Our St. Baldrick’s Foundation Research Outcomes blogs highlight examples of the progress your donations are supporting. This quarterly edition focuses on a new FDA approval for neuroblastoma, strategies to improve T cell functioning, understanding why some kids with medulloblastoma get sicker, and how secondary cancers increase the likelihood of future heart problems. Thank you for making this research possible.
Repurposed Drug Receives FDA Approval to treat Neuroblastoma
The U.S. Food and Drug Administration (FDA) has approved a drug to treat neuroblastoma, an often-fatal childhood cancer, based on pioneering research by Dr. André Bachmann. Dr. Bachmann’s early work that helped lead to this approval was supported by the St. Baldrick’s Foundation.
“It can take 15-20 years and large amounts of money to develop a new drug,” says Dr. Bachmann, “and I thought, why not try to repurpose a drug that is already used in the clinic for another disease?” With this in mind he looked at Difluoromethylornithine, or DFMO, which was developed in 1978 and later used to treat West African sleeping sickness.
Dr. Bachmann’s preclinical work demonstrated DFMO’s remarkable ability to stop cancer cell growth. Building on his findings, pediatric oncologists developed clinical trials that led to the December 2023 FDA approval. This achievement offers hope to kids battling neuroblastoma, providing a potentially life-saving treatment option.
Helping T Cells Do Their Job Better
In the body, there are special cells called T cells that help fight against tumors. These T cells need to be in good shape and able to do their job properly to fight cancer. St. Baldrick’s funded researcher, Dr. Alex Huang and colleagues have found a protein called Piezo1 that seems to help T cells work better. They wanted to see what happens when they remove Piezo1 from T cells and then expose them to cancer. They found that models without Piezo1 in their T cells developed more aggressive tumors and didn’t respond well to treatments that usually help the immune system fight cancer. This study suggests that Piezo1 helps T cells do their job better when fighting cancer. While it’s not feasible at the moment to target Piezo1 in T cells for medical purposes, this research provides important groundwork for researchers to investigate further the possibility of therapies that target Piezo1 specifically in T cells.
Dr. Huang’s research was supported through a partnership with the Osteosarcoma Collaborative.
Understanding ecDNA in Medulloblastoma
Circular extrachromosomal DNA (ecDNA) found in tumors plays a big role in making cancer cells grow, resist drugs, and make patients sicker. St. Baldrick’s Scholar, Dr. Lukas Chavez and colleagues studied tumors from kids with the brain tumor medulloblastoma. They found circular ecDNA in about 18% of samples. Patients with this ecDNA were more likely to get sicker again and more likely to die within 5 years after being diagnosed. This study shows how common and diverse ecDNA is in medulloblastoma and how it can make the cancer more dangerous. Further research will focus on understanding how ecDNA contributes to cancer progression and treatment resistance, so researchers can explore how to help these patients.
Dr. Chavez’s grant is named for Hannah’s Heroes, a St. Baldrick’s Foundation Hero Fund established to honor Hannah Meeson.
Secondary Cancers Increase Likelihood of Heart Problems
Childhood cancer survivors face a high risk of serious health issues as they age, such as second cancers and heart problems. St. Baldrick’s Scholar Dr. Hari Narayan is working to find ways to better determine which children will develop heart problems. He recently looked at data and found that survivors who developed a second cancer between 15 and 25 years after their initial diagnosis were more likely to experience heart problems later on.
Dr. Naryan’s findings suggest that factors like the treatments used for the second cancer, such as chemotherapy and radiation, could contribute to this increased risk. These findings highlight the importance of monitoring childhood cancer survivors for heart problems and considering ways to reduce their risk, such as adjusting treatment and promoting healthy lifestyles.
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:
The best gifts are the ones that make us smile and fill our hearts with joy. When we are lucky enough to receive such a gift, we find ourselves filled with gratitude.
Jonah is this kind of gift — to his family, to his friends and to those he meets every day. Despite having faced childhood cancer, he is, in his mum’s words, “the most kind, inclusive, loving, welcoming, forgiving heart while also being the hardest working person you’ll ever meet.”
It doesn’t seem to add up—Jonah has every reason to be discouraged, defeated and even angry because of the impact childhood cancer has had on his life. But when you meet this survivor, you can see that childhood cancer doesn’t define him and you can’t deny that he is a gift. This is Jonah’s story.
Jonah at the beach in 2021.
St. Baldrick’s Ambassadors represent the wide diversity of kids diagnosed with childhood cancers. Their stories highlight the importance of supporting the best childhood cancer research so all kids diagnosed can live long, healthy, productive and happy lives.
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).
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.
Scientific research continues at a great pace thanks to your tireless support. Pediatric cancer researchers proceed to make new discoveries and provide hope for children with cancer. See five examples of the many research outcomes you’ve made possible below:
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.
On Mother’s Day, we celebrate all moms, each special in her own way. Mothers of kids who have fought childhood cancer have traveled a journey no one would have chosen. May is also Brain Tumor Awareness Month. We asked Gaylene Meeson to share her story of being mom to a very special brain tumor survivor, Hannah.Gaylene Meeson and her daughter Hannah, survivor of an aggressive brain tumor called anaplastic meduloblastoma.
Photo by [Kenneth Lim, kennethlimphotography.com].
With a long history of support from the St. Baldrick’s Foundation, Dr. Eric Raabe of Johns Hopkins University is a “Rockstar Researcher” in pediatric brain tumors.
As an undergraduate student, Dr. Raabe volunteered at a children’s hospital where a pivotal moment influenced his decision to become a pediatric oncologist. He vividly remembers a young boy who had relapsed and was being hospitalized after having one of his lymph nodes biopsied. The boy sat alone in his room with the shades down. In the dark room the boy became more and more withdrawn as he sat and waited for the results. He thought he was going to die.
No sooner had the results come back negative for recurrence of his cancer, than the blinds went up and he wanted a pizza with everything on it. The experience left a lasting impression and prompted Dr. Raabe’s decision to become a pediatric physician scientist. In that moment he realized the impact he could make in a scared and sick child’s life. He decided then and there that he wanted to be part of providing a path to hope and a path to a cure. He wanted to help guide these children from the darkness to a place of hope and light.
What is medulloblastoma?
Medulloblastoma is the most common malignant brain tumor in children. It originates in the back part of the brain called the cerebellum. In up to 1/3 of cases, it can spread to other parts of the brain and spinal cord. Most cases are diagnosed before age 10.
Editor’s Note: Today on the St. Baldrick’s blog, we’ve decided to hand the microphone over to Dan Butler, whose son, Sullivan, was diagnosed with cancer in 2016, at the age of 10. June is Cancer Survivors Month at St. Baldrick’s, and the first Father’s Day after Sullivan’s diagnosis and treatment was especially meaningful for Dan.
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