Pharma Focus Asia

SHEPHERD Therapeutics and Mayo Clinic Announce Collaboration to Advance Research in Rhabdomyosarcoma Using SHEPHERD's Precision-Oncology Platform, DELVE

Friday, February 26, 2021

SHEPHERD Therapeutics, a company dedicated to catalyzing lifesaving treatments for rare cancer patients, announced a collaboration with the Mayo Clinic to advance research in rhabdomyosarcoma (RMS), a rare pediatric cancer with few treatment options. As part of the collaboration, SHEPHERD and Mayo clinicians and researchers, Dr. Patricio Gargollo, Dr. Candace Granberg, and Dr. Fabrice Lucien-Matteoni, will use SHEPHERD's proprietary, next-generation precision-oncology platform, DELVE, to identify mechanisms for drug response and resistance in RMS patients, and to identify new single drug and multi-drug combinations that have the potential to impact RMS care.

Although 380 out of 400 cancers are considered rare, treatments for rare cancers are lacking. As of 2018, almost 80% of cancer patients without an approved therapy had a rare cancer, and from 2012 to 2016, 75% of clinical trials did not specifically include any rare cancers. All cancers that predominately affect pediatric patients are considered rare.

"SHEPHERD's mission is to save the life of every rare and pediatric cancer patient by connecting them with next-generation, lifesaving therapies," said David Hysong, CEO of SHEPHERD. "This partnership showcases a new paradigm for how rare cancers can be treated; we are honored to work alongside such a brilliant team at the Mayo Clinic to bring hope to these young patients and their loved ones."

The Schafran family is one of millions that has experienced the devastating loss of a loved one as a result of not having a treatment for a rare cancer, when their beloved Finn passed away at age 3 from RMS. Although RMS can occur at any age, it most often affects children. The disease forms in soft tissue — specifically skeletal muscle tissue or sometimes hollow organs such as the bladder or uterus. BrandiLee Schafran, Director of Patient Advocacy at the SHEPHERD Foundation and Finn's mother, shared, "Together, Mayo Clinic and SHEPHERD will be a powerhouse of promise in the fight against the devastating disease that is rhabdomyosarcoma, accelerating research that could save lives that do not need to be lost."

This rhabdomyosarcoma research project is being led by the experienced and dedicated Mayo Clinic clinicians and scientists:

  • Patricio Gargollo, M.D., Associate Professor Urology, Pediatric Urologist, Mayo Clinic
  • Candace Granberg, M.D., Assistant Professor Urology, Pediatric Urologist, Mayo Clinic and SHEPHERD Foundation Advisory Council member
  • Fabrice Lucien-Matteoni, Ph.D., Assistant Professor of Urology, Mayo Clinic

"Started in 2018, our multi-disciplinary team has been invested in understanding the reasons why rhabdomyosarcoma is such an aggressive cancer and identifying molecular vulnerabilities that can be targeted with next-generation therapies. Our research is somehow limited by the scarcity of fresh specimens and biological tools to study this disease. Our collaboration with SHEPHERD Therapeutics and access to the DELVE platform represents a unique opportunity to extrapolate from more heavily-studied cancers and accelerate rhabdomyosarcoma research. Our clinical and scientific expertise will synergize with DELVE's platform to open new opportunities and options for more efficacious treatments in rhabdomyosarcoma patients," said the researchers.

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