FDA Approves Sodium Thiosulfate to Prevent Chemo-Induced Hearing Loss in Kids
An excerpt from our 2018 Voice of the Patient Report on Childhood Cancer Hearing Loss
In big news for kids receiving the chemotherapy agent cisplatin now and in the future, the Food and Drug Administration (FDA) recently approved sodium thiosulfate (brand name PedMark) to reduce the risk of chemo-induced hearing loss in pediatric cancer patients.
In 2018, when Children’s Cancer Cause hosted its first externally-led Patient Focused Drug Development (EL-PFDD) meeting on the topic of chemo-induced hearing loss, sodium thiosulfate (STS) was a hot topic of conversation. We hope that parents and survivors who spoke directly to the FDA by participating in that meeting feel a sense of pride and accomplishment in this huge victory for kids receiving treatment today.
Approximately one third of children with cancer will receive a platinum therapy such as cisplatin as their first or second line treatment. In the U.S., approximately 5,000 children receive platinum chemotherapy annually. Several pediatric cancers are typically treated with platinum, and these include brain and central nervous system cancers, neuroblastoma, hepatoblastoma, osteosarcoma, germ cell tumor, and retinoblastoma.
Up to 75 percent of children who receive cisplatin suffer from some degree of permanent hearing loss. The time of onset of the hearing loss is variable but can occur as early as the first or second cisplatin cycle. Hearing loss is a permanent lifetime disability that, in children, has ramifications for speech and language development, learning, social development, employment, and lifetime earnings.
Sodium thiosulfate is the first FDA-approved treatment to protect the inner ear from cisplatin treatment and reduce the likelihood that a child will experience hearing loss. The FDA approval of this therapy covers pediatric patients from one month old through 18 years old with localized tumors (meaning the cancer has not spread beyond where it began). We’ve heard from treating physicians that clinics have already begun administering this therapy for kids currently receiving cisplatin.
A number of cancer survivors at our 2018 meeting reported positive experiences as participants in clinical trials of STS. Some parents reported that after being administered STS, their child did not experience any, or any further, hearing loss.
Most were unaware until after they had experienced hearing loss that there was a drug in clinical trials that might have prevented their hearing loss. Many seemed to have only learned about it during the EL-PFDD meeting and expressed anguish that the drug had, at the time, not yet been FDA-approved.
“We used STS after round two [of chemo],” said one mom. “It worked wonders. I wish we knew about it before we started."
After the EL-PFDD meeting, we heard from another mom who expressed hope that the FDA would take note of what she observed at the meeting: “Our daughter was seated with two other survivors with hearing loss they got 12 to 15 years ago. Directly behind them in the next row was a boy that received the preventative medication [STS] for hearing loss 17 years ago. He was with his mom and the doctor who gave him the preventative medication. It must have been so frustrating for the doctor to have the knowledge that the three girls seated in front of him could have received the preventative medication and not have hearing loss as they do now.”
While we have much more work to do in this area - including stronger support and better treatment options for survivors who’ve already suffered hearing loss - we are grateful to the FDA for this approval, to the clinical trial researchers and participants, and to all the families who took part in our 2018 meeting. Your voice was heard, and your stories made a difference.