Clinical Trials: Rhabdomyosarcoma
Rhabdomyosarcoma (RMS) is a rare soft tissue tumor found most often in children. Surgery and radiation are generally used to treat the site of the tumor, while chemotherapy is used to prevent further spread of the cancer and to treat disease at all sites in the body.
For decades, the long-term survival for patients with high-risk Rhabdomyosarcoma, a soft tissue tumor found most often in children, remained at 30 percent.
Dr. Weigel and the team at the University of Minnesota, in conjunction with the Children’s Oncology Group (COG), have been working tirelessly to improve survival rates. Over a decade ago, Dr. Weigel started investigating a more intensive combination of chemotherapy combined with radiation to kill more tumor cells.
Based on positive early research through Phase I and II trials, our team led a national multi-center Phase III trial, which showed a 20 percent higher survival rate. This is a major breakthrough for this cancer, and Dr. Weigel’s protocol is now the standard of treatment for patients with high-risk RMS.
Dr. Weigel and her team at the University are also conducting other clinical trials to develop better treatments and create better outcomes for kids and adults who are diagnosed with all stages of rhabdomyosarcoma. They include:
Phase I Trial using Seneca Valley Virus
This Phase I trial is studying the side effects and identifying the best dose to deliver of Seneca Valley virus in treating young patients with relapsed or refractory neuroblastoma, rhabdomyosarcoma, or other rare tumors with neuroendocrine features. A virus called Seneca Valley virus-NTX-010, which is in the same virus family as the common cold virus, may be able to kill tumor cells without damaging normal cells. This trial is led by the U of M’s Dr. Michael Burke and is open nationally at select Children’s Hospitals.
Pilot Study involving new combination of chemotherapy agents
This clinical trial is studying the side effects and how well giving chemotherapy drugs — temozolomide and cixutumumab — together with combination chemotherapy works in treating patients with high-risk rhabdomyosarcoma. This trial builds upon previous studies by adding a new chemotherapy drug to the treatment regimen developed by Dr. Weigel.
Cixutumumab is a monoclonal antibody that blocks some tumor growth factors. It is hoped that giving cixutumumab with chemotherapy will prevent the tumor from growing or spreading to other parts of the body. Temozolomide interacts with irinotecan, another chemotherapy drug, and it is hoped that this synergy will help kill tumor cells more effectively.
Phase II Trial that studies use of cell-cyle inhibitor drug (Sorafenib)
University researchers are taking part in the study of a new class of drugs, called cell-cycle inhibitors. This type of drug is different from chemotherapy because it can target and stop (inhibit) specific pathways of tumor growth. Unlike chemotherapy, cell cycle inhibitors have fewer side effects because they do not kill “good cells.” This Phase II trial studies how well Sorafenib works in treating younger patients with relapsed or refractory rhabdomyosarcoma. Researchers believe Sorafenib will stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.