NHS offers new drug to stop children and teens losing hearing after chemotherapy

By Tim Gunn

A first-of-its-kind drug that protects the hearing of babies, children and young people undergoing cancer treatment has been approved for NHS use in England, based on the results of one of our trials.??

Our SIOPEL-6 study showed that sodium thiosulfate (Pedmarqsi) almost halves the percentage of children and teenagers who lose hearing as a side effect of the platinum-based chemotherapy drug cisplatin. ?

Now the National Institute of Health and Care Excellence (NICE) has recommended the drug to NHS England, almost 60 children and teenagers (one month to 17 years old) should be eligible to receive it over the next year.

Dr Penelope (Peppy) Brock, who led the SIOPEL-6 trial from Great Ormond Street Hospital in London, called the decision “crucial”.?

“As cure rates increase into the high 90s for several cancers, the need to resolve these permanently disabling effects becomes more and more pressing,” she added. “I am delighted that finally we have something to offer to counter this life-impacting side effect and can give children the opportunity to live healthy, happy and fully integrated lives after overcoming cancer.”?

Adding sodium thiosulfate to cisplatin

Cisplatin is a very effective treatment for many solid tumours, and has contributed to a dramatic improvement in survival since it was first approved in the 1970s (on the basis of another of our clinical trials). ?

However, while cisplatin is rapidly removed from most of the body following treatment, it lingers in the cochlea, the portion of the inner ear responsible for hearing. The damage it can cause there has an especially big impact on people’s ability to hear high-pitched (or high-frequency) sounds.?

Around 6 in 10 children and young people treated with cisplatin alone will develop some level of irreversible hearing loss.??

“We’re lucky to have such an effective treatment,” said Brock, an expert in hearing loss as a side effect of cancer treatment (ototoxicity). “But a huge proportion of children treated with cisplatin alone are left with permanent hearing loss, which can be utterly debilitating.?

“Even mild hearing loss can severely impact a child’s future development. Key consonants like ‘s’, ‘f’, ‘t’ and ‘k’ are heard at high frequencies, and their loss can be particularly difficult for children who haven’t yet developed speech.”

Cutting side effects across cancer types

Although SIOPEL-6 focused on children being treated for hepatoblastoma, the NICE guidance covers multiple cancer types. Sodium thiosulfate is recommended for all children and young people from one month to 17 years old who are receiving cisplatin for solid tumours that have not spread to other parts of the body.???

This?reflects evidence from another trial carried out in the US and Canada, which found that sodium thiosulfate contributed to a similar reduction in hearing loss in this wider group.?

The drug is given as an infusion (through an IV drip) six hours after cisplatin chemotherapy. This is to ensure it doesn’t interfere with the way cisplatin kills cancer cells.?

As a result of the NICE decision, NHS England will start offering sodium thiosulfate for children and teenagers being treated with cisplatin over the next three months.?

NICE decisions are also usually adopted in Wales and Northern Ireland, so the drug should become available there soon too. Scotland has separate process for approving drugs and has not yet evaluated sodium thiosulfate for this use on the NHS.?