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Massachusetts General Hospital

in collaboration with Duke University School of Medicine, The Hospital for Sick Children and the National Institutes of Health

CureSearch Acceleration Initiative Award: 2022-2025 

Focus: Rhabdomyosarcoma

Project title: Targeting p53 in childhood rhabdomyosarcoma

Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma of childhood and adolescence. This cancer forms in skeletal muscle tissue or sometimes hollow organs, such as the bladder or uterus, and can spread from where it started to other areas, including lungs and lymph nodes, making treatment and recovery more difficult. While it can occur at any age, it most often affects children and adolescents with more than 350 pediatric cases diagnosed annually in the U.S. Despite decades of study, the standard of care for RMS remains a chemotherapy regimen developed in the 1970s. Associated toxicities include bone marrow suppression, infection, liver damage, cardiotoxicity, and decreased kidney function. Long-term survival for high-risk RMS has remained less than 30% for over 40 years. 

David Langenau, PhD, is a CureSearch Acceleration Initiative Awardee conducting research at Massachusetts General Hospital. TP53 is a transcription factor, the mutation of which is thought to be critical to the growth of a wide variety of cancers. TP53 mutations are associated with poor outcome in several pediatric cancers including choroid plexus carcinoma, osteosarcoma, medulloblastoma and RMS. For decades academic and industry partners have been investigating the clinical and phenotypic consequences of TP53 mutations, and druggability of p53, largely in adult cancers. However, because it is a transcription factor, p53 has been difficult to pharmacologically target. Dr. Langenau and his colleagues will test novel therapeutics that reactivate p53 protein function for their ability to restore treatment sensitivity in RMS. By identifying p53 stabilizing therapies that synergize with chemo-and radiation therapies in p53-mutant RMS, they aim to develop a combination therapy that requires less chemotherapy and radiation, reducing treatment-related toxicities, and improving outcomes for patients with RMS. 

“We are grateful to CureSearch for funding this consortium grant focused on developing new understanding and cures for aggressive pediatric muscle cancers - commonly called rhabdomyosarcoma.”

- David Langenau, PhD

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