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March 2017 Affiliated with Columbia University Medical Center and Weill Cornell Medicine
Medulloblastoma Mouse Model May Help Reduce Treatment-Related Toxicity, Minimize Risk for Pediatric Brain Tumor Recurrence

A novel mouse model for studying brain tumors in mice eventually may help researchers pinpoint the molecular features of cancer cells remaining after therapy and identify ways to decrease the risk for cancer recurrence in pediatric patients.

Praveen Raju, MD, PhD, Assistant Professor of Pediatrics in the Division of Child Neurology at Weill Cornell Medicine and Attending Pediatrician at the Komansky Center for Children’s Health at NewYork-Presbyterian/Weill Cornell Medical Center, has spent over five years developing a unique mouse model of medulloblastoma in order to identify and eliminate treatment-resistant tumor cells as a means to minimize or prevent cancer relapse, as well as to find novel therapies for pediatric patients with recurring disease who are often drug-resistant.

“We hope that utilizing the sophisticated medulloblastoma mouse model that we have developed, we can make the bench-to-bedside process more efficient for identifying the most promising candidate drugs or treatment approaches to advance to clinical trials in children with medulloblastoma,” Dr. Raju said.

The leading cause of all cancer-related deaths in children in the United States is brain tumors. About 15% of the nearly 4,500 children who are diagnosed with brain tumors every year have malignant medulloblastoma. Unfortunately, one-third of these patients die within five years of diagnosis despite aggressive therapy, according to the American Brain Tumor Association.

Dr. Raju said a major hurdle that he and his colleagues encounter in the treatment of medulloblastoma is that the toxicity of current therapies including surgery, chemotherapy and radiation therapy may cause pediatric patients to experience neurocognitive deficits, growth and endocrine adverse effects, and other significant long-term consequences that impair their quality of life.

With this in mind, Dr. Raju and his colleagues developed the medulloblastoma mouse model to identify new treatment strategies for pediatric patients that will improve overall survival outcomes, yet minimize treatment-related toxicities. The model displays several key clinical features including diverse tumor histologies and metastases frequently found in patients, but rarely found in mouse medulloblastoma models to date. Importantly, the model allows the identification of medulloblastoma cancer cells at single-cell resolution so he can identify drugs or therapies that hopefully will target all the tumor cells and not just the majority of them.

Dr. Raju and his team are currently designing drug treatment studies to test if the model’s sophisticated drug-testing approach will translate successfully to clinical trials in patients. His main goal is to detect cancer recurrence earlier using noninvasive techniques, find out why certain cancer cells are resistant to therapies, and make the bench-to-bedside process more efficient for identifying promising drugs or treatments to advance to clinical trials in children with medulloblastoma.

The innovative research by Dr. Raju on pediatric patients with brain cancer has been recognized by The Hartwell Foundation, St. Baldrick’s Foundation for Childhood Cancer, Unravel Pediatric Cancer Foundation, American Brain Tumor Association and Hyundai Hope on Wheels Foundation. In addition, he and his colleague David Lyden, MD, PhD, recently were awarded a Starr Cancer Consortium grant as well as an Alex’s Lemonade Stand Foundation Innovation Award.

“Research such as ours takes time, but we hope that with additional resources and research support that we can advance this approach to improve the lives of children afflicted with this devastating disease,” Dr. Raju said.

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