Case CCC has received over $1 million in combined support from the Children’s Tumor Foundation and Gateway for Cancer Research to investigate a targeted treatment approach for malignant peripheral nerve sheath tumor (MPNST), a rare cancer.
The study's principal investigator Gary Schwartz, MD, Case CCC’s Director, was recognized at the Gateway for Cancer Research award ceremony this week. He collaborates with Ankit Mangla, MD, member of Case CCC’s Developmental Therapeutics Program, who is principal investigator for the clinical trial.
The clinical trial, currently conducted at University Hospitals Seidman Cancer Center, part of the Case CCC consortium, evaluates BMS-986504, an investigational drug, to determine whether it can slow or shrink MPNST tumors with a genetic alteration called MTAP deletion.
The loss of MTAP may make tumor cells more sensitive to the study drug. BMS-986504 inhibits PRMT5, an enzyme involved in cellular processes that support tumor growth. Researchers are testing whether this molecular vulnerability can provide a basis for treatment.
The study focuses on patients ages 12 and older with locally advanced or metastatic MPNST that cannot be surgically removed. The protocol requires measurable disease and confirmation of homozygous MTAP deletion—loss of both copies of the gene—through next-generation sequencing, along with additional clinical and laboratory criteria.
Researchers will assess tumor response, safety and tolerability, progression-free survival and overall survival. The drug’s effectiveness in this setting remains under investigation.
Support from the Children’s Tumor Foundation and Gateway for Cancer Research strengthens Case CCC’s investigation of whether a tumor’s molecular characteristics can help guide treatment development for this rare cancer.
Plans are underway to extend the trial to Cleveland Clinic, another Case CCC consortium member, and other sites across the United States. Find information about this trial here.