An international collaboration targets a rare cancer

Torsten Nielsen

A privately-funded collaborative that includes UBC’s Torsten Nielsen will create the world’s largest collection of primary tumour samples and cell models of myxoid liposarcoma.

The collaborative, which also includes researchers at Leiden University in the Netherlands and the University of Texas, will use the tissue samples to better understand myxoid liposarcoma and its response to various treatments. They will also work to identify pathways that can be targeted with existing and experimental drugs.

The Liddy Shriver Sarcoma Initiative, through a peer-reviewed selection process, has awarded a two-year, $250,000 grant for the collaborative. A comprehensive experimental plan for the study has been published in April’s issue of the Electronic Sarcoma Update Newsletter, and a study report will be published when the project is completed.

“We’re combining resources from multiple countries to get enough samples of a relatively rare disease that we can make sense out of them — a scientifically large enough collection,” says Dr. Nielsen, an Associate Professor in the Department of Pathology and Laboratory Medicine, and a clinician-scientist at the Vancouver Coastal Health Research Institute and the BC Cancer Agency.

Myxoid liposarcomas account for about 10% of all soft tissue sarcomas, which are rare cancers of the connective tissues. They typically affect adults between the ages of 30 and 50 years old and are prone to metastasize. There are currently no effective systemic therapies for the disease, which can be highly lethal.

One of the difficulties in studying rare diseases is a lack of tissue specimens and patient data. This study addresses that challenge by creating the world’s largest collection of primary tumor samples and cell models of myxoid liposarcoma, a resource that will be made available to qualified researchers worldwide.

Dr. Nielsen’s work has already led to two new, widely-used diagnostic biomarkers in sarcoma and to two clinical trials that are currently underway.

“In myxoid liposarcoma, we know that there is a very specific, causative mutation called FUS-DDIT3,” Dr. Nielsen says. “The scientific community has yet to translate this finding into new therapies for patients — partly because there have not been enough researchers working on it, and partly because no one research team has access to enough materials (cancer cells and tumor specimens) to do this work effectively. I believe that with this funding we have a great opportunity to connect the underlying molecular biology to new treatment opportunities.”

Cancer research can be a highly competitive field, but sarcoma researchers share a unique sense of teamwork within the cancer research community. Dr. Nielsen explains, “Sarcoma researchers have a real sense of community and cooperation. Sometimes it feels like there are more types of sarcoma than there are sarcoma research teams! We are keenly aware that we need to combine resources to achieve critical mass for the study of rare diseases.”

This grant was made possible by donations to an annual fund-raising event, the Wendy Walk, that is being held this spring in Miami, New York and Los Angeles to raise money for liposarcoma research. The event is named for Wendy Landes, of Pacific Palisades, California, who was diagnosed with multi-focal dedifferentiated liposarcoma in 2008.

Proceeds from the Wendy Walk were given to the Liddy Shriver Sarcoma Initiative for distribution. The all-volunteer organization, based in Ossining, New York, was created by Liddy Shriver’s family shortly before she succumbed to Ewing’s sarcoma in 2004, at the age of 37. Dedicated to increasing global public awareness of sarcoma, raising funds to award research grants, and providing support and timely information to sarcoma patients, their families, and medical professionals, it has raised $3.25 million, and awarded $2 million in grants.