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New cancer treatment effectively targets tumors, limits hearing loss in children

Results of clinical trial show that sodium thiosulphate reduces ototoxicity by 50 percent
man in suit and tie, sitting in front of microscope, smiling
Edward Neuwelt, M.D., has spent more than 20 years researching pediatric hearing loss caused by platinum-based chemotherapies. A clinical trial based on his work has found a treatment protocol that reduces hearing loss by nearly 50 percent when treating a form of pediatric liver cancer. (OHSU/Kristyna Wentz-Graff)

A common side effect of platinum-based chemotherapies, such as cisplatin or carboplatin, is damage to the inner ear that may result in long-term hearing loss.

A clinical trial led by Cancer Research UK funded researchers at London’s Great Ormond Street Hospital, with contributions from OHSU in Portland, Oregon, has found that administering the drug sodium thiosulphate, or STS, following cisplatin to treat a form of pediatric liver cancer reduces hearing loss by nearly 50 percent without impeding the impact of the chemotherapy.

The findings published today in the New England Journal of Medicine.

The SIOPEL-6 trial is based on more than 20 years of preclinical research conducted by the study’s senior author Edward Neuwelt, M.D., and colleagues at OHSU. Penelope Brock, M.D., Ph.D., a pediatric consultant at GOSH, served as the trial’s principal investigator.

Using human and animal models, Neuwelt, a professor of neurology and neurological surgery in the OHSU School of Medicine, first determined that STS could prevent hearing loss caused by platinum-based chemotherapies. He and his team then determined that delaying the dosage of STS would avoid interference with the treatment’s impact on cancerous tumors.

Edward Neuwelt M.D.
“This advancement has the potential to impact other forms of pediatric cancers treated with platinum-based chemotherapy, as well. We look forward to the day when – following treatment – patients can be one step closer to just being kids,” says Neuwelt. (OHSU/Kristyna Wentz-Graff)

“We are allowing young patients to have the best of both worlds: no cancer and little to no hearing loss that could cause difficulties with speech recognition, learning and social engagement,” said Neuwelt. “This advancement has the potential to impact other forms of pediatric cancers treated with platinum-based chemotherapy, as well. We look forward to the day when – following treatment – patients can be one step closer to just being kids.”

Based on the results of the SIOPEL-6 trial, the research team believes that STS should be considered a new standard of care for treating hepatoblastoma, as well as other childhood cancers where cisplatin is used. The treatment regimen has received breakthrough therapy designation by the U.S. Food and Drug Administration and researchers are seeking marketing authorization from both the FDA and European Medicines Agency.

Additional findings of the SIOPEL-6 trial are available here.

The study was funded by Cancer Research UK and others; SIOPEL 6 ClinicalTrials.gov number, NCT00652132; EudraCT number, 2007-002402-21.

In the interest of ensuring the integrity of our research and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our researchers may hold with entities outside of OHSU. In regards to this research, OHSU, the Portland Veterans Affairs Medical Center and the Department of Veterans Affairs have a financial interest in Fennec Pharmaceuticals, a company that may have a commercial interest in the results of this research and technology. Neuwelt, an inventor of technology licensed to Fennec Pharmaceuticals, has divested himself of all potential earnings. Review details of OHSU's conflict of interest program to find out more about how OHSU manages these business relationships.

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