Make an Impact this Childhood Cancer Awareness Month
As Childhood Cancer Awareness Month approaches this September, we are focused not only on many battles for progress, but on winning the war against all forms of cancers that attack babies, children, teens and young adults.
Thanks to St. Baldrick’s supporters, researchers are making advancements and discoveries daily. And the work of our advocates has also changed the game, resulting in the National Cancer Institute more than doubling its investment in childhood cancer research.
But now is the time to take more action, push forward and use the momentum we have to reach our vision of living in a world where kids no longer die of cancer, and survivors live longer, healthier lives.
As a volunteer and donor powered charity, every bit of progress St. Baldrick’s makes possible is a direct result of individuals like YOU.
This year, for Childhood Cancer Awareness month, our goal is to raise $1.5 million dollars. This money will save lives, starting with the next round of St. Baldrick’s grants this October.
Here are some ways you can get involved for Childhood Cancer Awareness Month. It’s time to take action and prove that we can #conquerkidscancer!
Advocate
Childhood Cancer Awareness Month is also an exciting time for advocacy. Throughout the month of September, advocates across the country will be speaking up for kids with cancer. Here are a few upcoming advocacy opportunities in September:
- Tuesday, September 13: Members of the Alliance for Childhood Cancer, which St. Baldrick’s co-chairs, are heading to Capitol Hill to advocate for the Childhood Cancer STAR Reauthorization Act. Join the St. Baldrick’s Speak Up advocacy network to receive updates on how YOU can amplify their advocacy!
- Thursday, September 22: The Childhood Cancer Caucus is holding its annual Summit to discuss policies impacting the childhood cancer community.
- Friday, September 23: The White House is holding a Childhood Cancer Forum, which you can livestream from home. Sign up here for more information on how to participate in the Forum virtually.
Get Social and Start a Fundraiser
Today, there are 4.7 billion social media users around the world! How great it is that so many of us are connected. That’s why this September, you are encouraged to create your own Facebook fundraiser during Childhood Cancer Awareness Month – to rally your network about your passion to support lifesaving childhood cancer research through St. Baldrick’s.
We’ve got a Toolkit full of instructions and goodies for everyone thinking of hosting a Facebook Fundraiser. Click here to access it.
Head-shaving
Every St. Baldrick’s event starts with the passion of a single volunteer. Bring your community together to raise funds for childhood cancer research by organizing a signature head-shaving event. As a volunteer event organizer, you’ll work directly with foundation staff to ensure your event is not only a great time, but a success, to help kids with cancer.
If you’re not ready to organize an event but still want to participate, that’s terrific, too! Check your area for any upcoming events near you and sign up to shave or volunteer. There are options for everyone!
To learn more about head-shaving events, click the link below!
St. Baldrick’s Head-shaving Events
September 12th is National Video Game Day
You can help #ConquerKidsCancer by hosting a charity stream on Tiltify for St. Baldrick’s. Click this link and join one of our “Current Fundraising Events” to help raise awareness and funds in your community.
We even have a Toolkit filled with some great info to help you promote your stream. For questions or to request the toolkit, contact Creates@StBaldricks.org.
Style With a Purpose
St. Baldrick’s is offering limited edition St. Baldrick’s “Research is Hope” shirts, but only until September 30. Raise awareness and support the mission in style!
A minimum of $10 per shirt will go to St. Baldrick’s Foundation for this limited time campaign, ending September 30, 2022.
Shop our Bonfire campaign here
Coworkers Unite!
September is a great month to rally your co-workers and host an employee giving campaign or engage your team in a company-wide fundraiser. Psst, we have loads of ideas. Let’s talk!
Corporate.Partnerships@StBaldricks.org
Give Shoppers a Choice
Online and in-store retailers, we have a turnkey solution for you to offer shoppers a way to give at checkout. Customers will have an opportunity to help conquer kids’ cancer by giving a donation after their purchase, making their shopping experience more meaningful.
Contact Corporate.Partnerships@StBaldricks.org for more information.
Childhood Cancer Awareness Month is the ideal time to join forces in the battle to give kids a lifetime by conquering childhood cancer. Taking part in one or more of the above items is a way to demonstrate that everyone can make an impact, together.
Read more on the St. Baldrick’s blog:
Research Outcomes: Your Dollars at Work
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
Read more on the St. Baldrick’s blog:
Announcing St. Baldrick’s Foundation July 2022 Grants
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.
- University of Alabama at Birmingham, Birmingham, Ala.
- 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.
- University of California San Diego, San Diego, Calif.
- 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
- University of Colorado Anschutz Medical Campus, Aurora, Colo.
- Florida
- University of Florida, Gainesville, Fla.
- St. Baldrick’s Scholar (continuing): Lan Hoang-Minh Ph.D.
- University of Florida, Gainesville, Fla.
- Georgia
- Emory University School of Medicine Department of Pediatrics and Children’s Healthcare of Atlanta, Atlanta, Ga.
- Illinois
- University of Chicago, Chicago, Ill.
- Pediatric Cancer Data Commons: Samuel Volchenboum M.D., Ph.D., M.S.
- University of Chicago, Chicago, Ill.
- 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.
- Massachusetts General Hospital (Mass Gen), Boston, Mass.
- 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.
- Johns Hopkins University School of Medicine, Baltimore, Md.
- 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.
- University of Minnesota School of Public Health, Minneapolis, Minn.
- New York
- Weill Cornell Medicine, New York, N.Y.
- St. Baldrick’s Scholar: Claire Vanpouille-Box, Ph.D.
- Weill Cornell Medicine, New York, N.Y.
- 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.
- The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio
- Ontario, Canada
- The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
- International Scholar (continuing): Anirban Das D.M., M.D.
- The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
- 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
- St. Jude Children’s Research Hospital, Memphis, Tenn.
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
Read more on the St. Baldrick’s blog:
Childhood Cancer: A parent’s role, finding a voice, and a path through advocacy
This was written by Abby’s mom, Patty Furco.
Cancer can make you feel helpless. You want to fix things for your child and you can’t. During and after treatment, parents have little control. Yes, we can advocate for our kids, find the best medical team, the best treatments, hospital, and love on them. But how can we help?
I want to help others that are on the path we lived on for years. Witnessing the devastation and disruption that childhood cancer families go through, it’s impossible to not be changed by what I’ve seen and experienced. My journey as a hands-on, living it day to day, childhood cancer caregiver is over. My incredible daughter Abby, forever 15, passed away last October after a valiant 11-year battle with cancer and its effects. However, my role in the childhood cancer world is not over. I need to help. I do not want other families going through things that we did. How can I (and we as a childhood cancer community) make things better for children and families? How do we create more survivors? Where do we go from here?
In 2012, we learned about the St. Baldrick’s Foundation funding childhood cancer-specific1 research and knew we wanted to join their efforts. Before we were part of the childhood cancer world, St. Baldrick’s had funded two trials that Abby had access to during her treatments – one drug helped her gain remission in 2011, and the other brought her out of heart failure in 2014/2015. Both of these drugs helped her live and love longer. We have fundraised, participated, and organized St. Baldrick’s Foundation events since.
We have been champions of childhood cancer research federal funding by visiting the halls of Congress in person and virtually over the years and by serving as a 2017 Ambassador family.
The St. Baldrick’s Foundation co-chairs a childhood cancer coalition called the Alliance for Childhood Cancer, which brings together other organizations and families to advocate for initiatives to help increase federal funding and federal childhood cancer support as a whole. We have introduced why childhood cancer-specific funding is needed and have secured support and funding for the Childhood Cancer STAR Act, the most comprehensive childhood cancer bill ever. This funding has created programs that help facilitate more research in childhood cancers and survivorship, ultimately in hopes to increase survivor rates and one day find cures.
Participating in these advocacy days has allowed me to meet other incredible families of both bereaved children and survivors. Not only are we able to support each other with a deep understanding, these meetings continue to fuel my fire to do more.
I was honored this year to be selected to nationally represent childhood cancer advocates and attend the American Association of Cancer Research (AACR) Scientist <-> Survivor Program. I joined 30 other patient advocates in person in New Orleans along with 20,000 researchers, doctors, medical representatives and overall just smart people. We learned so much as advocates and were exposed to exponentially more information about where cancer research is today and where it is headed in the future. Our program was founded, created, and facilitated by the incredible Dr. Anna Barker, past deputy director of the National Cancer Institute (NCI). Dr. Barker is a firm believer that a well-educated patient advocate role within cancer research is a pivotal piece of the puzzle. Our program was created to build us up, help us learn from one another and the thousands of educators at the conference, and then let us go out and do good things.
As a part of this program, I had the honor of presenting the St. Baldrick’s Foundation’s current action plan during a poster session and educating anyone that wanted to listen about the great things St. Baldrick’s does for children battling cancer.
But I am just one person. How can I be a bigger voice and spread the impact? I’m learning there isn’t one straightforward answer. The power of many is a key part of this. How can we as parents, family members or friends in the childhood cancer world help?
One way to help is by building support for the Childhood Cancer STAR Reauthorization Act. This bill will allow the programs from the STAR Act – including the investments in childhood cancer research – to continue for 5 more years. I invite you to click the link below, or text STAR4KIDS to 52886 to take action today.
For those of you in the trenches, I’ve got your back. You love on your child, you plan those appointments, scans, hospitalizations, medicines, treatments…do what you do each day. If there is a day that you have a moment to take a breath, do that first. Do something for yourself or other family members and friends. After that, if you feel the need for action, share your story. Take a moment to write or talk about your every day.
Even though I lived it every day for 11 years, I never quite grasped how powerful our stories can be. They are meaningful and can be helpful to others, not only in getting funding and sharing the importance of research, but also instilling hope.
We continue to fundraise, raise awareness, fund research, and love on our children, our families, other families in the battle, while we support one another. My story isn’t ending here, I’m still thinking, wondering, acting…what is next? How can I help even more? Stay tuned…
Click the link below, or text STAR4KIDS to 52886 to take action today
Ask Your Lawmakers to Cosponsor the STAR Reauthorization Act Today!
Read more on the St. Baldrick’s blog:
Q&A On Fertility After Adolescent or Young Adult Cancer
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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Advocacy Is Making a Difference
The childhood cancer community is filled with passionate, dedicated advocates rallying together to make a difference for kids with cancer and childhood cancer survivors. Thanks to the tremendous advocacy of the childhood cancer community, Congress, the Administration, and the National Cancer Institute (NCI) are taking notice.
Over the last five years, childhood cancer advocates have successfully lobbied for meaningful policies and changes. Back in 2018, Congress unanimously passed the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act, the most comprehensive childhood cancer legislation in history.
And the community’s advocacy didn’t stop there. Each year since the STAR Act was signed into law, Congress fully funded the programs STAR created, providing $30 million each year in new resources for childhood cancer. Now Congress is poised to extend the STAR Act further. Just last month, childhood cancer champions introduced H.R. 7630/S. 4120, the Childhood Cancer STAR Reauthorization Act to allow the programs from the STAR Act to continue for five more years.
On the heels of the success of the Childhood Cancer STAR Act, the Childhood Cancer Data Initiative (CCDI) was created in 2019 with the goal of funding $50 million in new childhood cancer research funding each year for ten years. As with the STAR Act, the childhood cancer community has successfully advocated for full funding for CCDI each year. That means Congress has provided $270 million in new resources for childhood cancer since the STAR Act and CCDI were created.
These accomplishments in advocacy are also changing the research landscape at NCI. For many years, the childhood cancer community has rallied around a particularly startling statistic: Less than 4% of the National Cancer Institute’s (NCI) budget funds childhood cancer research. This number so strikingly sums up the frustration about the lack of investment in treatments for kids with cancer and the slow pace of research. The 4% statistic has been a rallying cry for the childhood cancer community to demand better for kids with cancer – and after years of advocacy for more federal research funding, that 4% statistic has indeed changed. Thanks to the tremendous advocacy of St. Baldrick’s advocates and the childhood cancer community, NCI has more than doubled its investment in childhood cancer research in recent years.
The groundswell of advocacy and the investments foundations like St. Baldrick’s makes in early-career researchers and groundbreaking research have paved the way for more funding for childhood cancer research at NCI. In federal fiscal year 2020, NCI spent more than $500 million on childhood cancer for the first time, representing nearly 8% of its total budget that year.
But the work doesn’t stop here. The St. Baldrick’s Foundation will continue to keep up the pressure for more funding to develop new, safer therapies for kids with cancer. Join us today by urging your members of Congress to cosponsor the Childhood Cancer STAR Reauthorization Act, which will allow the programs – and funding – from the STAR Act to continue for five more years.
Click the link below, or text STAR4KIDS to 52886 to take action today.
Ask Your Lawmakers to Cosponsor the STAR Reauthorization Act Today!
Read more on the St. Baldrick’s blog:
Research Outcomes: Incredible Impact and Hope
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
Read more on the St. Baldrick’s blog:
Rolling Up Their Lab Coat Sleeves: The 2022 St. Baldrick’s Fellows
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:
2022 Ambassador – Meet Natasha, St. Baldrick’s First International Ambassador
Meet Natasha, a 13-year-old from Uganda, and St. Baldrick’s first International Ambassador.
Natasha in her school uniform.
Research Outcomes: Novel Discoveries
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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