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
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Each year, the St. Baldrick’s Foundation picks five kids to serve as Ambassadors. In this role, they represent the thousands of kids affected by childhood cancers and remind us of the importance of supporting childhood cancer research.
Take any group of kids and they’ll all have their own way of talking, their own opinions on books, movies, and video games, their own favorite foods.
But there is one thing the St. Baldrick’s 2019 Ambassadors have in common: childhood cancers. Beyond that, they share the support of loving families and a desire to inspire others to raise money for childhood cancer research.
Our 2019 Ambassadors, from left to right: Aiden, Arianna, Sullivan, Gabby, and Brooke.
With the holiday season upon us and another year drawing to a close, it’s a great time to reflect on some of the major research accomplishments of doctors and scientists whose work on childhood cancers benefited from the support of St. Baldrick’s donors like you.
There’s much to be thankful for. All things considered, 2018 was a remarkably successful year for childhood cancer research, with much of that success spurred on by grants funded by St. Baldrick’s. Of course, none of this would have been possible without our generous donors.
Dr. Kohanbash’s cutting-edge research on ependymomas is supported by a Hero Fund in memory of Henry Cermak, who passed away in 2008 after a long, 2-year fight that included many surgeries, chemo regimens, and 93 rounds of radiation.
St. Baldrick’s Fellow Dr. Elliot Stieglitz is a big reader, but not in the way that you might think. Over three years, he read the DNA of one hundred children with JMML, a rare leukemia, and he discovered something major. Read on to learn how his discovery could lead to better treatments for kids with this rare disease.
RESEARCH DEVELOPMENT: Dr. Elliot Stieglitz has brought his St. Baldrick’s-funded research to a Phase 2 clinical trial for kids with relapsed JMML. In this trial, which is first of its kind in the United States, researchers will be testing whether an oral targeted medication used in the treatment of melanoma in adults slows or even kills leukemia cells in kids with persistent JMML. In addition, Dr. Stieglitz developed a test that predicts which JMML patients have the best prognosis and therefore need less intense therapy. He’s now in the process of establishing a clinical test that will eventually be available to patients. This test will help kids get just the treatment they need and avoid damaging long-term effects from harsh therapies. Keep up the great work, Dr. Stieglitz!
For St. Baldrick’s Fellow Dr. Elliot Stieglitz, being a pediatric oncologist is the perfect blend of emotional satisfaction and intellectual stimulation.
His heart is with the kids and their families, guiding them through the toughest time in their lives. His head is in the lab, trying to find better treatments for childhood cancer.
Dr. Elliot Stieglitz is a St. Baldrick’s Fellow at the University of California, San Francisco. He’s researching ways to help kids with JMML who don’t respond to standard treatment. He explains JMML symptoms, treatment options, and how your support is moving research forward.
What is JMML?
Juvenile myelomonocytic leukemia (JMML) is a type of blood cancer that affects young children.
When Gary Crays found out his grandson, Ezra, had childhood cancer, he was devastated. Read Gary’s account of what it was like to witness Ezra’s childhood cancer journey and how St. Baldrick’s gave him a way to fight back.
Ezra was diagnosed with juvenile myelomonocytic leukemia (JMML) when he was 4 months old.
Denial. That’s what I felt when I first learned that my newborn grandson, Ezra, had abnormal blood counts.
His parents and his big brother were as healthy as could be, and his mother was careful to extremes during her pregnancy. I thought, “Whatever this is, it will quickly resolve itself.”
Since his birth four years ago, little Ezra has learned to love airplanes, chocolate milk, and his big brother. He’s also learned what it’s like to have childhood cancer. Read what Ezra’s mom, Elisabeth, has to say about his early diagnosis and how some bad news ended up saving Ezra’s life.
Ezra, now in remission, was diagnosed with juvenile myelomonocytic leukemia (JMML) when he was 4 months old.
On the morning of April 20, 2013, we welcomed our second son, our sweet, little Ezra. For a couple hours, all was well with the world.
We can’t remember the exact moment, or the exact words that first brought news of Ezra’s medical issues, but nothing could have prepared us for the frightening journey we embarked upon that day.
Caemon was a happy, inquisitive child, full of energy and love. Not even childhood cancer could change that. “He was a boy who was ready to live, and whether at home or at the children’s hospital, he was going to keep making meaning of his life,” writes his mom, Timaree. She tells his story below.
Photo courtesy In Her Image Photography
On a sunny afternoon in September 2009, my wife and I, a couple for 11 years, were transformed into a family with the birth of our son Caemon.
He looked nothing like we expected with his downy blonde hair and Pacific Ocean eyes, and in an instant, we knew that any expectations we had of this chubby little cherub had to be released. The only certainty was that after two years of trying so hard to make a family, our son was finally here. He was ours to cherish, and cherish him we did.