Jakefest

Dr. Mary Beckerle of the Huntsman Cancer Institute began developing a novel epigenetic strategy to treat Ewing sarcoma. The project, completed in 2016, demonstrated so much potential for its anti-tumor effects that it was licensed to Salarius Pharmaceuticals, which is conducting a phase 1 trial in Ewing sarcoma. The trial will recruit up to 50 patients with Ewing sarcoma at clinical sites across the country. In addition, Salarius Pharmaceuticals is now expanding clinical trials into relapsed and refractory Ewing sarcoma and Ewing-related sarcomas, as well as myelodysplastic syndromes and chronic myelomonocytic leukemia, both cancers that can progress into acute myeloid leukemia.

In 2013, Dr. William Weiss of the University of California, San Francisco, led an international study to determine if drugs that reprogram the epigenome can improve outcomes for children with high-risk medulloblastoma. In doing so, Dr. Weiss and his team identified previously unknown targets in medulloblastoma, building a knowledgebase that will enable identification of new therapeutic options against epigenetic targets for medulloblastoma. During the course of his work, he was unable to identify a drug that crossed the blood-brain barrier, but a new CureSearch Young Investigator project that is targeting epigenetics in pediatric brain tumors with more modern therapies means that the discoveries made by Dr. Weiss' lab are continuing to make an impact on pediatric therapy development.

Dr. Andrew Kung of Memorial Sloan Kettering Cancer Center began a study in 2017 to identify new therapies for pediatric sarcoma. At the conclusion of his project, Dr. Kung’s lab has identified a pediatric Master Regulator (MR)-targeting therapy with significant efficacy in cell and animal models of Wilms tumor and rhabdoid tumor. As a result, they are currently negotiating a clinical trial proposal with the company that produces the drug. Ultimately, Dr. Kung aims to have this new therapy in clinical trials in 2022.

Dr. Jason Yustein of Baylor College of Medicine began a project in 2013 to explore the genetic changes that occur when cancer spreads. In doing so, he studied mice with rhabdomyosarcoma in order to track the development of the cancer, and identify the genes responsible for the disease’s progression and subsequent metastasis. Dr. Yustein is actively using the mouse models developed through his CureSearch-funded project to test novel therapies and immune contributions. In fact, in two 2021 publications, Dr. Yustein identifies new promising targets for treatment of high-risk rhabdomyosarcoma. He is using his models to move the new targeted therapies into preclinical testing.

In addition, Dr. Ranjit Bindra of Yale University began a CureSearch-funded project in 2017 to identify novel targeted agents for alveolar rhabdomyosarcoma. He created an automated drug screening process, which can be applied to many other pediatric cancers.

In 2018, Dr. Ranjit Bindra received additional funding from CureSearch to test a novel drug in pediatric patients with a specific subtype of glioma that makes it sensitive to chemotherapy by inhibiting the PARP gene that is involved in repairing DNA mutations. The clinical trial is now active at the University of California San Francisco, the Dana-Farber Cancer Institute, Washington University St. Louis, Johns Hopkins University, Oregon Health & Sciences University, St. Jude Children’s Hospital and Yale University. Dr. Bindra has treated his first six patients, three of which have received the first dose level of combination BGB-290 and temozolomide. Over the next six months, Dr. Bindra’s team will recruit additional patients for the second dose
cohort of the trial.

Additionally, CureSearch partnered with PRA Health Sciences and members of the Coalition Against Childhood Cancer to develop a White Paper about the RACE for Children Act that features an overview of the rapid speed to clinical trials of his new therapeutic regimen. This accelerated pace to the clinic was enabled by the screening platform developed by Dr. Bindra.