British girl’s hearing restored in pioneering gene therapy trial
A British girl has had her hearing restored after becoming the first person worldwide to take part in a pioneering new gene therapy trial.
Opal Sandy was born totally deaf due to auditory neuropathy, which disrupts nerve impulses from the inner ear to the brain.
But the 18-month-old’s hearing is now almost normal – and could improve further – after having the one-time treatment.
Opal was treated at Addenbrooke’s Hospital in Cambridge and the head of the trial, Professor Manohar Bance, said results were “better than I hoped or expected” and he hopes medics might be able to cure others with this type of deafness.
“We have results from (Opal) which are very spectacular – so close to normal hearing restoration. So we do hope it could be a potential cure,” he said.
Opal (second left) with her parents and sister Nora, who has the same condition. Pic: PA
Auditory neuropathy can be caused by a fault in the OTOF gene, which makes a protein called otoferlin and enables cells in the ear to communicate with the hearing nerve.
Opal, from Oxfordshire, had an infusion of the working gene into her right ear during surgery in September – the treatment developed by biotech firm Regeneron.
Her parents noticed changes in only four weeks and the improvements were especially noticeable 24 weeks later.
Jo and James Sandy, both 33, said they were “gobsmacked” when she responded to sound tests at home without her cochlear implant, the usual way to treat the condition.
Opal had her operation in September. Pic: Cambridge University NHS Trust
“I thought it was a fluke or like a change in light or something that had caught her eye, but I repeated it a few times,” said Mrs Sandy.
They say she now enjoys the sound of slamming her cutlery on the table and playing with toy drums and wooden blocks.
“We were told she had near normal hearing last time,” she added. “I think they got responses at sort of 25 to 30 decibels.
“I think normal hearing is classed at 20 decibels, so she’s not far off. Before, she had no hearing whatsoever.”
Opal’s surgery was very similar to fitting a cochlear implant, according to Prof Bance.
He said the inner ear (cochlea) was opened and the treatment infused using a catheter over 16 minutes.
“We have to make a release hole in another part of the ear to let the treatment out because it has to go all the way through the ear,” he said.
“And then we just repair and close up, so it’s actually a very similar approach to a cochlear implant, except we don’t put the implant in.”
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Experts say it could mark a ‘new era’ in treating deafness. Pic: PA
He said the gene therapy potentially “marks a new era in the treatment for deafness”.
“It was just the fact that we’ve been hearing about this for so long, and there’s been so much work, decades of work… to finally see something that actually worked in humans… It was quite spectacular and a bit awe-inspiring really,” he said.
The treatment was developed especially for children with OTOF mutations, and a second child who has had the same surgery is also seeing positive results.
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The trial has three parts – with three deaf children, including Opal, getting a low dose in just one ear.
Another three children will get a high dose on one side. Then, if safe, a new set of children will get a dose in both ears at the same time.
Up to 18 people from the UK, US and Spain are being recruited and will be followed up for five years.
Results of the study were presented on Wednesday to the American Society of Gene and Cell Therapy conference in Baltimore.
Conservative Party leader Kemi Badenoch has called on Sir Keir Starmer to sack Treasury minister Tulip Siddiq over allegations she lived in properties linked to allies of her aunt, Sheikh Hasina, the deposed prime minister of Bangladesh.
It comes after the current Bangladeshi leader, Muhammad Yunus, said London properties used by Ms Siddiq should be investigated.
He told the Sunday Times the properties should be handed back to his government if they were acquired through “plain robbery”.
Tory leader Ms Badenoch said: “It’s time for Keir Starmer to sack Tulip Siddiq.
“He appointed his personal friend as anti-corruption minister and she is accused herself of corruption.
“Now the government of Bangladesh is raising serious concerns about her links to the regime of Sheikh Hasina.”
Ms Siddiq insists she has “done nothing wrong”.
Her aunt was ousted from office in August following an uprising against her 20-year leadership and fled to India.
Ms Siddiq is also named with her aunt in Bangladesh court documents about meetings with the Russian government.
Tory leader Kemi Badenoch has called on Sir Keir to sack the minister
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As economic secretary to the Treasury, Ms Siddiq is responsible for policy on both the City and tackling corruption.
She referred herself to the prime minister’s ethics watchdog on Monday following the reports about the properties.
On the same day, the prime minister said: “Tulip Siddiq has acted entirely properly by referring herself to the independent adviser, as she’s now done, and that’s why we brought into being the new code.
“It’s to allow ministers to ask the adviser to establish the facts, and yes, I’ve got confidence in her, and that’s the process that will now be happening.”
Police in Aberdeen have widened the search area for two sisters who disappeared four days ago in the city.
Eliza and Henrietta Huszti, both 32, were last seen on CCTV on Market Street after leaving their home on Tuesday at around 2.12am.
The sisters – who are part of a set of triplets and originally from Hungary – crossed the Victoria Bridge to the Torry area and turned right on to a footpath next to the River Dee.
Eliza and Henrietta Huszti were last seen on CCTV in Market Street. Pic: Police Scotland/PA
They headed in the direction of Aberdeen Boat Club but officers said there is no evidence to suggest the missing women left the immediate area.
Specialist search teams, police dogs and a marine unit have been trying to trace the pair.
The sisters crossed Victoria Bridge before walking along a footpath next to the River Dee
The pair were heading in the direction of Aberdeen Boat Club on the south side of the River Dee
Further searches are being carried out towards the Port of Aberdeen’s South Harbour and Duthie Park.
Police Scotland said it is liaising with authorities in Hungary to support the relatives of the two sisters.
Chief Inspector Darren Bruce said: “Eliza and Henrietta’s family are understandably extremely worried about them and we are working tirelessly to find them.
“We are seriously concerned about them and have significant resources dedicated to the inquiry.”
The sisters, from Aberdeen city centre, are described as slim with long brown hair.
Henrietta Huszti. Pic: Police Scotland
Eliza Huszti. Pic: Police Scotland
Officers have requested businesses in and around the South Esplanade and Menzies Road area to review their CCTV footage for the early morning of Tuesday 7 January.
Police added they are keen to hear from anyone with dashcam footage from that time.
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