While treatments like CAR T cell immunotherapy have changed the landscape of treatment for kids with some types of childhood leukemias, researchers have had less success translating immunotherapy to solid tumors. Alex’s Lemonade Stand Foundation (ALSF) Young Investigator grantee, Adam Wolpaw, MD, at Children’s Hospital of Philadelphia, set out to understand why and see how he could translate those insights into treatments for children with neuroblastoma.
When Edie Gilger was 2 years old, frontline treatment for neuroblastoma failed. Her family was faced with a choice: more toxic treatments that might not work and if they did, they’d leave Edie with myriad long-term side effects, or try an experimental targeted treatment, funded by Alex’s Lemonade Stand Foundation.
Her family choose the latter.
Edie turns 13 years old this June, celebrating over 10 years of survivorship. She is doing it without the long-term side effects that years of high-dose chemotherapy and radiation can bring.
All thanks to Alex Scott.
But the story doesn’t stop with Edie.
Oncology researcher Dr. Meenaskshi Hegde, from Texas Children’s Hospital is working to change the outcome for kids facing ependymoma and to ensure that treatments are available at several institutions. Using ALSF Center of Excellence (COE) grant funding, Dr. Hegde is leading a Phase 1 study of CAR T cells for patients with refractory or relapsed ependymoma. The study, which opened in early 2022 at two children’s hospitals, will give patients access to treatment closer to home. Local approval is also underway at four more hospitals.
The National Institutes of Health define rare diseases as conditions that affect fewer than 200,000 people. But for families facing one of these diseases, like Beckwith-Wiedemann Syndrome (BWS), these diagnoses don’t feel rare. BWS affects approximately 1 in 10,000 kids and is linked to increased risk of certain childhood cancers, including Wilms tumor and hepatoblastoma. With proper treatment and monitoring, these cancers are treatable, which makes it critical to properly identify kids with BWS. Jennifer Kalish, MD, PhD, a pediatric geneticist at Children’s Hospital of Philadelphia, has focused her career on studying rare diseases like BWS.
As a young trainee, many people gave Dr. Michelle Monje, Alex’s Lemonade Stand Foundation (ALSF) grantee from Stanford University the (unsolicited) advice that one cannot have a big career in medicine or science and also have children.
She ignored the advice.
Dr. Monje (mother of four) was just awarded a research grant from ALSF. This grant is a $1 million commitment over two years to study CAR T cell immunotherapy for spinal cord diffuse midline gliomas in a Phase 1 clinical trial. Diffuse midline gliomas are inoperable, lethal, high-grade central nervous system tumors primarily affecting children and young adults.
It is estimated that 400,000 children under the age of 20 are diagnosed with cancer each year. However, there are most likely more cases of childhood cancer that go unreported. Not every country has a universal childhood cancer registry or a public health protocol for tracking cases. And in poorer countries, diagnosis and treatment delays not only limit the knowledge of cases but limit long-term survival for these children. In high-income countries, 80% of children diagnosed with cancer are cured and in some low and middle-income countries, only 20% of children survive. There is work to be done to help these children, and together, we can do it by investing in collaborative research. Childhood cancer researchers, funded by Alex’s Lemonade Stand Foundation (ALSF), work together on innovative, cutting-edge projects aimed at finding safer treatments and cures for all children facing cancer. Here’s how research works to find cures for all children:
While the pandemic continues to bring uncertainty in the world, Alex’s Lemonade Stand Foundation (ALSF) funded researchers are continuing to push ahead and make strides in the search for safer treatments and cures for all children. Fueled by ALSF funded research grants, these researchers utilize collaboration, innovation and cutting-edge science in their labs all around the world. These researchers are aiming to not just level the playing field — but to give children what they need: cures and safer treatments. Meet a few of these researchers to watch in 2022:
At 3-and-a-half years old, Sam's parents found a large lump on Sam's abdomen. He underwent surgery to remove it and was cancer-free for a short time. When the cancer returned, it was treated like stage 4 neuroblastoma. Sam has been cancer-free since 2017!
