A closer look at Dr. Elvin Wagenblast’s AML study

Leukemia is the most common type of cancer in children. Even before a child is born, genes can undergo changes that are linked to the development of leukemia. There is a specific type of leukemia called acute myeloid leukemia (AML) that has unique gene changes, including when two genes called NUP98 and NSD1 combine. About 15% of children with AML have this gene fusion, and it makes their prognosis worse. Sometimes, another gene, called WT1, also has changes in cases of AML where children experience chemotherapy-resistance. 

Doctors and scientists are not yet sure why this type of leukemia doesn’t respond well to treatment. In studies with mice, they observe mixed results when trying to recreate this leukemia. 

To understand this leukemia better and find new treatments, Dr. Elvin Wagenblast will create a special model that imitates the gene changes found in patients. His team is going to use different types of human blood cells to see how leukemia develops and if it can resist standard chemotherapy. They will study the genes and molecules involved and find ways to stop the leukemia from growing. The goal is to identify targets for new treatments that can be tested in more studies and help improve the lives of children with AML and their families. 

A Novel Technique: Dr. Wagenblast is working to create better treatments 

His team will use a special technique called CRISPR/Cas9 to study leukemia in the lab using normal blood cells. This will help him understand how the disease grows and changes over time. By trying new experiments, he hopes to find out why children get leukemia and develop new ways to treat it. 

Think of genes as a recipe book that tells your body how to grow and function. Sometimes, there may be mistakes in the recipe that cause health problems. CRISPR/Cas9 is a tool that allows scientists to edit the recipe book. It’s a new technology that has shown a lot of promise in helping us understand and potentially treat genetic diseases in the future. 

By using CRISPR/Cas9, scientists can remove or add specific pieces of the genetic code. This can help them study how genes work, find out which genes are linked to certain diseases, or even try to fix genetic disorders.  

Dr. Wagenblast’s work is supported by a CureSearch Young Investigator Award. With this support, he will try new and unique ways to figure out why leukemia happens in children. His ultimate goal is to make important progress in understanding and treating childhood leukemia, which will help many children with this disease.  

Your donation can help fund the next breakthrough in 2023 and beyond! Make a gift today and bring new treatments for the children who are counting on us.  

The post Research blog: Uncovering Why and How Pediatric Leukemia Develops  appeared first on CureSearch for Children's Cancer.

Dr. Patrick Grohar receives Fight Osteosarcoma Together Super Grant to address critical, unmet need in pediatric osteosarcoma treatment 

Bethesda, Md. – June 6, 2023 – CureSearch for Children’s Cancer announced today a three-year, $1.5 million grant to Patrick Grohar, MD, PhD, of Children’s Hospital of Philadelphia to develop a new targeted treatment approach for pediatric osteosarcoma, an aggressive form of bone cancer.  

The award – the Fight Osteosarcoma Together Super Grant – is a joint funding award with St. Baldrick’s Foundation, CureSearch, Battle Osteosarcoma, Michael and April Egge; The Osteosarcoma Collaborative, and the Zach Sobiech Osteosarcoma Fund of Children’s Cancer Research Fund. 

“I have dedicated my life to the care of patients with bone tumors,” said Dr. Grohar. “My team has a history of translating therapies to pediatric bone tumor patients, and are now seeing responses in the clinic. The work proposed has the potential to be highly impactful for the patients we serve. Our group is honored and humbled to have been selected for this award.”  

Treatments for osteosarcoma haven’t changed in 30 years, and few targeted agents have successfully translated to the clinic for patients. Dr. Grohar’s team will study the role of the MYC gene, a common gene in osteosarcoma tumors, and determine how MYC makes osteosarcoma aggressive.  

If a novel and effective MYC inhibitor is developed as a result of this project, it could potentially improve the treatment of many cancers, including osteosarcoma. 

“Real progress in childhood cancer research requires collaboration, so we are honored to be a funding partner on this award,” said Holly Zink, Director of Research and Programs at CureSearch. “Dr. Grohar’s project aligns with our laser focus on addressing critical, unmet need in pediatric cancer treatment. We look forward to seeing the impact of this groundbreaking research on pediatric osteosarcoma patients who are counting on novel treatment options.” 

To learn more about how you can support innovative projects like this and help advance childhood cancer research, visit curesearch.org/donate. 

About CureSearch for Children’s Cancer 
CureSearch for Children’s Cancer, a national nonprofit organization that works to end childhood cancer by driving targeted and innovative research with measurable results in an accelerated time frame. Using a unique funding strategy, CureSearch only funds translational research projects with the strongest potential to become a new treatment and quickly reach patients. To learn more visit curesearch.org. 

The post CureSearch and funding partners award $1.5M for osteosarcoma research appeared first on CureSearch for Children's Cancer.