NFCR Translational Research: Childhood Cancer

New Treatment for Rhabdomyosarcoma in Children: Clinical Trial Beginning Soon

Cesare Spadoni, Ph.D., MBA University of London

Rhabdomyosarcoma is the most common pediatric soft tissue sarcoma and is aggressive and highly malignant.  It develops from skeletal muscle cells that have failed to fully differentiate. The aggressive subtype, alveolar rhabdomyosarcoma, results from the abnormal fusion of two genes.

NFCR support for Dr. Cesare Spadoni has advanced the preclinical research of the drug, volasertib, to treat rhabdomyosarcoma. As a highly potent inhibitor of the enzyme PLK1, volasertib reduces activity and stability of the abnormal fused proteins. Significantly, the anti-cancer effects of volasertib are more pronounced when combined with the chemotherapy, vincristine. Volasertib has already been studied as single agent in Phase I clinical trials in children with leukemia and refractory solid tumors. A Phase I clinical trial to treat rhabdomyosarcoma with volasertib will begin soon.

BASIC RESEARCH PROJECTS

Developing New Treatment for Childhood Brain Cancer, Meduloblastoma

NFCR’s support also reaches the discovery program for Medulloblastoma, a malignant childhood brain cancer. Medulloblastoma accounts for 20% of all childhood brain tumors and has high heterogeneity (caused by different genes) and a tendency to metastasize (spread). Ongoing research is identifying a lead compound that inhibits two targets (gene abnormalities or vulnerabilities) in the cancer – this will increase efficacy and reduce toxicity and resistance to the treatment. The research aims to select and characterize a candidate to enter into the pre-clinical phase in late 2022.