


Read more on the St. Baldrick’s blog:

Real-Life Stories

The Top 2021 Messages Worth Remembering

by St. Baldrick's Foundation
December 16, 2021

As we close a difficult year, we reflect on lessons shared by childhood cancer patients and survivors who have faced adversity with grace, courage, and resilience. From their words, come some messages worth remembering now.

Sarah Swaim, age 31, two-time leukemia survivor & advocate

The St. Baldrick’s Foundation shares the top 2021 messages worth remembering. St. Baldrick’s Honored Kid, Sarah Swaim, and two-time leukemia survivor shares how having support during her cancer diagnosis helped her to keep going.

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What Is Retinoblastoma?

by Murali Chintagumpala, M.D. and Carlos Rodriguez-Galindo, M.D.
May 11, 2020

Dr. Chintagumpala’s specific interests include the management of children with all brain tumors, retinoblastoma, bone tumors and kidney tumors. He serves as chair of the Retinoblastoma Sub-Committee for the Children’s Oncology Group and is a leader in conducting clinical trials involving children with brain tumors and Retinoblastoma.

Dr. Rodríguez-Galindo is a member of the St. Baldrick’s Scientific Advisory Committee. His research focuses on retinoblastoma, bone sarcomas, histiocytic disorders and rare childhood cancers.

what is retinoblastoma

What is Retinoblastoma?

Retinoblastoma is a cancer of the eye that only occurs in children and typically in very young children. Two-thirds of retinoblastoma patients are diagnosed before they’re 2 years old and more than 90% are diagnosed before turning 5.

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Events and Fundraisers

National Volunteer Week: Meet Jessica and Dr. Schultz

by St. Baldrick's Foundation
April 11, 2019

As we wrap up National Volunteer Week, today we’ll meet two more volunteers with very different stories: one is the mom of a child diagnosed with cancer; the other, a researcher who’s devoted her career to early detection of childhood cancers.

Both will show us that it doesn’t matter how you get involved – just that your involvement is crucial to the work of the St. Baldrick’s Foundation.

Jessica’s daughter, Mya, and friend Brendan are the faces of “Two Tiny Heroes”.

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What Does it Take to Beat Cancer, Fly on a Zip Line and Go to School Without Sight? Bravery Every Day

by Katie Vescelus
August 31, 2018

Honored Kid Matthias was diagnosed with retinoblastoma when he was just 3 months old, leaving him blind. But that hasn’t stopped him. Today, Matthias is a childhood cancer survivor and an independent 10-year-old who is learning to navigate the world without sight. As his mom, Katie, shares, he’s one brave kid.


Matthias was just a baby when doctors had to remove his eyes to save his life. Since then, he’s learned to read braille and use a cane to help him get around. Photo by Jen Sherrick Photography

When Matthias lost his eyes to bilateral retinoblastoma nine years ago, we never could have imagined how happy and full his life would be. He is a hiker, traveler, and adventurer who has visited 23 states and two countries. He loves sports, especially hockey. He is obsessed with learning about weather, and he is a good student. He is a loyal friend and a fierce advocate for pediatric cancer research and disability rights. He has shaved his head five times for St. Baldrick’s, traveled to Washington D.C. twice to talk to legislators about the STAR Act and is a guest speaker for a disability awareness organization.

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Childhood Cancer

St. Baldrick’s Helps Researchers Envision a Bright Future for Kids with Retinoblastoma

by Erinn Jessop, St. Baldrick's Foundation
April 6, 2018

A child’s eyes see the love in their parents’ faces and the joy of their siblings. They see the vibrant colors in a box of crayons and the sparkle of rain on a flower. Sight helps kids navigate their classroom, their playground and their world. But what happens when pediatric cancer attacks that precious sense?

With retinoblastoma – an eye cancer in children that is usually diagnosed before the age of 3 years old – a kid can lose their vision to the cancer and their long-term health to the harsh treatment. They can even lose their lives.

That’s what tumor immunologist Dr. Vanessa Morales-Tirado and her St. Baldrick’s Summer Fellow, Zachary Goldsmith, are working to change.

Our Summer Fellow grants give students the chance to work in childhood cancer research labs with expert mentors. Think that’s pretty cool? Then learn more about St. Baldrick’s grants >

Dr. Morales-Tirado and Summer Fellow Zach

Dr. Vanessa Morales-Tirado, with the University of Tennessee, works with Zachary Goldsmith, a St. Baldrick’s Summer Fellow and PhD candidate, in the lab.

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Babies and Cancer [Q&A]

by Erin Breese, M.D.
August 11, 2015

Anyone can get cancer — even babies. Dr. Erin Breese, a St. Baldrick’s Fellow studying infant leukemia, explains the signs, symptoms and treatment of babies with cancer, and how research is helping pinpoint better therapies so babies with cancer can grow up to live long, healthy lives.

a baby's feet

Can babies get cancer?

Unfortunately, cancer can occur at any age including during infancy. According to recent statistics, roughly 23 of every 100,000 babies are diagnosed with cancer each year.

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Kids with Cancer

‘Miss Tough Enough’ Takes on Second Childhood Cancer Diagnosis

by Erinn Jessop, St. Baldrick's Foundation
October 7, 2014

Tacey Raye first battled cancer as a baby, losing her sight in the process. Now, years later, the high school freshman and Texas rodeo queen is facing yet another diagnosis. Help kids like Tacey. Donate today.

Tacey Raye fought retinoblastoma soon after she was born. Eight years after being declared cancer free, she's fighting again.

Tacey Raye fought retinoblastoma soon after she was born and had both eyes removed by the time she was in first grade. Eight years after being declared cancer free, she’s fighting childhood cancer again.

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Cancer Free, But Never Free From Cancer

by Katie Vescelus
September 8, 2014

2013 Ambassador Matthias was diagnosed with retinoblastoma, a type of childhood cancer, when he was just 3 months old. The treatment left him permanently blind, but five years later, he’s cancer free and starting kindergarten. His mom, Katie, shares this update.

Matthias and Magnus starting school

Matthias (left) and his older brother, Magnus, on the first day of school this year.

On July 13, 2009, I held our son, Matthias, as he recovered from surgery to remove his right eye. His left eye had been removed two weeks before.

This surgery allowed him to be cancer free for the first time in his life, but it left him permanently and irreversibly blind.

I lifted him from the recovery room bed as he slept, and the nurses helped me navigate the tangled web of tubes so we could settle into a rocking chair. I rocked and sang softly to him as he awoke for the first time to a new world, one in which he was healthy but completely blind.

On August 4, 2014, just five short years later, I hugged Matthias and held his hand as he boarded the bus for kindergarten. I kissed him goodbye and again guided him into a new world. It was almost as terrifying as the day he lost his vision.

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Seeing Christmas in a New Light

by Katie Vescelus
December 5, 2013

After childhood cancer took Matthias’ sight, Christmas changed forever.

Cancer changed us in many ways; sometimes I don’t even recognize our lives anymore. Many of these changes aren’t bad — we have a deeper respect for life, an appreciation of health, and a conscious enjoyment of every moment with loved ones.

Some of the changes were difficult, and we’ve accepted them. Even the way we celebrate holidays has changed.

I’ve always enjoyed Christmas, and having children makes this time of year even more magical. In 2008, we were excited to celebrate Matthias’ first Christmas, along with Magnus, who was 2 at the time, and just starting to participate in all of the excitement of the season.

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