A team led by Gary Schwartz, MD, Case CCC Director, received a grant from the Children’s Tumor Foundation (CTF)—a nonprofit dedicated to improving care for millions affected by genetic conditions—to support a multi-site study across Ohio, including Case CCC’s NCI-designated consortium, The Ohio State University Comprehensive Cancer Center, and Siteman Comprehensive Cancer Center. Case CCC consortium partners, University Hospitals and Cleveland Clinic, will be the first to open the trial to children and adolescents impacted by neurofibromatosis type 1 (NF1).
This genetic disorder causes tumors to form on nerves anywhere in the body—including the skin, brain, and spinal cord. In NF1, a mutation in the NF1 gene on chromosome 17 makes a protein called neurofibromin. This protein helps regulate cell growth; when faulty, cells grow uncontrollably and can cause tumor growth on nerve endings that can become cancerous.
The Phase II clinical trial aims to reduce disease burden for patients with unresectable NF1-associated malignant peripheral nerve sheath tumors (MPNST) and homozygous methylthioadenosine phosphorylase (MTAP) deletions because traditional cancer therapies, such as surgery, chemotherapy, or radiation, cannot be used for the following reasons:
MPNSTs are aggressive soft tissue sarcomas with a five-year survival rate of only 20% in locally advanced or metastatic disease, and cannot be treated with surgery due to tumor proximity to critical nerve structures.
Radiation therapy may help control local tumor growth, but it has no significant overall survival benefit.
Response rates to chemotherapy can be lower than 20% in inoperable disease.
By trialing BMS-986504, a therapy supplied by Bristol Myers Squibb following a competitive process, Schwartz and his team believe they might have an answer with the potential to not only improve patient outcomes but also transform the standard-of-care for this disease.