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

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Research

Research Outcomes: Novel Discoveries

by St. Baldrick's Foundation
February 15, 2022

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:

Lab Equipment with text: Research Outcomes

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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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Real-Life Stories

Being the Mom of a Hero Named Hannah

by St. Baldrick's Foundation
May 7, 2021

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 HannahGaylene Meeson and her daughter Hannah, survivor of an aggressive brain tumor called anaplastic meduloblastoma.
Photo by [Kenneth Lim, kennethlimphotography.com].

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Research

DR. ERIC RAABE VS. PEDIATRIC BRAIN TUMORS

by St. Baldrick's Foundation
July 20, 2020

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.

doctor in mask

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Facts

What Is Medulloblastoma?

by Elias Sayour, M.D., Ph.D. and Agnes Petrosiute, M.D.
May 1, 2020

Dr. Sayour and Dr. Petrosiute are both St. Baldrick’s Scholars. This blog was written by Dr. Petrosiute in May 2014 and updated in April 2020 by Dr. Sayour.

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.

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Real-Life Stories

A Dad Shares His Son’s Cancer Story Part 1: Discovery and Treatment

by Dan Butler
May 31, 2019

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.

Dan reading to his son Sullivan in the hospital.

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Childhood Cancer

Why the Right Diagnosis Matters: Meet a Doctor Working to Get it Right and Hear How Avery Inspires Him

by St. Baldrick's Foundation
March 15, 2019

Brain tumors in children can sound especially daunting – but some brain tumors can be malignant and not especially life-threatening. Therefore, getting an accurate diagnosis is key, and Dr. James Olson of Seattle Children’s Hospital is working on more accurately diagnosing and treating brain cancers in children.

Dr. James Olson is photographed alongside Avery, one of his pediatric cancer patients.Dr. James Olson with Avery, one of his patients.

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Childhood Cancer

Meet the St. Baldrick’s 2019 Ambassadors!

by St. Baldrick's Foundation
January 7, 2019

Each year, the St. Baldrick’s Foundation picks five kids to serve as Ambassadors. In this role, they represent the thousands of kids affected by childhood cancers and remind us of the importance of supporting childhood cancer research.

Take any group of kids and they’ll all have their own way of talking, their own opinions on books, movies, and video games, their own favorite foods.

But there is one thing the St. Baldrick’s 2019 Ambassadors have in common: childhood cancers. Beyond that, they share the support of loving families and a desire to inspire others to raise money for childhood cancer research.

Images introducing the St. Baldrick's 2019 Ambassadors, including Aiden, Arianna, Sullivan, Gabby, and Brooke.

Our 2019 Ambassadors, from left to right: Aiden, Arianna, Sullivan, Gabby, and Brooke.

 

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Families

One Sibling Was Diagnosed with a Brain Tumor and Then the Unthinkable Happened

by Erinn Jessop
October 9, 2018

Kalea

Kalea and Noah snuggle in one hospital bed during treatment.

From waking up in the morning to getting tucked in at night, siblings Noah and Kalea were practically inseparable.

They’d eat their breakfast together – whatever 6-year-old Kalea had, 4-year-old Noah wanted too – and brush their teeth together. The two kids would get so immersed in playing together, and so quiet, that their parents, Duncan and Nohea, would get nervous and go check on them. Inevitably, the adults would interrupt some elaborate imaginary adventure and the kids would shoo them away.

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