Research

We call these “infrastructure grants” because they provide support for programs and personnel that are necessary to open and coordinate clinical trials. (No, we’re not building roads and bridges here!).

Clinical trials are vital to getting drugs and new treatments approved, resulting in access to effective medications for children with cancer. In 2021, the FDA used Children’s Oncology Group clinical trial data to authorize pediatric labeling indications for four chemotherapeutics, more than any prior year.

While vital, clinical trials are very labor intensive and require significant resources and staff and to ensure all children with cancer who are eligible for studies can be enrolled on these studies.

Most of these new grants support personnel to do the work to manage clinical trials – to enroll patients, manage data, and all the other “behind the scenes” work.

Research truly is a team effort, thank you for being part of it.

This $1.2 million in vital support goes to:

• Arizona  
  • Phoenix Children’s Hospital, Phoenix 

• California
  •  Loma Linda University, Loma Linda         
  • The Regents of the University of California, San Francisco, San Francisco                 
  • Valley Children’s Healthcare, Madera         
  • Children’s Hospital of Orange County, Orange         
  • The Regents of the University of California, Los Angeles 

• Connecticut 
  • Connecticut Children’s, Hartford 

• District of Columbia 
  • Children’s National Medical Center (WDC), Washington  

• Florida 
  • St. Joseph’s Children’s Hospital of Tampa, Tampa 

• Georgia 
  • Children’s Healthcare of Atlanta, Inc., Atlanta 

• Illinois 
  • Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago 

• Indiana  
  • Indiana University, Bloomington 

• Iowa 
  • Blank Children’s Cancer and Blood Disorders Center, Des Moines 

• Louisiana 
  • Ochsner Hospital for Children, New Orleans 
  • Children’s Hospital New Orleans, New Orleans 

• Michigan 
  • Children’s Hospital of Michigan Foundation, Detroit  

• Missouri 
  • SSM Health Cardinal Glennon Children’s Hospital, St. Louis 

• New Jersey 
  • St. Joseph’s Health, Paterson      

• New Mexico 
  • University of New Mexico HSC, Albuquerque 

• New York 
  • The Melodies Center for Childhood Cancer and Blood Disorders at the Bernard & Millie Duker Children’s Hospital at Albany Medical Center, Albany         
  • SUNY Upstate Medical University, Syracuse     

• North Carolina 
  • Wake Forest University Health Sciences, Winston-Salem 

• South Carolina 
  • Bon Secours St. Francis Health System, Greenville 

• Texas 
  • University of Texas Health Science Center at San Antonio (UT Health San Antonio), San Antonio 
  • El Paso Children’s Hospital Foundation, El Paso 

• Virginia 
  • Children’s Hospital of The King’s Daughters, Norfolk       

• Wisconsin 
  • HSHS St. Vincent Hospital, Green Bay         

Explore all the new grants to learn how your donations are helping make a lasting change in childhood cancer research, and helping children and teens of every background. And look for more grants soon!

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

Donate

Read more on the St. Baldrick’s blog:

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

This research is powered by you. This quarterly edition of the St. Baldrick’s Foundation Research Outcomes recognizes promising research to improve survival rates, provide holistic therapies, and translate adult drugs into pediatric trials.

Thank you for making research possible.

Reducing the Deaths from Infection after Bone Marrow Transplant

A bone marrow transplant can cure difficult to treat pediatric leukemia but has many short-term and long-term side effects. One major risk after transplant is viral infections, which can be hard to treat with available medicine, and can be deadly. St. Baldrick’s Fellow Dr. Jeremy Rubinstein and colleagues have had great success in tackling these viral infections by taking T-cells (a type of infection fighting cell that is part of the immune system) donated by children’s personalized stem cell donors and engineering them to attack and kill certain viruses.

Recently published results from their clinical trial offering this therapy as a preventative measure suggest that this treatment has been very safe and effective. Next steps include a randomized trial comparing this approach to the standard of care to see which is best at preventing and limiting the complications of viral infections. This clinical trial has the potential to decrease the number of pediatric cancer survivors who die from infection while also shortening hospitalizations and decreasing the need for other anti-viral medications.

This research was generously supported by the Rally for Ryan Fund, a St. Baldrick’s Hero Fund.

Translating Adult Drugs to Treat Incurable Brain Tumors

St. Baldrick’s Fellow Dr. Nathan Dahl uses genetic screening to identify new therapeutic targets in diffuse midline gliomas (DMGs), childhood brain tumors that are currently incurable. He found that CDK9 inhibitors, a class of drugs already moving forward in adult clinical trials, are a potential new treatment.

With St. Baldrick’s support, Dr. Dahl found that these inhibitors effectively treated models of DMG with minimal toxic side effects. This identified a new class of effective drugs ready for rapid translation into pediatric trials for this devastating disease. This discovery has now formed the basis for a phase 1 clinical trial, providing hope for future patients.

This research was supported by the Kids Shouldn’t Have Cancer Foundation, a St. Baldrick’s non-profit partner.

A Holistic Approach to Childhood Cancer

With St. Baldrick’s support, Dr. Ashraf Mohamed was able to bring Integrative Oncology to Cook Children’s Medical Center in Ft. Worth, Texas. Integrative Oncology brings specialists together and provides complimentary therapies for pediatric cancer patients. These evidence-based practices include interventions like animal-assisted therapy, rehabilitative services, psycho-education, psychosocial screenings, behavioral health strategies, creative art therapies, chaplain services, safe herbs/supplements program, and pain management strategies.

Information collected from psychosocial screenings will show staff trends in psychosocial distress which will aid in patient diagnosis and treatment decisions, and recommendations for the appropriate complementary therapeutic interventions. Next steps include expanding the psychosocial screenings to caregiver screenings which will meet all the same goals as the patient and aid in a family centered intervention. With the use of a caregiver distress screening, it will ensure that psychosocial risk is adequately assessed, evaluated, and strategic steps are taken to mitigate psychosocial harm to the family.

More Pediatric FDA Approvals as a Result of Clinical Trial Data

When St. Baldrick’s makes a grant to the Children’s Oncology Group (COG) these funds help to open and maintain lifesaving clinical trials. In 2021, the FDA used COG clinical trial data to authorize pediatric labeling indications for four chemotherapeutics, more than any prior year. These pediatric labeling indications demonstrate the high data quality and reliability that COG can achieve. More importantly, these studies were pivotal to approvals which allow broad access to effective medications for children with cancer.

Not every publication or outcome 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: Your Dollars at Work
• Research Outcomes: Incredible Impact and Hope
• Research Outcomes: Novel Discoveries

EXCITING UPDATE November 1, 2022: The largest academic collaboration of its kind was announced last month, and it builds off this St. Baldrick’s supported work. The Broad Institute of MIT and Harvard, Dana-Farber Cancer Institute and St. Jude Children’s Research Hospital will collaborate to transform and accelerate the identification of vulnerabilities in pediatric cancers and translate them into better treatments.

Dr. Stegmaier said, “The Pediatric Cancer Dependency Map provided our community with a treasure trove of new data. However, the next critical step was to validate candidate gene targets emerging from this project. The St. Baldrick’s Foundation Robert J Arceci Innovation Award provided the critical funding for us to demonstrate the power of the Pediatric Cancer Dependency Map. 

With St. Baldrick’s support, we validated new candidate therapeutic targets in childhood cancers, such as Ewing sarcoma and neuroblastoma, which have led to the launching of drug discovery efforts, and we have gained novel insights into the mechanistic underpinnings of these diseases.  We were able to show our community the power of the project. I think we are just at the tip of the iceberg.  We are all very excited about the promise of what is to come through this new large-scale collaboration.”

We are excited to see the impact this collaboration has in helping to conquer childhood cancer.

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.

Read more »

Of the many incredible things St. Baldrick’s donors have made possible for kids with cancer, one stands out to me like no other. The return on investment from this one grant has already far exceeded expectations, and each year its impact continues to grow. 

A Seed Planted

With the goal of training doctors from low- and middle-income countries, the first St. Baldrick’s International Scholar was awarded in 2013. But while Dr. Joseph Lubega of Uganda was in training at Baylor College of Medicine in Houston, he was also planting a seed: He set up the first pediatric oncology program for pediatricians in his home country. 

With St. Baldrick’s support, Dr. Lubega became one of the first trained pediatric oncologists in all of Uganda, where the need is great – as it is across Africa.

And the ability for pediatricians in the region to specialize in childhood cancer without leaving Africa was a game-changer.

The first four doctors graduated from the program at Makerere University in Kampala, Uganda in August 2018. Including Dr. Lubega, there were then five trained pediatric oncologists in Sub-Saharan Africa – with more following in their footsteps.

A Tree Grows: An Update

Dr. Lubega’s St. Baldrick’s funding ended in 2019, but he often sends updates, always thanking St. Baldrick’s donors.

In August 2022, he wrote after several visits with colleagues, accompanied by Dr. David Poplack, Director of Global Hematology Oncology Pediatric Excellence (HOPE), Texas Children’s Cancer Center. Dr. Lubega writes:

Over the last couple of weeks, we touched base with several of the pediatric cancer teams that are led by the fellows we trained, and we continue to support. It is mind blowing how much progress has been made! The passion! The compassion! The effort these teams are putting into improving things for these children is so inspiring.

I have attached some photos for you.

Dr. Joseph Lubega and 2023 St. Baldrick’s Ambassador Natasha at her 14th birthday party

Natasha, our St. Baldrick’s International Ambassador, is now over three years since she completed her therapy for acute myeloid leukemia, and we can confidently say she is cured, since AML very rarely relapses over three years later. More importantly, July 27 was her 14th birthday, and we hosted a birthday party for her in Kampala – you can see the city in the background.

Dr. Fred Lutwama, current St. Baldrick’s International Scholar (center) with Dr. David Poplack (left), and Dr. Joseph Lubega (right)

We touched base with Dr. Fred Lutwama, current St. Baldrick’s International Scholar. He is making lots of progress in building the molecular diagnostic capacity in Kampala.  

Ten of the pediatric oncologists transforming care for children fighting cancer in Africa

We visited Dar es Salaam in Tanzania where Dr. Lulu Chirande (next to Dr. Poplack) and Dr. Nana Nakiddu (extreme right), both graduates of our training program at Makerere University in Uganda, are now leading another training program. This one is at Muhimbili University (MUHAS) in Dar es Salaam, with a total of six fellows: three in year 1 and three in year 2 (graduating this year). Dr. Fat-Hiya Al-Oufy, second right, will be the first pediatric oncologist in Zanzibar.

Five more pediatric cancer leaders with Drs. Lubega and Poplack

The training program in Uganda at Makerere University College of Health Sciences is thriving. Dr. Ruth Namazzi (far left), one of our very first graduates there, is the Fellowship Director. Dr. Joseph Gore (front center) is one of the fellows and will be the first pediatric oncologist in South Sudan.

The St. Baldrick’s mustard seed is now a massive tree!

Branching Out Across Africa

Twenty people have now graduated from the program in Uganda, and they are leading pediatric cancer care and research at nine different centers across Africa. These pediatricians are now seeing more than 2,500 new children each year, in Uganda, Kenya, Tanzania, Malawi, and Botswana. Natasha is just one of them.

And it all started with one grant, made possible by St. Baldrick’s donors. Thank you.

Donate in honor of Natasha and others fighting cancer in Africa

Donate

Read more on the St. Baldrick’s blog:

• New Pediatric Oncology Training Program Bridges the Gap for Kids in Africa
• Commemorating International Childhood Cancer Day
• International Scholar in Uganda Gives Kids With Cancer Hope [VIDEO]

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 

Donate

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:

Read more »

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:

Read more »