A new drug developed by researchers at Georgetown University Medical Center may be able to stop the progression of a rare bone cancer that affects children and adolescents.
In preclinical rat studies, researchers found that the agent (S)-YK-4-279, developed by Dr. Jeffrey Toretsky and his colleagues, halted the progression of the malignant bone tumor Ewing sarcoma. The drug also caused some rat tumors to regress so much that researchers could not microscopically detect cancer cells. Toretsky established TDP Biotherapeutics to manufacture the drug.
Ewing sarcoma is caused by the exchange of DNA between two chromosomes, and (S)-YK-4-279 works by stopping the two proteins--EWS-FLI1 and RHA--from interacting. The research is set to be presented at the American Society of Clinical Oncology (ASCO) annual meeting next week.
"This agent has the potential to be more effective, and considerably less toxic, than the current drugs now used to treat this rare cancer," Toretsky, a pediatric oncologist and researcher at Georgetown Lombardi Comprehensive Cancer Center, part of the university's medical center, said in a press release.
Annually, about 500 children and young adults are diagnosed with Ewing sarcoma, and few patients whose cancer progresses are cured. Typical treatment consists of a combination of several different chemotherapy drugs. One of these, Lundbeck's Cosmegen, is highly toxic and can cause severe skin and muscle damage if it leaks into surrounding tissue when administered.
The FDA has granted TDP Biotherapeutics orphan drug status for (S)-YK-4-279, and the company is seeking an investigational new drug application for the agent in hopes of moving it toward a clinical trial.
- here's the press release