Research

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

Donate

Read more on the St. Baldrick’s blog:

• Research Outcomes: Incredible Impact and Hope
• Research Outcomes: Novel Discoveries
• Research Outcomes: Advancing Research to Improve Treatment

St. Baldrick’s donors have just funded over $8.9 million to advance research to find cures for childhood cancers and give survivors long and healthy lives. This investment brings the total granted by St. Baldrick’s for research since 2005 to more than $322 million.

These 23 new grants include:

• 7 new St. Baldrick’s Scholars
• 9 current Scholars receiving continuing funding for another year of research
• 2 new International Scholars, one from Egypt and one from Jordan
• 1 current International Scholar from India receiving funding for another year of research

The remaining grants support the clinical trials of the Children’s Oncology Group and another year of funding for 2 team science (consortium) projects and for the Pediatric Cancer Data Commons.

The St. Baldrick’s Foundation supports lifesaving research throughout the world, awarding grants that focus on all major types of childhood cancers. Read on for more about just 3 of these new research projects. Explore the links at the bottom to view all the grants.

Understanding Radiotherapy and Pediatric Brain Tumors

St. Baldrick’s Scholar, Dr. Claire Vanpouille-Box, is advancing research into radiotherapy treatment for brain tumors. Recently scientists found that radiotherapy can activate the immune system against multiple tumors. However, the tumors of patients who undergo radiotherapy always regrow, which suggest that radiotherapy is not activating immunity against these tumors. Dr. Vanpouille-Box will work to understand why this is happening to develop strategies that will improve treatment for pediatric brain cancer patients.

Developing an Inexpensive and Accurate Cancer Detecting Tool

St. Baldrick’s International Scholar, Dr. Anirban Das is developing a new, inexpensive tool to identify children with a genetic variation that can lead to deadly cancers which may not respond to conventional treatments. Many older tests often fail to detect this genetic trait accurately. With this new tool, Dr. Das has found that these cancers may be more common than previously thought, and can also develop in adolescents and young adults. The tool also helps detect patients whose cancers do not respond to chemotherapy and radiation but could respond to immunotherapy. With this additional year of funding, Dr. Das is now expanding the use of his tool to identify additional patients and cancer types who may benefit from this approach. This is important especially for developing countries, where such cancers are more prevalent. The low cost, and the ability to diagnose the genetic condition from tumors directly, or even from saliva without additional blood tests, are distinct advantages in these resource limited settings.  

Advancing Research into Rare Cancers like Histiocytosis

The St. Baldrick’s Foundation has supported the North American Consortium for Histiocytosis (NACHO) since 2014. Histiocytosis is a group of rare disorders, most common in children, in which there is an over-production of white blood cells known as histiocytes that can lead to organ damage and tumor formation. Before NACHO, there was little progress in knowledge and new therapy development for this wide variety of conditions. NACHO has 63 member institutions working together and with this additional year of funding they aim to keep growing so that children all over North America will have access to clinical trials that could save their lives. 

Thank you for supporting the best research, no matter where it takes place, to help all kids with cancer survive and thrive.

The full list of institutions receiving grants:

• Alabama
  • University of Alabama at Birmingham, Birmingham, Ala.
    • St. Baldrick’s Scholar (continuing): Emily Johnston, M.D.
• California
  • University of California San Diego, San Diego, Calif.
    • St. Baldrick’s Scholar (continuing): Lukas Chavez, Ph.D.
    • St. Baldrick’s International Scholar: Jehad Almaliti, Ph.D.
• Colorado
  • University of Colorado Anschutz Medical Campus, Aurora, Colo.
    • St. Baldrick’s Scholar: Kelly Faulk, M.D.
    • St. Baldrick’s International Scholar: Eman Elsabbagh, M.D., MSc
• Florida
  • University of Florida, Gainesville, Fla.
    • St. Baldrick’s Scholar (continuing): Lan Hoang-Minh Ph.D.
• Georgia
  • Emory University School of Medicine Department of Pediatrics and Children’s Healthcare of Atlanta, Atlanta, Ga.
    • Consortium Research Grants
• Illinois
  • University of Chicago, Chicago, Ill.
    • Pediatric Cancer Data Commons: Samuel Volchenboum M.D., Ph.D., M.S.
• Massachusetts
  • Massachusetts General Hospital (Mass Gen), Boston, Mass.
    • St. Baldrick’s Scholar: David Sykes, M.D., Ph.D.
  • Dana-Farber Cancer Institute, Boston, Mass.
    • St. Baldrick’s Scholar: John Prensner, M.D., Ph.D.
  • Boston Children’s Hospital, Boston, Mass.
    • St. Baldrick’s Scholar (continuing): Yangming Ou, Ph.D.
• Maryland
  • Johns Hopkins University School of Medicine, Baltimore, Md.
    • St. Baldrick’s Scholar (continuing): Challice Bonifant M.D., Ph.D.
  • National Cancer Institute, Center for Cancer Research, Pediatric Oncology Branch, Bethesda, Md.
    • St. Baldrick’s Scholar (honor award, no funding associated with this grant): Rosa Nguyen, M.D., Ph.D.
• Minnesota
  • University of Minnesota School of Public Health, Minneapolis, Minn.
    • St. Baldrick’s Scholar: Tianzhong Yang, Ph.D.
    • St. Baldrick’s Scholar (continuing): Mark Osborn, Ph.D.
• New York
  • Weill Cornell Medicine, New York, N.Y.
    • St. Baldrick’s Scholar: Claire Vanpouille-Box, Ph.D.
• Ohio
  • The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio
    • St. Baldrick’s Scholar: Margot Lazow, M.D.
    • St. Baldrick’s Scholar (continuing): Benjamin Stanton, Ph.D.
• Ontario, Canada
  • The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
    • International Scholar (continuing): Anirban Das D.M., M.D.
• Tennessee
  • St. Jude Children’s Research Hospital, Memphis, Tenn.
    • St. Baldrick’s Scholar (continuing): Kelsey Bertrand, M.B.B.S.
    • St. Baldrick’s Scholar (continuing): Paulina Velasquez, M.D.
    • Consortium Research Grants

The next set of grants will be announced in November, supported by donations between now and October. Visit the St. Baldrick’s grants page to learn more about all the research you’re making possible.

Donate now and help support research into better treatments for kids with cancer

Donate

Read more on the St. Baldrick’s blog:

• Rolling Up Their Lab Coat Sleeves: The 2022 St. Baldrick’s Fellows
• Research Outcomes: Incredible Impact and Hope
• Announcing St. Baldrick’s July 2021 Grants

Each survivor’s risk of late effects of cancer treatment depends on their tumor, specific treatments, age, genetic makeup and other factors. Surgeries, chemotherapies, radiation, stem cell transplants and other treatments take a toll on the body – and sometimes the mind – in many ways. Some late effects make life more difficult; others are life-threatening.

Heart and lung problems are common, as are secondary cancers.

Other late effects can include hearing problems, hormonal imbalances, difficulty growing, mental health needs or cognitive deficiencies, bone density issues and easy bone fractures, fertility and reproductive problems, and more.

We asked St. Baldrick’s Scholar Dr. Hazel Nichols to tell us about some of the reproductive health issues faced by adolescent and young adult (AYA) cancer survivors:  

How does cancer treatment impact fertility?

Cancer treatments can potentially affect future fertility. For example, radiation therapy to or near the abdomen, pelvis, or spine can harm nearby reproductive organs. Radiation therapy to the brain can also damage the pituitary gland, which helps control the production of certain hormones needed for pregnancy.

Some types of chemotherapy can affect the ovaries, reducing the number of eggs and changing hormone levels. Having been treated for cancer during adolescence and young adulthood can also affect sexual health, body image, and financial stability during childbearing years.

Read about 2012 St. Baldrick’s Ambassador Sarah’s dreams of becoming a mom here 

Do patients or their families receive counseling on these options?

Counseling patients on the effects of cancer treatment on fertility and options for fertility preservation is recognized as a critical part of high-quality cancer care. National guidelines recommend fertility counseling for AYA patients before cancer treatment.

However, fertility counseling has been described as one of the most under prescribed and least implemented services in cancer care. More than half of AYA cancer survivors report needing more information for reproductive planning both before and after cancer treatment. This unmet need has been associated with lower emotional functioning and health-related quality of life.

How is your St. Baldrick’s supported research helping childhood cancer survivors?

Despite advances in fertility preservation options and recognition of fertility counseling as a part of high-quality cancer care, the incidence of post-diagnosis childbirth has remained stable for many years.

My research is working to understand what the needs and challenges are for accessing fertility-related services.

Specifically, I am examining AYA cancer survivors’ age, race, and rural residence in relation to using fertility preservation options. We hypothesized that fertility preservation will be more common at older ages and in more recent diagnosis years, and will be less common for AYAs with a rural residence or African American race. Our research helps identify barriers to use of fertility services to inform strategies to improve cancer care delivery.

We showed that, during 2004-2015, only 1.2% of female AYA cancer survivors froze eggs or embryos for fertility preservation after cancer diagnosis in North Carolina. Younger women were 6 times more likely to use fertility preservation than older women. Women who were Black or who lived in rural areas or had lower socioeconomic status or had children at diagnosis were less than half as likely to use fertility preservation. We believe these results highlight the barriers that that cost creates for accessing fertility preservation, and caution that women who have children already may less often receive fertility counseling around having additional children in the future.

June is National Cancer Survivor Month, a time to celebrate childhood cancer survivors – and to keep the focus on progress. St. Baldrick’s will continue to support research not only to find new cures, but better ones.

(2022). Disparities in fertility preservation use among adolescent and young adult women with cancer. Journal of cancer survivorship : research and practice, 10.1007/s11764-022-01187-y. Advance online publication. https://doi.org/10.1007/s11764-022-01187-y

Help kids to survive and thrive. Support research into better treatments for kids with cancer 

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Read more on the St. Baldrick’s blog:

• A Dad Shares His Son’s Cancer Story Part 2: Survivorship
• The Long and Winding Road to Survivorship
• Surviving Cancer – Through the Eyes of a 16-year-old

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.

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

Donate

Read more on the St. Baldrick’s blog:

• Research Outcomes: Novel Discoveries
• Research Outcomes: Advancing Research to Improve Treatment
• Research Outcomes: Progress and Hope

The next generation of childhood cancer researchers is rolling up their lab coat sleeves and doubling down on the fight to end childhood cancers.

Thanks to donors like you, these doctors will train with leaders in the field and launch new research projects to answer pressing questions in the quest to conquer childhood cancers.

Explore the new research you’re supporting:

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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:

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What was once just a concept is now a reality: Precision pediatric cancer care to improve treatment.

Researchers can now sequence all the genes in the genome in an individual patient’s cancer to find gene changes or targets, and then in some cases, identify drugs that match those targets to improve treatment. (A genome includes all the genes in a cell, in this case, a cancer cell.)

This has absolutely transformed how doctors treat children with cancer in some cases.

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What is osteosarcoma?

Osteosarcoma is the most common type of bone cancer in children. It often originates in the long bones of the body that include the thigh bone, the shinbone, or the bone that runs from the shoulder to the elbow, called the humerus.

While osteosarcoma frequently starts in a particular bone, it can potentially move to other sites in the body, such as the lungs and other bones. This movement, known as metastasis, often makes the disease more difficult to treat.

A type of childhood cancer, osteosarcoma is commonly found in children children, teens, and young adults between the ages of 10 and 30. Patients who are 10-20 years of age account for 60% of osteosarcoma cases.

You can help to find the best treatments for kids with cancer. Donate now to help them grow up and live long, healthy lives >

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Enrollment on a clinical trial is often a child’s best hope for a cure. And every child enrolled on a trial is helping kids in the future to have a better outcome.

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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.

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