CHOP Welcomes World-Renowned Sarcoma Expert

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Oncology Update

Patrick J. Grohar, MD, PhD, recently joined Children's Hospital of Philadelphia (CHOP) as an attending physician with the Cancer Center and the Director for Translational Research with CHOP’s Center for Childhood Cancer Research. Dr. Grohar’s research focuses on developing therapies for pediatric sarcomas, particularly Ewing sarcoma, a form of cancer that’s rare in adults but relatively common in the pediatric population. It most often occurs in the pelvis and less commonly in the long bones of the arms and legs.

Dr. Grohar comes to CHOP from the Van Andel Research Institute in Michigan, where he was the program leader of Skeletal Disease and Cancer Therapeutics in their Center for Cancer and Cell Biology. He is currently a member of the Children’s Oncology Group Bone Tumor Steering Committee and is vice chair for biology of the Bone Tumor Committee, co-chair of the Combined Sarcoma Biology Committee and chair of Ewing Sarcoma Biology Committee.

For sarcoma patients, the current therapeutic approach of chemotherapy, radiation and surgery is effective in many patients, but less so in those whose cancer has spread or relapsed. New therapies are desperately needed for this patient population. Dr. Grohar’s work in this area is truly translational: bench to bedside and back again.

The sarcoma field needs a better understanding of what Dr. Grohar calls “the bad actors.” “Neuroblastoma has really good biomarkers that subdivide patients into high, medium and low risk,” he explained. “Sarcoma in general doesn’t have that.”

Many sarcomas are driven by a particular mutation called chromosomal translocation that generates a transcription factor. The resulting dysregulated transcription factor alters the expression of more than 500 genes to drive tumorigenesis and progression. “These transcription factors are felt to be undruggable targets — because of their physical shape, there’s no obvious place to put a molecule to block function,” said Dr. Grohar. “We are working hard on trying to find ways to drug these targets because the tumor completely depends on them.”

One reason Dr. Grohar is excited to be at CHOP is the relationship with Penn Medicine’s Abramson Cancer Center. “Dr. Grohar can synergize with existing sarcoma researchers at CHOP and Penn,” said Rochelle Bagatell, MD, the solid tumor section chief at CHOP. “He’ll take things to another level.”

He is also optimistic: “In today’s day and age, I don’t think that changing outcomes for patients is unrealistic,” Dr. Grohar said. “We’ve made progress. We have some exciting data in preclinical models. It’s all about serving the patients, and coming to CHOP felt like an opportunity to make an impact.”

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