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Rhabdomyosarcoma (RMS)

Rhabdomyosarcoma research

Rhabdomyosarcoma (RMS) is a skeletal muscle cancer that can be found anywhere in the body, from the head to the limbs. It is a rare, though often fatal, disease that primarily affects children.

RMS is one of many types of sarcoma, cancers derived from connective tissues like muscle and fat. There are two different types of RMS, known as embryonal and alveolar. Embryonal RMS is found mainly in children under age five and has a five-year survival rate of about 40%. Alveolar RMS has a five-year survival rate of less than 20% and affects children of all ages.

RMS has challenged scientists and clinicians as they work to understand what causes the disease and how best to treat it. The disease is rare and the genomic landscape of RMS tumors is varied. Because of this, RMS is understudied. But recent research suggests that the disease holds great promise for a cure.

RMS research at CSHL is part of a larger effort called the Sarcoma Research Project. This highly targeted effort aims to understand what causes sarcomas and to identify new therapeutics to treat them. The initial phase of the project is focused on RMS.

Cold Spring Harbor Laboratory (CSHL), a world leader in cancer biology and genetics, is applying its unique interdisciplinary approach and cutting-edge technology to RMS in an effort to develop effective therapeutics for the disease.

To search for a cure, CSHL Assistant Professor Chris Vakoc is collaborating with Oregon Health and Science University Professor Charles Keller. Keller is one of the world leaders in RMS research and has created dozens of mouse models for the disease that will be instrumental in future research. Vakoc is using cells from these mouse lines in an test he has developed to pinpoint genetic weaknesses within a population of cancer cells. Vakoc’s cutting-edge approach has already been successful in developing a drug for acute myeloid leukemia, with a treatment now in clinical trials.

The Vakoc-Keller collaboration was spurred by a 2014 Banbury Center symposium on RMS. The first-ever meeting of its kind, the symposium brought together researchers from around the world to chart a path forward to better diagnosis and treatment of RMS. The research presented during the meeting suggests that development of a drug for RMS is indeed a realistic objective.

Read the report: Rhabdomyosarcoma: A critical review of research and implications for developing therapies (pdf).

The Sarcoma Research Project has been made possible by the generous support of local foundations. Your contributions provide ongoing support in CSHL’s search for a cure for RMS. See foundations to the left.

The National Cancer Institute is a resource for additional information on RMS.

Christina Renna

Friends of TJ

Clark Gillies Foundation

Michelle Paternoster