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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Real-Life Stories

One Last Christmas: A Mother Shares Her Son’s Cancer Story

by Erica Bonner, Aiden's Mom
December 4, 2019

My name is Erica, and I never imagined that I would have a childhood cancer story to tell.

I’ll never forget the moment I heard the words “your son has cancer”—it hit our family like a ton of bricks. Now I’m sharing my son Aiden’s story because I believe in the critical need to support childhood cancer research with the St. Baldrick’s Foundation. And this holiday season, you can join me in giving hope to the next child faced with a cancer diagnosis.

First, cancer took away my little boy’s smile. Then, it took his life.

Boys on a swingAiden on the right (in the green shirt), on the swing with his brother Evan, before his diagnosis. That smile sums him up–grinning, being Aiden, just loving life.

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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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Childhood Cancer

Childhood Cancer Research You Helped Fund in 2018

by St. Baldrick's Foundation
November 29, 2018

With the holiday season upon us and another year drawing to a close, it’s a great time to reflect on some of the major research accomplishments of doctors and scientists whose work on childhood cancers benefited from the support of St. Baldrick’s donors like you.

There’s much to be thankful for. All things considered, 2018 was a remarkably successful year for childhood cancer research, with much of that success spurred on by grants funded by St. Baldrick’s. Of course, none of this would have been possible without our generous donors.

Dr. Kohanbash’s cutting-edge research on ependymomas is supported by a Hero Fund in memory of Henry Cermak, who passed away in 2008 after a long, 2-year fight that included many surgeries, chemo regimens, and 93 rounds of radiation.

Dr. Kohanbash’s cutting-edge research on ependymomas is supported by a Hero Fund in memory of Henry Cermak, who passed away in 2008 after a long, 2-year fight that included many surgeries, chemo regimens, and 93 rounds of radiation.

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Childhood Cancer

New Immunotherapy Breakthrough Could Bring Hope to Kids With DIPG

by Erinn Jessop, St. Baldrick's Foundation
September 21, 2018

Maddy

Honored Kid Madelyn was diagnosed with DIPG in 2010. A fashionista with a big spirit and an equally big heart, Maddy was an inspiration to those around her. She passed away in November 2011 after an 18-month battle with DIPG.

No child has ever survived a diffuse intrinsic pontine glioma brain tumor, commonly called DIPG.

This aggressive pediatric brain cancer multiplies in the brainstem, which controls some of our most basic functions for living – our breathing, heart rate, blood pressure and ability to swallow and speak.  And because the cancer is so intertwined with its delicate surroundings, DIPG is often inoperable.

A DIPG diagnosis and a zero percent survival rate has remained the reality for kids and families for decades.

But what if there might be hope? What if that hope came from within the child’s own body?

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Research

An Injection of Hope: Researcher Studies Innovative Potential Therapy for DIPG

by Erinn Jessop, St. Baldrick's Foundation
June 18, 2018

For kids diagnosed with a rare and fatal type of brain tumor called DIPG, or diffuse intrinsic pontine glioma, there is no cure and treatments are heartbreakingly scarce. St. Baldrick’s researcher Dr. Mark Souweidane is on a mission to change the bleak statistics on DIPG survival. Learn about his groundbreaking work so far and what’s coming next.

BREAKING NEWS: The promising results of Dr. Souweidane’s groundbreaking research have just been published in the peer-reviewed journal Lancet Oncology! Supported by St. Baldrick’s, this Phase 1 clinical trial involved the injection of a cancer-fighting drug directly into the tumors of children with DIPG. There were exciting results — no serious side effects or dose-limiting toxicities were observed in the kids who participated, which means that the therapy has been deemed safe for use in pediatric patients. Thanks to St. Baldrick’s support, this promising trial will now expand to multiple institutions, giving hope to kids with this currently incurable, fatal tumor and to their families.

Dr. Mark Souweidane

DIPG life expectancy is devastatingly short — with many kids dying within two years of diagnosis. Dr. Mark Souweidane wants to change that.

For kids with DIPG, treatment with radiation just lets them live a little while longer. Traditional chemo doesn’t work because of the blood-brain barrier. Tumor removal with surgery is out of the question, because the cancer is intertwined with the delicate tissues of the brainstem, which regulates breathing and other vital functions.

So, what does a doctor working on DIPG do to help these kids?

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Childhood Cancer

St. Baldrick’s Top 10 Highlights of 2017

by Erinn Jessop, St. Baldrick's Foundation
January 8, 2018

2017 was a big year, full of breakthroughs, incredible stories and some pretty amazing achievements in the childhood cancer world. Join us as we reflect on St. Baldrick’s top 10 highlights of the past year — and make sure you give yourself a pat on the back, because much of this was possible because of YOU!

St. Baldrick's 2017 Highlights

Are you ready to take a trip down memory lane? Here we go …

1) Passage of the RACE Act

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Families

Researcher Works to Crack the DIPG Code with Help from McKenna Claire

by Erinn Jessop, St. Baldrick's Foundation
December 15, 2017

McKenna Claire was 7 years old when she was diagnosed with DIPG

McKenna Claire was 7 years old when she was diagnosed with a rare brain tumor called DIPG. The McKenna Claire Foundation was established in her memory and in 2013, St. Baldrick’s partnered with the McKenna Claire Foundation to fund DIPG research, like the work done by Dr. Rameen Beroukhim at the Dana Farber Cancer Institute.

Honored Kid McKenna Claire was bright, spirited, and loved soccer and gymnastics. McKenna was full of grace, joy and grit through it all, even as her childhood cancer progressed and she could no longer run across a soccer field, jump on a trampoline, talk or swallow. She died just six months after her diagnosis with a rare, fatal type of brain tumor called DIPG  – weeks before her birthday. She would have been 8 years old.

Learn more about McKenna and her cancer journey from her mom, Kristine >

Stories like this are why St. Baldrick’s researcher Dr. Rameen Beroukhim studies DIPG, otherwise known as diffuse intrinsic pontine glioma. In fact, McKenna’s photograph hangs in his lab.

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Facts

What Is Diffuse Intrinsic Pontine Glioma (DIPG)?

by Adam Green, M.D.
September 22, 2017

What is DIPG
Dr. Green is a St. Baldrick’s Scholar at the University of Colorado. He explains DIPG symptoms, treatment, and how research is helping kids with this type of childhood cancer.

What is DIPG?

DIPG stands for diffuse intrinsic pontine glioma. It is a type of high-grade glioma, a brain tumor that comes from cells called glia that surround, protect, and otherwise support the nerve cells in the brain.

DIPG is always found in the brainstem. This part of the brain controls many basic functions like breathing and swallowing, as well as muscles that help with speech and eye movements.

It is most common in elementary school-aged children, but it can affect children of any age.

Learn more about childhood cancer >

About 250 kids in the U.S. are diagnosed with DIPG each year.

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Advocacy

What I Learned From My Daughter’s DIPG Diagnosis

by Kristine Wetzel
September 21, 2017

When Kristine’s daughter McKenna was diagnosed with a rare pediatric brain tumor that no child has ever survived, she learned there was no known cure because of a lack of funding for research. You can help — get involved.

McKenna Claire

McKenna was diagnosed with DIPG, a deadly brain tumor, when she was 7.

In January 2011, our healthy, active, intelligent 7-year-old daughter, McKenna, came down with what we thought to be a stomach virus. After a week of doctor visits, seeing her left eye begin to stray and her mouth begin to droop, we insisted on having a CT scan.

Childhood cancer was never even a consideration in our minds before that scan, but less than 24 hours and one MRI later, we found ourselves surrounded by doctors at the nurses’ station in the PICU waiting to hear the diagnosis.

It was in the midst of that chaos that we were told our daughter had diffuse intrinsic pontine glioma, or DIPG, an extremely rare pediatric brain tumor that typically strikes between the ages of 5 and 7, infiltrates the brain stem, and has a 0% survival rate.

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